The Journey

2009-04-14T18:35:00.003-05:00

Our Drew lost his battle with ALS on Tuesday April 7, 2009. His family would like to thank all of the people who made Drew's journey a little more bearable by following his blog, leaving notes of encouragement and comments. He will not be forgetten.

Thank You,

The Schemera Family

2009-03-21T18:07:00.001-05:00

The natural flights of the human mind are not from pleasure to pleasure, but from hope to hope.--Samuel Johnson

Some states push back against stem cell research

2009-03-11T18:41:00.000-05:00

Some states push back against stem cell research
By SHANNON McCAFFREY, Associated Press Writer Shannon Mccaffrey, Associated Press Writer 1 hr 31 mins ago
ATLANTA – A showdown is shaping up in some of the nation's most conservative states over embryonic stem cell research, as opponents draw language and tactics from the battle over abortion to counter President Barack Obama's plan to ease research restrictions.
Legislation granting fertilized embryos "personhood" has gained momentum in at least three state legislatures. The strategy — which has been used to try to undermine the Roe v. Wade decision legalizing abortion — is now aimed at embryonic stem cell research. The scientific field uses stem cells from human embryos, which can develop into different kinds of adult cells, to seek answers about human health.
Opposition to both abortion and stem cell research hinges on the same issue: When does life begin? As a result, embryonic stem cell research has become the latest front in the decades-old battle over abortion.
"If you are someone who believes that a single cell embryo is a person then you are looking for any opportunity you can to make that argument. But as a country, legally, we've never accepted that," said Michael Werner of the Coalition for the Advancement of Medical Research. "The legislative tactics are the same."
Abortion opponents believe embryonic stem cell research is an assault on life in its earliest form. Fertilized embryos are destroyed when stem cells are extracted from them for research.
"No one's right for a cure supersedes someone else's right to life," said Dan Becker, president of Georgia Right to Life.
The opponents expect to push for restrictions in conservative-leaning states. And they say states must take the lead in pushing the abortion and stem cell issues into the increasingly conservative federal courts.
Legal experts said the state measures restricting stem cell research raise constitutional concerns in a largely untested area of law.
Alta Charo, professor of law and bioethics at the University of Wisconsin, said a new line of legal thought holds that scientific inquiry should be protected by the First Amendment, "like a political or religious statement or activity."
She said the measures restricting the use of fertilized embryos also raise questions about the right to procreation.
"The courts haven't settled this yet," Charo said.
While Louisiana already bans the destruction of fertilized embryos, the courts have not yet weighed in, Charo said.
In Georgia, a measure that would ban some forms of stem cell research on fertilized embryos is moving quickly through the state Senate. The bill would outlaw the destruction of fertilized embryos, which the legislation defines as a person. It is expected to face a vote in the full state Senate on Thursday.
Similar "personhood" measures have cleared one chamber each in Montana and North Dakota.
They come in the wake of a Colorado ballot initiative that said human life begins at conception. It failed to win voter approval last year.
David Prentice, senior fellow for life sciences at the Washington, D.C.-based Family Research Council, said Obama's announcement Monday that he will free federal funds for embryonic stem cell research will rally conservatives.
"This is the beginning," Prentice said. "I think there will be more to come."
In 2001, President George W. Bush limited federal funding for embryonic stem cell research to 21 stem cell lines already in existence. Because they were already being used for research, Bush allowed work on them to continue.
Obama's new approach will enable federally funded researchers to use hundreds of new embryonic stem cell lines. Supporters believe the research could lead to treatments for major disorders, such as Parkinson's disease and spinal injuries.
Eight states bucked the Bush administration limits and allowed state money to be spent on the research: California, Connecticut, Illinois, Iowa, Maryland, Massachusetts, New Jersey and New York.
Some of them, struggling with gaping budget deficits, may rein in state funding for those research programs, now that federal dollars will again be flowing.
Sean Tipton, director of public affairs at the American Society for Reproductive Medicine, said legislation that would affect stem cell research has been introduced in several states, including Alabama, Georgia, Maryland, Montana, North Dakota and South Carolina.
"It's clearly part of a national strategy and at some point it will probably succeed," Tipton said.
Tipton said advocacy groups are targeting states where they have the best chance of success.
One of those is Georgia, where Gov. Sonny Perdue has said he opposes embryonic stem cell research, even as he tries to lure biotech companies to state.
"I am absolutely opposed to creating embryos to cure a disease," Perdue told reporters this week.
The Georgia bill cleared the Senate Health and Human Services Committee by a close 7-6. The religious conservatives pushing it are influential with Georgia's Republican-led Legislature.
Opponents say the Senate bill would be a blow to the state's thriving research universities, as well as fertility clinics that perform thousands of in-vitro treatments every year.
"We have the president of the United States saying he is going to put science ahead of politics and unfortunately in Georgia we are moving in the opposite directions," said state Sen. David Adelman, a Democrat from Decatur.
___
National Conference of State Legislatures Genetic Technologies Project: http://www.ncsl.org/programs/health/genetics

European Commission Awards Orphan Designation to Knopp Neurosciences' KNS-760704 in ALS

2009-03-11T16:57:00.000-05:00

European Commission Awards Orphan Designation to Knopp Neurosciences' KNS-760704 in ALS
PITTSBURGH--(BUSINESS WIRE)--Knopp Neurosciences Inc. ("Knopp") today announced that the European Commission ("EC") has designated KNS-760704 as an orphan medicinal product for the treatment of Amyotrophic Lateral Sclerosis.
The EC decision, based on a favorable opinion from the European Medicines Agency (EMEA), follows the designation of KNS-760704 as an orphan drug for the treatment of ALS by the U.S. Food and Drug Administration in 2007. KNS-760704 is currently in Phase 2 clinical trials for ALS, a universally fatal neurodegenerative disease with limited treatment options.
"We are very pleased with the European Commission's designation of KNS-760704 as an orphan medicinal product and the recognition of its potential to be of significant benefit for patients with ALS," said Michael Bozik, M.D., president and CEO of Knopp. "Orphan medicinal product designation will significantly facilitate our efforts to develop a safe and effective treatment for patients suffering from this relentless disease."
To stimulate the research and development of orphan drugs, the European Union ("EU") has established a centralized procedure for the designation of orphan medicinal products and provides incentives for the development of medicinal products for rare disorders. Companies with an orphan designation for a medicinal product benefit from incentives that include fee reductions, a 10-year market exclusivity period following authorization for designated products, scientific advice to optimize development, and direct access to the EMEA centralized procedures for marketing authorization.
Knopp plans to initiate Phase 3 development of KNS-760704 in ALS in late 2009.
About KNS-760704
KNS-760704 is a low molecular weight benzothiazole shown to improve mitochondrial function and confer significant cellular protection in neurons under stress. The chirally pure form of the synthetic benzothiazole (6R)-2-amino-4,5,6,7-tetrahydro-6-(propylamino)benzothiazole, KNS-760704 is highly orally bioavailable, water soluble, renally excreted, and only moderately protein bound. In Phase 1 studies, the compound was shown to be safe and well tolerated in healthy human subjects. Phase 2 studies of KNS-760704 in ALS are ongoing. The compound has received orphan drug designation from the U.S. Food and Drug Administration and the European Commission for the treatment of patients with ALS.
About ALS
Amyotrophic lateral sclerosis, also known as Lou Gehrig's disease and Charcot's sclerosis, is a rapid, universally fatal neurodegenerative disorder characterized by progressive muscle weakness and wasting. ALS affects adults in the prime of life and creates a substantial burden for caregivers. U.S. prevalence is approximately 20,000 and the global incidence is approximately two per 100,000. Only one drug has been approved for the treatment of ALS. Life expectancy after symptom onset is usually three to five years.
About Knopp Neurosciences Inc.
Knopp Neurosciences is a drug discovery and development company focused on delivering breakthrough treatments for neurological disorders through innovation, experience, and partnership. The company's lead product candidate is KNS-760704, an orally bioavailable small molecule in development for the treatment of ALS. Knopp's leadership includes experienced neuroscience drug development and discovery executives formerly associated with major pharmaceutical companies. Knopp's financing has been led by Saturn Capital Inc. of Boston as placement agent and Saturn Partners II as lead funder.
This press release contains "forward-looking statements," including statements relating to Knopp's planned regulatory filings and clinical development programs for KNS-760704. All forward-looking statements are based on management's current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including the uncertainties inherent in clinical trials and product development programs, the availability of funding to support continued research and studies, the availability or potential availability of alternative therapies or treatments, the availability of patent protection for the discoveries and strategic alliances, as well as additional factors that may cause Knopp's actual results to differ from our expectations. There can be no assurance that KNS-760704 will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market the products. Knopp undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise

finally

2009-03-10T17:51:00.000-05:00

FDA, Insmed, Inc. Announce Special Program for IPLEX and ALS
The U.S. Food & Drug Administration granted approval for a limited number of people with ALS in the United States to receive IPLEX, a drug that combines insulin-like growth factor (IGF-1) and IGF binding protein 3. IPLEX, which has not been approved for use in ALS, is manufactured by Richmond, VA-based Insmed, Inc. The new program is the result of an agreement between the FDA and Insmed.
The ALS Association hopes that the FDA-approved program will develop informative data about IPLEX that can lead to a better understanding of its efficacy and safety and enable both patients and clinicians to make more informed decisions about the use of IPLEX and its potential as a therapy for ALS. To this end, The Association encourages the FDA and Insmed to establish partnerships with the ALS community to ensure that the program yields meaningful results that will guide the next steps in determining whether IPLEX is effective and safe for ALS.
IPLEX originally was approved in the United States as a treatment for children with growth failure, but it is now discontinued and no longer available for this population. The drug is being tested in a now-closed study involving myotonic muscular dystrophy (MMD). Insmed is supplying IPLEX to the Italian government under an “expanded access program,” but it continues to be an untested and unproven treatment for ALS in the United States.
Based on existing clinical and scientific evidence, The ALS Association cannot encourage or recommend the off-label use of this medication without substantive evidence of its efficacy through a rigorous clinical trial. The ALS Association is continuing to monitor and assess information about IPLEX as it becomes available to provide the public with the most up-to-date reports about its potential for ALS.
Additional information on today's announcement is available here: http://www.fda.gov/cder/drug/infopage/mecasermin_rinfabate/default.htm.
For additional information from The ALS Association, please contact our Patient Services Department at alsinfo@alsa-national.org.

finally-will there be enough time.....??

2009-03-09T16:57:00.001-05:00

Obama overturns Bush policy on stem cells
By PHILIP ELLIOTT, Associated Press Writer Philip Elliott, Associated Press Writer 26 mins ago
WASHINGTON – Reversing Bush policy, President Barack Obama on Monday cleared the way for a significant increase in federal dollars for embryonic stem cell research and promised no scientific data will be "distorted or concealed to serve a political agenda."
Obama signed the executive order on the divisive stem cell issue and a memo addressing what he called scientific integrity before an East Room audience packed with scientists. He laced his remarks with several jabs at the way science was handled by former President George W. Bush.
"Promoting science isn't just about providing resources, it is also about protecting free and open inquiry," Obama said. "It is about letting scientists like those here today do their jobs, free from manipulation or coercion, and listening to what they tell us, even when it's inconvenient especially when it's inconvenient. It is about ensuring that scientific data is never distorted or concealed to serve a political agenda and that we make scientific decisions based on facts, not ideology."
He said his memorandum is meant to restore "scientific integrity to government decision-making." He called it the beginning of a process of ensuring his administration bases its decision on sound science; appoints scientific advisers based on their credentials, not their politics; and is honest about the science behind its decisions.
Fulfilling a campaign promise, Obama signed the order that on stem cell research that supporters believe could uncover cures for serious ailments from diabetes to paralysis. Proponents from former first lady Nancy Reagan to the late actor Christopher Reeve had pushed for ending the restrictions on research.
Obama paid tribute to Reeve, calling him a tireless advocate who was dedicated to raising awareness to the promise of research.
Obama's action reverses Bush's stem cell policy by undoing his 2001 directive that banned federal funding for research into stem lines created after Aug. 9, 2001.
The president said his administration would work aggressively to make up for the ground he said was lost due to Bush's decision, though it can't be known how much more federal money will be spent on the research until grants are applied for and issued.
"Medical miracles do not happen simply by accident," Obama declared.
Embryonic stem cells are master cells that can morph into any cell of the body. Scientists hope to harness them so they can create replacement tissues to treat a variety of diseases — such as new insulin-producing cells for diabetics, cells that could help those with Parkinson's disease or maybe even Alzheimer's, or new nerve connections to restore movement after spinal injury.
House Minority Leader John Boehner, R-Ohio, criticized Obama, saying in a statement that the president had "rolled back important protections for innocent life, further dividing our nation at a time when we need greater unity to tackle the challenges before us."
Bush limited the use of taxpayer money to only the 21 stem cell lines that had been produced before his decision. He argued he was defending human life because days-old embryos — although typically from fertility clinics and already destined for destruction — are destroyed to create the stem cell lines.
The Obama order reverses that without addressing a separate legislative ban, which precludes any federal money for the development of stem cell lines. The legislation, however, does not prevent funds for research on those lines created without federal funding.
Researchers say the newer lines created with private money during the period of the Bush ban are healthier and better suited to creating treatment for diseases.
Obama called his decision a "difficult and delicate balance," an understatement of the intense emotions generated on both sides of the long, contentious debate. He said he came down on the side of the majority of Americans who support increased federal funding for the research, both because strict oversight would prevent problems and because of the great and lifesaving potential it holds.
"Rather than furthering discovery, our government has forced what I believe is a false choice between sound science and moral values," Obama said. "In this case, I believe the two are not inconsistent. As a person of faith, I believe we are called to care for each other and work to ease human suffering."
Obama warned against overstating the eventual benefits of the research, but he said his administration "will vigorously support scientists who pursue this research," taking another slap at Bush in the process.
"I cannot guarantee that we will find the treatments and cures we seek. No president can promise that. But I can promise that we will seek them actively, responsibly, and with the urgency required to make up for lost ground," he said.
It's a matter of competitive advantage globally as well, the president argued.
"When government fails to make these investments, opportunities are missed. Promising avenues go unexplored," Obama said.
But the president was insistent that his order would not open the door to human cloning.
"We will develop strict guidelines, which we will rigorously enforce, because we cannot ever tolerate misuse or abuse," Obama said. "And we will ensure that our government never opens the door to the use of cloning for human reproduction. It is dangerous, profoundly wrong, and has no place in our society, or any society."

2009-03-09T14:53:00.000-05:00

http://news.prnewswire.com/ViewContent.aspx?ACCT=109&STORY=/www/story/03-09-2009/0004984875&EDATE=

Please review/double-click, the hyperlink above.
It contains some interesting stuff, to say the least.
Quick excerpt:
"This is the first demonstration of transplanted human neurons synapsing, or making mature structural connections, with the rat motor neurons, something which has not been demonstrated before," said Dr. Karl Johe, Neuralstem's Chief Scientific Officer and a study co-author. "Our earlier work with this ALS model showed that the stem cells delayed onset of the disease and played a neuroprotective role. Now we have clear evidence that they can become an integral part of the rat nervous system that controls the muscles. I would expect these cells to be readily accepted by and integrated into a human nervous system, such as in an ALS or a spinal cord injury patient."

iplex info

2009-03-09T14:49:00.000-05:00

To:
teamiplex@googlegroups.com

Randy and Bob,

I believe you both saw this on ALSTDI forum, where I posted it March 2. Nothing has changed. The FDA will do what they’re going to do—with or without Italian data. This is no longer an issue of science (data about whether Iplex is or is not pre-proven and good for those with ALS). It’s simply an issue of whether or not they can deny a terminal population a medication that is already FDA approved—nothing else. That’s an issue of state statutes, federal law and FDA practice—all of which they (FDA) are now violating. And those physicians who will write off-label Rx’s or INDs will do so with or without Italian data—which they find suspect under the best of situations. And those who won’t write off-label or INDs will not start doing so because they see data from an Italian “observation.”

Anyone reading this can use their time more constructively by writing to your legislators and to FDA, using as a template that which you have previously seen from several people—me, Eddie Esparza and others. Or, write a new and different message demanding the right to use Iplex.

Regards,
Steve Byer

From ALSTDI FORUM March 2, 2009

Dear Randy,When reading the following, please bear in mind that I am neither an apologist nor spokesperson for Insmed. Because of my interest in Iplex as a potential treatment for ALS, I have been able to make some communication inroads with Insmed and others related to this issue. I think, from your post quoted just below, and others from you more recently, it is advisable to share some thoughts on the subject.“I feel like a broken record and apologies if this has been covered in another post, but it has now been almost 3 months since the Iplex rally, at which time we were told (those of us who attended) that the Italian data would be released soon. I understood that it was this data that was to be the predicate of further activities and pursuits of obtaining it in the U.S. and maybe elsewhere. Have I missed something? Has this data been released or, if not, any indication as to when this will happen? I suppose "soon" could be anytime, but with each passing day an no news on this front, it looks more and more like either there was misinformation, miscommunication or attempts by one or more parties to mislead and spread false hope. I hope I'm wrong about this, of course.” Questioner ALS-TDI forum 02/09/09In response to the above and other of your posts and emails:1. When I asked for the data on December 22, Insmed stated they have turned over the results to two separate researchers for analysis and comparison to historical placebos. They had, just then, received the data from Italy after “numerous requests.”2. When I asked again on January 19, 2009, they stated both companies were having difficulty analyzing the information received from Italy because of the difficult nature of translating data from many different areas of Italy (20), many different age and progression groups, many different analyses by physicians treating the patients (I think it was 20 physicians) and few patients in the study (only 60 because they excluded all others who weren’t on Iplex for at least a certain time period). In other words, it was never purported to be a clinical test study—but rather “an observation.”3. While the information is unsatisfactory to me, and perhaps to you, it’s also understandable even by those of us who would prefer a clear set of study data and results from a well-conducted double-blind test study.4. Those with ALS don’t always have the luxury of time that a clinical test study requires. In the case of Myotrophin, an inferior drug for the purpose of treating ALS, it took from 1997 to 2009 for the results to be finalized. Even then, the results were unclear and marred by ineffective dosages and contradictory results from three different trials (in US, EU and again US).5. Therefore, those with diseases such as ALS (little-understood, no apparent cure, almost always treated unsuccessfully) are sometimes required to try a medication even without the benefit of prior test data if there is some, or any, reason to feel the treatment has promise. In the case of Iplex, there are several foundations, or at least suggestions, to feel the drug has promise. 6. You already know what those are, but just in case: prior usage by eight US patients in early 2007; continuing usage by now of up to 180 Italian patients paid for by the Italian government (a cost I doubt they take lightly); the results thus far of use by those with Myotonic muscular dystrophy (University of Rochester—a continuing study); scientific foundations previously provided you and others regarding the underlying benefits of IGF-1/IGFbp-3 vs free IGF-1; hope for Iplex when other hope doesn’t exist.7. Insmed has nonetheless stated that, from the little information on hand, there appears to have been a positive effect from at least 50% of those who were in the 60-patient group. That is my recollection of the specific words, and while it may be ever-so-slightly off, the meaning was clear—50% of those with ALS treated with anything is a success. I didn’t think to ask, at the time, if the remaining 50% were “stable, declining, or otherwise.”8. I was asked, at the time of the conversations above, not to publicize the information because it was unconfirmed and to some extent conjecture. Further, Insmed, as a public company, is unable to release such information without qualification because it can be construed as “forward-looking statements”, a legal issue for public companies. I do feel that enough time has passed from the conversations that it can be disseminated.9. If this is, in anyone’s view, “misinformation, miscommunication or attempts by one or more parties to mislead and spread false hope”, then so be it. Steve Byer

Stem cells can help regrow diseased muscles

2009-03-08T17:22:00.000-05:00

Stem cells can help regrow diseased muscles
Sydney, March 6 : An experimental procedure that dramatically strengthens stem cells' ability to regenerate damaged tissue could offer new hope to victims of muscle-wasting diseases like myopathy and muscular dystrophy.
The first-ever procedure has been successfully used to regrow muscles in a mouse model, but it could be applied to all tissue-based illnesses in humans in the liver, pancreas or brain, the researchers say. The research team, which is based at University of New South Wales (UNSW) and formerly from Sydney 's Westmead Children's Hospital, adapted a technique currently being tried in bone marrow transplantation. Adult stem cells are given a gene that makes them resistant to chemotherapy, which is used to clean out damaged cells and allow the new stem cells to take hold. The ability of adult stem cells to regenerate whole tissues opens up a world of new possibilities for many diseases, according to the study co-authors, Peter Gunning and Edna Hardeman, both professors, and Antonio Lee, from UNSW's School of Medical Sciences. "What has been the realm of science fiction is looking more and more like the medicine of the future," Gunning said. The procedure solves one of the major hurdles involving stem cell therapy - getting the cells to survive for more than an hour or so after inserting them into damaged tissue, said an UNSW release. These findings were detailed in the journal Stem Cells.

How stem cells turn into blood cells

2009-03-08T17:20:00.000-05:00

How stem cells turn into blood cells
Washington, Mar 6 : A research team led by an Indian origin scientist has shed light on how stem cells turn into blood cells.
Stem cells are the building blocks of every organ and tissue in the body. They have a unique ability to become any type of cell in the body including bone, muscle and blood cells.Dr. Mick Bhatia, director of the McMaster University Stem Cell and Cancer Research Institute claim to have identified a particular cell pathway, known as the noncanonical Wnt that prompts stem cells to specialize and become blood cells.The pathway appears to organize the stem cells so that they can respond to signals telling them what to turn into."By directing cell differentiation, this method provides the most efficient way to produce blood cells that we are aware of to date," said Bhatia."This finding is exciting because it may provide a new way to make blood from human stem cells that could be used to regenerate the blood system in patients, including those with leukemia or those undergoing cancer treatments

that indirectly destroy the immune and blood system," said Dr. Christine Williams, Director of Research Programs at the Canadian Cancer Society Research Institute.The findings are published in Cell Stem Cell.

my cousin sent me this, thanks sally

2009-03-06T17:02:00.001-05:00

Source: Obama to reverse limits on stem cell work
By BEN FELLER and LAURAN NEERGAARD, Associated Press Writers Ben Feller And Lauran Neergaard, Associated Press Writers 1 min ago
WASHINGTON – President Barack Obama expected to sign an executive order on Monday reversing restrictions on federal funding of embryonic stem cell research.
The long-expected move is likely to stir up not only the promise of scientific breakthrough but also the controversy over where government crosses a moral line.
Obama will hold an event at the White House to announce the move, a senior administration official said Friday. The official spoke on condition of anonymity because the policy had not yet been publicly announced.
Under President George W. Bush, federal money for research on human embryonic stems cells was limited to those stem cell lines that were created before Aug. 9, 2001. No federal dollars could be used on research with cell lines from embryos destroyed from that point forward.
Obama's move is expected to lift that restriction. The official said the aim of the policy is restore "scientific integrity" to the process.
Embryonic stem cells are master cells that can morph into any cell of the body. Scientists hope to harness them so they can create replacement tissues to treat a variety of diseases — such as new insulin-producing cells for diabetics or new nerve connections to restore movement after spinal injury.
"I feel vindicated after eight years of struggle, and I know it's going to energize my research team," said Dr. George Daley of the Harvard Stem Cell Institute and Children's Hospital of Boston, a leading stem cell researcher.
Such research is controversial because embryos must be destroyed to obtain the cells; they typically are culled from fertility-clinic leftovers otherwise destined to be thrown away. Once a group of stem cells is culled, it can be kept alive and propagating in lab dishes for years.
There are different types of stem cells, and critics say the nation should pursue alternatives to embryonic ones such as adult stem cells, or those found floating in amniotic fluid or the placenta. But leading researchers consider embryonic stem cells the most flexible, and thus most promising, form — and say that science, not politics, should ultimately judge.
"Science works best and patients are served best by having all the tools at our disposal," Daley said.
Obama made it clear during the campaign he would overturn Bush's directive.
During the campaign, Obama said, "I strongly support expanding research on stem cells. I believe that the restrictions that President Bush has placed on funding of human embryonic stem cell research have handcuffed our scientists and hindered our ability to compete with other nations."
He said he would lift Bush's ban and "ensure that all research on stem cells is conducted ethically and with rigorous oversight."
"Patients and people who've been patient advocates are going to be really happy," said Amy Comstock Rick of the Coalition for the Advancement of Medical Research.
The ruling will bring one immediate change: As of Monday, scientists who've had to meticulously keep separate their federally funded research and their privately funded stem cell work — from buying separate microscopes to even setting up labs in different buildings — won't have that expensive hurdle anymore.
Next, scientists can start applying for research grants from the National Institutes of Health. The NIH already has begun writing guidelines for what embryonic stem cell lines will qualify under Obama's ruling. Among other things, the guidelines are expected to demand that the cells were derived with proper informed consent from the woman or couple who donated the original embryo.

http://www.google.com/search?sourceid=navclient&aq=h0&oq=&ie=UTF-8&rlz=1T4GGLL_enUS302US302&q=Italians+find+ALS+gene The 1.5-million-euro study, which

2009-03-04T16:39:00.000-05:00

http://www.google.com/search?sourceid=navclient&aq=h0&oq=&ie=UTF-8&rlz=1T4GGLL_enUS302US302&q=Italians+find+ALS+gene

The 1.5-million-euro study, which looked at the so-called 'sporadic' type of ALS, identified a gene called Sunc1 which appears to play a predominant role in regulating ALS. But Chio' said Sunc1 was "probably just the tip of the iceberg." He said the study will now move into a third phase in which 300 new patients will be examined, all of them Italian.

epidermal growth factor

2009-03-04T16:26:00.001-05:00

Epidermal growth factor promotes the differentiation of stem cells derived from human umbilical cord blood into neuron-like cells via taurine induction in vitro.

Jin W, Xing YQ, Yang AH.Department of Ophthalmology, Renmin Hospital of Wuhan University, Jiefang Road 238#, Wuhan, Hubei Province, 430060,

People's Republic of China.Results of recent investigations have demonstrated the plasticity of mesenchymal stem cells (MSC) can differentiate into neural lineages. In this study, we explored the experimental condition of differentiation into neuron-like cells or rhodopsin (RHOS)-positive cells induced by epidermal growth factor (EGF) and taurine in vitro and to investigate their biological characteristics. MSC were obtained from umbilical cord blood (UCB) of term deliveries. Cultured cells were treated with Dulbecco's modified Eagle's medium/F12 (pH 7.0-7.2) supplemented with 30 ng/ml EGF. After the third cell passage, the cells were trysinized and analyzed with a flow cytometer using the following monocloned antibodies: CD90, CD29, CD34, CD44, and CD45. Taking another MSC of the third passage, its basal medium was replaced with alpha minimum essential medium supplemented with taurine (50 micromol/L). Cells were cultured for an additional 8-10 d, fixed, and then immunocytochemicall y analyzed. Primary antibodies included the following: neuron-specific enolase (NSE), RHOS, and nestin. In our study, we isolated a cell population derived from UCB, which possesses morphological characteristics similar to those of MSC isolated from bone marrow. In the cytometric analysis, MSC did not present labeling for the hematopoietic line (CD34 and CD45) and were positive for CD29, CD44, and CD90. After induction by taurine, 80.5 +/- 16.2% of the cell population expressed NSE, 36.8 +/- 9.6% expressed RHOS, and 29.6 +/- 9.3% expressed Nestin, while only 7.9 +/- 3.5% expressed NSE in the control group. This study demonstrates that partial MSC induced by taurine and EGF can differentiate into neuron-like cells or RHOS-positive cells in vitro, which may provide a promising therapeutic strategy for the treatment of some forms of retinal degeneration.

2009-03-02T13:33:00.003-05:00

Stem cell breakthrough now goes one step further
Sun Mar 1, 1:20 pm ET
PARIS (AFP) – Pioneering work by Japanese stem-cell researchers two years ago has taken a major step forward, helping the quest for versatile, grow-in-a-dish transplant tissue, according to papers published on Sunday.
Two teams have combined ideas to devise a safer technique for reprogramming skin cells so that they become "pluripotent" stem cells -- fundamental cells that then grow into specialised organs.
Their effort builds on an award-winning breakthrough in 2007 by Shinya Yamanaka of Kyoto University.
He and his team introduced four genes into skin cells, reprogramming them so that they became indistinguishable from embryonic stem cells.
That achievement conjured the distant vision of an almost limitless source of transplant material that would be free of controversy, as it would entail no cells derived from embryos.
But the downside of the technique for creating these so-called induced pluripotent stem cells (iPS) is that the genes are delivered by a "Trojan horse" virus.
Reprogramming cells using a virus modifies their DNA in such a way that they cannot be given to patients without boosting the risk of cancer.
In the new studies, published by the British-based journal Nature, two squads of researchers from Britain and Canada recount a method by which the four genes are delivered into the cell without using a virus -- and then are removed after the reprogramming is done.
The insertion is carried out using "piggyBac," a tried-and-tested technique in genetically-modified crops in which mobile genetic sequences called transposons are slotted into the genome.
In the iPS work, it has been tested successfully on mouse and human skin cells. Tests on the reprogrammed cells lines show they faithfully reproduce the behaviour of embryonic stem cells.
"I was very excited when I found stem cell-like cells in my culture dishes. Nobody, including me, thought it was really possible," said Keisuke Kaji from the Centre for Regenerative Medicine at the University of Edinburgh, Scotland.
In a press release issued by Britain's Medical Research Council, Ian Wilmut -- the "father" of Dolly the cloned sheep -- stressed that the new iPS cells would have to be tested thoroughly for safety before being used in any human trials.
And, "crucially," scientists were still hunting for a way of coaxing pluripotent cells into the specialised tissues that could be used in transplants, said Wilmut, who heads the unit where Kaji works.
Even so, "there is hope that the promise of regenerative medicine could soon be met," he said.
Stem cells have excited huge interest over the past decade. Promoters say this material could reverse cancer, diabetes, Alzheimer's and other regenerative diseases.
But the dynamic has been sapped by opposition from religious conservatives, who argue that research on embryos -- the prime source of stem cells so far -- destroys human life.

