Tuesday, September 30, 2008

Procrastination is the thief of time.--Edward Young

a great article

Insmed Receives FDA Orphan Drug Designation For IPLEX(TM) In The Treatment Of Myotonic Muscular Dystrophy

Insmed Inc. (Nasdaq: INSM), a developer of follow-on biologics and biopharmaceuticals, announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation for IPLEX(TM) for the treatment of Myotonic Muscular Dystrophy (MMD). Insmed is currently conducting a 24-week Phase III enabling trial for IPLEX(TM) in MMD, and recently was awarded a grant of approximately $2.1 million from the Muscular Dystrophy Association (MDA), which is expected to cover a substantial portion of the external costs associated with the trial."This Orphan Drug Designation is another milestone for Insmed in our development and commercialization plan for IPLEX(TM) in MMD," said Geoffrey Allan Ph.D., Insmed's President and Chief Executive Officer. "Orphan status, combined with the recent $2.1 million MDA grant, positions us well to continue advancing this important product candidate through clinical development, and maximize the market opportunities for MMD available to us."Orphan status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases affecting fewer than 200,000 Americans annually. This orphan drug designation for IPLEX(TM), grants Insmed seven years of market exclusivity upon approval for the MMD indication. In addition, Insmed is eligible for tax credits relative to its development costs, as well as assistance from the FDA in advancing the drug candidate through the regulatory process.MMD affects approximately 37,000 Americans, and nearly 60,000 people in the European Union. MMD is a genetic disease characterized by endurance loss, muscle wasting, weakness, pain, cognitive impairment and gastro-intestinal dysfunction. There is currently no cure for the disease, and no specific treatment has been developed to satisfactorily reverse or ameliorate the common symptoms associated with the disease.About IPLEX(TM)IPLEX(TM) is a complex recombinant human insulin-like growth factor-I (rhIGF-I) and its predominant binding protein IGFBP-3 (rhIGFBP-3). The drug was approved in the United States in December 2005 for the treatment of children with growth failure due to severe primary IGF-I deficiency (Primary IGFD). IPLEX(TM) is currently being studied as a treatment for several serious medical conditions, including two neuromuscular disorders, Myotonic Muscular Dystrophy (MMD) and ALS (Lou Gehrig's Disease); HIV-Associated Adipose Redistribution Syndrome (HARS); and retinopathy of prematurity (ROP).About InsmedInsmed 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 StatementsThis 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 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 entrance into the follow on biologics market may be unsuccessful, our common stock could be delisted from the Nasdaq Global Market and other risks and challenges detailed in our filings with the U.S. Securities and Exchange Commission, including our Quarterly Report on Form 10-Q for the quarter ended September 30, 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.Insmed Inc.http://www.insmed.com
Article URL: http://www.medicalnewstoday.com/articles/92188.php
Main News Category: Muscular Dystrophy / ALS

Monday, September 29, 2008

People with ALS pass through a doorway into a different world. It's a one way trip: you may survive, but you can never come home to the same old life.

And however much they want to be with you, Moms, Dads, Sisters, Brothers, Spouses, Friends & Lovers can't come into your world on an equal footing. But when people with ALS meet, it's different.

They don't have to explain how it feels
They don't have to explain anything

"Victory is always possible for the person who refuses to stop fighting."

Wednesday, September 24, 2008

pics from vegas

phil and i
robyn and i
me taking a nap
w/ robyn
the venetian


josh from hart & huntington tattoo
phil, josh and me
the gang at dinner
joe, bob, robyn and me
joe, bob, jodi and me

me having a pseudobulbar moment
presley and i
(my future goddaughter )






