Tuesday, April 14, 2009

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

Saturday, March 21, 2009

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

Wednesday, March 11, 2009

Some states push back against stem cell research

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

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

Tuesday, March 10, 2009

finally

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.

Monday, March 9, 2009

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

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."

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

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

Sunday, March 8, 2009

Stem cells can help regrow diseased muscles

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

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.

Friday, March 6, 2009

my cousin sent me this, thanks sally

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.

Wednesday, March 4, 2009

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

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

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.

Monday, March 2, 2009

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.

Saturday, February 28, 2009

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.

Thursday, February 26, 2009

Public release date: 23-Feb-2009

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

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.”

Wednesday, February 25, 2009

email me at schemera@yahoo.com for attachments

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
Having hope makes every day easier, more gentle

Friday, February 20, 2009

NeuralStem Inc.'s ALS Trial on Clinical Hold

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

Thursday, February 19, 2009

great website

http://www.alsworldwide.net/

From: alscenter@jhmi.edu

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.orgwww.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

Wednesday, February 18, 2009

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

Tuesday, February 17, 2009

email me for attachment schemera@yahoo.com

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.

Sunday, February 15, 2009

Obama to lift ban on stem cell research soon: aide

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

http://www.popularmechanics.com/science/health_medicine/4303407.html

Saturday, February 14, 2009

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

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

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."

Thursday, February 12, 2009

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

Wednesday, February 11, 2009

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

Monday, February 9, 2009

Scientists Heartened at Prospect of End to Stem Cell Ban

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

Sunday, February 8, 2009

from eddie

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
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."

Tuesday, February 3, 2009

Selecting Potent Immune Suppressive Mesenchymal Stem Cells

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

£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

Monday, February 2, 2009

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

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.

Saturday, January 31, 2009

i got this email from my buddy eddie

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

Friday, January 30, 2009

Stem Cell Research: The Quest Resumes

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

'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

Thursday, January 29, 2009

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.

K

from my friend kirshner

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.

K

Wednesday, January 28, 2009

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

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."
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