2009-02-28T16:48:00.000-05:00

Stem cell research supporters offer U.S. Senate bill
By Maggie Fox Maggie Fox Fri Feb 27, 3:35 pm ET
WASHINGTON (Reuters) – Two prominent supporters of stem cell research said on Thursday they had reintroduced a Senate bill that would allow federal funding for human embryonic stem cell research, in anticipation of President Barack Obama's support for the work.
Senators Tom Harkin, an Iowa Democrat, and Arlen Specter, a Republican from Pennsylvania, said their bipartisan measure would allow federal funding for research using stem cells taken from human embryos left over from fertility treatments.
"It is the same bill that both houses of Congress approved in 2007, but was vetoed by President Bush," they said in a statement.
Obama has promised to overturn Bush's policy that strictly limited the use of federal funds for embryonic stem cell research.
Many groups that support embryonic stem cell research have been eagerly waiting for him to do so, but White House spokesman Robert Gibbs has hinted that Obama would prefer to wait and do something in concert with Congress.
"For too long, political interference has delayed research that holds the promise for millions of Americans who suffer from a wide range of diseases," Harkin said in a statement.
"President Obama has promised to lift the restrictions on embryonic stem cell research that were put in place by President Bush, and I hope and expect that he will do so soon, but we have to make sure that the freedom to pursue this research is also protected by federal law, not merely by an executive order that can be reversed during a future administration."
Specter said legislation would protect the policy "so that it does not ping-pong back and forth with each successive president."
Other senior senators co-sponsored the bill, including liberal Democrats Edward Kennedy of Massachusetts and Dianne Feinstein of California, as well as conservative Republican Orrin Hatch of Utah.
Stem cells are the body's master cells and those taken from days-old embryos are especially powerful, with the ability to change into all the various cell-types in the body.
Researchers are studying them to try and find ways to regenerate tissues and treat diseases such as Parkinson's, diabetes, cancer and also injuries.
Opponents such as Bush say it is immoral to experiment on or destroy human embryos and say U.S. taxpayers should not have to pay for such research.

Public release date: 23-Feb-2009

2009-02-26T18:48:00.000-05:00

Public release date: 23-Feb-2009[
Print Article E-mail Article
Close Window ]Contact: Donna Perrydonna@biologists.com44-122-343-3319The Company of Biologists
Human stem cells provide a new model for Lou Gehrig's disease
Motor neurons derived from embryonic stem cells mimic the progress of familial ALS
February 23, 2009, Cambridge, UK – Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a devastating condition in which motor neuron degeneration causes progressive loss of movement and muscle tone, leading to death. Overcoming the limited success of previous models, a report published in Disease Models & Mechanisms (DMM), dmm.biologists.org describes how neurons can be derived from human stem cells, and engineered to mimic inherited ALS.
Researchers at the University of California Los Angeles developed an optimized protocol to generate motor neurons from human embryonic stem cells (ES cells), which express normal or mutant forms of the SOD-1 gene, which is linked to inherited, familial ALS. Resulting cells exhibit hallmark characteristics of motor nerve cells, and neurons expressing mutant SOD-1 display abnormalities typical of ALS. Defects included shortened cell projections and a reduced life span compared to cells containing the normal SOD-1 gene.
This human cell-derived model of ALS provides a new method of studying this disease and testing novel therapeutics. This is especially helpful as only one drug is approved to help slow ALS progression, and animal models currently used in drug development have had limited success. Additionally, this research may aid other gene-linked neurodegenerative diseases, as they too may benefit from studies in a human cell-derived model.
###
Commentary on this work will be featured in the DMM Podcast for Volume 2, Issue 3/4 of DMM. Podcasts are available via the DMM website at: dmm.biologists.org.
The report was written by Saravanan Karumbayaram, Theresa K. Kelly, Andres A. Paucar, Anne J.T. Roe, Joy A. Umbach, Andrew Charles, Harley I. Kornblum, and Martina Wiedau-Pazos at the David Geffen School of Medicine at the University of California Los Angeles, and Steven A. Goldman at the University of Rochester Medical Center in Rochester, NY. The report is published in the March/April issue of Disease Models & Mechanisms (DMM), a research journal published by The Company of Biologists, a non-profit based in Cambridge, UK.
About Disease Models & Mechanisms:
Disease Models & Mechanisms (DMM) is a new research journal publishing both primary scientific research, as well as review articles, editorials, and research highlights. The journal's mission is to provide a forum for clinicians and scientists to discuss basic science and clinical research related to human disease, disease detection and novel therapies. DMM is published by the Company of Biologists, a non-profit organization based in Cambridge, UK. The Company also publishes the international biology research journals Development, Journal of Cell Science, and The Journal of Experimental Biology. In addition to financing these journals, the Company provides grants to scientific societies and supports other activities including travelling fellowships for junior scientists, workshops and conferences. The world's poorest nations receive free and unrestricted access to the Company's journals.

Breaking News

2009-02-26T18:01:00.000-05:00

Breaking News
New Gene Mutation Discovery by ALS AssociationConsortium is Major Research Breakthrough
(February 26, 2009) -

In one of the most significant breakthroughs in the recent history of ALS research, a consortium of scientists organized and funded by The ALS Association has discovered a new gene, ALS6 (Fused in Sarcoma), responsible for about 5 percent of the cases of inherited ALS. The discovery will provide important clues to the causes of inherited ALS, which accounts for 10 percent of all cases, and sporadic ALS, which occurs in individuals with no family history of the disease and accounts for the other 90 percent of cases diagnosed.
“This is a momentous discovery in furthering our understanding of ALS,” said Lucie Bruijn, Ph.D., senior vice president of Research and Development at The ALS Association. “A new gene provides a new piece of the puzzle we can use to shed light on why ALS develops, and where to focus our efforts on creating new treatments and finding a cure.”
The results of this groundbreaking research are published in the Friday, February 27 issue of the prestigious journal Science. The project was led by Tom Kwiatkowski M.D., Ph.D., at Massachusetts General Hospital, and Robert Brown, M.D., of the University of Massachusetts School of Medicine, and ALS Association-funded researchers Caroline Vance, Ph.D., and Christopher Shaw, M.D., of Kings College in London. The project was supported by a consortium of leading ALS researchers from around the world, formed as part of The Association’s Gene Identification Project. Their success reflects an unprecedented effort to accelerate the search for genetic mutations linked to all forms of ALS.
Dr. Brown noted, “We are particularly delighted because our trans-Atlantic consortium has pursued the chromosome 16 gene for more than six years. The ALS Association has been an all-important partner in this search. This discovery should lead to new cell and animal models of ALS, which will accelerate drug development.”
“Global partnerships between investigators and funding agencies, such as the Motor Neuron Disease Association in the United Kingdom, are crucial to making these kinds of breakthroughs,” Dr. Bruijn commented. “This finding has opened up a whole new avenue of research and has the potential to uncover a common mechanism for most forms of ALS.”
The gene mutations were first identified by Dr. Kwiatkowski and were immediately confirmed by Dr. Vance, who also demonstrated abnormal accumulations of the mutant protein in cells cultured in the laboratory and the motor neurons of people carrying FUS mutations.
The gene, called FUS (“fused in sarcoma”), normally carries out multiple functions within motor neurons. These include regulating how gene messages (called messenger RNAs) are created, modified, and transported in order to build proteins. Some of these same functions also are performed by another gene called TARDPB encoding the protein TDP43, and mutations in the TDP-43 gene were recently linked to ALS as well.
“The fact that these two genes help perform the same function suggests that problems in this function may be critical in the development of ALS,” Dr. Bruijn said. “More research into exactly how these two genes work could ultimately lead to new treatments that are effective in slowing or stopping the progression of ALS and extending the lives of people with the disease.”
The mutations in the ALS6 gene were identified by detailed genetic sequencing in several families with an inherited form of ALS (familial ALS). Normally, the ALS6 protein works in the cell’s nucleus, but the mutations caused it to instead cluster outside the nucleus. Further work will be needed to determine precisely how this leads to ALS. With the gene in hand, scientists will be able to create cell and animal models containing the mutated gene, to examine in detail how the mutation operates and how it causes ALS.
“This suggests there may be a common mechanism underlying motor neuron degeneration,” according to Dr. Shaw. Motor neurons are nerve cells in the brain and spinal cord that control muscles. Motor neurons degenerate in ALS.
This is the second ALS-causing gene to be discovered in the past 12 months. SOD1, discovered in 1993, accounts for 20 percent of inherited cases of the disease. Mutations in the TARDP gene account for another four to five percent. The only well-defined causes of ALS are genetic. In both inherited and sporadic ALS, the disease symptoms and pathology are the same.
The possibility that ALS may be caused by several factors is the rationale for The Association’s policy of funding multiple genetic projects around the world and encouraging these leading geneticists to work together and share information to help locate disease-linked genes for faster, more accurate scientific results. By funding research on a global level, The Association helps put together “genetic pieces” of the ALS puzzle.
“Through our support of research such as this study, The ALS Association is committed to finding the causes of ALS, and using that knowledge to develop a cure as rapidly as possible,” Dr. Bruijn said. “We will build on the discovery of this new gene to carry that effort forward.”

email me at schemera@yahoo.com for attachments

2009-02-25T19:30:00.001-05:00

Dear friends, family, & supporters,

I am once again, requesting help from any and all family, friends, & supporters . Please Review the above attachments we (Team IPLEX) have provided...

You can... cut and paste certain talking points from the attachments, or... you can just cut and paste the entire document - as is. (of course don't forget to, change the address, name, and salutation's.) then double-click each Web link below, (which will open up that particular FDA officer's e-mail voicing your support for this (PALS) cause.

All We Are Asking ... is that they (the FDA hierarchy), provide us (PALS) a more data-driven reason/argument, for denying American PALS a drug/therapeutic, that the Italian government are paying Millions of Dollars annually (on behalf of their citizenry), for the specific Treatment of ALS. Is That Really Too Much to Ask ?... Just show us the proof, that -- IPLEX Is Dangerous, and we'll all go away and shut up.

What's with all the secrecy ? What ever happened to openness and transparency? - What's with the... MY WAY OR HIGHWAY attitude of Dr. Katz? ... Finally, why haven't the Italian government, refused to stop paying, the Millions of Dollars (annually), for reasons that, IPLEX Is Dangerous and or Ineffective.?

Yes, roadblocks (FDA ) exist that prevent immediate access to Iplex ,but just like the last time we went up against Goliath, we are making progress. Last week, Stephen Byer (Team IPLEX Capt.) & his nonprofit organization - ALS worldwide, met with two Illinois legislators, who have promised, and are already showing, their support, of our effort to secure Iplex.

Therefore, we (Team IPLEX) are attaching documents in both PDF format and Microsoft Word format for your immediate use:

Please Take 10-15 minutes and Help, because for myself & other PALS, it truly is - A Race against Time.

Either of these documents can be copied and pasted into either media or congressional websites by using the Microsoft Word versions attached. Or, they can be sent, as is, to those for whom you have an email address by using the PDF files as attachments.

At the latter part of 2008, we (Team IPLEX & supporters) were collectively successful in doing what some said, was impossible - we can do it again but not without your help.

Immediately below are FDA contacts beginning with Dr. Russell Katz, the individual who appears responsible for the current FDA decision to deny Iplex to the ALS community. We urge that you send a copy of both the Congressional letter and the press release to each and every one of the following FDA personnel.

Please... contact Drs Katz, Temple , Jenkins, Woodcock, Torti and Ms. Behr -- Today. Together we can change the FDA decision and gain access to Iplex immediately.

Dr Russell Katz
Division Director, Neurology Products
301-796-2250 Tel
301-796-9842 Fax
russell.katz@fda.hhs.gov

His Superior :
Dr Robert Temple
Office of Medical Policy
301-796-2270 Tel
301-796-9840 Fax
robert.temple@fda.hhs.gov

His Superior :
Dr John Jenkins
Office of New Drugs
301-796-0700 Tel
301-796-9856 Fax
john.jenkins@fda.hhs.gov

His Superior :
Dr Janet Woodcock
Office of the Center Director
301-796-5400 Tel
301-847-8752 Fax
janet.woodcock@fda.hhs.gov

Her Superior :
Dr Frank M Torti
Acting Commissioner, FDA
10903 New Hampshire Avenue
Silver Spring, MD 20993-0002
Frank.torti@fda.hhs.gov

FDA Ombudsman:
Virginia L. Behr Office of the Chief of Staff FDA Center for Drug Evaluation and Research
301-796-3436 Tel
301-847-8452 FaxEmail: CDERombudsman@fda.hhs.gov

2009-02-25T19:21:00.000-05:00

Having hope makes every day easier, more gentle

NeuralStem Inc.'s ALS Trial on Clinical Hold

2009-02-20T15:29:00.000-05:00

NeuralStem Inc.'s ALS Trial on Clinical Hold 2/20/2009
ROCKVILLE, Md., Feb. 20 /PRNewswire-FirstCall/ -- Neuralstem, Inc. announced today its spinal cord stem cell trial to treat ALS is on clinical hold and that the Federal Drug Administration (FDA) has provided the Company with specific comments, questions and recommendations for modifications to its protocol.
(Logo: http://www.newscom.com/cgi-bin/prnh/20061221/DCTH007LOGO )
"The FDA has presented us with their review of our entire Investigational New Drug (IND) application," said Richard Garr, Neuralstem's President & CEO. "They have asked for some additional information regarding our product manufacturing process and pre-clinical studies, as well as our novel clinical delivery injection device and technique. The Company believes that it can provide this information in an expeditious manner."
"The Agency has also requested various modifications to the protocol and eligibility criteria for patients in the trial, as well as slight changes to the timing of the surgeries," Mr. Garr continued. "We are evaluating these changes and will respond accordingly. The Agency had extensive 'non hold' comments, requests for information, and recommendations. These primarily concerned issues that will need to be addressed for final product manufacturing and testing. We are appreciative of their work in this area."
"Over all we believe the Agency's comments and recommendations are extremely helpful," Garr concluded. "We are evaluating them carefully, and expect to reach agreement with the Agency on all matters so that the trial can be approved and move forward."
About Neuralstem
Neuralstem's patented technology enables, for the first time, the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells into mature, physiologically relevant human neurons and glia. Major Central Nervous System diseases targeted by the Company with research programs currently underway include: Ischemic Spastic Paraplegia, Traumatic Spinal Cord Injury, Huntington's disease and Amyotrophic Lateral Sclerosis (ALS).
In pre-clinical work, the company's cells have extended the life of rats with ALS (Lou Gehrig's disease) as reported the journal TRANSPLANTATION, in collaboration with Johns Hopkins University researchers, and also reversed paralysis in rats with Ischemic Spastic Paraplegia, as reported in NEUROSCIENCE on June 29, 2007, in collaboration with researchers at University of California San Diego.
Cautionary Statement Regarding Forward Looking Information
This news release may contain forward-looking statements made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such forward-looking statements in this press release regarding potential applications of Neuralstem's technologies constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward- looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstem's periodic reports, including the annual report on Form 10- KSB for the year ended December 31, 2007 and the quarterly report on form 10-Q for the period ended September 30, 2008.
CONTACT: Richard Garr, President of Neuralstem, Inc., +1- 301-366-4960; orMedia: Deanne Eagle of Planet Communications, +1-917-837-5866; orInvestors: Ina McGuinness of ICR, Inc., +1- 310-954-1100
Read at BioSpace.com

great website

2009-02-19T19:14:00.001-05:00

http://www.alsworldwide.net/

From: alscenter@jhmi.edu

2009-02-19T15:19:00.000-05:00

From: alscenter@jhmi.edu To: The ALS Community Date: February 18, 2009Subject: Robert Packard Center ALS News Network

TRIALS OF CELL-BASED THERAPY DON'T COME OUT OF THE BLUE

Some thoughts on Geron's start of stem cell-based therapy for spinal injury.http://www.alscenter.org/news/briefs/090218.cfm Late last month, ALS sufferers' hearts had to quicken at the announcement by=the biotech firm, Geron, hailing the start of clinical trials of a stem cel=-based therapy for new spinal injuries. Though the trial is a Phase I study meant to see if injected cells are safe =or humans, and though its patient subjects have had their very specific spi=al cord injuries only one-to-two weeks, the appearance of an authentic tria= involving potentially therapeutic cells - ones aimed at lessening or preve=ting paralysis - is exciting.What about trying out those cells or similar ones for ALS injuries to the ne=vous system?"Packard researchers in this country and Europe are doing an enormous amount=of work to sort out the many steps necessary to discover which cells to use=one day in ALS patients," says Packard Director Jeffrey Rothstein. "But eve= after that most essential work, after the appropriate cell type stands wel= out from the others, questions remain. "These are not simply safety questions," Rothstein says: Many of our ALS pat=ents would be willing to try a new therapy and fully understand the risks.What needs answering are questions of benefit and of procedure. "How do you =dminister the cells," he asks, "so the risk of getting them is justified by=their potential to relieve the disease or improve quality of life? "The impediments to our progress now are typical of what you'd encounter for=any potential new therapeutic drug," he explains. How many cells are approp=iate to deliver? That's the equivalent of finding the appropriate dose of a drug.What's the best way to distribute the cells throughout the brain and spinal =ord? That is akin to asking how often you give a drug.Do you give the cells through an IV or by direct surgical injection? What ha=pens to cells after we put them in? Do they divide like tumors? Do they die=and cause a scar that would hasten ALS effects? "Those are questions we mu=t first sort out in animals," says Rothstein.And a cell therapy carries its own unique concerns: How do we know that the =good' cells we put into one patient are identical to those we'd culture and=give a patient a year later? Quality assurance issues are well-established =or drugs like penicillin, Rothstein adds, but they have to get worked out f=r every new type of stem cell. The Packard Center has been doing its homework, however - not just for its o=n scientists but for the world's.A DECADE OF GROUNDWORK For almost 10 years, our researchers have been laying the groundwork for pos=ible cell therapy for ALS. Many of their published studies have likely infl=enced the Geron trial-planners. Here are a few:- Packard scientist Doug Kerr was the first to show that an animal model of = paralytic disease could be helped by injections of stem cells. - Packard Director Jeff Rothstein and his team solidified the suspicion that=motor neurons' natural protectors are its neighboring glial cells - that th=y remove harmful toxins as they collect in nervous system tissues under ass=ult. (The cells injected in the Geron study have the potential to become gl=al cells, those widespread nervous system cells in close proximity to motor=neurons.) - Work by Don Cleveland and Jean-Pierre Julien sent researchers scurrying mo=e surely to explore the concept of glial cells as protectors. They revealed=that keeping glia healthy can protect even motor neurons carrying an ALS ge=e. - Nicholas Maragakis and Mahendra Rao showed investigators everywhere how to=isolate, culture and precisely identify glial-restricted precursor (GRP) ce=ls - the broad class of "undeveloped" cells akin to the cell types Geron is=injecting. - Maragakis and Rao demonstrated that GRPs are neuroprotective - both in cul=ures that mimic the nervous system and in live animal models of ALS. Maraga=is and Rothstein have gone on to clarify much of the underlying biology. MORE RECENT WORK Because they specialize in ALS biology, Packard scientists know to target st=m cell and related therapies where they'd potentially do the most good. Las= November, for example, they reported a key step, an animal study in which =hey injected glial-restricted precursors precisely around the cervical spin=l cord home to motor neurons that permit breathing. Paralysis of those neur=ns precipitates death in ALS. The rat ALS models in the study survived significantly longer. Their forelim=s and breathing muscles stayed strong and functional far longer, while moto= neuron loss slowed. "This shows us that targeting cell delivery to critica= spinal areas is certainly worthy of investigating as a therapy for ALS, as=a way to slow specific types of motor neuron loss," says researcher Nichola= Maragakis. Now work needed to inch closer to clinical trials is underway.* * *In the last six months, Maragakis and colleague Hongjun Song have been worki=g at what can only be described as ALS's leading edge: they've begun studyi=g human pluripotent stem cells created from the skin of a patient with fami=ial ALS. One of the few recent, legitimate scientific breakthroughs, the technique in=olves reprogramming the patient's skin cells to behave like stem cells. The= the stem cells are coaxed to become motor neurons. This means that Packard=scientists will be able to study the progress of real ALS disease in real h=man cells, something that was out of reach before. ___________________________________ About The Robert Packard Center for ALS Research at Johns Hopkinswww.alscenter.org Located in Baltimore, the Robert Packard Center for ALS Research at Johns Ho=kins is a worldwide collaboration of scientists aimed at developing therapi=s and a cure for amyotrophic lateral sclerosis (ALS), also known as Lou Geh=ig's disease.The Center is the only institution of its kind dedicated solely to the disea=e. Its research is meant to translate rapidly from the lab bench to the cli=ic, largely by eliminating time spent waiting for grants and lowering insti=utional barriers to sharing scientific results.Scientists and clinician members of the Packard Center have moved drugs reli=bly and rapidly from preclinical experiments to human trials. Direct or ind=rect links to international biotech or pharmaceutical companies bring the i=frastructure and experience needed to make promising drugs into therapies.Packard scientists are the first to propose and test a combination approach =o drug therapy, a tactic that has worked for AIDS, cancer and other disease=.ALS is a progressive, disabling neuromuscular disease that causes complete p=ralysis and loss of function - including the ability to eat, speak and brea=he. ALS progresses quickly and is not curable. Most patients die within fiv= years of diagnosis._________________________________________Rebecca BergerResearch Program CoordinatorRobert Packard Center for ALS Research at Johns Hopkins5801 Smith Avenue McAuley Suite 110Baltimore, MD 21209410.735.7678 direct410.735.7680 faxrberger6@jhmi.edu www.alscenter.org www.fiesta5K.org****************************************************************************=***Click on the link below to unsubscribemailto:sympa@lists.johnshopkins.edu?subject=unsubscribe%20alscenter&body=3DPLEASE%20SEND%20THIS%20EMAIL%20OUT%20FOR%20UNSUBSCRIBINGPlease follow the instructions below if the above link does not work:Compose an email to: sympa@lists.johnshopkins.eduWith Subject: unsubscribe alscenterNote: 'unsubscribe alscenter' should be on the SUBJECT line, NOT IN THE MESS=GE BODY.To contact the list owner: mailto:alscenter-request@lists.johnshopkins.edu

2009-02-18T17:06:00.001-05:00

Courage is a special kind of knowledge: the knowledge of how to fear what ought to be feared and how not to fear what ought not to be feared.-- David Ben-Gurion

email me for attachment schemera@yahoo.com

2009-02-17T15:46:00.001-05:00

Dear Brothers and Sisters,

We ( Team IPLEX ) are respectful requesting help from any and all family, friends, & supporters . Please Review attachments I have provided You can... cut and paste certain talking points from the attachments , or you can just cut and paste the entire document -- as is... of course,change the address , name, and salutation's. -- Then utilize the Web link that I have provided, to send your elected representatives (2) senators & ( 1) Congressperson a quick e-mail voicing your support for this (PALS) cause. All we are asking is that they (our elected representatives) look into the shady nature of this matter -- end of story... is that too much to ask?... just looked into the matter, on behalf of terminally ill people? -- Please Take 10-15 minutes and Help, because for PALS, it truly is -- A Race against Time. http://www.visi.com/juan/congress/
http://www.senate.gov/general/contact_information/senators_cfm.cfm

The whole Iplex story significantly highlights the lack of care and concern by institutes such as the FDA. The human factor has been totally removed. The FDA's response has been unbelievable & shameful. They (FDA) are in effect shutting patients out of a drug/therapeutic , that they themselves have already (2006) approved for infants/children. Now, they (FDA) are claiming that it is necessary to shut/turn down all IND applications for IPLEX for reasons that it -- kills people? And get this, all based on unsubstantiated data. A Drug Well-Tolerated by Babies? In plain speak, there is something very fishy going on here. We, (Team IPLEX & supporters) feel that the FDA should be making a more concise data-driven argument, or at least before they start making politically motivated & bogus claims that IPLEX kills people... IPLEX has been supplied to Italian, PALS for over 2 years now, over 100 patients, how many of the 100 have died? Faster by using IPLEX.

Stephen & Barbara Byer parents of Ben Byer (famed, movie producer of the award-winning film "Indestructible" ) explain how their son, Ben, after 14 days of taking the drug (IPLEX ), went from "oatmeal and pudding to a Whoppers ." But was subsequently forced to stop taking the drug (IPLEX) when it was pulled/forced off the market by the terms of a patent lawsuit. We (team IPLEX & supporters) fought too hard in 2008, to get the companies to come to terms, (to make IPLEX available ) to shut up and go away now... .

Bottom line, WHAT DO WE (PALS ) HAVE TO LOSE? -- If our (PALS & supporters) current letter writing campaign, is able to inspire, motivate, and or raise awareness to the plight of PALS. I will definitely feel like --Mission Accomplished!! Even if we aren't able to get the FDA to immediately change, there bogus position, I still feel that victory is just around the corner, this fight will not be over... not by a long shot!! -- unless we give up and accept our fate.

If whatever we're able to accomplish with our current e-mail/ letter writing campaign helps the next generation of PALS dream bigger, work harder and once and for all, finish the work we've started, I'll always believed in my heart that we have made our mark as true champions. But Don't Get Me Wrong, I Want to Win This Thing ASAP... If It's Wrong and or Selfish to Want to Live Longer, well then,... I'm Wrong, & Selfish -- End of Story. PS: We Really Need Your Help

http://www.visi.com/juan/congress/

Thanks ,

Eddie spaghetti) Esparza.

