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.