Obama to lift ban on stem cell research soon: aide

2009-02-15T14:32:00.000-05:00

Obama to lift ban on stem cell research soon: aide
Sun Feb 15, 10:01 am ET
WASHINGTON (Reuters) – U.S. President Barack Obama will soon issue an executive order lifting an eight-year ban embryonic stem cell research imposed by his predecessor, President George W. Bush, a senior adviser said on Sunday.
"We're going to be doing something on that soon, I think. The president is considering that right now," Obama adviser David Axelrod said on "Fox News Sunday."
In 2001, Bush limited federal funding for stem cell research only to human embryonic stem cell lines that already existed. It was a gesture to his conservative Christian supporters who regard embryonic stem cell research as destroying potential life, because the cells must be extracted from human embryos.
Embryonic stem cells are the most basic human cells which can develop into any type of cell in the body.
Scientists believe the research could eventually produce cures for a variety of diseases, including Parkinson's disease, diabetes, heart disease and spinal cord injuries.
Obama vowed to reverse Bush's ban during his presidential campaign and in his inaugural address last month promised to return science to its proper place in the United States.
The U.S. Food and Drug Administration last month cleared the way for the first trial to see if human embryonic stem cells could treat people safely.
The trial will try to use stem cells from already existing lines to regrow nerve tissue in patients with crushed spinal cords.
Stem cells are the body's master cells, giving rise to all the tissues, organs and blood. Embryonic stem cells are considered the most powerful kinds of stem cells, as they have the potential to give rise to any type of tissue.
(Reporting by Alan Elsner; Editing by Eric Beech)
Sun Feb 15, 10:01 am ET
WASHINGTON (Reuters) – U.S. President Barack Obama will soon issue an executive order lifting an eight-year ban embryonic stem cell research imposed by his predecessor, President George W. Bush, a senior adviser said on Sunday.
"We're going to be doing something on that soon, I think. The president is considering that right now," Obama adviser David Axelrod said on "Fox News Sunday."
In 2001, Bush limited federal funding for stem cell research only to human embryonic stem cell lines that already existed. It was a gesture to his conservative Christian supporters who regard embryonic stem cell research as destroying potential life, because the cells must be extracted from human embryos.
Embryonic stem cells are the most basic human cells which can develop into any type of cell in the body.
Scientists believe the research could eventually produce cures for a variety of diseases, including Parkinson's disease, diabetes, heart disease and spinal cord injuries.
Obama vowed to reverse Bush's ban during his presidential campaign and in his inaugural address last month promised to return science to its proper place in the United States.
The U.S. Food and Drug Administration last month cleared the way for the first trial to see if human embryonic stem cells could treat people safely.
The trial will try to use stem cells from already existing lines to regrow nerve tissue in patients with crushed spinal cords.
Stem cells are the body's master cells, giving rise to all the tissues, organs and blood. Embryonic stem cells are considered the most powerful kinds of stem cells, as they have the potential to give rise to any type of tissue.
(Reporting by Alan Elsner; Editing by Eric Beech)

20 New Biotech Breakthroughs that Will Change Medicine

2009-02-15T14:26:00.000-05:00

Insmed Sells Follow-on Biologics Platform to Merck & Co., Inc. for Gross Proceeds of $130 Million

2009-02-14T17:37:00.000-05:00

Insmed Sells Follow-on Biologics Platform to Merck & Co., Inc. for Gross Proceeds of $130 Million
--Insmed to Retain IPLEX(TM) portfolio Company to host conference call at 8:30 am ET today
RICHMOND, Va., Feb 12, 2009 /PRNewswire-FirstCall via COMTEX News Network/ -- Insmed Inc. (Nasdaq: INSM), a developer of follow-on biologics and biopharmaceuticals focused on niche markets with unmet medical needs, announced today that it has entered into a definitive agreement with Merck & Co., Inc. whereby Merck, through an affiliate, will purchase all assets related to Insmed's follow-on biologics platform. Under the terms of the agreement, Insmed will receive a total of $130 million for the assets. After fees, taxes and other costs related to the transaction, Insmed expects net proceeds of approximately $123 million as a result of this agreement.
As part of this transaction, Insmed will receive initial payments of up to $10 million for Insmed's lead follow-on-biologic candidates and the remaining balance upon closing of the transaction, which is targeted for March 31, 2009. These initial payments will allow the Company to maintain its normal business operations throughout the closing period without the need for dilutive financing.
Insmed's follow-on biologics assets include INS-19 and INS-20, whose development and commercial rights will now belong to Merck, as well as the Boulder, Colorado-based manufacturing facility. Merck intends to assume the facility's lease and ownership of all the equipment in the building. In addition, upon closing of the transaction, Merck intends to offer positions to employees of the Boulder facility. Insmed will retain its Richmond, VA corporate office, which houses its Clinical, Regulatory, Finance, and Administrative functions, in support of the continuing IPLEX(TM) program.
"We have long maintained that our follow-on biologics assets hold substantial value, and this agreement with Merck, one of the largest pharmaceutical companies in the world, is a testament to that value," said Dr. Geoffrey Allan, President and CEO of Insmed. "We are pleased that over the past two years our team has been successful in developing such a valuable asset, which, as a result of this agreement, will generate a substantial return to the Company."
"This transaction will transform and strengthen our balance sheet in a completely non-dilutive fashion, and provides us with substantial financial flexibility in a market where cash, especially for small biotech companies, is scarce," continued Dr. Allan.
The proceeds from the transaction will be used to support the continued development of IPLEX(TM), and the Company will carefully evaluate other options, which could include the distribution of a portion of the cash to shareholders.
As of December 31, 2008, Insmed had $2.4 million in cash on hand with an ongoing net cash burn of approximately $1.2 million per month. This transaction, in accordance with Virginia corporate law, does not require a shareholder vote, though it is conditional on certain closing requirements, including obtaining necessary consents.
RBC Capital Markets served as exclusive financial advisor to Insmed on the transaction and the review of strategic alternatives, and provided a fairness opinion to the Company's Board of Directors.
Conference Call
Insmed will host a conference call today at 8:30 AM ET in order to further discuss this transaction. To participate in today's 8:30 AM ET live conference call, please dial 800-891-3155 (U.S. callers) or 617-597-5527 (international callers), and provide passcode 54149478. A live webcast of the call will also be available at: http://phx.corporate-ir.net/playerlink.zhtml?c=122332&s=wm&e=2097821
Please allow extra time prior to the webcast to register, download and install any necessary audio software.
The webcast will be archived for 30 days, and a telephone replay of the call will be available for seven days beginning today at 12:30 PM ET at 888- 286-8010 (U.S. callers) or 617-801-6888 (international callers), using passcode 91512481.
About Insmed
Insmed Inc. is a biopharmaceutical company with unique protein process development and manufacturing experience and a proprietary protein platform aimed at niche markets with unmet medical needs. For more information, please visit http://www.insmed.com.
Forward-Looking Statements
This release contains forward-looking statements which are made pursuant to provisions of Section 21E of the Securities Exchange Act of 1934. Investors are cautioned that such statements in this release, including statements relating to planned clinical study design, regulatory and business strategies, plans and objectives of management and growth opportunities for existing or proposed products, constitute forward-looking statements which involve risks and uncertainties that could cause actual results to differ materially from those anticipated by the forward-looking statements. The risks and uncertainties include, without limitation, risks that closing conditions under our agreement with Merck & Co., Inc. may not be met, product candidates may fail in the clinic or may not be successfully marketed or manufactured, we may lack financial resources to complete development of product candidates, the FDA may interpret the results of studies differently than us, competing products may be more successful, demand for new pharmaceutical products may decrease, the biopharmaceutical industry may experience negative market trends, our continuing efforts to develop IPLEX(TM) may be unsuccessful, our common stock could be delisted from the Nasdaq Capital Market and other risks and challenges detailed in our filings with the U.S. Securities and Exchange Commission, including our Annual Report on Form 10-K for the year ended December 31, 2007. Readers are cautioned not to place undue reliance on any forward-looking statements which speak only as of the date of this release. We undertake no obligation to publicly release the results of any revisions to these forward-looking statements that may be made to reflect events or circumstances that occur after the date of this release or to reflect the occurrence of unanticipated events. Investor Relations Contact:
Brian Ritchie - FD
212-850-5683
brian.ritchie@fd.com
Media Contact:
Irma Gomez-Dib - FD
212-850-5761
Irma.gomez-dib@fd.com

There's Definitely Something Wrong with This Picture Fiscal year 2008, Actual (not estimated) NIH (National Institute of Health) allocation of spendi

2009-02-14T16:06:00.002-05:00

There's Definitely Something Wrong with This Picture

Fiscal year 2008, Actual (not estimated) NIH (National Institute of Health) allocation of spending

DISEASE (NIH)-RESEARCH BUDGET
HIV/AIDS research -- 2.9 billion.
Substance abuse research -- 1.76 2 billion.
Drug abuse (NIDA only) -- 1 billion
Obesity research -- 664 million.
Alcohol research -- 452 million.

Depression research -- 402 million

Tobacco research -- 311 million.

Sexually transmitted diseases 245 million.

Food safety research -- 244 million.

Arthritis 232 million.

Sleep research 225 million.

Multiple sclerosis research 169 million

Parkinson's disease research 152 million.

Youth violent research 115 million.

Pneumonia research 93 million.

Infertility research 73 million.

Crohn's disease research, 51 billion.

ALS research 43 million.

Suicide research -- 39 million

Teenage Pregnancy research 21 million
http://report.nih.gov/rcdc/categories/

ALS - Woefully Under-Funded (NIH) Compared to Other Diseases

MS (multiple sclerosis ) seems to offer a reasonable standard of comparison as it has about the roughly similar incidence as ALS (10,000 new cases per year for MS vs.7000 for ALS). There are approximately 400,000 people in the US living with MS vs. about 30,000 with ALS. Why the difference in prevalence if the diseases have a somewhat similar incidence rate? The major factor is lethality. MS patients live a normal life span while, ALS patients have a much shorter life span. If ALS were not such an effective killer, its prevalence rate would obviously be much higher. It seems reasonable that the two diseases should have nearly equal public funding, since they both occur with the similar frequency, but this is not the case. NIH budgets 169 million on MS research but $43 million on ALS? . It is immoral to base funding on prevalence in the context of similar incidence rates, thereby penalizing ALS patients for the lethality of their disease

The next disease for comparison is Huntington’s disease, another disorder caused by degeneration of brain cells, i.e. neurons, in certain areas of the brain. Its incidence and prevalence rate are much lower than ALS and patients usually live 10-25 years after diagnosis. Yet, this disease has an NIH budget of $51 million versus, $43 million for ALS.
For the, 3rd and final comparison, HIV/AIDS. This illness has a large incidence (85,000) and prevalence (1,100,000) The number of patients who die from this disease each year is only a bit more than twice the number of ALS deaths, yet HIV/AIDS has a research budget of nearly $3 BILLION! vs. $41m for ALS-?

CONCLUSION I: Given its incidence rates and high lethality, ALS is woefully under-funded compared to other diseases or and this may explain, at least in part, why so little has been learned about this disease since Lou Gehrig’s death 65 years ago and why there have been no significant advances in treatment. It does not get the recognition it deserves because its high lethality severely limits the number of Americans who are living with the disease at any one point in time.

RECOMMENDATION I: The NIH budget for ALS research should be immediately increased to $110m or the same amount as MS.

The fact that the low prevalence rate of ALS, is directly due to its high lethality, therefore does not provide any incentive for major pharmaceutical companies to search for a cure. The only drug for ALS, Rilutek, about 15 years old now, extends life only 2-3 months, is used by many patients at high cost, yet the maker of the drug, Aventis, claims that it loses substantial money on Rilutek. MS, in comparison, with its 400,000 prevalence in the US alone, presents significant incentive for drug companies to invest in research. In fact, there are 5 meds that have proven very beneficial and sales are big. Avonex, for example, had close to $1b in sales in the last year. Another drug, Copaxone, may have the greatest potential to alter the course of MS and sales, once the issue of side effects is resolved, should be huge. If we accept the view that it costs the companies $800 m over 10 years to develop and bring to market a successful drug, then about $4 billion has been spent to produce these disease mitigating medications, or $400 m per year, not including current research.
The FDA offers an orphan disease program to provide incentives to companies to invest in research of diseases having a prevalence of fewer than 200,000. The program provides tax incentives and other advantages for only small grants; unfortunately the incentives offered are too small to interest large companies with all their research potential.

Conclusion II: Because of ALS’s high lethality, prevalence will always be low. As a consequence, big pharma will not have the incentive to invest in ALS research and the high tech power of drug companies won’t be harnessed to find new therapeutic agents in the fight against ALS.

Recommendation II: Federal health agencies must provide the incentive to big pharma to invest in ALS research. The FDA Orphan Disease Program must be substantially increased to accomplish this. $80m per year in incentives must be allotted to get major companies interested in working on this research. Funding could include a combination of grants, tax credits and exclusive marketing rights, etc. The grants could be reduced as companies come closer to marketing the new agents.

Under-funding of ALS will not be righted without our ( PALS ) active intervention. I am asking for your help on behalf of both the 35,000+ Americans living with the disease today and the estimated 70,000 that ill die every 10 years , and the 7000 that will die just this year, until a cure is found. ALS has been neglected far too long.
Sincerely,
Edward W. Esparza
(pals since 2005)
"For PALS, the cost of waiting/doing nothing, is simply too high."

2009-02-12T17:07:00.000-05:00

Change and growth take place when a person has risked himself and dares to become involved with experimenting with his own life.--Herbert Otto

2009-02-11T21:29:00.001-05:00

Dear Brothers and Sisters,

We ( Team IPLEX ) are respectful requesting help from any and all family, friends, & supporters . Please Review attachments I have provided

You can... cut and paste certain talking points from the attachments , or you can just cut and paste the entire document -- as is... of course,change the address , name, and salutation's. -- Then utilize the Web link that I have provided, to send your elected representatives (2) senators & ( 1) Congressperson a quick e-mail voicing your support for this (PALS) cause. All we are asking is that they elected representatives) looked into the shady nature of this matter -- end of story... is that too much to ask?... just looked into the matter, on behalf of terminally ill people? --

Please Take 10-15 minutes and Help, because for PALS, it truly is -- A Race against Time.

http://www.visi.com/juan/congress/
http://www.senate.gov/general/contact_information/senators_cfm.cfm

The whole Iplex story significantly highlights the lack of care and concern by institutes such as the FDA. The human factor has been totally removed. The FDA's response has been unbelievable & shameful. They (FDA) are in effect shutting patients out of a drug/therapeutic , that they themselves have already (2006) approved for infants/children. Now, they (FDA) are claiming that it is necessary to shut/turn down all IND applications for IPLEX for reasons that it -- kills people? And get this, all based on unsubstantiated data. A Drug Well-Tolerated by Babies?

In plain speak, there is something very fishy going on here. The company (INSMED) that developed & owns the property rights, refuses to release any data, from a 100 patient, 2 year trial/Italian program. We, (team IPLEX & supporters) feel that the FDA should be forcing this information out, in order to make a more concise data-driven argument, or at least before they start making claims that IPLEX kills people... Insmed has been supplying Iplex to Italian, PALS for over 2 years now to over 100 patients, respectively review this is Insmed has issued many, a statement that IPLEX is safe and well tolerated. IPLEX is already an FDA approved drug and has already been proven safe for an indication for infants/kids dwarfism ).

Stephen & Barbara Byer parents of Ben Byer (famed, movie producer of the award-winning film "Indestructible" ) explain how their son, Ben, after 14 days of taking the drug (IPLEX ), went from "oatmeal and pudding to a Whoppers ." But was subsequently forced to stop taking the drug (IPLEX) when it was pulled/forced off the market by the terms of a patent lawsuit. We (team IPLEX & supporters) fought too hard in 2008, to get the companies to come to terms, (to make IPLEX available ) to shut up now... .
Bottom line, WHAT DO WE (PALS ) HAVE TO LOSE? --

If our (PALS & supporters) current letter writing campaign, is able to inspire, motivate, and or raise awareness to the plight of PALS. I will definitely feel like --Mission Accomplished!! Even if we are able to get the FDA to immediately change, there bogus position, I will still feel that victory is just around the corner, this fight will not be over... not by a long shot!!

If whatever we're able to accomplish with our current e-mail/ letter writing campaign helps the next generation of PALS dream bigger, work harder and once and for all, finish the work we've started, I'll always believed in my heart that we have made our mark as true champions.

But Don't Get Me Wrong, I Want to Win This Thing ASAP... If It's Wrong and or Selfish to Want to Live Longer, well then,... I'm Wrong, & Selfish -- End of Story.

PS: We Really Need Your Help
EDDIE

2009-02-11T14:45:00.000-05:00

No person was ever honored for what he received. Honor has been the reward for what he gave.--Calvin Coolidge

Scientists Heartened at Prospect of End to Stem Cell Ban

2009-02-09T19:05:00.000-05:00

Scientists Heartened at Prospect of End to Stem Cell Ban
By Amanda GardnerHealthDay Reporter
MONDAY, Feb. 9 (HealthDay News) -- Researchers are rejoicing over President Barack Obama's anticipated lifting of the eight-year ban on embryonic stem cell research imposed by his predecessor, President George W. Bush.
The anticipation moved one step closer to reality Thursday, with media reports that Obama gave House Democrats at a closed-door Virginia retreat a "guarantee" that he would sign an executive order overturning Bush's policy.
"It's going to remove an embarrassment for American science," said Dr. Darwin Prockop, director of the Texas A&M Health Science Center College of Medicine Institute for Regenerative Medicine at Scott & White Hospital in Temple. "It's a statement that we're going to again believe in science."
Yet those same experts are aware that the sobering state of the economy could impose its own restrictions on this type of research.
"This clearly is a very important part of our medical future," said Paul Sanberg, distinguished professor of neurosurgery and director of the University of South Florida Center of Excellence for Aging and Brain Repair in Tampa. "[But] to clear the path for this without giving additional money to the National Institutes of Health will be disappointing. I hope the stimulus package also includes an increase in embryonic stem cell funding."
Sanberg also expressed concern that any monies redirected to stem cell research could divert funds from other critical avenues of research. "If it's a normal competitive process, it will take money away from other programs," he said.
Stem cell research received a big boost in January, when the U.S. Food and Drug Administration approved the first-ever human trial using embryonic stem cells as a medical treatment.
Geron Corp., a California-based biotech company, was given the OK to implant embryonic stem cells in eight to 10 paraplegic patients who can use their arms but can't walk.
In 2001, then-president Bush limited federal funding for stem cell research only to human embryonic stem cell lines that already existed.
The decision prompted some scientists to worry that the United States would fall behind other countries in the drive to unlock the potential of stem cell research.
Embryonic stem cells are the most basic human cells, believed to be capable of growing into any type of cell in the body. Working as a sort of repair system for the body, they can theoretically divide without limit to replenish other cells. The scientific hope is that stem cells may, at some point in the future, become capable of treating a variety of diseases and conditions, such as Parkinson's disease, diabetes, heart disease and spinal cord injuries, according to the U.S. National Institutes of Health.
National polls continue to find that the majority of Americans favors embryonic stem cell research, although some surveys have found that that support has declined somewhat in recent years.
Many people object to the use of embryonic stem cells, contending that the research requires the destruction of potential life, because the cells must be extracted from human embryos.
The stem cells being used in the recently approved Geron trial were obtained from one of the Bush administration's approved stem cell lines. And no federal funds were used in the development of this treatment.
Since the restrictions on embryonic stem cell research took effect, many research institutions have redirected their focus to other types of stem cells. Prockop's institution, for instance, deals only with adult stem cells.
Adult stem cells can give rise to all the specialized types of cells found in tissue from which they originated, such as skin. But, scientists don't agree on whether adult stem cells may yield cell types other than those of the tissue from which they originate, according to the National Institutes of Health.
Prockop said embryonic stem cells "are mainly of interest as a research tool and a biological experimental system. Their use in patients in spite of that recent approval for Geron is really very questionable because of the potential for tumors."
Still, the anticipated lessening of restrictions by the Obama administration may help funnel more private money into stem cell research, the experts said.
"This should give more general acceptance to stem cell research, because now, there won't be this stigma associated with it as much," Sanberg said.
And, perhaps, a new federal policy would spur organizations such as the American Heart Association -- which currently does not fund research involving human embryonic stem cells or stem cells derived from fetal tissue -- to channel funds into this line of research, Sanberg added. (The heart association said it "recognizes the value of all types of stem cell research and supports federal funding of this research.")
Still, Sanberg pointed out, some ethical issues surrounding stem cell research and its application will remain.
For instance, he said, "There still needs to be some oversight on the uses of stem cells and cloning."
More information
To learn more about stem cells, visit the U.S. National Institutes of Health.

2009-02-09T17:14:00.002-05:00

EMAIL ME FOR THE LETTER TO SEND TO THE SENATOR OF YOUR CHOICE
SCHEMERA@YAHOO.COM

from eddie

2009-02-08T14:04:00.000-05:00

February 6, 2009
Dear Friends,
Below is a recap of recent activity regarding our mutual quest for securing Iplex and a suggested course of action. Some of what is written below is already known to many of you, but others may not be as up-to-date, so please excuse any repetition.

1. Between January 16-30, 2009 the initial (8) Iplex Investigational New Drug applications were summarily rejected by Dr. Russell Katz, FDA Director, Neurological Products Division. The reasons cited were “reports of increased mortality” about another drug, Myotrophin, and concern about ALS patients networking and their resultant determination to seek and use Iplex prior to a clinical test study. The reasons cited were judgmental, fallacious, and destructive to the ALS community for obvious reasons.
2. In order to provide legal foundation for several additional patient families and to further test the FDA waters, five additional IND applications, with Institutional Review Board support, were submitted to the FDA on February 3, 2009 by ALS WORLDWIDE. These IND applications incorporated logic and fact to counter the FDA reasons for rejecting the original group (submitted by individual physicians directly) but we nonetheless expect these new applications will also be denied.
3. We, and others on Team Iplex, have had extensive conversations with all interested parties including patient families, FDA, legal advisors, involved and uninvolved pharmaceutical companies, judiciary and elected officials (both state and federal levels). The consensus is that the quickest, & best way of successfully countering current FDA posture is to apply ongoing political pressure on Dr. Katz and his superiors by both US Senators and Representatives and their state-level counterparts.
4. Immediately following this email will be another separate email with attachments. This should be sent to each of your (2) US Senators and your (1) US Representative. It should be directed to them at both their Washington and in-state offices at their respective email addresses. Some of you may not know who your elected officials are and, if so, I have provided you with the Web link...http://www.visi.com/juan/congress/ Now at this point, all you have to do is double-click the Web link, find your particular state, then click your state and follow the prompts. The attachment/cover letter has been written to apply “across the board” or, as is said, “one size fits all” or if you choose as a template the. In addition to your federal officials, we believe the email should also be directed to your state’s Governor and the elected state officials from your area.
5. Brothers and sisters, please... be aware that this effort will not work , if any of you reading this assumes that it will suffice if others , do the work. This will only work... if you assume that your emails are the only ones going out. Please, I beg you... spend part of this weekend preparing the emails to go out on -- Monday morning -- February 9. They won’t be in their offices on the weekend and Monday is always the best time for new issues to be presented. When sending the email and any attachments, mark it “Urgent” and follow it with the same email on Wednesday, February 11 and Friday, February 13 with the following sentence typed at the top of Monday’s email: Senator (or Governor, representative or otherwise) – Did you read my prior email and will you act on behalf of our family and every other ALS family by forcing the FDA to allow Iplex for patients dieing ALS? Please tell me immediately you are doing to stop this travesty. I am your constituent, supporter and part of an ALS family.
6. After locating your elected officials contact information, also find and record their office telephone numbers, both in Washington DC (for the federal officials) and within your state for them and the state elected officials. We (PALS & supporters) will begin a coordinated (follow-up) calling, beginning on Monday, February 16, followed up with a repeated coordinated calling on Wednesday, February 18 & Friday, February 20, if you have not heard from them with a responsible answer by email, US mail or telephone. A responsible answer does not include “Thank you for your inquiry. We always listen to our constituents” or anything like that. A responsible answer will tell you exactly what they are doing, when and with whom. In your telephone efforts beginning on Monday, February 16, first ask for the elected official and when you’re unable to get that person on the phone, then ask for the Administrator of Medical Affairs or the generalist who fits that category. Don’t get off the phone until someone has listened, you have an individual’s email to whom you can re-direct the original email and attachments, and you feel they are serious about doing something.
7. We are not saying this is the only way, what we are saying is that we think it is the best way to get Iplex for all of our loved ones. You may wish to slightly modify the cover email but the attachments are in PDF files so they cannot be changed. The letter has been written to appeal to a busy, and undoubtedly preoccupied, elected official who will then hopefully forward it and the detailed attachments to the staff person specializing in medical affairs (for whom the attachments will be meaningful). The cover email and attachments are also intended to be courteous, accurate, honest, legible and personalized enough to instigate quick and responsible action by the elected officials. The email and its attachments are not intended to be offensive to the FDA or Dr. Katz. Rather, it is to get the FDA to agree that Iplex does not, and should not, require formal clinical test studies prior to its usage by the ALS community. Further, the letter and attachments are intended to clearly refute the reasons stated by Dr. Katz and FDA for declining Iplex usage in the sample denial letter included.
8. Some of you have asked whether this type of communication should be posted on the various forums. I personally think that doing so may diffuse our collective efforts because none of you or me has the time to argue the merits or demerits of Iplex and, even more importantly, free choice by a physically devastated patient community if we are using our time more effectively by organizing political pressure. Understand there is nothing secretive or contradictory to anyone else’s rights by the suggested application of political pressure for a just cause, but I am certain that most of you reading this remember the periodic condemnation of free choice decision-making by Drs Bedlack, Silani and Cudchavitz in the Journal of ALS, Steve Gibson of ALSA on his blog site and certain always-negative posters on various forums. I suggest instead that your and our efforts be confined, or at least directed primarily, toward the application of political pressure on the FDA.
9. Finally, our immediate goal is to both secure the annulment of the FDA denial of IND applications submitted and equally as importantly, to allow Iplex to be used by the ALS community from this point forward through simple cross-label prescription by licensed physicians. This was the case when Iplex was initially used by those with ALS is early 2007 with unilaterally excellent results. We want the same appropriate plan implemented now, as it was then, in that the only differences between February 2007 and February 2009 are a) the settlement of the legal dispute between Genentech, Tercica and Insmed, and, b) the loss of thousands of ALS lives and the dramatic further decline of those still living whose death or decline might have been dramatically lessened by their use of Iplex.
10. Now look for the next email which needs to be carefully read, then filled in to the political figures as discussed above and emailed this upcoming Monday morning, February 9 as outlined. Please help..., for people with ALS, it really is a race against time

Thanks ,
Eddie spaghetti) Esparza.
Team IPLEX Capt.-, PALS since 2005

2009-02-08T13:37:00.000-05:00

A man walked into a bar and sat down, and ordered a beer. As he sipped the beer, he heard a soothing voice say "nice tie!" Looking around, he noticed that the bar was empty except for himself and the bartender at the end of the bar. A few sips later the voice said "beautiful shirt".At this, the man called the bartender over. "Hey...I must be ... Read Morelosing my mind," he told the bartender. "I keep hearing these voices saying nice things, and there's not a soul in here but us.""It's the peanuts," answered the bartender."Say what?""You heard me," said the barkeep. "It's the peanuts ... they're complimentary."

Selecting Potent Immune Suppressive Mesenchymal Stem Cells

2009-02-03T17:57:00.000-05:00

Selecting Potent Immune Suppressive Mesenchymal Stem Cells
Paris, France -
It is widely known that mesenchymal stem cells, whether derived from the bone marrow, cord blood, or menstrual blood all possess some degree of immune suppressive activity. This is one of the reasons why mesenchymal stem cells are being used in clinical trials in a "universal donor" manner, and why the first marketing approval more than likely will be the BLA for Osiris using mesenchymal stem cells to treat the inflammatory condition graft versus host disease.
An important question is what if more potent immune suppressive mesenchymal stem cells can be collected from a population of mesenchymal stem cells by selecting for a specific surface marker? For example, selection of adipose derived mesenchymal stem cells for CD9 is associated with increased angiogenic activity.
In a recent paper (Nasef et al. Selected Stro-1-enriched bone marrow stromal cells display a major suppressive effect on lymphocyte proliferation. Int J Lab Hematol 2009 Feb;31(1):9- 19) selection of mesenchymal stem cells with increased immune suppressive activity was performed based on sorting of cells for higher expression of the molecule STRO-1.
The authors purified bone marrow derived mesenchymal stem cells into Stro-1 high expressing cells and Stro-1 low expressing cells and found that Stro-1 expression was associated with:
1. Increased suppressive activity on ongoing mixed lymphocyte reaction.
2. Increased suppression of T cell proliferation in a contact dependent manner.
3. Upregulated expression of IL-8, LIF, IDO, HLA-G, and VCAM1.
4. Expression of TGF-beta and IL-10 was induced only after contact with T cells.
These data suggest two things: firstly it may be beneficial to select for subsets within subsets of mesenchymal stem cells when trying to treat a specific condition; and secondly, when doing in vitro experiments looking at gene products made by mesenchymal stem cells, it is important to culture the mesenchymal stem cells with other cells, since the mesenchymal stem cell in its basal state may not be producing cytokines you are looking for.

£120,000 plea for laser to fight fatal brain disease

2009-02-03T15:08:00.001-05:00

£120,000 plea for laser to fight fatal brain disease

Published Date: 30 January 2009
THE Royal Hallamshire Hospital in Sheffield is to buy cutting edge laser equipment, which scientists hope may one day lead to a cure for motor neurone disease.
The £120,000 needed to bring the laser to the neurosciences department at the Hallamshire will be raised in partnership with hospital charity Neurocare. The Veritas Microdissection System will help researchers at the hospital's world- leading mot
or neurone disease (MND) research unit to analyse and compare cells from patients more quickly and accurately.A laser will harvest single cells or tens of thousands of cells in seconds. By monitoring cell behaviour and trialling the effects of drugs across different tissue samples, researchers hope to one day find the causes of MND and a cure.Paul Heath, senior scientific officer at the Royal Hallamshire Hospital, is one of the specialists who will be using the equipment.He said: "MND is a truly horrible, fatal disease for which there is no cure. Yet this equipment would enhance Sheffield's reputation as one of the world's leading centres for research, which is a real source for regional pride. The equipment has the potential to offer real hope to all sufferers."MND is a fatal group of diseases which cause the death of motor neurones – the nerve cells through which the brain connects with the muscles to control movement.Degeneration of the motor neurones leads to weakness and wasting of muscles, causing increasing loss of mobility in the limbs, and difficulties with speech, swallowing and breathing.Barrie Butterton, aged 62, from Gainsborough, Lincolnshire was diagnosed with motor neurone disease four years ago. After diagnosis, he was referred to specialists at the Hallamshire. Barrie said: "Motor neurone disease has wrecked my life. I had worked in the construction and civil engineering industry all my working life and was looking forward to retiring and enjoying life with my family."Now I don't just suffer from MND and the effects of muscle wastage, I suffer from all the things connected to it. Symptoms like deep depression, severe cramp, excess saliva, poor circulation and terrible sleeping patterns are a part of my every day life."I heard about Neurocare from specialist nurse Hanna Nixon and Professor Pam Shaw in Sheffield, who is one of the world's leading MND experts. She told me about Neurocare and the project they were funding and now I want to do anything I can to support the charity and the doctors and scientists who are working so hard to rid the world of this devastating disease."To raise money or to donate call Neurocare on 0114 2676464 or email: appeals@neurocare.org.uk

2009-02-02T16:12:00.001-05:00

2009 Feb;15(2):RA23- 31.
Related Articles
From the basics to application of cell therapy, a steppingstone to the conquest of neurodegeneration: A meeting report.

Park DH, Eve DJ, Borlongan CV, Klasko SK, Cruz LE, Sanberg PR.Center of Excellence for Aging and Brain Repair, Dept. Neurosurgery, Tampa , FL , U.S.A.The annual meeting of the American Society for Neural Therapy and Repair (ASNTR) showcases the latest research trends in neurodegenerative disease and the related medical regenerative science. The 2008 ASNTR meeting covered a variety of different topics ranging from basic research to exploration of currently unknown pathogenesis and mechanisms for specific neurodegenerative disease such as Parkinson's disease, Alzheimer's disease, or stroke. This included studies to characterize stem cells, such as neural stem cells, embryonic stem cells, bone marrow mesenchymal stem cells, and human umbilical cord blood cells, for transplantation and the conditions necessary to maximize the efficacy of endogenous and exogenous stem cells, such as isolation, purification, differentiation, and migration. Moreover, a number of studies looked at methods for more advanced application of transplantation of cells or specific factors, through tissue engineering or manipulation beyond simple injection. Finally, well-known or previously un-known dietary supplementation or pharmacological materials that can affect the nervous system positively or negatively, were also important topics.
PMID: 19179980 [PubMed - in process]
http://www.ncbi. nlm.nih.gov/ pubmed/19179980? dopt=Abstract

International Stem Cell Corporation Begins Pre-Clinical Trials On Human Corneal Epithelial Cells

2009-02-02T16:09:00.000-05:00

International Stem Cell Corporation Begins Pre-Clinical Trials On Human Corneal Epithelial Cells
Article Date: 01 Feb 2009 - 0:00 PSTInternational Stem Cell Corporation (OTCBB:ISCO) , the first company to perfect a method of creating human "parthenogenetic" stem cells from unfertilized eggs, is planning pre-clinical trials aimed at applying its laboratory-grown human corneal epithelial cells to improve photorefractive keratectomy (PRK), a form of corrective laser eye surgery. These trials are the first step toward Food and Drug Administration (FDA) clinical trials to test the efficacy of using ISCO cells to improve healing after corneal surgery, and are part of the company's efforts to increase the clinical utility of its discoveries in culturing corneal cells and tissues. This work is being done in collaboration with Dr. Paul H. Chen, M.D., who has developed the cell transfer technology. Dr. Chen is an eye surgeon at North County Laser Eye Associates, and he is on staff at Scripps Memorial La Jolla and Scripps Encinitas Hospitals . "This collaboration is an excellent opportunity for ISCO to use its cell culture and manufacturing expertise to create therapeutic human cells that can enter the market relatively quickly and improve patient's quality of life," said Jeffrey Janus, ISCO's president. "We are fortunate to be working with Dr. Chen on this exciting project." About International Stem Cell Corporation (ISCO.OB) International Stem Cell Corporation is a California biotechnology company focused on developing therapeutic and research products. ISCO's technology, Parthenogenesis, results in the creation of pluripotent human stem cell lines from unfertilized human eggs. ISCO scientists have created the first Parthenogenetic homozygous stem cell line (phSC-Hhom-4) that can be a source of therapeutic cells that will minimize immune rejection after transplantation into hundreds of millions of individuals of differing sexes, ages and racial groups. These advancements offer the potential to create the first true "Stem Cell Bank" and address ethical issues by eliminating the need to use or destroy fertilized embryos. ISCO also produces and markets specialized cells and growth media worldwide for therapeutic research through its subsidiary Lifeline Cell Technology. For more information, visit the ISCO website at: http://www.internat ionalstemcell. com. Forward-Looking StatementsStatements pertaining to anticipated future financial and/or operating results, future growth in research, technology, clinical development and potential joint venture and other opportunities for the company and its subsidiary, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company's business, particularly those mentioned in the cautionary statements found in the company's Securities and Exchange Commission filings. The company disclaims any intent or obligation to update these forward-looking statements.

i got this email from my buddy eddie

2009-01-31T19:37:00.001-05:00

The path to a cure; ALS specialist Dr. Robert H. Brown Jr. joins elite team at UMass.
Byline: Elizabeth Cooney When Dr. Robert H. Brown Jr. says it, it sounds so simple. "These diseases will yield," he said. "We just can't take no for an answer." The new head of neurology at University of Massachusetts Medical School UMMS is ranked fourth in primary care education among the nation’s 125 medical schools in the 2006 U.S.News & World Report annual guide, “America’s Best Graduate Schools”. UMMS is also a major center for research. and UMass Memorial Medical Center is single-minded in his determination to find better treatments for such devastatingdev·as·tate tr.v. dev·as·tat·ed, dev·as·tat·ing, dev·as·tates1. To lay waste; destroy.2. To overwhelm; confound; stun: was devastated by the rude remark. ..... Click the link for more information. neuromuscular diseases as amyotrophic lateral sclerosis amyotrophic lateral sclerosis (ALS) (ā'mīətrōf`ik, sklĭrō`sĭs) or motor neuron disease, , better known as Lou Gehrig's disease Lou Geh·rig's diseasen.See amyotrophic lateral sclerosis. . In October he left Massachusetts General HospitalMassachusetts General Hospital Health care The major teaching hospital for Harvard Medical School, widely regarded as one of the best health care centers in the world ..... Click the link for more information. and Harvard Medical SchoolHarvard Medical School (HMS) is one of the graduate schools of Harvard University. It is a prestigious American medical school located in the Longwood Medical Area of the Mission Hill neighborhood of Boston, Massachusetts. ..... Click the link for more information. to find a faster pathway to treatments at the state's medical school The lure was RNA interferenceRNA interferencen.A process in which the introduction of double-stranded RNA into a cell inhibits the expression of genes. ..... Click the link for more information., or RNAi for short, the mechanism that can silence genes. UMass professor and Nobel laureate Craig C. Mello was one of two scientists to discover the biological process in 1998. Since then, the medical school has added to its cadre of RNAi researchers by recruiting distinguished scientists such as Dr. Brown, who was already collaborating with UMass experts. The school's proposed Advanced Therapeutics Cluster will include a center devoted to RNAi, part of the state's $1 billion life sciences initiative. RNAi holds promise for treating diseases by shutting off defective genes that make harmful proteins. In ALSAls (äls), Ger. Alsen, island, 121 sq mi (313 sq km), Sønderjylland co., S Denmark, in the Lille Bælt, separated from the mainland by the narrow Alensund. ..... Click the link for more information., mutant genes produce proteins that kill nerve cells needed to move muscles. Most people die within three to four years after first developing muscle weakness and then paralysis. "I spent 30 years at the Mass. General Hospital, which is a wonderful institution," Dr. Brown, 61, said last month in his UMass Medical School office, already adorned by images of Lou Gehrig and photos of Red Sox players at ALS fundraisers. "But it seemed to me that I would probably make more progress here by joining forces with the people who made the basic discoveries in this (RNAi) technology. I just thought the route would be more direct here." Dr. Brown, both a research scientists and a neurologist who sees patients, began studying ALS in 1979. It was through his clinical work that he began to investigate the genetic underpinnings of ALS. One of his patients, Oscar Horvitz, was the father of H. Robert Horvitz H. Robert Horvitz (born May 8, 1947) is an American biologist best known for his research on the nematode worm Caenorhabditis elegans. He is currently at the Massachusetts Institute of Technology where he is Professor of Biology and a member of the McGovern Institute for , a scientist at MIT MIT - Massachusetts Institute of Technology who later won a Nobel Prize Nobel Prize, award given for outstanding achievement in physics, chemistry, physiology or medicine, peace, or literature. The awards were established by the will of Alfred Nobel, who left a fund to provide annual prizes in the five areas listed above. for his work on the genetic regulation of organ development and programmed cell death pro·grammed cell deathn.See apoptosis.
programmed cell deathproposed system of cell death, often including poly(ADP)-ribosylation, ensures that a cell will not survive if it is so badly damaged that its recovery would harm the . After Dr. Brown had the sad task of confirming a diagnosis of ALS in his patient, he began discussing research with his patient's son. At the time, the technology to find genes and map them was just entering laboratories, where scientists were trying to divine what they did. "Bob and I started talking about this horrific disease, and what we might do about it," Dr. Horvitz said in an e-mail interview about Dr. Brown. "At that point we embarked upon collaborative studies.... We agreed that the emerging technologies of human genetics Human geneticsA discipline concerned with genetically determined resemblances and differences among human beings. Technological advances in the visualization of human chromosomes have shown that abnormalities of chromosome number or structure are surprisingly offered new approaches, and it was this work that led to the identification of the first ALS gene, known as SOD1. Since then Bob has continued to pioneer the application of ever-more sophisticated genetic methods to ALS research." SOD1 was identified in 1993, breaking open the world of ALS research. The gene produces superoxide dismutase, an enzyme that protects cells from damage wrought by toxic free-radical molecules. Defective forms of the gene were found in families who had ALS. Most cases of ALS are sporadic, meaning they strike people who have no family history of the disorder, but finding the gene implicated im·pli·cate tr.v. im·pli·cat·ed, im·pli·cat·ing, im·pli·cates1. To involve or connect intimately or incriminatingly: evidence that implicates others in the plot.2. in the inherited forms offers a keyhole through which to learn about all forms of the disease. The scientific paper reporting the breakthrough was dedicated to Oscar Horvitz. Once the gene was identified, the next step was to study it in specially bred laboratory mice and then devise ways to fix it. "It's been a long slog. It became painfully clear that the treatments would be very difficult to find. This was a very hard nut to crack, and there were no easy fixes," Dr. Brown said. "Because of the frustration of working for 10 years with conventional therapies without success, it seemed to me we needed to look for something new and to go as far upstream as one could to shut off the disease process right at the start." That's when Dr. Brown and his colleagues began to get very interested in gene silencing through RNAi. "There are several lines of study suggesting that the technology actually works in mice," he said. "The question is how best to get it in people." One way would be to inject active RNAi molecules into the spinal fluid of patients that would then thwart the mutant gene, but there are doubts about how effective that would be, he said. Another approach is more complicated: using gene therapy to deliver into the spinal cord a gene that uses the machinery of the cell to continuously make gene-silencing RNA RNA: see nucleic acid.
RNA in full ribonucleic acidOne of the two main types of nucleic acid (the other being DNA), which functions in cellular protein synthesis in all living cells and replaces DNA as the carrier of genetic . Both methods pose challenges. Of the two, Dr. Brown thinks the gene therapy-RNAi combination holds the most promise for achieving a sustained effect. It's no coincidence that the planned UMass therapeutics cluster includes a gene therapy center as well as an RNAi center. (The third is devoted to stem cell science.) "The new dean is a gene therapist, so suddenly it's like a perfect storm," Dr. Brown said. "This isn't just a laboratory exercise. He's extremely serious about seeing this happen and implementing programs. That feeds in perfectly to the idea of a more effective way to deliver RNAi therapy." Dr. Brown's sense of urgency comes from the patients he hopes to help. They don't have a lot of time. "Bob is somebody who is a very caring and compassionate physician," said Dr. Terence R. Flotte, dean of the medical school since April 2007. "His motivation is heavily centered around coming up with solutions for the patients who are so tragically affected. People say stuff like that a lot, but you can see in his eyes - that's what really matters to him." Dr. Brown's optimism about finding a cure was clear last month, two days after the presidential election. "What did someone say the night before last?" he asked. "`Yes, we can.'" CUTLINE

Stem Cell Research: The Quest Resumes

2009-01-30T16:15:00.000-05:00

Stem Cell Research: The Quest Resumes
Fri Jan 30, 11:30 am ET
Scientific inspiration can come from anywhere - a person, an event, even an experiment gone awry. But perhaps nothing can drive innovation more powerfully than the passion born of tragedy. Or, in Douglas Melton's case, near tragedy. The co-director of the Harvard Stem Cell Institute (HSCI) is one of the leading figures in the search for cures for presently incurable diseases, and his breakthrough work is challenging many long-held beliefs about the ways biology and human development work.
But it was a very personal experience that brought Melton to stem cells, one that 17 years later he still finds difficult to discuss. When his son Sam was 6 months old, he became ill with what his parents thought was a cold. He woke up with projectile vomiting and before long began taking short, shallow breaths. After several hours, he started to turn gray, and Melton and his wife Gail brought the baby to the emergency room. For the rest of that afternoon, doctors performed test after test, trying to figure out what was wrong. "It was a horrific day," says Melton. (See the top 10 medical breakthroughs of 2008.)
It was not until that evening that a nurse thought to dip a testing strip into Sam's urine and they finally got a diagnosis. The boy's body was flooded with sugar; he had Type 1 diabetes. Then, as now, the disease had no cure, and patients like Sam need to perform for themselves the duties their pancreas cannot - keeping track of how much glucose they consume and relying on an insulin pump to break down the sugars when their levels climb too high. The diagnosis changed not only Sam's life but the lives of his parents and older sister Emma as well. Throughout Sam's childhood, Gail would wake every few hours during the night to check his blood sugar and feed him sugar if his concentration fell too low or give him insulin if it was too high. "I thought, This is no way to live," says Melton. "I decided I was not just going to sit around. I decided I was going to do something."
Trained as a molecular biologist in amphibian development, Melton began the work he pursues today: trying to find a way to make insulin-producing cells by using stem cells. "It was a courageous thing to do because he was at the pinnacle of his career," says Gail. "He brought home textbooks on the pancreas to figure it all out." Nearly two decades later, Melton is convinced that stem cells will be a critical part of new therapies that will treat and maybe cure not only diabetes but also other diseases for which there are no answers today.
Melton's confidence is testament to the extraordinary advances in stem-cell science, some of which have brought the promise of breakthrough therapies for conditions like diabetes, Parkinson's and heart disease closer than ever before. The cells filling petri dishes in freezers and incubators in Melton's lab and others around the world are so vastly different - in provenance, programming and potential - from the stem cells of just two years ago that even the scientists leading this biological revolution marvel at the pace at which they are learning, and in some cases relearning, rules of development. Until recently, the field has revolved around either embryonic stem cells - a remarkably plastic class of cells extracted from an embryo that could turn into any of the body's 200 tissue types - or their more restricted adult cousins, cells taken from mature organs or skin that were limited to becoming only specific types of tissue. On Jan. 23, after nearly a decade of preparation, the Food and Drug Administration approved the first trial of an embryonic- stem-cell therapy for a handful of patients paralyzed by spinal-cord injuries.
But today the field encompasses far more than just embryonic and adult stem cells; it has expanded into the broader field of regenerative medicine, and Melton's lab at Harvard is at the vanguard, bringing the newest type of stem cells, which do not rely on embryos at all, closer to the clinic, where patients will actually benefit. Last summer, Melton stunned the scientific community with yet another twist, finding a way to generate new populations of cells by reprogramming one type of fully mature cell so it simply became another, bypassing stem cells altogether. "If I were in high school, I can't imagine anything more interesting than stem cells," says Melton. "This is so cool. It's so amazing that cells in the body have this potential that we can now unlock by asking question after question."
A Battle Joined That hidden power in each of us did not become obvious until 1963, when Canadian researchers Ernest McCulloch and James Till first proved the existence of stem cells, in the blood. These cells possess the ability to divide and create progeny - some of which will eventually expire, others that are self-renewing. The pair irradiated mice, destroying their immune cells. They then injected versatile bone-marrow cells into the animals' spleens and were surprised to see a ball of cells grow from each injection site. Each mass turned out to have emerged from a single stem cell, which in turn generated new blood cells.
That discovery led, 35 years later, to James Thomson's isolation of the first human embryonic stem cells, at the University of Wisconsin in 1998. And that milestone in turn inspired researchers to think about directing these cellular blank slates to eventually replace cells that had been damaged or were depleted by disease. The key lay in finding just the right recipe of growth factors and nutrients to induce a stem cell to become a heart cell, a neuron, an insulin-making cell or something else. It would take decades, the researchers all knew, but new therapies were sure to come.
Then, in 2001, everything changed. The use of discarded embryos made embryonic-stem-cell research deeply controversial in the U.S. Citing moral concerns, then President Bush restricted federal funding for the study of human embryonic stem cells. Under the new policy, U.S. government funds could be used only to study the dozens of embryonic cell lines already in existence - many of which proved not to be viable.
Read Stem Cells: The Hope and The Hype.
See the Year in Health, from A to Z.
The decision sent some leading scientists abroad, to Britain, Singapore and China, where the governments were more receptive to their work. Others who stayed behind but lacked private funding shifted their attention from embryos to the less versatile adult stem cells. Federally backed scientists, like Melton, who continued embryonic work were forced to adopt a byzantine system of labeling and cataloging their cell cultures and equipment so that government money was not used to grow forbidden cells - and government microscopes were not even used to look at them.
Those days may soon be over. Barack Obama campaigned on a promise to lift the research ban and support "responsible oversight" of the stem-cell field. For scientists, that means "we can stop the silliness," says Melton.
As welcome as that change will be, it may be less urgent now - owing primarily to the work of scientists like Melton. While embryonic stem cells remain the gold standard for any treatments that find their way into the clinic, newer techniques using the next-generation stem cells may soon surpass the older ones.
The Fighter In looks and demeanor, Melton is the quintessential professor, soft-spoken and thoughtful, someone who appears more mentor than maverick. Born and raised on the South Side of Chicago, he developed an early fascination with animal development; that curiosity led to a bachelor's degree in biology at the University of Illinois in 1975, then a second undergraduate degree, in the history and philosophy of science, at Cambridge University on a Marshall Scholarship. Melton remained there for his Ph.D. work, studying under Sir John Gurdon - the first to clone a frog. At Harvard, Melton teaches a frequently oversubscribed undergraduate course on science and ethics, in which he uses his keen sense of logic to provoke. When the class discussed the morality of embryonic-stem-cell research, Melton invited Richard Doerflinger of the U.S. Conference of Catholic Bishops to present arguments against the field. Melton asked Doerflinger if he considered a day-old embryo and a 6-year-old to be moral equivalents; when Doerflinger responded yes, Melton countered by asking why society accepts the freezing of embryos but not the freezing of 6-year-olds.
Clearly, Melton does not shrink from a fight. As Washington's squeeze on stem-cell research tightened in the early part of this decade, he decided to take action, providing life support for what remained of the U.S. stem-cell community. Not convinced that an entire field could make much progress relying on a few dozen cell lines of questionable quality, in 2004 he used funds HSCI receives from the Juvenile Diabetes Research Foundation and the Howard Hughes Medical Institute, as well as from Harvard alumni, and developed a more streamlined method for generating stem-cell lines from embryos. He created more than 70 new ones and has since distributed 3,000 copies to scientists around the country for free.
"Doug drew a line in the sand," says Alan Trounson, president of the California Institute of Regenerative Medicine, the organization charged with dispensing state money for embryonic-stem-cell research. "He turned the tables on an Administration that was incredibly negative toward stem cells and showed [it] we are not going to tolerate being put out of this field by ideological views that we don't think are correct." Melton's motivation was, again, both professional and intensely personal. Two months after Bush announced his ban, Melton's daughter Emma, then 14, also received a diagnosis of Type 1 diabetes.
In part owing to the restrictive U.S. policy, the momentum in stem-cell research seemed to shift overseas. In 2004, South Korean researcher Hwang Woo Suk announced that he had generated the first human embryonic stem cells from healthy people - and in the following year, from afflicted patients themselves - using an abbreviated cloning method. The latter feat would mean that cardiac patients could essentially donate themselves a healthy new heart without fear of rejection.
The news was huge - but it was also a lie. In 2006, Hwang admitted he had falsified his results. (Melton's colleague at HSCI, Kevin Eggan, finally created embryonic stem cells from patients in 2008.) Although Hwang became a pariah, he had the right idea. Melton and others had been trying to do just what the Korean scientist claimed to have done - grow a new population of a patient's own cells. The key to the process is a supply of fresh, good-quality human eggs, which incubate skin cells taken from a patient. Building up such a stockpile, however, proved practically impossible. The egg-extraction process is invasive and carries certain risks; after the state of Massachusetts prevented donors from being compensated for their eggs, out of fear the women would feel coerced, HSCI ended up with only one volunteer out of more than two years of recruiting.
Melton faced mounting political pressure too. In 2004, voters in California approved a measure providing $3 billion in state funding to embryonic-stem-cell research. That threatened to draw scientists in the stem-cell community west, and Melton took pains to foster a "band of brothers" mentality. "I tried to create a cocoon here," he says, "and tell people that your job is to focus on the science. Don't worry what the politicians say." By then, Melton's team was one of only a handful in the country working on embryonic stem cells and was making headway in teasing apart the myriad critical steps needed to guide these impressionable cells into becoming insulin-generating cells. Both as a scientist and as a father, Melton remained convinced that the federal restrictions simply could not survive. He continued to insist that "the science is so significant that it will change the policy."
And then, astonishingly, it did. In June 2006 a modest researcher from Japan made a startling announcement at the International Society for Stem Cell Research conference in Toronto. Shinya Yamanaka quietly described a study in which he took skin cells from a mouse and stirred them in with varying genetic cocktails made from a recipe list of 30 genes known to be important in development. When he hit on the right four genes and inserted them into the cells aboard retroviruses, he wiped the cells clean, reprogramming them and returning them to an embryo-like state without ever creating the embryo. Four genes, he told his audience, was all it took to undo a lifetime's worth of delicate genetic tapestry. No need for eggs, no need for embryos. Could it be that easy? Were the debate and controversy over embryonic stem cells now rendered moot? "It was unquestionably unexpected," says Melton of the breakthrough.
Read a TIME cover story on Stem Cells.
See the Year in Health, from A to Z.
A year later, Yamanaka followed up his work by reporting success with the same four factors in turning back the clock on human skin cells. At about the same time, in Wisconsin, Thomson achieved the same feat using a different cocktail of genes. With those studies, what became known as induced pluripotent stem cells (iPS cells) were suddenly a reality. Never mind the frustratingly fickle process needed to create embryonic stem cells; this was something any molecular-biology graduate student could do. "We figured somebody would have success with reprogramming. We just thought that somebody would come along a generation from now," says Dr. David Scadden, Melton's co-director at HSCI. "Yamanaka threw a grenade at all of that, and now all of the doors are open."
Beyond Stem Cells Melton, for one, isn't wasting any time before running through those doors. The iPS technology is the ultimate manufacturing process for cells; it is now possible for researchers to churn out unlimited quantities of a patient's stem cells, which can then be turned into any of the cells that the body might need to repair or replace.
Before that can happen, however, Melton wants to learn more about how diseases develop. And iPS cells make that possible too. For the very first time, he can watch Type 1 diabetes unfold in a petri dish as a patient's cells develop from their embryonic state into mature pancreatic cells. The same will be true for other diseases as well. "There is a good reason we don't have treatments for diseases like Parkinson's," says Melton. "That's because the only way science can study them is to wait until a patient appears in the office with symptoms. The cause could be long gone by then, and you're just seeing the end stages." No longer. Now the major steps in the disease process will be exposed, with each one a potential target for new drugs to treat what goes wrong. "This is a sea change in our thinking about developmental biology," says Dr. Arnold Kriegstein, director of the Institute for Regeneration Medicine at the University of California, San Francisco. "I consider it a real transformative moment in medicine."
The true power of reprogramming, however, does not stop with the stem cell. This summer, Melton flirted with the rules of biology once again when he generated another batch of history-making cells, switching one type of adult pancreatic cell, which does not produce insulin, to a type that does - without using stem cells at all. Why, he thought, do we need to erase a mature cell's entire genetic memory? If it's possible to reprogram cells back to the embryo, wouldn't it be more efficient in some cases to go back only part of the way and simply give them an extreme makeover? Using mouse cells, Melton did just that, creating the insulin-producing pancreatic cells known as islets. "The idea now is that you can view all cells, not just stem cells, as a potential therapeutic opportunity," says Scadden. "Every cell can be your source."
Realizing that potential - and with it, the prospect of successful treatments for conditions like Parkinson's or diabetes - may still be a few years away. Even iPS cells have yet to prove that they are a safe and suitable substitute for the diseased cells they might eventually replace in a patient. Ensuring their safety would require doing away with dangerous genes that can also cause cancer, as well as the retroviral carriers that Yamanaka originally used. Melton's team has already replaced two of the genes with chemicals, and he anticipates that the remaining ones will be swapped out in a few years. There are also hints that the iPS cells' short-circuited development makes them different in some ways from their embryonic counterparts. In mice, embryonic stem cells can generate a new mouse clone; iPS cells from the animals have so far stopped short of the same feat, aborting in midgestation, suggesting that some development cues may be missing. "It certainly makes me cautious," says Eggan.
Even if iPS cells do not prove as stable and as versatile as embryonic stem cells when they're transplanted into patients, they remain a powerful research tool. And if nothing else, they will have opened our eyes to the remarkable plasticity of biology and made possible new ways of thinking about repairing and replacing damaged tissues so we may consider not only treating but also curing disease. "It's a wonderful time," says Scadden. "Keep your seat belt on, because this ride is going to be wild."
For patients like Sam and Emma Melton, that ride carries with it the possibility of being free of the insulin pumps and injections they endure to keep their blood sugar under control. "I definitely think about how my life would be different if there is a cure," says Sam. His father is keenly aware that the ability of stem cells and reprogramming science to provide that cure is far from guaranteed. But his initial confidence in the power of the technology hasn't waned. "Everything we learned about stem cells tells us this was a really powerful approach," he says. "It would be a great shame if we let it wither and just go away." Melton, for one, is determined not to let that happen.
Science in Steps
A decade of conflicts and breakthroughs
1998 James Thomson, U of Wisconsin, isolates human embryonic stem cells
2001 President Bush restricts federal funding for research on human embryonic stem cells
2004 Douglas Melton of Harvard creates more than 70 embryonic-stem-cell lines using private funding and distributes free copies of the cells to researchers around the world
2006 Shinya Yamanaka, Kyoto University, turns back the clock on mouse skin cells to create the first induced pluripotent stem (iPS) cells, or stem cells made without the use of embryos. He uses only four genes, which are inserted into a skin cell's genome using retrovirus vectors
2007 Yamanaka and Thomson separately create the first human iPS cells
2008 July Kevin Eggan at Harvard generates the first patient-specific cells from iPS cells - motor neurons from two elderly women with ALS
August Melton bypasses stem cells altogether and transforms a type of mouse pancreatic cell that does not produce insulin into one that does
September Konrad Hochedlinger at Harvard creates iPS cells in mice using the common-cold virus rather than retrovirus vectors - an important step in making the technology safer for human use
October Melton's team makes human iPS cells by replacing two of the four genes, known to cause cancer, with chemicals. All four must be swapped out before iPS-generated cells can be transplanted into people
October Yamanaka creates mouse iPS cells using safer plasmids of DNA instead of retrovirus vectors
Read Stem Cells: The Hope and The Hype.
See the Year in Health, from A to Z.
View this article on Time.com
Related articles on Time.com:
A Leap Forward for Stem Cells
Cautious Optimism for the First Stem Cell Human Trial Approved by the FDA
Why Harvard Is Recruiting Egg Donors for Stem Cell Studies
A Breakthrough on Stem Cells
One Small, Small Step Toward Stem-Cell Nirvana

2009-01-30T15:37:00.002-05:00

'Life is too short to wake up in the morning with regrets, so love

the people who treat you right,

forget about the ones who don't

and believe that everything

happens for a reason. If you get a

chance, take it.

If it changesyour life, let it.

Nobody said life would be easy, they just promised it would be worth it

2009-01-29T15:53:00.000-05:00

If you are trusting the FDA to protect you then you are following blindly. The FDA recalls several medicions every year secondary to adverse effects such as death that it had at some time in the past approved. The FDA approves unsafe drugs, products and such every year and people are injuried and die every year because of thier mistakes.Last year the FDA finally addmitted on record that they have no idea how to handle products involving stem cells. They finally came clean that thier guidelines are outdated and not appropriate when involving stem cells and that they need to set up all new polices to govern stem cells. They also admitted that they did not even have anyone within the FDA to guide them. They then annouced that they would hire new experts and set up a brand new panel to create stem cell policies and guidelines. Since this anouncement last year they have not publically made a statement as to what if any progress they have made.The FDA is far from perfect and only a fool would think that they are not subject to the making of mistakes. Do not trust blindly and do not be that fool. Be smart, be educated and look not only at what your goverment tells you as we all know our goverment is far from perfect, honest and angelic.BTW everything I have said above is fact not theroy and all of it is a matter of public record. Goggle and yahoo searches can reveal all of these things.

from my friend kirshner

2009-01-29T15:48:00.002-05:00

That info was sent to me by Don Margolis. I have known him for some time and he often sends me info. Don can be a little soap boxy but he is also very good at uncovering some interesting and often overlooked information. The "theroy" may or may not be correct but history does show us some very interesting facts as does sceince. Embryonic stem cells have been proven to be unsafe. That is just a fact. Another fact is that studies involving humans and embryonic stem cells have been approved before and then pulled. This has happened more than once. To my knowledge only one has gone through for Batten's.The world of stem cells is very nasty and cut throat so do not be fooled by what you think you know as there is a very unpleasent underworld to it that you do not see or read about everyday. Stem cells are big big money and people will do some very strange and often inapprpriate things when bilions of dollars are involved.

Cautious Optimism for the First Stem Cell Human Trial Approved by the FDA

2009-01-28T15:42:00.000-05:00

Cautious Optimism for the First Stem Cell Human Trial Approved by the FDA
Mon Jan 26, 7:05 pm ET
It was nearly a decade in the making, but the first human trial using embryonic stem cells was approved on Friday.
The trial, which will test a stem-cell based treatment for spinal cord injury, will begin later this summer and will use cells generated by Geron Corporation. The approval marks the first time human stem cells, extracted and grown from embryos, will be transplanted into patients. Adult stem cells, which are present in many types of tissue, have been used in treatments for years - the most common being bone marrow transplants in cancer care - but an embryonic study is a whole new thing. There's good reason it's being greeted with so much excitement. (See the top 10 medical breakthroughs of 2008.)
Scientists believe that embryonic stem cells are more versatile than adult cells in generating the more than 200 different tissue types in the body. The need for healthy new cells is particularly acute in the case of spinal cord injury, because once central nervous system tissue is destroyed, it does not regenerate - not in any significant way at least. The Geron team began its work with what is known as a Presidential stem cell line - stem cells derived from discarded in vitro fertilization embryos that already existed in 2001 when former President Bush decided to prohibit the use of federal funds to pursue human embryonic stem cell work. At the time, fewer than two dozen of these stem cell lines were of good enough quality to use as a basis for human treatments.
The therapy revolves around coaxing the stem cells to develop into a type of cell known as an oligodendrocyte, which in turn gives rise to critical insulation and growth factors that can repair neurons damaged by a trauma to the spinal cord. Geron has named its version GRNPOPC1. By having more of these cells around the spinal cord, more nerves might be repaired and can then potentially re-establish proper connections. Simply by working with the GRNOPC1 cells, Geron scientists have learned more about how they operate, which will only expand their understanding of how the central nervous system might be healed. Says Geron's president and CEO Dr. Thomas Okarma: "They make dozens of factors that can stimulate nerve function, growth and regeneration." (Read "Scientists Reach Stem Cell Milestone.")
The trial will enroll patients with injury to the thoracic region, high in the spinal cord between the third and tenth vertebrae. Doctors will be trained to inject the cell treatment at specific locations, where the cells will remain to do their nerve-nurturing work. "I think it's incredibly exciting," says Dr. Susan Fisher, a stem cell scientist and a professor of obstetrics, gynecology and reproductive science at University of California San Francisco. "This really provides a blueprint for how to do these sorts of trials. It really proves the principle that these sorts of human embryonic stem cell therapies can survive the FDA approval process."
And that process wasn't such an easy hurdle to overcome. Because no one has used embryonic stem cells, or the cells derived from these stem cells, in people before, the FDA was particularly cautious. The trial that it did approve is what's known as a Phase 1. It will involve no more than a dozen patients and is not designed to test the effectiveness of the cells. Rather, it will simply monitor the safety of inserting them into people. The researchers will be looking for whether the cells cause tumors, trigger an immune response, or start to migrate away from the spinal cord area. "There are certainly unknowns that we can't predict," says Dr. David Scadden, co-director of the Harvard Stem Cell Institute. "We don't know whether or not these cells might grow abnormally in a person. We don't know if things might occur just by these cells being present that could result in an outcome we don't want. This really is a first go, with a lot on the line."
Dr. Ronald Crystal, chairman of the department of genetic medicine at New York-Presbyterian Hospital/Weill Cornell Medical College, knows those stakes all too well. He is a veteran of the last revolution in medical technology, gene therapy, which, after some hyped expectations in the 1990s, fell into disfavor after some unsuccessful trials. Crystal is cautiously optimistic about the potential for this trial to open the door to future stem cell therapies. "I think this is a very positive start, but the expectations and hype I see around stem cell therapies are the same that I saw around gene therapy. We just have to remember that these take a long time to develop. But you will never know if they work unless you study them in humans, so this is an important first step."
Stem Cells: The Hope and The Hype
See the Year in Health, from A to Z.
View this article on Time.com
Related articles on Time.com:
A Breakthrough on Stem Cells
Banking on Stem Cells
Finding a Master Heart Cell
Eeek! There's a Mouse Cell in My Stem Cells!
Scientists Reach Stem Cell Milestone

SCHEMERA@YAHOO.COM

2009-01-27T15:41:00.001-05:00

Drew,

So glad you won!! Nasty cut...must have given you one whopper of a headache!I have been following your blog since June...thank you for providing so much up to date information.I have a family member who is very ill ...yet to be definitely diagnosed (symptoms all of ALS, of course, we continue to pray for a miracle that has any other name)Do you have an email address that I could contact you at to ask some questions?

Take CareLee

Monday, January 26, 2009, 12:43pm EST | Modified: Monday, January 26, 2009, 1:05pm

2009-01-26T18:22:00.003-05:00

Monday, January 26, 2009, 12:43pm EST Modified: Monday, January 26, 2009, 1:05pm
Neuralstem wins key patent for stem cell work
Washington Business Journal - by Vandana Sinha Staff Reporter

Joanne S. Lawton
Richard Garr, CEO of Neuralstem.View Larger
Neuralstem Inc. said Monday it has nailed down a key patent for its core technology, which grows neural stem cells from the brain to replace damaged spinal cord cells.
The patent comes in time for Neuralstem’s first clinical trials, planned for this year if the Food and Drug Administration gives them the green light. The Rockville company intends to test the ability and safety of injecting fetal stem cells into the spinal cords of patients suffering from amyotrophic lateral sclerosis, more commonly known as Lou Gehrig’s Disease.
Under the new patent, Neuralstem (AMEX: CUR) can grow almost unlimited numbers of stem cells from all parts of the brain, all in a controlled and consistent manner. The company has been honing its technology for the past 13 years, relying on fetal stem cells rather than their more controversial counterpart, embryonic stem cells.
Neuralstem filed its request in December to launch its first human clinical trials this spring. It expects to hear the FDA’s decision by the middle of next month, but another competitor’s recent news has raised its hopes. On Friday, the FDA approved a request by Geron Corp. to test embryonic stem cells in human trials for the first time, illustrating to Neuralstem leaders a growing comfort level at the agency with using previously untested stem cells to treat non-fatal diseases.
“It removes a tremendous amount of uncertainty,” said Neuralstem CEO Richard Garr. “It makes it clearer what’s required to make it forward [to trials]. From what we’ve seen, we’ve done what’s required.”
While he said President Barack Obama’s entry in the White House is more coincidental than influential in the FDA’s decision for Geron, he does foresee it opening more doors for stem cell discovery and science.
“There’s no question that the tone will be different,” Garr said. “The tone from the top will always have an impact.”
Garr points out the difference between the two companies’ techniques, however, saying a green light for Geron doesn’t necessarily guarantee one for Neuralstem. Whereas Geron is working to essentially restrip the neural “wires” that connect spinal cells to help heal traumatic spinal cord injury, Neuralstem is opting to fully replace destroyed spinal cord cells to treat ALS.
Before the end of the year, Neuralstem also hopes to start clinical trials of its technology on improving traumatic spinal cord injury, the same disease area as Geron, potentially placing Neuralstem in direct competition with the Menlo Park, Calif., company.
The local company raised more than $1.7 million in a stock offering at the end of last year --enough, Garr said, to pay for the ALS trials on an anticipated 15 patients.
All contents of this site © American City Business Journals Inc. All rights reserved.

2009-01-25T20:28:00.000-05:00

Dear friends,
News came out this past Friday that the Food And Drug Administration (FDA) had okayed its first (ever) embryonic stem cell test. This test took several months to get through the review process because of the dogmatic roadblocks, the Bush administration put in place.
In an interview with CNN on January 18, Obama said: "I like the idea of the American people's representatives expressing their views on an issue like this." As President,,, it is Pres. Obama has the power to issue an Executive Order opening federal funding for stem cell research. ." Let's hold him to his campaign pledge!!
Only after this fruitless & obstructive red tape is eliminated and federal funding for stem cell research is allowed, can life saving research once again resume. . Although President Obama pledged to reverse the Bush administration's ban on federal funding of stem cell research during the campaign trail, he seems to be backing off on that pledge now.
If we leave this issue alone for any length of time, it will definitely be relegated to the back burner, and research for diseases like ALS, M.S., Parkinson's, and Alzheimer's, spinal cord injuries, and organ and tissue damage will definitely be set back.
President Obama must issue an Executive Order to open federal funding for stem cell research. ." Let's hold him to his campaign pledge!!
Right now, there is no bigger supporter than myself of Pres. Barack Obama, but if he starts passing the buck on such important issues as federal funding for stem cell research, there will be no greater critic. People's Lives are at stake here, let's do the right thing.

All family & friends of loved ones suffering from diseases that stem cell research funding could help, should take the time and get involved ?. We need your support
http://www.visi.com/juan/congress/
http://www.senate.gov/general/contact_information/senators_cfm.cfm.
Thanks ,
Eddie spaghetti) Esparza.
They came first for the Communists, And I didn't speak up because I wasn't a Communist;
And then they came for the trade unionists, And I didn't speak up because I wasn't a trade unionist;
And then they came for the Jews, And I didn't speak up because I wasn't a Jew;
And then . . . they came for me . . . And by that time there was no one left to speak up."

Richard John Neuhaus

FDA OKs 1st Embryonic Stem Cell Trial

2009-01-23T18:59:00.000-05:00

FDA OKs 1st Embryonic Stem Cell Trial
By Steven ReinbergHealthDay Reporter by Steven Reinberghealthday Reporter 1 hr 56 mins ago
FRIDAY, Jan. 23 (HealthDay News) -- The first human trial using embryonic stem cells as a medical treatment has been approved by the U.S. Food and Drug Administration.
Geron Corp., a California-based biotech company, has been given the OK to implant embryonic stem cells in eight to 10 paraplegic patients who can use their arms but can't walk. Stem cell injections will be given within two weeks of the injury. The study will begin this summer, and will be conducted at up to seven different medical centers.
"This marks the dawn of a new era in medical therapeutics," Dr. Thomas B. Okarma, Geron's president and CEO said during a Friday morning teleconference. "This approach is one that reaches beyond pills and scalpels to achieve a new level of healing."
Ultimately, this type of therapy might have the power to restore permanent organ and tissue function, Okarma said. The goal of this first trial is to see if injecting embryonic stem cells into humans is safe. However, the researchers will also be looking for signs of improvement in the patients' ability to feel sensation in or move their legs.
Patients will receive injections at the site of the injury. It is hoped these cells will mature into cells that will repair damaged nerves and produce chemicals that nerve cells need to function and grow.
This phase I trial will be limited to patients whose injury is located in the middle of the spine. If the trial is successful, Okarma said, the hope is to extend the treatment to patients with cervical spine injuries who are paralyzed from the neck down.
Okarma said the injections must be given early after the injury, before scar tissue has developed that would prevent the cells from growing, but after the initial swelling has subsided.
In addition, patients will receive anti-rejection drugs for about two months, after which they should no longer need those drugs. Patients will be followed for at least one year, he said.
The treatment is not expected to restore full function to patients, but the researchers hope to see modest gains. "Any return of bladder or bowel function, a return of sensation, or a return of lower extremity locomotion would be a very exciting finding," Okarma said.
In experiments with rats, researchers found these cells were safe and did restore some function. "These cells insulate as well as stimulate nerve fibers, leading to restoration of function in animal models of spinal cord injury," Okarma said.
The cost of this therapy isn't known yet, but Okarma said it would be "affordable."
Embryonic stem cells are the most basic human cells. These cells are believed to be capable of growing into any type of cell.
The controversy surrounding the use of these cells has become a political issue, with some objecting that the use of these cells destroys potential life because they must be extracted from human embryos. This belief resulted in the Bush administration banning federal funding for embryonic stem cell research.
While the Obama administration has indicated that it will lift the ban, the stem cells used in this trial were obtained from one of the Bush administration's approved stem cell lines. However, no federal funds were used in the development of this treatment.
A decade has passed since the first embryonic stem cells were isolated at the University of Wisconsin, in groundbreaking research that was funded by Geron Corp.
Geron is also working on using embryonic stem cells to treat failing hearts and to create insulin-producing islets for type 1 diabetics, Okarma said.
"Embryonic stem cells are really nature's own way of making more of ourselves," Okarma said. "We are simply harnessing the biology of normal human development in our attempts to achieve permanent cures to chronic disease and injury."
Peter Kiernan, chairman of the Christopher And Dana Reeves Foundation, said he's excited about this latest development in stem cell research.
"This is not just a comet across our sky, this is really more like dawn," Kiernan said. "We are beginning a vast human experiment, and we have been waiting an extremely long time to get to this point. This is a very significant development."
At the same time, Kiernan said he does not overestimate what can be expected from this trial.
"Of the millions of people dealing with paralysis in our nation, they are all delighted with subtle increases in function," Kiernan said. "We eat, drink, sleep getting people out of wheelchairs, but the reality of the world we are in is if people get bowel function, some sexual function, some ability for movement, that is a wonderful outcome."
More information
For more on embryonic stem cells, visit the U.S. National Institutes of Health.

FDA allows first test of human stem cell therapy

2009-01-23T18:57:00.000-05:00

FDA allows first test of human stem cell therapy
By Maggie Fox, Health and Science Editor Maggie Fox, Health And Science Editor Fri Jan 23, 11:51 am ET
WASHINGTON (Reuters) – The U.S. Food and Drug Administration has cleared the way for the first trial to see if human embryonic stem cells can treat people safely, a company involved in the controversial research on Friday.
Geron Corp, a California biotechnology company, said it plans a clinical trial to try to use the stem cells to regrow nerve tissue in patients with crushed, but not severed, spinal cords.
The issue of human embryonic stem cell research has been a political touchstone, with anti-abortion forces backed by former president George W. Bush arguing the technique involves the destruction of human embryos. Advocates say it could transform medicine.
"For us, it marks the dawn of a new era in medical therapeutics. This approach is one that reaches beyond pills and scalpels to achieve a new level of healing," Geron Chief Executive Dr. Thomas Okarma said in a telephone briefing.
Shares of Geron rose more than 53 percent to $8 in mid-morning trading on Nasdaq after touching $8.38.
Geron will recruit eight to 10 recently injured patients and inject them with small numbers of human embryonic stem cells manipulated to become the oligodendrocyte cells that insulate nerves, and that produce compounds to stimulate the growth of nerve cells.
Okarma said there was no political significance to the announcement coming the same week as President Barack Obama took office. Obama has been widely expected to lift restrictions on federal funding and support of human embryonic stem cell research.
An FDA spokeswoman said, "Before FDA allowed the study to proceed, Geron worked with FDA to address important scientific questions."
CHEAP AND EASY
Okarma said the treatment should eventually become cheap and easy to mass produce because the cells can be grown in vats. He believes the cells may be useful for other diseases such as multiple sclerosis, in which nerve cells are stripped of their insulating sheaths, and perhaps strokes.
Financial analysts celebrated. Stephen Brozak and Daniel Mallin of WBB Securities LLC said it could "as important to drug therapy as the discovery of ... penicillin."
Dr. Robert Lanza of the rival Massachusetts-based Advanced Cell Technology, which is also seeking to create therapies using human embryonic stem cells, called the decision a "huge advance for the entire field". This sends a message that we're ready at last to start helping people," he said.
Stem cells are the body's master cells, giving rise to all the tissues, organs and blood. Embryonic stem cells are considered the most powerful kinds of stem cells, as they have the potential to give rise to any type of tissue.
Researchers are also trying to find ways to use so-called adult stem cells, taken from bone marrow and elsewhere in the body, and have learned how to transform ordinary skin cells into stem-like cells. But scientists argue that no one knows which route will work so all avenues must be pursued.
The Phase I trial will be designed to show that patients do not develop tumors, or damage to their nervous systems. But Okarma believes it will also indicate whether the stem cells might repair the damaged spinal cords.
"These are living cells that will divide, make more of themselves and migrate throughout the lesion after injection," he said.
While the patients will get low doses of immune-suppressing drugs for the first two months, Okarma is confident the cells will escape immune system recognition and patients will not have to endure the treatments that organ and tissue transplant recipients usually do. Treatment on the first patient should begin this summer.
Okarma said the company has a strong balance sheet, free of debt, and "ample to fund the company through this trial".
(Additional reporting by Susan Heavey, Doina Chiacu and Toni Clarke; Editing by Alan Elsner and Julie Steenhuysen)

British trials to use stem cells for stroke, blindness

2009-01-20T22:04:00.000-05:00

British trials to use stem cells for stroke, blindness
Mon Jan 19, 11:29 am ET
LONDON (AFP) – Two separate trials are set to begin in Britain utilising cutting edge stem cell research in a bid to help treat victims of strokes and blindness, medical experts announced Monday.
Doctors are hoping to launch the world's first trial for a treatment that aims to improve the quality of life for thousands of stroke victims on patients in Glasgow in June, although the procedure must still be approved by an ethics committee.
The treatment, which uses cells taken from an aborted foetus that are to be injected into the brains of stroke victims to see if they can effectively regenerate damaged areas, was developed by Britain-based company ReNeuron.
"That single cell was expanded by means of technology so we can have something to treat many, many thousands of patients," said ReNeuron founder John Sindon, who is working with consultant doctor Keith Muir on the planned trial at Southern General Hospital in Glasgow.
"You could make the argument that it would have otherwise gone to waste. The reality is that we're trying to turn that into something with a lasting effect."
Separately, a two-year trial involving 20 patients with corneal blindness will begin this month at the Princess Alexandra Eye Pavilion in Edinburgh and the Gartnavel General Hospital in Glasgow.
The treatment being used involves using the stem cells of dead adult donors, rather than the more controversial research involving embryonic stem cells, and if successful could help millions of people around the world who suffer from corneal blindness, around 80 percent of whom are elderly.
As part of the process, adult stem cells are cultivated and then transplanted onto the cornea's surface.
"This study is the first of its kind anywhere in the world and it is exciting to be involved in such groundbreaking work," said Professor Bal Dhillon, who is heading the trial.
"I probably see two or three new cases of corneal disease every month. On a larger scale, it's a significant problem."
A similar study by the University of Pennsylvania in the United States last year found that people with inherited blindness saw dramatic improvements in their vision when a corrective gene was injected into their eyes.
Scientists believe stem cells, which are capable of developing into almost every tissue of the body, could prove key in finding a cure for a number of serious diseases, including also diabetes and cancer.

me vs. the night stand-i won....

2009-01-18T15:22:00.001-05:00

Obama wants Congress to act on lifting stem cells ban

2009-01-17T17:22:00.000-05:00

Obama wants Congress to act on lifting stem cells ban
Fri Jan 16, 6:11 pm ET
WASHINGTON (AFP) – President-elect Barack Obama said Friday he wanted legislation in Congress to permit federal funding on stem cell research and overturn a ban imposed by President George W. Bush.
Obama, who favors medical research on stem cells derived from human embryos, told CNN he was still exploring an executive order to revoke Bush's ban.
"But I like the idea of the American people's representatives expressing their views on an issue like this," he said, lauding a "bipartisan" consensus in Congress that such research is ethical and potentially life-saving.
If the research could yield hope for victims of degenerative diseases such as Parkinson's and Alzheimer's, "I think that sends a powerful message," he said.
In blocking federal funding for stem cell research, Bush sided with religious conservatives who argue that research on embryos destroys human life, albeit at its earliest stage of development.
Stem cells are primitive cells from early-stage embryos capable of developing into almost every tissue of the body.
Scientists believe they could prove key in finding a cure for a number of serious diseases, including also diabetes and cancer.

2009-01-17T17:11:00.001-05:00

Dear brothers and sisters,

Please review the attachment/article, well all I can say about yet another bogus, don't get out of line, hopeless message ... -- WELCOME TO THE 21st CENTURY !! -- The Old Way of Doing Things Are Almost over for you guys. As Stephen Byer articulately pointed out the Internet is king, no longer will people be blindly corralled like cattle to their doom, at least not progressive minded fighters .

Articles like this are just a last, desperate attempt by the ALS establishment to keep control . For too long they've been making us believe that, the end all gospel of medical evidence and facts, are in the hands of the holy Grail = "experts" and no one, especially mere mortals who surf the Internet, should ever dare try their hand at questioning /challenging the established order.

Let's face it we (PALS) have been at the mercy of waiting for unending research, big pharma interest, orphan disease politics, etc. They also think that calling something a "myth" will belittle the efforts of those who educate themselves & fight for their own health freedoms. Sorry, that may have been last century, but not this one.

I love this statement from the article/attachment (Red):
We need to do a better job of distributing information to PALS on the many benefits of attending specialized ALS clinics, on the many benefits of participating in the research studies offered there, and on the dangers of self-experimentation. We should refrain from off-label prescribing of unproven therapies. Most importantly, we need to educate PALS on how to distinguish myth from realityAllow Me to Sort This Ridiculous Paragraph into Sections
1. Do a better job of distributing information to PALS??? -- What information are they referring to? Oh, let me think of a classic "Mr. Johnson, your breathing capacity has dropped to dangerous levels, you may want to consider a trache" but then again only 10% of all patients at this neuromuscular center have ever taken that option. WOW! Could you repeat the second option again? Or what about this priceless tidbit (information distribution ) -- Mr. Johnson there are no available treatment options except Rilutek/Riluzole , which is highly recommended by this neuromuscular team, nevertheless you probably have anywhere from 2-5 years of your life left, I suggest you get your affairs in order.. would you like to speak to our an M D A representative?.

2. Many benefits of attending specialized ALS clinics -- Oh Really, Would You Care to Expand on What They Are? --I am not clear what are the benefits of specialized ALS clinics. Oh, I know, refills for Rilutek, prescription for XYZ depression, anxiety, order lab work to check if my liver is cooked by so much Rilutek, prescription for drooling, referral to ENT, prescription for a wheelchair, or just to tell me every three months that my disease is progressing and there are no options/is NOTHING out there. I call that the trip to HOPELESS LAND...it is more of a hussle. A typical day at a neuromuscular clinic includes... bathing time, dressing time, loading time, driving time, waiting time, (in between specialist's) and finally it's time to see the "specialist/neurologists" to hear a 25 minute no-hope message that includes "see you in three months." So, if I am missing something, I need these authors to tell me what other benefits are there at specialized ALS clinics!!!!

3. On the many benefits of participating in the research studies offered there -- Which one? The one that generates the most donations or the one where I share a cage with the mouse? Tell me Mr. authors, which of the research studies are moving fast enough to generate my interest? What I see mainly is tons of money wasted by big name university researchers to ensure job security for centuries to come. Or the real warrior scientists wanting to participate on research funds that get push aside because they are not affiliated with a big name university or organization. How about "creating false hope" dear authors!

4. And on the dangers of self-experimentation. I recently heard of two scientists who have been spending their OWN money working around the clock to find a cure for ALS, out of their OWN pocket! They know that rare diseases like ALS will not be a one size fits all cure. We all know that ALS is just an umbrella term for a number of different types of motor neuron diseases. And with the exception of the 5% familial type, treatments will need to be customized to the individual and the only way is to do it on trial and error basis. If I am willing to use my body for science sake, why should any article stop me from doing it?

5. We should refrain from off-label prescribing of unproven therapies ... What ? ? ? -- Do these authors know that new drugs are often not tested for safety and efficacy specifically in children. Therefore between 50 percent and 75 percent of all medications prescribed by pediatricians in the U.S. are for off-label applications. Are they proposing to tell these pediatrician not to continue this practice? Another example, the standard of care for a particular type or stage of cancer involves the off-label use of one or more drugs. Another example is the use of tricyclic antidepressants to treat neuropathic pain. According to medical literature, this old class of antidepressants is now rarely used for clinical depression due to its side effects but the tricyclic are often effective for treating pain on those patients with neuropathic conditions. Should we share the authors recommendations with the neuro community too? Oh forgot, how about Provigil an off label drug used for MS patients....the list goes on and one. Do we stop this practice all together for everyone or are the authors suggesting just IPLEX for PALS to be restricted and not prescribed off label. THAT will be discrimination BIG TIME!!!! Law suit, law suit....

6. Most importantly, we need to educate PALS on how to distinguish myth from reality - MAN!... Does anyone else feel insulted?... what about Rilutek/Riluzole? -- Do they honestly think that PALS are a bunch of dummies without brain? Actually, PALS brains are very active and perform best without the distractions of the other body parts it is called ENHANCED functioning....and last time I check Rilutek was really a MYTH....prove me wrong!

Summarizing: These gents need to accept the fact that the world has changed, and the game is almost up. Blessed be cyber technologies that allow people to break through special interests, share information (good or bad) and take over a cause and become engaged activists. Next step, We All Write Letters to Those Well Intended Authors who are just misguided and cannot see that the world has changed and remind them that whenever writing an article just directed to PALS, they must be reminded that, although PALS bodily functions are steadily declining, the one thing that's left intact is the brain . Should our (PALS) families, not have the right to any and all possible therapies including stem cells, IPLEX, or whatever other reasonable therapeutic that might have even marginal efficacy? -- save our lives-? Should it not be our doctors responsibility to adopt the attitude that, all reasonable treatments/therapies including stem cell should be considered , instead of the fatalistic “oh sorry, Mr. Charlie - . Life isn't fair, that’s just the way it is, there's nothing we can do -- SEE YOU IN THREE MONTHS!!

this video is amazing

2009-01-16T14:11:00.001-05:00

http://www.youtube.com/watch?v=Cmiq1eGmsxE&feature=channel_page

ER stop

2009-01-15T18:13:00.003-05:00

i ended up rolling off my bed and i hit my head. my night stand took the brunt of it. i recieved 10 stitches. my sister was "cool hand luke" throughout. i'm ok. never better....,
drew

Downtown Mannequin Torsos Symbolic Of Disease

2009-01-15T18:07:00.002-05:00

Downtown Mannequin Torsos Symbolic Of Disease

By JOHN W. ALLMAN The Tampa Tribune
Published: January 15, 2009
Related Links
More On Lou Gehrig's
More Health News

TAMPA - Most people know the name, but not the disease.
"Piece by Piece," a new campaign by the ALS Association Florida Chapter, hopes to change that by increasing public awareness of Amyotrophic Lateral Sclerosis, or Lou Gehrig's disease.
The campaign's official debut is today at Joe Chillura Courthouse Square Park in downtown Tampa.
Across the grass, volunteers worked in early morning cold to set up 150 mannequin torsos. Each torso bears a black T-shirt. Some have names of Lou Gehrig's patients, both those still living and some who have died.
The display will be open to the public until 6 p.m.
The mannequin bodies have no arms or legs, which is symbolic of the devastating impact of the disease, said Kamden Kuhn, spokeswoman for the Florida chapter.
Lou Gehrig's is a progressive neurodegenerative disease that causes victims to become completely paralyzed. The disease is gradual, however, and can slowly take away the ability to walk, speak and, finally, breathe.
"There are cancer survivors. There are heart attack survivors. There is no such thing as an ALS survivor," said Kuhn, who lost her grandfather to the disease prior to joining the association. "That's why we think this needs a lot of attention."
The "Piece by Piece" campaign was conceived by Tampa advertising agency Dunn & Co. It will travel throughout Florida this year with stops in seven cities.
The mannequins will be on display in Tampa through February. They will be in Lykes Gaslight Square Park next Thursday and Centennial Park in Ybor City on Saturday, Jan. 31.
The ALS Association Florida Chapter is accepting donations for research and patient care. People wishing to give money can have the name of a loved one put on a T-shirt used in the display.
Volunteers will be on hand at each stop of the campaign to provide information.
Steve Franks' name is on a T-shirt.
The Pinellas Park resident, diagnosed with Lou Gehrig's in 2003, considers himself "one of the lucky ones."
Most people die within five years of being diagnosed, Franks said. The disease has limited his ability to walk for long stretches, and affected his speech, but he is still able to drive. Franks, 50, is in charge of transporting the mannequins to each city during the campaign.
"My biggest frustration is that people don't know what ALS is and what it's doing to the people who have it," he said Thursday morning. "That's why I want to be part of this. It's going to educate so many people."
For More Information:To learn more about Amyotrophic Lateral Sclerosis, or Lou Gehrig's disease, and the "Piece by Piece" campaign, go to: http://www.stealingpieces.org/.
To donate to the ALS Association Florida Chapter and sponsor a T-shirt in the campaign, call 888-257-1717, ext. 107.

Reporter John W. Allman can be reached at jallman@tampatrib.com or (813) 259-7915.

http://www2.tbo.com/content/2009/jan/15/downtown-mannequin-torsos-symbolic-disease/

2009-01-14T21:23:00.000-05:00

--------------------------------------------------------------------------------

Nature Biotechnology 27, 10 (2009)

doi:10.1038/nbt0109-10

Pfizer's $100 million stem cell stake

Nayanah Siva

Pfizer has launched Pfizer Regenerative Medicine, an independent research unit focused exclusively on using stem cells to develop new medicines. The New York-based company will spend more than $100 million over the next 3-5 years on the new initiative, which will employ 70 researchers based at two facilities, in Cambridge, Massachusetts, and Cambridge, UK. The UK group will focus on neural and sensory disorders, whereas the US team will concentrate on endocrine and cardiac research. In-house researchers will work with both embryonic and adult stem cells, but significant collaborations are also planned. Chief Scientific Officer Ruth McKernan, who will head the UK site, says: "We are keen to take advantage of successful work done by other companies and academic labs. We will be working with several collaborators and these will be announced in the new year." In the past, big pharma has shied away from investing in stem cell research, but Pfizer's move confirms that attitudes are changing. London's GlaxoSmithKline recently signed a $25 million four-year deal with Harvard University, and the venture funds of Basel-based Novartis and Roche helped bankroll Cellerix, a Madrid company testing stem cells from fat to treat rare skin conditions. Stanford University, California, also recently announced the construction of the world's largest stem cell research building to house over 600 scientists by 2010.

All NIH human embryonic stem cell registry lines now deposited at NSCB

2009-01-13T16:57:00.002-05:00

All NIH human embryonic stem cell registry lines now deposited at NSCB
Jan. 12, 2009
by Janet Kelly

The U.S. National Stem Cell Bank (NSCB) has announced that it has received deposits of two human embryonic stem cell lines from Cellartis AB, a biotechnology company based in Sweden. With the addition of the new lines, the National Stem Cell Bank now has received all 21 cell lines from the six providers listed on the National Institutes of Health (NIH) federal registry.

Currently, 16 of these lines have completed the NSCB's extensive quality control process and are available for distribution to research scientists around the world. The NSCB's initial testing process, which can take several months or longer to finalize, begins upon receipt of a new cell line and is carried out to ensure the identity of the cell line, cell characteristics and that the starting cell material is free from contaminants.

The NIH established the country's first National Stem Cell Bank at the WiCell Research Institute, a private, nonprofit supporting organization to the University of Wisconsin-Madison, in September 2005. Its mission is to obtain, characterize and distribute the 21 human embryonic stem cell lines that currently may be used in U.S. federally funded research. All six providers of the NIH-registry stem cell lines — WiCell at UW-Madison, University of California, San Francisco, and Novocell in the U.S.; ES Cell International (ESI) in Singapore; Technion in Israel; and Cellartis in Sweden — were invited to deposit their cells by the NSCB shortly after it was established.

Derek Hei, a UW-Madison researcher and leader of the NSCB, says the availability of a variety of human embryonic stem cell lines for study is critical to advancing the field. "The addition of the Cellartis lines to the National Stem Cell Bank is extremely important because now we'll be able to distribute these lines to the worldwide research community," he says. "We'll also be able to generate data unique to these lines that is valuable to the advancement of stem cell research."

Mats Lundwall, CEO of Cellartis, says, "We are delighted to have this collaboration with the U.S. National Stem Cell Bank that will increase the amount of NIH eligible lines readily available in the U.S. The Cellartis cell lines are among the most extensively characterized in the world and now their distribution within the U.S. has been further facilitated through this partnership."

In addition, the host organization for the NSCB, the WiCell Research Institute, recently began its own bank, the WiCell International Stem Cell (WISC) Bank, to study and distribute stem cells that currently cannot be offered through its current contract with the NIH. "Due to the NSCB's deposits of all 21 NIH-registry lines and WiCell's ability to offer induced pluripotent and other stem cells through our new WISC Bank, our facility provides a tested operation with experienced staff that is a unique source for stem cell researchers worldwide," says Erik Forsberg, executive director of WiCell.

WiCell researchers are working to characterize the cells in the National Stem Cell Bank to support the development of human embryonic stem cells for research and therapeutic applications. Their efforts include trying to understand how each cell line behaves under different conditions in the laboratory, testing the cells for potential pathogens and assessing their potential to become specific tissues, such as heart, neuronal or insulin-producing islet cells. The results of these studies and tests are made available to the scientific community on the National Stem Cell Bank Web site.

Scientists from nonprofit and academic institutions anywhere in the world can request the human embryonic stem cell lines deposited in the bank. The site provides research protocols and in-depth information on specific cell lines. The NIH Registry Lines available through the National Stem Cell Bank are priced at an affordable $500 per two vials, which contain approximately six million cells capable of establishing multiple new colonies.

The WiCell Research Institute, founded in 1999, is dedicated to expanding the frontiers of science and medicine by unlocking the potential of stem cells. As a private, nonprofit supporting organization of UW-Madison, WiCell conducts research, supports research at UW-Madison, hosts the National Stem Cell Bank and the WISC Bank, provides training for scientists and offers educational outreach programs for K-12 students and the community.

WiCell has provided free human embryonic stem cell licenses to more than 500 researchers in 32 countries and 42 states. WiCell offers an ongoing schedule of stem cell training classes and workshops customized for participants ranging from youth to non-technical adults to lab technicians to top researchers. Since 2002, WiCell has trained more than 600 individuals in stem cell technologies and methodologies.

Cellartis AB is a premier provider of human embryonic stem cell (hES)-derived products and technologies that will drive unprecedented innovation in drug discovery and regenerative medicine today and in the future. Today, alongside fully characterized, ethically derived human embryonic stem cell lines, the company offers custom-prepared human cardiomyocytes and mesenchymal progenitors derived from hES cells along with stem cell antibodies and tools. Cellartis is engaged in programs for the production of homogenous populations of hepatocytes suitable for use as bio-tools in industrial applications. In addition, Cellartis has built the world's first large-volume automated production facility for human ES cells and can provide human ES cells for screening campaigns. The company was founded in 2001, has more than 50 employees and is located in Gothenburg, Sweden and Dundee, Scotland/UK. For more information, e-mail info@cellartis.com.

2009-01-09T16:22:00.002-05:00

New drug combo may boost stem cell production
By Michael Kahn Michael Kahn
Thu Jan 8, 11:51 am ET

LONDON (Reuters) – A novel drug combination using Genzyme Corp's Mozobil shows it may be possible to spur bone marrow into releasing extra adult stem cells into the bloodstream to repair the heart and broken bones, researchers said on Thursday.

The study of mice raises hope that researchers could use the same technique to tackle autoimmune diseases such as rheumatoid arthritis in which the body confuses healthy tissues for foreign substances and attacks itself, they said.

"We hope that by releasing extra stem cells, as we were able to do in mice in our new study, we could potentially call up extra numbers of whichever stem cells the body needs," said Sara Rankin of Imperial College London, who led the study.

"Our work could lead to new treatments to fight various diseases and injuries which work by mobilizing a person's own stem cells from within."

Stem cells are the body's master cells, giving rise to various tissues and the blood. They are found throughout the organs, blood and tissue and are in immature form until they generate needed cell types.

Doctors hope to use them some day in a new field called regenerative medicine in which tailor-made transplants of tissues and perhaps organs can be grown from a patient's own cells.

Rankin and her team looked at mesenchymal stem cells -- immature cells than can give rise to bone, muscle or blood vessels -- and endothelial cells that help make blood vessels in the heart.

They treated healthy mice with one of two proteins that occur naturally in bone marrow called VEGF and G-CSF growth factor. Following this treatment the mice received Genzyme's stem-cell transplantation drug Mozobil.

Researchers know that G-CSF in combination with Mozobil mobilizes one kind of stem cell used in bone marrow transplantation known as hematopoetic stem cells, which give rise to blood cells. But Rankin and colleagues wanted to see if VEGF growth factor could stimulate other types of stem cells involved in building heart and bone tissue and blood vessels.

The team, which reported the findings in the journal Cell Stem Cell, found that mice given VEGF and Mozobil released around 100 times as many endothelial and mesenchymal stem cells into the bloodstream compared to mice that had no treatment.

While trials of the drug combinations in humans is years away, the researchers said the next step is determining in mice whether the technique actually helps repair damage, Rankin said.

"One of the exciting aspects is this would be a non-invasive treatment," she said in a telephone interview. "With this you are just giving a drug to promote what is a natural process."

(Reporting by Michael Kahn; editing by Maggie Fox and Elaine Hardcastle)

thanks yvonne

2009-01-08T14:56:00.001-05:00

Have you ever seen a palm tree in the midst of a great storm or hurricane? That tree may be bent so far over that it’s almost touching the ground, but when the wind finally stops, that palm tree bounces right back up. And do you know that while that palm tree is hunched over under the pressure of the storm, it is actually growing stronger?

The reason God said we’d flourish like a palm tree is because He knew there would be difficult times. He knew things would come against us to try to steal our joy and victory. God said, “You’re going to be like a palm tree because the storms of life will come, the winds will blow, but you are going to come right back up again stronger than before.” Nothing can hold you back! No weapon formed against you will ever prosper. No matter what’s happening in the world around you, keep standing. Keep praying. Keep believing. Your brightest days are right out in front of you, and God’s plan is to bring you blessing and victory all the days of your life.

2009-01-07T16:20:00.000-05:00

January 5, 2009
Time Magazine Recognizes ALS Stem CellResearch as No. 1 Medical Breakthrough in 2008
Time magazine has named the creation of motor neurons using ordinary skins cells from people with ALS as the No. 1 medical breakthrough in 2008. The research was reported last summer by The ALS Association.
"Researchers at Harvard and Columbia reported a milestone experiment in July, using a new method - one that doesn't require embryos at all - to generate the first motor neurons from stem cells in two elderly women with Lou Gehrig's disease, or ALS," wrote Alice Park in the magazines Top 10 Everything of 2008 issue published in December.
"The technique, developed by Kyoto University scientist Shinya Yamanaka in 2006, involves reprogramming a patient's ordinary skin cells to behave like stem cells, then coaxing them into the desired tissue-specific cells," continued Park. "Using the motor neurons created from ALS patients, scientists can now study the progress of the disease as the affected cells develop, degenerate and die in a dish - something researchers could never do before for such slow-moving conditions. Once scientists understand the development of ALS, they may be able to create more effective treatments, or perhaps even a cure."
"The recognition by a major news organization of the importance of this research is gratifying," said Dr. Lucie Bruijn, science director and vice president of The ALS Association. "Advances such as this make it possible to achieve a more detailed understanding of the ALS disease process, which we need in order to design therapies."
In an article dated August 1, The Association described how stem cells had been generated from individual patients with ALS, and that the accomplishment was likely to lead to development of new models of ALS and new understanding of disease mechanisms. "They will also provide a potential resource for drug discovery and the development of new treatments for ALS," Bruijn said at the time.
"Model systems to date have focused on the SOD1 mutations linked to 2 percent of ALS. These findings enable the development of cell lines from ALS patients, even those for which the specific causative genes remain unknown," continued Bruijn. "The ability to generate human motor neurons from ALS patients carrying genes linked to the disease is a very exciting accomplishment building on novel technology and the work of several groups." Motor neurons are the nerve cells that die in ALS.
In the study recognized by Time magazine, researchers at Harvard University and Columbia University took skin cells from patients with a genetic form of ALS, caused by mutation in the superoxide dismutase (SOD1) gene. The skin cells were treated with a small set of genes that scientists have recently learned will reprogram adult cells to become stem cells capable of developing into many cell types.
The researchers showed that the genes "deprogrammed" the skin cells, reverting them to an earlier stage in their development, turning them into stem cells. Stem cells, which are formed normally during human development, have the ability to become many different kinds of cells. The researchers showed the new stem cells could transform into motor neurons.
Parallel Efforts Underway by The ALS Association
One important next step will be to make sure the motor neurons can mature, since it is mature motor neurons that are affected by ALS. Such cells could then be examined to determine what factors make them susceptible to the disease process. These studies will need to be done in cells derived from many different patients in the current work, but also in cells derived from other patients, to make sure the results are widely applicable. Another important step will be to generate stem cells from patients with non-genetic (sporadic) forms of the disease. Motor neurons derived from ALS patients will be compared with those derived from people that do not have ALS. These studies are currently underway in parallel efforts funded by The Association. See http://www.alsa.org/research/grants.cfm?grant_id=192
Mature motor neurons would likely be useful for drug discovery, looking for compounds that improve their survival. While much publicity has surrounded the possible use of stem cells to replace dying motor neurons in ALS, the potential of such a treatment is unknown and was not attempted in the study highlighted by Time magazine.
More information on stem cells in ALS can be found HERE (http://www.alsa.org/research/article.cfm?id=715).

2009-01-07T16:08:00.000-05:00

We both had done the math. Kelly added it all up and... knew she had to let me go. I added it up, and knew that I had... lost her. 'cos I was never gonna get off that island. I was gonna die there, totally alone. I was gonna get sick, or get injured or something. The only choice I had, the only thing I could control was when, and how, and where it was going to happen. So... I made a rope and I went up to the summit, to hang myself. I had to test it, you know? Of course. You know me. And the weight of the log, snapped the limb of the tree, so I-I - , I couldn't even kill myself the way I wanted to. I had power over *nothing*. And that's when this feeling came over me like a warm blanket. I knew, somehow, that I had to stay alive. Somehow. I had to keep breathing. Even though there was no reason to hope. And all my logic said that I would never see this place again. So that's what I did. I stayed alive. I kept breathing. And one day my logic was proven all wrong because the tide came in, and gave me a sail. And now, here I am. I'm back. In Memphis, talking to you. I have ice in my glass... And I've lost her all over again. I'm so sad that I don't have Kelly. But I'm so grateful that she was with me on that island. And I know what I have to do now. I gotta keep breathing. Because tomorrow the sun will rise. Who knows what the tide could bring?" ~Chuck Noland(Tom Hanks) "Castaway"

2009-01-05T16:46:00.000-05:00

There comes a point in your life when you realize who matters, who never did, who won't anymore... and who always will. Don't worry about people from your past, there's a reason why they didn't make it to your future.

'Be kinder than necessary because everyone you meet is fighting some kind of battle.'

News from SUNY Upstate Medical University-LITHIUM

2009-01-05T13:01:00.000-05:00

News from SUNY Upstate Medical University
For more information contact: Darryl Geddes, 315-464-4828
SUNY Upstate Medical University to Study If Lithium Can Slow Progress of ALS in Humans
Patients sought for clinical trial
SYRACUSE, NY (01/05/2009; 1040)(readMedia)-- SUNY Upstate Medical University is participating in a national clinical trial to determine if lithium, a mood stabilizer, can slow the disease progression of early stage Amyotrophic Lateral Sclerosis (ALS), in humans.
According to Jeremy Shefner, M.D., Ph.D., findings from a study conducted in Italy demonstrated that lithium showed neuroprotective properties in experimental animals and promising results in a small trial of ALS patients. Shefner is SUNY Upstate's lead investigator of the study and professor and chair of the university's Department of Neurology.
"To more fully study the effects of lithium in ALS, the National Institutes of Health /National Institute of Neurologic Diseases and Stroke, the ALS Association and the Canadian ALS Association has funded this important trial," Shefner said. "We will recruit volunteers to participate in the trial. Patient volunteers may be included if they are within three years of their diagnosis and are not already taking lithium. Treatment will be for up to one year."
Lithium is a simple chemical substance approved for use in humans and prescribed as a mood stabilizer. It protects neurons in the brain in animal models of neurodegenerative diseases, including Alzheimer's disease and Parkinson's disease, and has been recently shown to do the same in a mouse model of ALS. In that study, lithium prolonged survival and protected cells in both the brain and spinal cord. While the exact mechanism of lithium's effect is unknown, researchers have proposed it promotes clearance of toxic protein accumulation.
The multi-site investigation will be a double-blind, placebo-controlled trial with 84 patients who will be randomized to either lithium or placebo. A review of data will occur after the 84th person is enrolled, and then a decision will be made on whether to expand the study to 250 patients. The disease course and safety assessments will be measured at regular intervals over that time.
The trial is being supported through the ALS Association's TREAT ALS (Translational Research Advancing Therapies for ALS) initiative, a drug discovery program and clinical trials process created by the association that accelerates discovery and testing of clinical candidates. In addition to SUNY Upstate, investigators from Massachusetts General Hospital, the University of Toronto and Columbia University will participate in the trial.
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost, leading to progressive paralysis.
For more information about the study or to participate in the study, call 315-464-5004.
-30-

2009-01-04T16:20:00.000-05:00

Open Federal Funding for Stem Cell Research
Target: President-Elect Barack ObamaSponsored by: Care2
The Bush Administration's ban on federal funding for human embryonic stem cell research has set back medical research on cures for Parkinson's disease, cancer and other diseases. Fear of violating Bush's edict (even for something as minor as buying office supplies with federal funding) has caused many researchers and institutions logistical headaches. Lifting these restrictions would free researchers to spend less time being concerned about government red tape and more time focusing on research. President-elect Obama has pledged to reverse the restrictions, opening federal funding for medical research. Tell Obama now that one of his first acts as president should be to act on his commitment to stem cell research!

2009-01-03T16:11:00.001-05:00

Dear Arthur and others,

Yes, we know of the terribly unfortunate and too early death of the mother of Maria Mouskos. May the soul of Paradisa Mouskos rest in peace. I believe she is the third person involved with Team Iplex to pass away during the period of November 8, 2008 until January 1, 2009. That represents the time in which those seeking Iplex have been waiting for Insmed to release it under the extraordinarily difficult and time consuming measures specified.

The article you have forwarded: I personally feel it is well-written and a relevant view that should be considered. It has been submitted by three well-qualified representatives of the ALS neurological establishment and there are many substantiated quotations and references from other documents and resources. And, left to the article only, there could be a foundation for saying “we are all wrong in embracing this treatment called Iplex”.

I also feel it is a clear example of the ALS neurological establishment attempting to disprove or contravene the efforts of the ALS community to be self-directed in their treatment protocols when they (the ALS community) have been unable to secure appropriate treatment through their physicians. Please don’t misunderstand—that lack of “appropriate treatment” may still be, and probably is, due to the lack of any efficacious medication or other intervention devised to deal with ALS. It isn’t because of some unholy cabal that is, in any way, attempting to thwart the well-being of those with ALS. No one could possibly be that callous or lacking in humanity to do such a thing.

But there is another alternative as to why this situation exists—a “possible” pharmaceutical treatment that is embraced by the patient base and ignored or even rejected by the ALS neurological establishment. That has to do with the medical and technological barriers between physicians and patients (ALS and otherwise) that have been breaking down in the approximate 11 years of internet access by patient populations. During that time, the information boundaries that previously existed have been gradually breaking down, more patients and their families have become more aggressive in determining their own treatments, and there has actually been an osmosis of data and knowledge that has been diffused in both directions—the previously single-directed transmission of information from physician to patient to a now-existing transmission of data in both directions. There is a price to be paid for this, or rather “prices to be paid for this”. Those include: possibly erroneous information transmitted, inappropriate use of otherwise good information transmitted, a lynch-mob mentality that possibly forces the medical establishment to embrace or at least reluctantly provide such treatment, and a gradual softening of the prior hard line of “I prescribe/you listen” mentality of the medical community. I doubt that any professional group would take that lightly, least of all the medical community that has regularly “earned its stripes” through many years of training, practice and frequent sacrifice—both personal and professional. There are exceptions, of course, to this and any generalization.

Which brings me to another form of “sacrifice”—that experienced by the ALS patient community and their loved ones, caregivers, families and friends. In this form of sacrifice, they and we have watched the decline of ALS bodies from prior normalcy to diminished and malnourished shadows of their former selves. We and they have watched and suffered as their respiration, eating, drinking, limb usage, muscle strength, ability to speak, walk, sleep and countless other functions have declined or disappeared. Those of us who fall into the category other than “patient” have been to too many funerals, wakes, shivas, memorial services, dedications, remembrances and otherwise to sit by and say, “that’s ok.” And far more significantly, while we, as caregivers or family or bystanders are able to walk away muttering, our loved ones don’t walk away at all.

So what is the “disconnect” and what is the “issue”? How and why should this situation be resolved? What is going on that causes three well-intentioned researchers to write an article denouncing or at least severely minimizing a grass roots approach for the use of a biotech treatment for which some very real claims of efficacy, or at least hopes of efficacy, exist? The answers, which are so apparent, are as follows:

1. The disconnect comes from the inability of the medical establishment to think, or even imagine, that a grass roots movement, developed by non-professionals via the internet and email, could possibly recognize a relevant treatment for a previously untreatable disease. That disconnect happened with HIV-AIDS and it is still the circumstance with ALS. But we all know that without the movement engendered by the HIV-AIDS patient community and their loved ones, that disease would still not have become a condition with which people live, as opposed to that from which they die.
And ALS is, unfortunately and horribly, a condition from which everyone still dies.

2. The issue is simply whether or not the ALS community should have the right to try a medication or other treatment protocol that has at least a suggestion of efficacy and a probability of safety at least as defined as the likelihood of disrepair and death with which they are otherwise faced. Is Iplex safe—“indisputably”, in the words of an FDA representative and clearly defined in the original safety studies of Iplex, which was, after all, approved for use by infants affected with severe short growth stature at the same mg/Kg dosage levels now proposed for use by those with ALS. I think that none of you reading this will forget that those with ALS, certainly one of the most under-served and ill-protected patient communities in the history of mankind, have too much to risk and too little to gain by being the “good patient” any longer—quietly and surely succumbing to this most devastating of diseases while following the only protocol that is absolutely approved and totally without any possibility of undue expense or possible side effects—nothing. I think Iplex is the better alternative.

Best Wishes,

Stephen Byer
bsbyer@mhtc.net
ALS WORLDWIDE

2009-01-02T22:38:00.002-05:00

The New Year
Posted: 01 Jan 2009 06:21 AM CST
The new year has commenced
Its momentous journey today.From today on, during the entire year,I shall not offer my volcano-ambition
To the world.
I shall offer the world
Only my moonlit heart’s flaming aspiration.
- Sri Chinmoy

I CAN'T STRESS THIS ENOUGH

2008-12-28T19:04:00.001-05:00

ALS ON ESPN

2008-12-24T14:04:00.000-05:00

http://www.youtube.com/watch?v=jvDiqIoHi5Q&feature=related

RIP MY FRIEND-HUMBERTO

2008-12-21T13:52:00.001-05:00

WE CAN HAVE MANY LIVES, OR JUST ONE, THE FACT IS THAT WE SHOULD LIVE IT AS FULLY AS POSSIBLE BECAUSE WHEN WE GO TO ANOTHER PLAN, FLOOR, ASTRAL, LIFE, OR WHATEVER IS THE NAME, THE ONLY THING THAT LAST IS LOVE, CARE, AFET , RESPECT FOR THOSE WHO LOVED US WHILE WE WERE HERE. I KNOW THAT THE PAIN STAYS, A VACCUM APPEARS, THE FEELING OF A LACK SHOW UP, BUT IT WILL BE LESS EVERY DAY, AND ONE DAY WILL BE JUST LOVE, GOOD MEMORIES AND GOOD MISS OF YOU..MISS YOUR LAUGHTER, YOUR FUNNY WAY, THE SONGS, THE MOVIES, THE LIFE WE LIVE.
I ASK MY BELOVED ONES NOT TO SUFFDER FOR ME, I WANT YOU TO UNDERSTAND I AM IN A BETTER PLACE, DIFFERT FOR SURE, A BEAUTIFUL GARDEN FULL OF FLOWERS, WITH WONDERFUL PEOPLE WAITING FOR ME, A PLACE WHERE PASSAPORT OR VISAS ARE NOT NECESSARY,FULL OF SUSHIS, SASHIMIS, TEMPURAS, KONIS AND SAQUES (AND WITH NO HANG OVER - PERFECT!).MAYBE THERE WILL BE THAT HOUSE, WITH WHITE CURTAINS AT THE WINDOW, A WHITE SHORT PICKED FENCE, OR MAYBE THAT BEACH WITH WHITE SAND, WITH BLUE AND ESMERALD GREEN WATER, WITH THE ETERNAL SUNSHINE, I DON'T KNOW, THE ONLY THING I KNOW IS THAT IT IS MY TURN TO TAKE CARE OF YOU AGAIN, TO WATCH AND PROTECT YOU FROM THE PLACE I AM, AND WHEN YOU FELL THAT COOL BREEZE OR THAT GOOD WARM...ITS ME HERE WITH YOU. BUT I WANT EVERY ONE TO KEEP SMILING, SO THEN I WILL BE HAPPY AND KEEP DOING HERE WHAT I ALWAYS WANTED TO DO... HELP YOU ALL.
I KNOW I AM STRONG ENOUGH FOR THIS, BECAUSE THEY GAVE IT TO ME WHEN I WAS THERE WITH YOU.
19 DECEMBER 2008. TIME OF PASS: 17:00 HS.

2008-12-19T16:53:00.000-05:00

Mozobil Boosts Stem Cells Before Bone Marrow Transplants
47 mins ago
FRIDAY, Dec. 19 (HealthDay News) -- The Genzyme Corp. drug Mozobil (plerixafor) has been approved by the U.S. Food and Drug Administration to boost a person's blood stem cell count before a bone marrow transplant, the agency said in a news release.
A bone marrow transplant is often performed in people with certain forms of cancer -- multiple myeloma and non-Hodgkin's lymphoma. Before getting high doses of chemotherapy or radiation, people with these forms of cancer may be advised to have blood stem cells collected so the cells can be re-infused after the therapy. Mozobil helps increase the number of stem cells before collection.
The most frequent side effects of Mozobil reported during clinical testing included diarrhea, nausea, fatigue, reactions near the injection site, headache, joint pain and dizziness.
More information
The U.S. Food and Drug Administration has more about this drug's approval history.

my friend eddie sent this to me

2008-12-19T14:02:00.002-05:00

http://money.cnn.com/news/newsfeeds/articles/prnewswire/200812020800PR_NEWS_USPR_____NETU034.htm

Dear Friends,
Please review/double-click, the hyperlink above.
It contains some very interesting/informative information, to say the least.
Quick Excerpt:
The technology is coming. . . . .

ROCKVILLE, Md., Dec 18, 2008 /PRNewswire-FirstCall via COMTEX/ -- Neuralstem, Inc. (NYSE Alternext US: CUR) announced this morning that it has filed an Investigational New Drug (IND) application with the U.SO. Food and Drug Administration (FDA) to begin a clinical trial to treat amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).
The Company is planning to treat ALS patients through spinal injections of its stem cells via its patented Human Neural Stem Cell technology."Like all first human trials, this proposed trial is primarily designed to test the safety and feasibility of both our stem cells and our method of delivering the cells to the spinal cord in ALS patients," said Neuralstem CEO and President, Richard Garr. "We are also proposing secondary endpoints which we hope will be able to measure a slowing down of the degenerative process."Neuralstem expects to conduct the trial at Emory University with Dr. Johnathan Glass, M.D., Director of the Emory Neuromuscular Laboratory and Director of the Emory ALS Center, as site Principal Investigator (PI). Dr. Eva Feldman, M.DO., Ph.D., Head of the A. Alfred Taubman Medical Research Institute and the De Jong Professor of Neurology at the University of Michigan Medical School, will be the overall PI for the ALS trial program. Formal approvals from these institutions to conduct the trial can come only after FDA approval of the trial protocol.."The filing of this IND is an important event for Neuralstem," said Garr, "but it marks only the beginning of a process which includes working together with the FDA to approve the first human ALS stem cell trial; refining our understanding of how to optimize delivery of our cells into patients; and ultimately delivering a new treatment for patients with this currently incurable disease."About NeuralstemNeuralstem's patented technology enables, for the first time, the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells into mature, physiologically relevant human neurons and glia. Major Central Nervous System diseases targeted by the Company with research programs currently underway include: Ischemic Spastic Paraplegia, Traumatic Spinal Cord Injury, Huntington's disease and ALS. The Company filed an IND (Investigational New Drug) application with the FDA for ALS clinical trials in December, 2008, and has entered into a collaborative agreement with Albert-Ludwigs-University, in Freiburg, Germany, to develop clinical trials for Huntington's disease..In pre-clinical work, the company's cells have extended the life of rats with ALS (Lou Gehrig's disease) as reported the journal TRANSPLANTATION, in collaboration with Johns Hopkins University researchers, and also reversed paralysis in rats with Ischemic Spastic Paraplegia, as reported in NEUROSCIENCE on June 29, 2007, in collaboration with researchers at University of California San Diego.Cautionary Statement Regarding Forward Looking Information

a step in the right direction

2008-12-18T19:47:00.001-05:00

Calif. university gets $25M for stem cell research
Wed Dec 17, 5:25 pm ET
SAN FRANCISCO – Billionaire philanthropists are donating $25 million to the University of California, San Francisco toward building a new stem cell research center.
University officials announced the donation Wednesday from the Eli and Edythe Broad Foundation.
The money will help fund a $123 million laboratory building to bring all the university's stem cell research under one roof.
The Broads, who live in Los Angeles, made their wealth in housing and financial services. They have donated hundreds of millions of dollars to disease research.
Last year, the couple pledged $20 million to the stem cell institute at the University of California, Los Angeles. In 2006, they gave $25 million to establish a stem cell center at the University of Southern California.

MDA/ALS Center Director Recommends Better Planning-noninvasive ventilation --- yes or no

2008-12-18T18:00:00.003-05:00

MDA/ALS Center Director Recommends Better Planning
Using a noninvasive ventilation device in ALS may actually weaken respiratory muscles faster than not using one, a preliminary study in the March issue of Muscle & Nerve suggests. On the other hand, the use of such devices was found by the researchers to relieve fatigue, to promote a sense of mastery over the illness, and even to prolong survival. Noninvasive ventilation is a term for air delivered through natural openings in the body, such as the nose or mouth, while invasive ventilation means air delivered through a surgical hole in the neck directly into the trachea (windpipe) through a tracheostomy tube. As ALS weakens respiratory muscles, those affected often need a form of assisted ventilation.
A BiPAP machine is a form of noninvasive ventilation that can be used part-time.
A common noninvasive ventilation device is the BiPAP, which stands for "bi-level positive airway pressure." One air pressure is delivered for inhalation, another for expiration. (The name is actually a registered trademark of the Respironics Company of Pittsburgh but is widely used in a generic sense.) BiPAP machines are generally portable and convenient compared with the type of machine usually used to deliver air with a tracheostomy tube. (For more on ventilation, see "What Everyone With ALS Should Know About Breathing," ALS Newsletter, vol. 5, no. 4, 2000; and two articles on breathing and ventilation in Quest, vol. 5, nos. 5 and 6, 1998.)

COULD BiPAP BE HARMFUL?
At least two recent journal publications - a letter in the Feb. 15 New England Journal of Medicine as well as the Muscle & Nerve article - have raised questions about whether noninvasive ventilation is helpful for people with ALS, whether it's dangerous to rely on it instead of moving to tracheostomy ventilation, and what effect noninvasive ventilation has on respiratory function.
The New England Journal letter recounts the story of a man with ALS who died after a mechanical failure turned off his BiPAP system. The Muscle & Nerve article reports a small study that found that measures of pulmonary (lung) function deteriorated in people with ALS who were using noninvasive ventilation while also finding that people felt better and lived longer when they were using it.
People who were able to sleep while using a noninvasive ventilation device lived an average of 20 months from the time of the study's beginning, while those who couldn't sleep with it on lived an average of five months. The study found that measures of pulmonary muscle function and blood oxygen and carbon dioxide levels, taken when the person was off the assistive device, declined over time despite the use of noninvasive ventilation for varying periods during the night and day.
Hiroshi Mitsumoto
IMPROVED QUALITY OF LIFE
Hiroshi Mitsumoto, a neurologist and neuromuscular disease specialist who directs the Eleanor and Lou Gehrig MDA/ALS Center at Columbia Presbyterian Medical Center in New York, was part of the Muscle &Nerve study. "It sounds contradictory," Mitsumoto said of the study. Noninvasive ventilation, he says, "may make pulmonary function worse, but the patient lives longer and his quality of life is better. So the question is, 'Is it good to have BiPAP or other noninvasive ventilation, or bad to have it?'"
Mitsumoto says the situation can be compared to that of someone with weak leg muscles who wears a brace. The leg muscles may weaken faster through disuse than they would if the person were trying to use them, but his function - walking - actually improves, because the leg is supported by the brace. "I think the [noninvasive] ventilator is doing the same thing," he says. "It's providing better ventilation but deconditioning the muscles at the same time." Mitsumoto noted that more studies need to be done to verify the results of these limited observations.
BiPAP WON'T WORK INDEFINITELY
The important thing for people with ALS and their caregivers to keep in mind, Mitsumoto said, is that BiPAP or other noninvasive ventilation won't be effective indefinitely in ALS. There will come a time when respiratory failure, even with a noninvasive vent system, forces the patient or caregiver to make an emergency choice - whether or not a tracheostomy should be performed and invasive ventilation started. This kind of ventilation can achieve more control and offer better assistance to failing respiratory muscles.
When to begin thinking about moving to invasive ventilation can be a "tough question," Mitsumoto says. "Increasing dependency on BiPAP during the night and even during the day is clearly one sign," he notes. "Increasing discomfort, despite using the BiPAP, is clearly another. Patients must be followed closely by a pulmonary doctor who has extensive experience with ALS. There's no simple, cookbook answer here."
Other experts have noted that morning headaches, increasing sleepiness, a sense of mental fuzziness, and increasing anxiety, particularly when lying down, are also signs that a person may not be getting adequate ventilatory support. Shortness of breath may or may not be perceived.
After a tracheostomy, air is delivered directly to the windpipe.
PLANNING AHEAD
It's best, Mitsumoto said, to have one's thoughts in writing in an advance directive before an emergency, such as a respiratory crisis or equipment failure, arises.
It's also important, he noted, to keep emergency resuscitation bags (often called Ambu bags) close by and to make sure someone can give temporary ventilation, if that's needed, while waiting for professional assistance.
Many people with ALS, Mitsumoto said, avoid discussing end-of-life issues with their doctors and families.
"How they want to deal with their life is not fully discussed," he said, "but they keep using [noninvasive] BiPAP. Meanwhile, their condition deteriorates, and serious problems can occur."
BiPAP and other noninvasive ventilation systems, he said, are "not an answer" for long-term survival.
"If they want to live [for a long time], they have to tell the doctor, and a prophylactic trach should be done," Mitsumoto said. "There is a lot of misunderstanding and miscommunication. BiPAP is not a machine to be used for long-term survival. Its primary effect is symptomatic relief. More and more people have started depending on BiPAP to live longer, but that's really the wrong usage. The BiPAP machine was not developed for that purpose.
"When they use BiPAP, they should have a good idea of what they want to do. Advance directives should be discussed early. When a BiPAP is utilized without discussing those issues, it's problematic." While I understand the obvious limitations of NIV, it would be useful to have current and clear clinical advice to lean on given the seriousness of respiratory muscle weakness in ALS/MND and its role in sustaining life and giving impoved quality of life.

2008-12-17T19:27:00.000-05:00

The cost of a thing is the amount of what I will call life, which is required to be exchanged for it, immediately or in the long run.--Henry David Thoreau

this is CRAP

2008-12-16T15:41:00.001-05:00

Vatican affirms 'dignity of human embryo'
by Martine Nouaille Martine Nouaille
VATICAN CITY (AFP) – The Vatican on Friday reopened ethical questions surrounding stem cell research and techniques such as cloning with a document affirming the "dignity of the human embryo."
"Dignitas Personae" (Dignity of the Person), the first "instruction" on reproductive technology in more than 20 years, comes as countries including the United States and France prepare to review policies in the controversial field.
The sweeping instruction lists biomedical techniques considered "illicit" by the Roman Catholic Church such as in vitro fertilisation, cloning, the therapeutic use of stem cells, producing vaccines from embryo cells and the use of the "morning-after" contraceptive pill.
Such practices go against the "fundamental principle" that the dignity of the person must be recognised from conception until natural death, it says.
Issued by the Congregation for the Doctrine of the Faith, the Vatican's doctrinal watchdog, the 33-page instruction updates a 1987 document, "Donum Vitae" (The Gift of Life), which asserted the integrity of the human embryo.
The new instruction virtually enshrines the embryo not only as a human being but also as a whole "person" with all the philosophical and legal consequences that such recognition might entail, according to Bishop Rino Fisichella, secretary of the Congregation for the Doctrine of the Faith.
"The recognition is implicit, but we don't get involved in the philosophical debate," Fisichella said as he presented the document.
The document, approved by Pope Benedict XVI, also reprises the Church's condemnation of in vitro fertilisation, while decrying methods that prevent implantation of the embryo or cause its elimination as "falling within the sin of abortion".
"The blithe acceptance of the enormous number of abortions involved in the process of in vitro fertilisation vividly illustrates how the replacement of the conjugal act by a technical procedure ... leads to a weakening of the respect owed to every human being," the document says.
The text also warns against a "eugenic mentality" arising from advances in genetic engineering, saying: "In the attempt to create a new type of human being, one can recognise an ideological element in which man tries to take the place of his Creator."
Catholics are called to abide by such "instructions," which have had practical consequences across the centuries.
The 1987 instruction, focussing on in vitro fertilisation, was signed by the pope, then Cardinal Joseph Ratzinger, during his 24-year tenure at the head of the Vatican's highest rule-making authority.
It had important consequences for Catholic hospitals around the world as they scrapped programmes to help infertile couples, and it affected funding for certain medical research.
While the techniques condemned by the Church are legal in many countries and widely practised, the new document says Catholic researchers have the duty to distance themselves from a "gravely unjust legal situation and to affirm with clarity the value of human life".
US president-elect Barack Obama, who is to take office on January 20, is expected to act quickly to reverse an executive order by President George W. Bush banning embryonic stem cell research.
Also, French bioethics law is set for review next year.
The Holy See is aware that it is challenging cutting-edge technology, led notably by British embryo researchers, and expects "a variety of reactions," Fisichella said.
"Some will prefer to ignore (the instruction), others will take the easier route of deriding it, and still others will file these pages away as a manifestation of obscurantism blocking progress and free research, but many others will share our concern and our analysis," he said.

Vatican condemns embryo stem cell research, cloning

2008-12-13T11:26:00.001-05:00

Vatican condemns embryo stem cell research, cloning
By Philip Pullella Philip Pullella Fri Dec 12, 10:39 am ET
VATICAN CITY (Reuters) – A Vatican bioethics document Friday condemned artificial fertilization and other techniques used by many couples and also said human cloning, "designer babies" and embryonic stem-cell research were immoral.
The long awaited document from the Vatican's doctrinal body marked a big step by the Vatican into the brave new world of biotechnology, an area in which governments around the world are struggling to formulate legislation.
The document also condemned new drugs that block pregnancy from taking hold, such as the so-called "morning-after pill" and the drug RU-486, which blocks the action of hormones needed to keep a fertilized egg implanted in the uterus.
These drugs, as well as the IUD (intrauterine device), which has been in use for decades, were deemed to fall "within the sin of abortion" and are gravely immoral.
"Dignitas Personae (dignity of a person), an Instruction of Certain Bioethical Questions," is an attempt to bring the Church up to date with recent advances in science and medicine.
The document, the most authoritative of its kind from the Vatican in 20 years, said human life deserved respect "from the very first stages of its existence (and) can never be reduced merely to a group of cells."
"The human embryo has, therefore, from the very beginning, the dignity proper to a person," said the Congregation of the Doctrine of the Faith's document, approved by Pope Benedict who headed the same office before his election in 2005.
It said most forms of artificial fertilization "are to be excluded" because "they substitute for the conjugal act ... which alone is truly worthy of responsible procreation."
Condemning in-vitro fertilization, it said the techniques "proceed as if the human embryo were simply a mass of cells to be used, selected and discarded."
The highly technical document said only adult stem cell research was moral because embryonic stem cell research involved the destruction of embryos. It also condemned freezing embryos.
GOVERNMENTS GRAPPLE
Governments in countries including the United States are grappling with legislation on embryonic stem cell research.
The outgoing administration of President George W. Bush has placed restrictions on federal funds for embryonic stem cell research but President-elect Barack Obama has promised to lift them.
The 35-page document also attacked the concept of "designer babies," either by pre-implantation diagnosis during in vitro fertilization where embryos are selected before being transferred to a woman's womb, or in attempts at human cloning in the future.
It branded as "shameful and utterly reprehensible" diagnosis aimed at ensuring that only embryos free from defects or having the desired sex or other particular qualities are transferred into a woman's womb.
It condemned the concept of human cloning "to satisfy certain specific desires, for example, control over human evolution, selection of human beings with superior qualities, pre-selection of the sex of a child to be born, production of a child who is the "copy" of another, or production of a child for a couple whose infertility cannot be treated in another way."
Saying life was sacred from the moment of conception to the moment of natural death, the document also defended the Roman Catholic Church's right to intervene on such matters.
"There are those who say that the moral teaching of the Church contains too many prohibitions. In reality, however, her teaching is based on the recognition and promotion of all the gifts which the Creator has bestowed on man: such as life, knowledge, freedom and love," it said.
(Editing by Janet Lawrence)41

IGF-1 Clinical Trial Completed

2008-12-12T11:22:00.000-05:00

IGF-1 Clinical Trial Completed
By Richard Robinson, Science Writer
Subcutaneous (under the skin) delivery of insulin-like growth factor 1 (IGF-1), known as the drug Myotrophin, does not benefit people with ALS at a dose of 0.5 milligrams per kilogram of body weight, according to a large clinical trial whose results were announced.
IGF-1 is a substance the body produces to sustain motor neurons, the nerve cells that die in ALS. Experiments in animal models of the disease suggested IGF-1 treatment may delay death of motor neurons. IGF-1 was tested in ALS a decade ago in two trials, but the results of the two were inconsistent, with one suggesting treatment was beneficial, and the other showing no benefit.
“These results are deeply disappointing to all of us in the ALS community,” said Lucie Bruijn, Ph.D., senior vice president, research and development, The ALS Association. “The subcutaneous delivery route may be the key problem, or it may be that IGF-1 alone is not sufficient to rescue motor neurons.”
The current trial involved 330 people with ALS from 20 ALS treatment centers across the United States. Patients were randomly assigned to receive either IGF-1 or a placebo, injected under the skin twice a day, for two years. The dose used was the highest tolerated dose from previous studies. Neither doctors nor patients knew which treatment the patient had received until the end of the study.
At the end of the two-year treatment period, there were no differences between people with ALS who received IGF-1 and those who received placebo in muscle strength, the need for a tracheostomy for breathing, or survival, indicating that IGF-1 provided patients no benefit.
The ALS Association is the only national, not-for-profit voluntary health organization devoted solely to fighting ALS through research, patient services, advocacy and public education and information. The Association is currently exploring multiple new avenues for treatment through its TREAT-ALS (Translational Research Advancing Therapies for ALS) drug discovery program and clinical trials process.
For more information about The ALS Association’s research program, visit www.alsa.org/research.

2008-12-11T16:36:00.000-05:00

Two Wolves

One evening an old Cherokee told his grandson about a battle that goes on inside people. He said, "My son, the battle is between two 'wolves' inside us all.One is Evil. It is anger, envy, jealousy, sorrow, regret, greed, arrogance, self-pity, guilt, resentment, inferiority, lies, false pride, superiority, and ego.The other is Good. It is joy, peace, love, hope, serenity, humility, kindness, benevolence, empathy, generosity, truth, compassion and faith."The grandson thought about it for a minute and then asked his grandfather, "Which wolf will win?"The old Cherokee simply replied, "The one you feed."

POWERFUL

2008-12-09T16:59:00.002-05:00

David Hauslaib's blog

http://www.jossip.com/right-to-dies-right-to-air-20081209/
HIS POWERFUL VIDEO
http://www.telegraph.co.uk/news/uknews/3690447/Assisted-suicide-to-be-shown-on-television-for-first-time.html
Craig Ewert, 59, a retired university professor, opted for assisted suicide rather than spend the rest of his life locked in a "living tomb."

2008-12-08T16:31:00.002-05:00

"Son, if the mountain was smooth, you couldn't climb it."

Another step forward in ALS and stem cell research

2008-12-08T15:46:00.000-05:00

Another step forward in ALS and stem cell research
Harvard Stem Cell Institute team replicates Lou Gehrig's disease process in lab dish
December 5, 2008
B. D. ColenHarvard News Office
A Harvard Stem Cell Institute research team has succeeded in deriving spinal motor neurons from human embryonic stem cells, and has then used them to replicate the Amyotrophic Lateral Sclerosis (ALS) disease process in a laboratory dish.
The researchers, lead by HSCI Principal Faculty member Kevin Eggan, found that human motor neurons exposed to glial cells carrying a known genetic mutation associated with ALS died, while other types of neurons exposed to the disease-carrying glial cells were unaffected.
"The logical next step is to ask what the glial cells are doing to kill the motor neurons," said Eggan, an assistant professor in Harvard's new inter-school Department of Stem Cell and Regenerative Biology.
Eggan said that the new findings are particularly important for ALS research because they answer the field's long-standing "murder or suicide" question. That is, do motor neurons in patients with ALS - an always-fatal, neurodegeneratative condition known as Lou Gehrig's Disease - die because of something inherent to the motor neurons, do they "commit suicide?" Or, are they "murdered" - is their something external killing them? This latest research strong suggests that the motor neurons are being "murdered" by something in the glial cells carrying a mutation of the SOD1 gene.
Additionally, Eggan said, this latest study is "important for stem cell science because one of the things we've been promising is that these stem cells would be important for drug discovery. We've produced industrial quantities of these motor neurons; we've shown that this does work, that you can overcome the technical limitations. This is a disease process in a petri dish," and that's what we've been promising.
Finally, he said, this experiment once again proves the utility of human embryonic stem cells, and confirms the value of continuing to use them to study both normal development and disease process, particularly as there have yet to be studies demonstrating that alternative types of cells are identical in all respects to human embryonic stem cells.
In addition to replicating the disease process, Eggan, post doctoral fellow Paolo DiGiorgio, and colleagues Gabriella L. Boulting and Samuel Bobrowicz, demonstrated that an inflammatory pathway plays a role in the disease process, and they found a small molecule that has at least some protective effect. Eggan noted, however, than when that same compound has been test on ALS patients it has slowed the disease process. "We don't know," he said, "whether there may be other issues there, whether there are problems involving the blood-brain barrier, or dosing levels."
Less than six months ago, using induced pluripotent stem cell (iPS) technology, a team lead by Eggan, who in addition to his Harvard titles is a Stowers Medical Institute Investigator, produced patient-specific stem cell lines from the skin cells of ALS patients.
In the spring of 2007. Eggan and colleagues created an ALS model using mouse stem cells. But research findings in animal models always beg the question of whether the results will translate directly to humans. In this case, the answer to that question is a resounding Yes!
The publication of this latest paper in the journal Cell Stem Cell is only the second report in the literature of human stem cell work resulting in the replication of a disease process in a laboratory dise. That work, involving Fragile X syndrome, did not involve the derivation of a specific cell type.
The Eggan lab's work was funded by Project ALS, with additional support from the Stowers Medical Institute and the New York Stem Cell Foundation.

Hi Team Iplex,

2008-12-06T20:54:00.000-05:00

Hi Team Iplex,

I have attempted to explain the process by which Iplex will be distributed in the USA. I am still unclear about out of country delivery of Iplex, and will follow up with Christine O’Neal first thing Monday to try to establish what the protocol for members of Team Iplex outside of the USA will be.I have set first to explain some of the language, which may be unfamiliar to some of us, with links and definitions. For those of you in the pharmaceutical industry, please jump in and fill in any missing links, and please feel free to try to simplify my definitions if you see a way to explain this more clearly. In brief, as many of us are aware, Iplex will be available in the USA by a process called a single patient IND, also commonly called Compassionate or Emergency use. This is a process which sounds very complex, but in reality is nothing more than a lot of paperwork, don’t be alarmed or frightened by it, it’s just paperwork…One of the first questions I have had is cost. When a drug is accessed by a single patient IND the FDA allows the drug manufacturer to recoup reasonable costs only. When I spoke to Christine Friday, she indicated that cost had not yet been set. I have no idea at this time what the monthly costs will be. Many also want to know about insurance reimbursement. At this point, I feel that insurance reimbursement is highly unlikely. I do not know of any insurance company that will pay for any of the costs associated with a single patient IND – that includes associated MD visits, labs, etc. I wish I had better news to report, but this is the unfortunate facts of the matter. A Single patient IND (again, AKA compassionate/emergency use) requires the approval of an IRB. Most of us aren’t familiar with what an IRB is. Here is a brief definition, as supplied by a Wikipedia link and my definition.Institutional Review Boards (IRB's): http://en.wikipedia.org/wiki/Institutional_review_boardEvery hospital/teaching facility that does research involving human subjects must have an IRB that initially reviews/approves proposed research. An IRB protects the ethical interests of humans involved in trial studies. Before it will approve a single patient IND, the IRB will determine that a our risks are reasonable in relation to anticipated benefits – as Iplex is already FDA approved, and has passed rigorous safety testing in other diseases, this shouldn’t be a very complex process. The FDA has already approved this process for Insmed, so their role should be simply shuffling of papers, I don't expect lengthy reviews on their end.Now, many of you may have physicians who are not affiliated with a teaching hospital, or do have a physician who conducts research and has association with an IRB, but who won’t write for Iplex, and are curious about what you can do to access an IRB. Fortunately for those of you, there are independent IRBs. One of the larger independent Institutional Review Boards that may be accessed by physicians not within the realm of academia is Western, their website is here http://www.wirb.com/. There are fees associated with this service, I cannot comment on the pricing, but would suggest you contact Western, or do a web search to locate any independent IRB. Your private physician may also be familiar with an independent IRB, and already have a established relationship with one?So, in summary, here is the process for a Single Patient IND, there are several forms which will be filled out, submitted to the FDA by your doctor, once all of the t’s are crossed and the I’s dotted, the drug will be shipped to your doctor. In my experience with government bureaucracy – it is important the forms be filled out completely and accurately. This is what you should expect your doctor to submit to the FDA.Request for a single patient IND for Compassionate or Emergency Use should be stated at the top of the correspondence. Brief Clinical History of the patient including the diagnosis, the disease status, prior therapy, response to prior therapy and the rationale for requesting the proposed treatment. Proposed Treatment Plan including the dose, route, planned duration, monitoring procedures and modifications (e.g. dose reduction or treatment delay) for toxicity. Reference a published protocol or journal article if appropriate. Drug Supply Reference Statement which would name the supplier or manufacturer and a statement that a Letter of Authorization to cross reference an appropriate IND of the supplier or Drug Master File (DMF) of the manufacturer is included. The treating physician must contact the supplier or manufacturer for such a statement. Informed Consent Statement that states that informed consent and approval of an appropriate Institutional Review Board (IRB) will be obtained prior to initiating treatment. There are some IRBs that have specific procedures for approving emergency requests. Investigator Qualification Statement that specifies the training, experience, and licensure of the treating physician. The first two pages of a Curriculum Vitae (a fancy name for resume) typically contain this information and are usually sufficient. FDA Form 1571 completed with the treating physician listed as the sponsor. Form 1571 and other forms can be downloaded from the Internet. Contact telephone number and facsimile number. If the request is approved, an IND number will be issued by the FDA and the treating physician will be contacted by phone or fax with a letter to follow. The IND is considered active upon issuance of the number. The IND sponsor (treating physician) will then contact the drug supplier and provide the IND number. The supplier may then ship the drug directly to the treating physician.Please feel free to email or call me if you want me to try to explain better. I have never been accused of being a good teacher, I apologize if this isn't as clear as it could/should be - but will be happy to answer any questions to the best of my ability.Andrea--"Being deeply loved by someone gives you strength, while loving someone deeply gives you courage." Lao Tzu Taoist Philosopher,600 BC-531 BC

--------------------------------------------------------------------------------

2008-12-05T19:11:00.001-05:00

Enclosed is the report from the government panel admitting the causes GULF WAR SYNDROME/ALS…

http://articles.latimes.com/2008/nov/18/science/sci-gulfwar18
http://articles.latimes.com/2003/sep/23/science/sci-als23

http://www.alsrecovery.com/lougehrigsdisease/?p=2806

http://news.google.com/news?hl=en&tab=wn&ned=us&ie=ISO-8859-1&ncl=1271386335

Dear Team Iplex, Dear Team Iplex,

2008-12-05T18:17:00.000-05:00

Dear Team Iplex,
Here is the briefest recap of my conversation with Dr Allan late yesterday:
1. Insmed is proceeding immediately with a Personal Physician IND (Investigational New Drug), or Compassionate Usage, program for Iplex availability by ALS patients world-wide.
2. This program is being offered after the consideration, and rejection for reasons of timeliness or other hindrances, of either "an approved drug being returned to the workplace" or a "Treatment IND". It is felt by Insmed that the prescribed method, a "Personal Physician IND", will accommodate the needs of the ALS community faster and easier for them.
3. Details of the program are being emailed directly to all patients and/or caregivers immediately. This includes both those who have contacted Insmed directly, usually to the attention of Christy O'Neal, and those whose contact information has previously been submitted by ALS WORLDWIDE or others.
4. Further information will be available on the www.Insmed.com website as of Monday, December 8, 2008.
5. The details of a physician letter that must be provided to the US FDA (Food and Drug Administration) will be linked on the Insmed website and in the individual letters being sent to all patients and caregivers whose names have been provided directly or through other channels. I am not including what I believe to be the accurate section(s) of the FDA.com website because I do not want to misstate or inaccurately misdirect anyone to inappropriate sections of a very large and complex website.
6. ALS WORLDWIDE will continue to provide either contact or organizational support to anyone in the ALS community who needs or requests that support. This can, and will, include suggested dosage information and the assistance or support of a physician who can oversee the use of Iplex in case the patient is unable to procure such assistance from their local family physician or neurologist. Please understand this assistance is in no way as an agent or representative of Insmed-it is strictly as volunteer support to the ALS community and is provided as a courtesy to an underserved patient community. There is no charge or compensation in any form.
7. A single-page letter from the attending physician will be required by FDA and its details will also be either directly stated in the Insmed website or linked to the FDA website as of Monday, December 8, 2008.
8. The results of the Italian Observational Study are not yet available. The observed patients' information is now being compared to historical placebo history for comparative understanding. This is a common technique when double-blind study comparisons are unavailable.
9. Patients from countries other than the US are advised to provide their individual country equivalent of US FDA with the same information as required by US FDA. Alternatively, their US physician representative can submit their request to US FDA if a US address is provided.
10. The final price charged for Iplex, and the method by which such charge is collected, have yet to be determined. My understanding is that "collection of money for the initial shipment(s) of Iplex will not stand in the way of its distribution to the ALS patients." I don't know how long this view will remain in effect, and because the likelihood of private, state or VA insurance coverage is always in question or even improbable, ALS Worldwide/Team IPLEX will be continuing our efforts to secure some form of corporate or foundational assistance for those not covered by insurance. We have not yet received any commitment of such participation and, as stated, we do not yet know what the eventual charge for Iplex will be or when it will be implemented.
Other points were discussed-they are either not relevant or less specific than the above information. We are traveling today through Wednesday, December 10 but are always reachable by email or cell phone. Please feel free to extract any or all parts of the above synopsis in your own individual communications to others within the ALS community.
Best Wishes,Stephen Byer
bsbyer@mhtc.net
ALS WORLDWIDE
ALS WORLDWIDE is a non-profit organization that provides support to ALS families internationally
through scientific research interpretation, individual patient advocacy and community activism.

Study raps Web sites touting stem cell therapies

2008-12-04T15:16:00.001-05:00

Study raps Web sites touting stem cell therapies
By MALCOLM RITTER, AP Science Writer Malcolm Ritter, Ap Science Writer Wed Dec 3, 3:10 pm ET
NEW YORK – Consumers should be wary of Web sites from clinics that offer stem cell treatments, says a study that found a lack of firm medical evidence to back up their claims. The Web sites in the study generally portrayed their therapies as safe, effective and ready for routine use, but published research doesn't support that "overoptimistic" picture, the study authors said.
The analysis is presented in the December issue of the journal Cell Stem Cell by scientists at the University of Alberta in Canada. They cautioned that their overall findings can't be applied to the claims of any individual clinic.
The study is "a very important wake-up call," said Dr. George Daley, past president of the International Society for Stem Cell Research, who had no role in the new report.
"I think these Web sites are dangerous," said Daley, a Boston stem cell researcher. "They overpromise effectiveness and safety of the therapy and they completely underestimate and underinform about risks. ... (Such) overhyped marketing directly to the patient is putting patients at risk of financial exploitation at the very least, and physical danger at the worst."
In recent years, desperate patients with few options have traveled to China and other countries where doctors offer stem cell or other cell treatments for such things as spinal cord injuries, Parkinson's disease and blindness.
The new study did not assess the Web site claims directly by checking on how well patients actually fared at the clinics in the study.
Instead, researchers began with the 19 Web sites they found through Google in 2007. Treatments were promoted in several countries, including China, Mexico and Russia. None promoted treatments within the United States; one didn't give a location for treatments.
Last July, the researchers looked for published studies in human patients about using stem cells to treat the medical conditions mentioned most often by the Web sites: multiple sclerosis, Parkinson's and Alzheimer's diseases, spinal cord injury, stroke and heart attack.
They reported finding some encouraging hints but no clear evidence of benefit.
In the same issue of the journal, a report from the international stem cell society describes new research guidelines that condemn the marketing of unproven therapies. The society has posted a patient handbook on its Web site to help people who are considering stem cell therapy.
The guidelines say that in limited cases, doctors may be justified in trying an experimental treatment outside of a formal study for small numbers of seriously ill patients. The guidelines recommend standards for that situation, such as approval from a group of experts with no vested interest in the treatment and a commitment by those offering it to proceed to a formal study.
___
On the Net:
Cell Stem Cell: http://www.cell.com/cell-stem-cell/home
Stem Cell society: http://www.isscr.org
Patient handbook: http://www.isscr.org/clinical_trans/pdfs/ISSCRPatientHandbook.pdf

Treatment of amyotrophic lateral sclerosis patients by autologous bone marrow-derived hematopoietic stem cell transplantation: a 1-year follow-up.

2008-12-03T16:16:00.000-05:00

Treatment of amyotrophic lateral sclerosis patients by autologous bone marrow-derived hematopoietic stem cell transplantation: a 1-year follow-up.
Deda H, Inci M, Kurekci A, Sav A, Kayıhan K, Ozgun E, Ustunsoy G, Kocabay S.
Department of Neurosurgery and Neurology, Akay Hospital, Ankara, Turkey.Background Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by progressive loss of spinal cord and cortical motoneurons. Despite improved understanding of the mechanisms underlying ALS, in clinical practice the management of ALS remains essentially supportive and focused on symptom relief. However, over the past few years stem cell research has expanded greatly as a tool for developing potential new therapies for treating incurable neurodegenerative diseases. Methods Thirteen patients with sporadic amyotrophic lateral sclerosis (SALS) were included in this study, and bone marrow (BM)-derived hematopoietic progenitor stem cells were used. We selected patients with bulbar involvement and severe loss of movement. Our aim was to put the stem cells into the end of the brain stem and at the beginning of the spinal cord because the blood-brain barrier is intact in ALS and this region was the most affected part in our patients. Under general anesthesia, a total laminectomy was performed at the C1-C2 level. Stem cells were injected to the anterior part of the spinal cord. Results During the follow-up of 1 year after stem cell implantation, nine patients became much better compared with their pre-operative status, confirmed by electro neuro myography (ENMG). One patient was stable without any decline or improvement in his status. Three patients died 1.5, 2 and 9 months, respectively, after stem cell therapy as a result of lung infection and myocardial infarction (MI). Discussion These results show that stem cell therapy is a safe, effective and promising treatment for ALS patients.

http://www.ncbi.nlm.nih.gov/pubmed/19012065?dopt=Abstrac

2008-12-02T21:55:00.000-05:00

First trial in patients with a potential treatment of the incurable ALS muscle disease
2008-12-01 09:00
Leuven (Belgium), Stockholm (Sweden) - Permission has been granted to start the first safety and tolerability trial on patients for a remedy for ALS. ALS is an incurable, paralyzing neurodegenerative disorder that strikes 5 persons in every 100,000. The disease commonly affects healthy people in the most active period of their lives - without warning. Researchers from VIB at the K.U.Leuven have previously shown the possibilities for the use of VEGF in the treatment of ALS through work in animal models. The Swedish Biopharmaceutical company NeuroNova has already built upon this research. Together with UZ Leuven they'll start the first evaluation of safety and tolerability of the drug in patients by the end of this year. This is an important step in the development of a new treatment. It will take several years before the protein can be made available as a medicine.
An incurable disease of the muscles
Amyotrophic Lateral Sclerosis (ALS) can strike anyone. The Chinese leader Mao Tse Tung, Russian composer Dimitri Shostakovich, the legendary New York Yankee baseball player Lou Gehrig, and astro-physicist Stephen Hawking have all been afflicted with ALS. About half of the patients dies within three years - some even in the first year - usually as a consequence of suffocation.
In ALS, the patient's nerve bundles that extend to the muscles deteriorate. As a result the patient loses control of the muscles, and progressively becomes paralyzed. The originating mechanism of this deadly disease of deterioration - which has an enormous medical and social impact - remains obscure. At present, the disease is totally untreatable.
VEGF: a promising candidate drug
VEGF is a substance that controls the growth of blood vessels. Unexpectedly, VEGF also helps neurons survive under stressful conditions. In 2001 Peter Carmeliet's team showed that too little VEGF causes ALS-like symptoms in mice. Later the group of Diether Lambrechts, Wim Robberecht and Peter Carmeliet showed that persons who produce too little VEGF - due to certain variations in the gene that codes for VEGF - have a higher risk of developing ALS. This was the starting point of a search for a possible treatment with the VEGF protein.
Testing the treatment on rats with a severe form of ALS and on rats with a milder form, the researchers found that, in both groups, the VEGF-treated rats manifested the disease later than the untreated animals, and they lived considerably longer.
Using a pump
The researchers also investigated what the optimal technique would be for administering VEGF. An ordinary injection proved to be ineffective. But continuous administration of the VEGF protein directly into the cerebrospinal fluid (the fluid that circulates around the brain and the spinal cord) was quite effective. This was possible by means of a small pump that continuously pumps the VEGF protein in the brain. Furthermore, this technique permits a patient-oriented approach by enabling the administered dose of the VEGF protein to be easily controlled.
A story with several players
These encouraging and promising results were only the first steps on the way to a new remedy. Anders Haegerstrand and his team of the Swedish company NeuroNova have taken the development of the treatment further. After additional studies this research has reached the stage of starting the first trial in patients. Wim Robberecht (UZ Leuven) and Markus Jerling (NeuroNova) will co-ordinate this first trial which is intended to evaluate the safety and tolerability of the drug and the infusion system. It is planned to start at the end of this year, and the investigator Dr Robberecht is currently looking for patients who are eligible for participation. These regulated studies on ALS patients will have to demonstrate the safety of the VEGF administration, and in a later stage the efficacy of VEGF as ALS therapy, before the protein can be made available as a medicine. Such procedures can easily last several years.
Questions
Given that this research can raise a lot of questions for patients, we ask you to please refer questions in your report or article to the email address that VIB makes available for this purpose: patienteninfo@vib.be . Everyone can submit questions concerning this and other medically-oriented research directly to VIB via this address.
For practical information concerning the clinical trials you can contact Petra Tilkin (petra.tilkin@uzleuven.be).
More information
For more information on this project, please contact
the VIB Communication Service: +32 9244 66 11
Peter Carmeliet: +32 16 34 61 42 of +32 475 87 13 79
Wim Robberecht: +32 16 33 07 70 (0486 09 85 69)
Anders Haegerstrand, CSO, NeuroNova: +46 8786 0900, anders.haegerstrand@neuronova.com
Markus Jerling, NeuroNova: + 46 8786 0900, markus.jerling@neuronova.com
For more information on NeuroNova, please contact
Ulf Ljungberg, NeuroNova: +46 8786 0900, ulf.ljungberg@neuronova.com
More info on
Relevant scientific publications
Lambrechts et al., Meta-analysis of VEGF variations in ALS: increased susceptibility in male carriers of the -2578AA genotype (J. Med. Genet., epub, July 2008)
Zacchigna et al., Neurovascular signalling defects in neurodegeneration (Nature reviews Neuroscience, Vol 9, March 2008, 169-181)
Lambrechts and Carmeliet, VEGF at the neurovascular interface: Therapeutic implications for motor neuron disease (Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, Vol 1762, Issues 11-12, November-December 2006, 1109-1121)
Storkebaum et al., Treatment of motoneuron degeneration by intracerebroventricular delivery of VEGF in a rat model of ALS (Nature Neuroscience, Vol 8(1), January 2005, 85-92)
Azzouz et al., Deletion of the hypoxia-response element in the vascular endothelial growth factor promoter causes motor neuron degeneration (Nature, vol 429,27 May 2004, 413-417)
Lambrechts et al., VEGF is a modifiere of amyotrophic lateral sclerosis in mice and humans and protects motoneurons against ischemic death (Nature Genetics, Vol 34(4), August 2003, 383-94)
Skene & Cleveland, Hypoxia and Lou Gehrig (Nature Genetics, vol 28, June 2001, 107-108)
Oosthuyse et al., Deletion of the hypoxia-response element in the vascular endothelial growth factor promoter causes motor neuron degeneration (Nature Genetics, Vol 28, June 2001, 131-138)
Note for the editor
The fundamental preclinical VEGF research was done by Diether Lambrechts, Wim Robberecht and Peter Carmeliet of the VIB Vesalius Research Center, K.U.Leuven, under direction of Peter Carmeliet (www.vib.be/Research/EN/Research+Departments/Vesalius+Research+Center, www.vib.be/Research/EN/Research+Departments/Vesalius+Research+Center/Peter+Carmeliet )
The further development of the research is done by NeuroNova (www.neuronova.com). The clinical trial is done in collaboration with Wim Robberecht, linked to UZ Leuven (www.neurology.kuleuven.be) and the research group ‘Neurobiology' of the VIB Vesalius Research Center, K.U.Leuven (More info: www.vib.be/Research/EN/Research+Departments/Vesalius+Research+Center/Wim+Robberecht).
VIB
VIB, the Flanders Institute for Biotechnology, is a non-profit research institute in the life sciences. Some 1100 scientists and technicians conduct strategic basic research on the molecular mechanisms that control the functioning of the human body, plants, and micro-organisms. Through a close partnership with four Flemish universities - Ghent University, the Katholieke Universiteit Leuven, the University of Antwerp, and the Vrije Universiteit Brussel - and a solid investment program, VIB unites the forces of 65 research groups in a single institute. Their research aims at fundamentally extending the frontiers of our knowledge. Through its technology transfer activities, VIB strives to convert the research results into products for the benefit of consumers and patients. VIB also develops and distributes a broad range of scientifically substantiated information about all aspects of biotechnology. More info at: www.vib.be.
K.U.Leuven
The University of Leuven is Belgium's largest university and one of the oldest universities in Europe, founded in 1425. It is a comprehensive university with 14 faculties, with a long tradition of high-quality interdisciplinary research and teaching. The University of Leuven has over 33,000 students (12 percent international) and over 17,000 staff members (8,600 in the various university departments and 8,700 at UZ Leuven, the university hospital). More info at: www.kuleuven.be
NeuroNova
NeuroNova (www.neuronova.com) is a Swedish bio-pharmaceutical company based in Stockholm, Sweden. NeuroNova has two drug candidates nearing clinical development for Parkinson's disease and ALS. NeuroNova works with neurogenesis and neuroprotection for the treatment of several currently incurable neurodegenerative diseases, including Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis (ALS) and Huntington's disease.
UZ Leuven
UZ Leuven is a university hospital with a reputation throughout Europe as a centre of medical excellence. High-quality, customised patient care, multidisciplinary cooperation, innovation and continuous training all go hand in hand at UZ Leuven. Thanks to the dedication and drive of our motivated staff, we are able to achieve our mission day in, day out.
Mention both VIB and the university
When reporting this research, please always mention VIB as well as the university concerned.

http://www.newsdesk.se/pressroom/neuronova/pressrelease/view/first-trial-in-patien...

2008-11-30T21:35:00.000-05:00

Read this post on a diferent forum and thought I should post it here. This letter was sent by Ron Gunn at INSMED who controll the IPLEX trial in Italy. INSMED Company Statement Regarding IPLEX™ (rhIGF-I/rhIGFBP-3) ALS Expanded Access Treatment Program in Italy This Company Statement is intended to communicate the activities of our ongoing IPLEXTM Expanded Access Treatment Program (EAP) for patients with Amyotrophic Lateral Sclerosis (ALS) in Italy. IPLEX (rhIGF-I/rhIGFBP-3) IPLEX is the trademark for the drug known as rhIGF-I/rhIGFBP-3. IPLEX is a synthetic complex of the growth factor, insulin like growth factor-I also known as IGF-I and the major protein in the bloodstream, IGFBP-3, which regulates the actions of IGF-I. IPLEX was approved by the US Food and Drug Administration in 2005 for a rare growth disorder unrelated to ALS. Until the initiation of the EAP, IPLEX had never been investigated for the treatment of ALS. ALS, often referred to as Lou Gehrig’s disease, is a progressive neurodegenerative disorder that affects nerve cells in the brain and spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The progressive degeneration of the motor neurons in ALS eventually leads to their death. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle movement action progressively affected patients in the later stage of the disease may become totally paralyzed. IGF-I is a neurotrophic factor which has been shown to be essential for normal development of the nervous system. In animal models, IGF-I has been shown to protect motor neurons from injury and promote muscle and nerve regeneration. IGF-I has been studied as a potential treatment for patients with ALS over the last decade. Two randomized controlled trials have been conducted with a product known as MyotrophinTM which is a synthetic version of IGF-I. The larger trial showed slowing of the progression of functional impairment and improvements in quality of life. The second, smaller trial, showed similar trends but these trends did not reach statistical significance. A third trial is currently underway in the United States with Myotrophin, the results of which may be reported sometime this year. Therefore at this point in time, it is unclear as to whether IGF-I is effective in slowing disease progression in patients with ALS. There is evidence that IPLEX is different from the synthetic forms of IGF-I. The binding protein IGFBP-3, which is a component of IPLEX, modulates the activity of IGF-I. One of the roles of IGFBP-3 is to prolong the length of time that IGF-I circulates in the bloodstream. Incorporating IGFBP3 also blocks the hypoglycemic action (blood glucose lowering action) of IGF-I which allows higher doses of IGF-I to be administered. In order to be an effective treatment of ALS it is important for IGF-I to cross the blood brain barrier. It is possible that by having higher amounts of IGF-I circulating in the bloodstream for a longer period of time, greater amounts of IGF-I may penetrate into the brain. In addition, it could be possible that the IGFBP-3 contained in IPLEX helps to transport IGF-I directly into the brain. For these reasons it is important to study the effects of IPLEX as a treatment for ALS. Expanded Access Program in Italy The Agenzia Italiana del farmaco (AIFA) first contacted Insmed in the summer of 2003 about providing IPLEX to patients in Italy. AIFA stated that the Italian Court had examined the clinical documents of an adult patient believed to be suffering from ALS and had decided that he must be treated with IGF-I. We agreed to provide IPLEX at a price to cover our cost to the first patient in Italy in the summer of 2004. In the fall of 2006, we began receiving several new requests from physicians of patients in Italy diagnosed with ALS who received court orders stipulating IPLEX treatment. At that time we, with AIFA’s guidance, established a formal Expanded Access Program to provide IPLEX to physicians for their patients. As part of this program, physicians are required to collect and provide safety information as well as information pertaining to the progression or improvement in the functional manifestations of the disease prior to beginning treatment and every 3 months thereafter. The functional manifestations of the disease are being evaluated using an ALS Functional Rating Scale which is designed to evaluate speech, salivation, swallowing, handwriting, cutting foods and handling utensils, dressing and hygiene, turning in bed and adjusting bed cloths, walking, climbing stairs and respiratory function. The purpose for collecting this information is to help ensure the safety of patients and to gain an understanding as to whether or not IPLEX treatment provides benefit to these patients. There are approximately 30 patients who are currently enrolled in the EAP with additional patients being added periodically. These patients are located throughout Italy from the Calabria region in the south to the Lombardy region in the north and in Sicily and Sardinia as well. Most patients have been receiving 1 injection of 1 vial of IPLEX per day. For a 60 kg person this is equal to 0.5 mg/kg of IPLEX which contains approximately 0.10 mg/kg of rhIGF-I. Patients in the Myotrophin studies received 2 injections of 0.05 mg/kg of rhIGF-I a day for a total daily dose of 0.10 mg/kg. Therefore, to date, most of the patients in the EAP have received the same total daily amount of rhIGF-I as the patients in the Myotrophin studies. So far this dose has been well tolerated and we have not had any reports of serious side effects related to the treatment. We are in the process of having physicians increase the dose to 1 mg/kg and maintain the patients at this dose in order to evaluate the effectiveness of the drug. For most of these patients, the process for obtaining IPLEX begins with patients petitioning the Italian Court for treatment. Following a court decision stipulating IPLEX treatment, Insmed is contacted by the treating physician or the patient’s pharmacist. We in turn, provide instructions to the physician and/or pharmacist along with the relevant documentation for the physician and pharmacist to fill out. Once all of the required documents and import licenses are in place we ship IPLEX to the patient’s pharmacy. Insmed recognizes that ALS is a devastating disease and that there are patients throughout the world who are in need of an effective treatment. However, the Expanded Access Program is restricted exclusively to Italy through license agreements with companies who hold the patent rights for the manufacture and use of IGF-I products in the treatment of ALS. Insmed does not currently have the rights to manufacture or distribute IPLEX for the treatment of ALS outside of Italy. Therefore we are unable to provide IPLEX to physicians for the treatment of their patients anywhere else in the world.

when you make the "THE PRAYER LIST" you know you're sick.....

2008-11-26T19:49:00.003-05:00

To all: Please pray for the following:
1-Jenna-Thanksgiving-successful surgery and clean MRI
2-Brian Moran, KM-repose of soul
3-Melody Garges-now in hospice-Melody has been battling cancer and side efects for so many years. Her parents, Tom and Sarah Flood, are two of the greatest people/parents in the world-and great examples of faith within the Order of Malta. Please pray and storm the heavens for special healing for Melody and God's Peace as well!
4-Baby Presley-special healing
5-Drew S.-special healing
6-Kirsten Lawley-38 year old mother with breast cancer and 4 children under 9.
7-JB-cataract surgery on Tuesday
8-A D'A--Alzheimer's disease
9-Seton Fell-special healing
10-Kim Renzi-special healing
11-Jim O'S-special healing
12-Kevin H-special intentions
13-Kennedy S.-special healing
14-Devon Lam-Thanksgiving- Devon has stabilized and may be returning home this week. Continue to storm the heavens for wee Devon!
15-Mike P.-guidance for right treatment of lung cancer
16-Kari-special intentions
17-Jonathan R.-special intentions
18-Keith Cassidy-ruptured appendix
19-Joanne P-Thanksgiving-prayers answered, but still 5 more days for novena!

2008-11-25T22:27:00.000-05:00

Drug used by ALS patients gets closer to distribution
By Elizabeth SimpsonThe Virginian-Pilot© November 26, 2008
A drug that some patients with a degenerative nerve disease have been clamoring to get for more than a year has moved a step closer to being freed for distribution.
Iplex, made by Richmond-based company Insmed, had been used in an off-label fashion by some patients with amyotrophic lateral sclerosis, commonly known as Lou Gehrig's disease.
But it was taken off the U.S. market in 2007 when California-based company Genentech sued Insmed, saying it used a component licensed exclusively to another drug company, Tercica.
That resulted in an outcry from patients who were using Iplex and others who wanted to. People with ALS progressively lose control of their voluntary muscles, such as swallowing and breathing, and eventually become paralyzed. Life expectancy after diagnosis is usually two to seven years.
In response to patients' concerns, Genentech, Tercica and Insmed signed a letter of intent earlier this month freeing Insmed to supply the drug if it receives regulatory permission from the U.S. Food and Drug Administration.
The drug still has not been through rigorous testing, nor has it been approved by the FDA for use by ALS patients. People who want the drug are hopeful that will be the next step. A phone call to Insmed for comment on such progress was not returned Tuesday.
Attorneys at the local office of the Richmond-based Williams Mullen recently got involved with the effort to free the drug for distribution after hearing that Josh Thompson, a Virginia Beach resident and son of prominent developer Bruce Thompson, wanted access to the drug.
Elizabeth Simpson, (757) 446-2635, elizabeth.simpson@pilotonline.com
http://hamptonroads.com:80/2008/11/drug-used-als-patients-gets-closer-distribution

the rebuttal

2008-11-25T22:08:00.001-05:00

Blu,
IPLEX is way more than just a growth hormone, better said it's -- IGF-I with a synthetic that binding protein BP3. -- I won't even go into the under dosing aspects of clinical trials .
I totally understand that most people are caught in the middle of uncertainty a doubt created by the so called "Scientific Community" which have as a job to burst any bubble of hope. No doctor is willing to support anything unless they see absolute & categoric "evidence" because lawyers are their biggest concern. I wonder if Dr. Sorensen from the Mayo Clinic or any other neurologists at any of the major neuromuscular centers in the United States (or the entire world for that matter) are willing to share their justification (or evidence) to support prescribing Rilutek, which again almost every major neurologist in the world and least officially supports.
Like I said, doctors are paralized by the fear of getting sue. Most are not willing to go outside their little comfort zone, in the name of "evidence" and are absolutely not willing to stick out their necks for what is right, concerning their patients.
Our medical model (just like the rest of our country's systems and processes) are being held hostage by lawyers, big pharma and special interests. It is a system that practices a profession based on fear, arrogance , cash, and absolutely no guts.
Very few doctors are brave enough to stand up to the official way of thinking, at the expense of being labeled quacks, getting sued or losing credibility with colleagues.
But they're are out there. There is an organization www.acam.org which lists many innovative doctors across the country, I believe that once they're brought up to speed on IPLEX they will be more than willing to prescribe it
But that's just one man's opinion .
Sincerely,
Eddie Spaghetti) Esparza
"Si Se Puede"(YES WE CAN!!)
Cesar Chávez

Disappointing Results from IGF-1 Clinical Trial

2008-11-25T22:05:00.000-05:00

Disappointing Results from IGF-1 Clinical Trial
By Richard Robinson, Science Writer
Subcutaneous (under the skin) delivery of insulin-like growth factor 1 (IGF-1) does not benefit people with ALS at a dose of 0.5 milligrams per kilogram of body weight, according to a large clinical trial whose results were released today.
IGF-1 is a substance the body produces to sustain motor neurons, the nerve cells that die in ALS. Experiments in animal models of the disease suggested IGF-1 treatment may delay death of motor neurons. IGF-1 was tested in ALS a decade ago in two trials, but the results of the two were inconsistent, with one suggesting treatment was beneficial, and the other showing no benefit.
“These results are deeply disappointing to all of us in the ALS community,” said Lucie Bruijn, Ph.D., senior vice president, research and development, The ALS Association. “The subcutaneous delivery route may be the key problem, or it may be that IGF-1 alone is not sufficient to rescue motor neurons.”
The current trial involved 330 people with ALS from 20 ALS treatment centers across the United States. Patients were randomly assigned to receive either IGF-1 or a placebo, injected under the skin twice a day, for two years. The dose used was the highest tolerated dose from previous studies. Neither doctors nor patients knew which treatment the patient had received until the end of the study.
At the end of the two-year treatment period, there were no differences between people with ALS who received IGF-1 and those who received placebo in muscle strength, the need for a tracheostomy for breathing, or survival, indicating that IGF-1 provided patients no benefit.
The ALS Association is the only national, not-for-profit voluntary health organization devoted solely to fighting ALS through research, patient services, advocacy and public education and information. The Association is currently exploring multiple new avenues for treatment through its TREAT-ALS (Translational Research Advancing Therapies for ALS) drug discovery program and clinical trials process.
For more information about The ALS Association’s research program, visit www.alsa.org/research.

Foes of stem cell research now face tough battle

2008-11-24T23:00:00.000-05:00

Foes of stem cell research now face tough battle
By KEVIN FREKING, Associated Press Writer Kevin Freking, Associated Press Writer Sun Nov 23, 12:05 pm ET
WASHINGTON – When the Bush presidency ends, opponents of embryonic stem cell research will face a new political reality that many feel powerless to stop.
President-elect Barack Obama is expected to lift restrictions on federal money for such research. House Speaker Nancy Pelosi, D-Calif., also has expressed interest in going ahead with legislation in the first 100 days of the new Congress if it still is necessary to set up a regulatory framework.
"We may lose it, but we're going to continually fight it and offer the ethical alternative," said Rep. Joe Pitts, R-Pa. "I don't know what the votes will be in the new Congress ... but it's very possible we could lose this thing."
Stem cells are the building blocks that turn into different kinds of tissue. Embryonic stem cells,e unlike more mature versions, are blank slates. If scientists could control them, they could direct regenerative therapy, perhaps allowing a diabetic's pancreas to begin produce insulin, for example.
Harvesting stem cells from four- or five-day-old embryos kills the embryo, which outrages opponents of this type of research. But supporters say hundreds of thousands of embryos stored in fertility clinics eventually will be destroyed anyway and that people should be allowed to donate them for research that could help others.
"I believe that it is ethical to use these extra embryos for research that could save lives when they are freely donated for that express purpose," Obama wrote during the campaign in response to 14 questions from scientists, doctors and engineers.
Under President George W. Bush, federal money for research on human embryonic stems cells was limited to those stem cell lines, or families of constantly dividing cells, that were created before Aug. 9, 2001. No federal dollars could be used on research with cell lines from embryos destroyed from that point forward. Federal regulations do not restrict embryonic stem cell research using state or private funds.
John Podesta, head of Obama's transition team, strongly hinted that the president-elect would deal with stem cell research soon after taking office Jan. 20. "As you know, he has said something specific about stem cell research, so I think you can expect that what he said in the campaign will be fulfilled once in office," Podesta said.
Obama made it clear during the campaign he would overturn Bush's directive.
"As president, I will lift the current administration's ban on federal funding of research on embryonic stem cell lines created after August 9, 2001, through executive order, and I will ensure that all research on stem cells is conducted ethically and with rigorous oversight," he said.
Opponents of such research say they will press their case on several fronts.
The main argument is that life begins at conception — that once fertilization occurred in the lab, so did a human being.
Secondly, they will argue that scientists are having success using other methods — adult stem cells that form specific tissues, or reprogramming skin cells to act like stem cells — so money should be directed where the biggest scientific breakthroughs have occurred. For example, this past week, doctors gave a woman a new windpipe with tissue grown from her own stem cells, eliminating the need for anti-rejection drugs.
"We still intend to try and talk about the real facts that it's the adult stem cells providing the actual treatments," said David Prentice, senior fellow at the Family Research Council.
Added Wendy Wright, president of Concerned Women for America: "There's a lot that's happened over the seven years that includes some remarkable scientific discoveries, which really should have made the issue of federal funding of embryonic stem cell research moot."
But Sean Tipton, director of public affairs at the American Society for Reproductive Medicine, took aim at those arguments.
"It's a little disingenuous for opponents who have effectively blocked federal funding of the work to then cite a lack of progress," Tipton said. "You hold someone at the starting line then you criticize them for not getting very far."
Dr. Chi Dang, professor of medicine at the Johns Hopkins University School of Medicine, agreed there have been tremendous advances with adult stem cells. But he said it is not yet clear that they have enough flexibility to be used in all the ways that an embryonic stem cell could be.
"From a scientific viewpoint, we would be cornering ourselves into generalizing things that may not be true," Dang said.
Dang also said these embryos would otherwise be discarded.
"The question is: Is it ethically more acceptable to destroy these embryos by pouring acid on them, or do you deploy these clusters of cells to create new cell lines that could benefit us in the future?"
Samuel Pfaff, a professor at the Salk Institute for Biologic Studies, said he also supports greater embryonic stem cell research to understand what makes them so special that scientists can endow other cells with similar properties.
"I think it's very fair to say that the long-term trajectory for this area of science is to understand embryonic stem cells so well that we don't have to use them anymore." Pfaff said.
___
On the Net:
Stem cell information at the National Institutes of Health: http://stemcells.nih.gov/info/basics
Candidates answers on embryonic stem cell research: http://www.sciencedebate2008.com

iplex update

2008-11-24T18:16:00.002-05:00

Dear friends,
Wanted to provide all concerned, a brief update on our current status with Iplex. Insmed has been in contact with, and assured the group leaders of Team IPLEX, that they have begun the process of clearing regulatory hurdles both in the USA and other countries. Insmed has also assured us that the production demands of those currently expressing an interest in Iplex are no barrier at all. While we are all anxious and ready to proceed at once with Iplex, alas, the wheels of the machine must turn and all regulatory obilgations must be met in order for Insmed to legally provide the drug to PALS. This process should be relatively quick, (all things considered).

I regret that there is little more to report at this time. There is little that we, Team Iplex, could, and probably should, do at this time to encourage the momentum of this process. Our continued efforts as a group have acheived what no one thought possible. Any further collective efforts, such as writing/calling, etc, may only serve to distract from Insmeds mandatory compliance with regulatory processes. We (Team IPLEX) have been assured that the second that the regulatory process is in place, it will be updated on Insmed's Corporate Webpages. We have been also assured that we would be notified/get a call outlining the details. However, I would still encourage everyone concerned, to check Insmeds web site on a regular basis.
I just can't tell youi how much it has meant to me and my family, to have been on this journey with all that have helped to make this possible.

To think that a small group of patients and caregivers fighting with all of our might, with a never say die attitude - could make such an incredible difference and have influence on multi-billion dollar companies is pretty incredible, at least that's the way I feel.

2008-11-21T22:10:00.000-05:00

www.alsmatters.org

Neuralstem enters stem cell collaboration for Huntington's disease in Germany

2008-11-21T21:28:00.000-05:00

The collaboration with Professor Nikkah will focus on Huntington's disease.
"We are pleased to have established this collaboration in Germany" said Richard Garr, Neuralstem President & CEO. "The goal of our work with Professor Nikkah will be to qualify our existing cGMP spinal cord cells into Professor Nikkah's human trial program to treat Huntington's disease. As we prepare to submit an IND to treat ALS with our stem cells in the U.S., we continue to look for strategic relationships in both Europe and Asia which will allow us to move the cells into humans. We believe that Professor Nikkah's program is the most advanced of its kind in Western Europe, and we are excited about his working with our cells."
Huntington's disease (HD) results from genetically programmed degeneration of brain cells, called neurons, in certain areas of the brain. This degeneration causes uncontrolled movements, loss of intellectual faculties, and emotional disturbance. HD is a familial disease, passed from parent to child through a mutation in the normal gene. Each child of an HD parent has a 50-50 chance of inheriting the HD gene.
Neuralstem's patented technology enables, for the first time, the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells into mature, physiologically relevant human neurons and glia.
Major Central Nervous System diseases targeted by the Company with research programs currently underway include: Ischemic Spastic Paraplegia, Traumatic Spinal Cord Injury and ALS. The company's cells have extended the life of rats with ALS (Lou Gehrig's disease) as reported the journal TRANSPLANTATION, in collaboration with Johns Hopkins University researchers, and also reversed paralysis in rats with Ischemic Spastic Paraplegia, as reported in NEUROSCIENCE on June 29, 2007, in collaboration with researchers at University of California San Diego. The Company expects to file its first IND (Investigational New Drug) application with the FDA for ALS in the fall.
http://www.neuralstem.com/