Researchers hope for change on stem cell politics By Julie Steenhuysen

Researchers hope for change on stem cell politics
By Julie SteenhuysenMon Sep 22, 10:54 PM ET
Stem cell experts said on Monday they hope the next U.S. president will end political curbs on embryonic stem cell research but some worry recent comments by Republican candidate John McCain suggest his past support for such research may be waning.
Both McCain and Democrat Barack Obama have said they favor easing restrictions on spending public money to finance embryonic stem cell research.
"My hopes are that politics get out of it," University of Wisconsin-Madison stem cell researcher James Thomson told reporters. "It's been a long eight years," he added, referring to President George W. Bush's staunch opposition to such research.
Stem cells taken from balls of cells that develop days after conception offer promise for regenerative medicine because they give rise to all tissues in the body.
But some oppose their use because they involve destruction of the embryo.
In 2001, Bush issued an executive order allowing only limited federal funding of work involving human embryonic stem cells. Congress has tried several times to loosen restrictions but Bush has vetoed every effort.
Obama supports embryonic stem cell research. McCain's stand is less clear.
"While I support federal funding for embryonic stem cell research, I believe clear lines should be drawn that reflect a refusal to sacrifice moral values and ethical principles for the sake of scientific progress," McCain said in a statement given September 15 to a website devoted to discussing science and the elections, http://www.sciencedebate2008.com/.
"Moreover, I believe that recent scientific breakthroughs raise the hope that one day this debate will be rendered academic."
Thomson said he was concerned about McCain's comments.
He said the worst thing that could happen is that McCain "gets painted into a corner" and makes a promise to oppose embryonic stem cell research that he would be forced to follow through on if elected president.
"It's too important to make it a Republican or Democratic issue," Thomson told reporters at the World Stem Cell Summit in Madison.
Both Thomson and Wisconsin Gov. Jim Doyle told reporters they were concerned about efforts by opponents of embryo research to offer stem cell advances as a reason to discontinue funding for embryonic stem cell research.
Researchers, including Thomson, have discovered ways to make powerful stem cells using ordinary skin cells. Called induced pluripotent stem cells or iPS cells, these new cells appear to behave like embryonic stem cells.
Other teams have directly reprogrammed one type of cell into another type, and other types of stem cells have also shown promise in experiments.
Thomson said that while promising, it is not clear if these cells are useful as human embryonic stem cells.
"Although I personally believe the future is in iPS cells, I could be wrong. There could be evidence to show these cells are fatally flawed. We have to keep an open mind," Thomson said.
(Editing by Maggie Fox and Philip Barbara)

Tuesday, September 23, 2008

YES!!!!!

Victory!
Senate Passes the ALS Registry Act
A few minutes ago, the United States Senate passed the ALS Registry Act!! Thanks to your outreach, we have just won a huge victory for people with ALS and their families across the country. We have made a difference!The bill now heads to the House, which is expected to quickly agree to the Senate version, which included technical changes made during the legislative process. We will keep you updated as the bill moves toward enactment into law.
Congratulations to everyone!

Stem cells open door for studying human disease By Julie Steenhuysen

Stem cells open door for studying human disease
By Julie SteenhuysenMon Sep 22, 10:05 PM ET
Advances in stem cell research offer a new way of studying human disease, allowing scientists to move beyond fruit flies and lab mice to see how human cells go awry and how drugs and other therapies might help, U. S. researchers said on Monday.
They said human embryonic stem cells and a newly invented type of cell called induced pluripotent stem cells or iPS cells -- made without the use of human embryos -- are helping to transform how researchers develop and test therapies.
"We've almost worn out what we can do with animals in disease biology such as fruit flies and mice," Lawrence Goldstein, a researcher at the University of California-San Diego, told about 900 scientists and stem cell advocates at the World Stem Cell Summit in Madison, Wisconsin.
"The problem at the end of the day is that humans are not just big mice."
Researchers such as Goldstein hope to use stem cells not only to create a new field of medicine called regenerative medicine -- and grow tissues, organs and blood for transplant -- but also to study diseases as they specifically affect humans.
Embryonic stem cells are the body's master cells, giving rise to all of the tissues in the body, and iPS cells, made using genes to transform ordinary cells, appear to have similar powers.
Stem cells made from people with various diseases can be used to grow batches of living tissue in the lab -- tissue with the same genetic defects that cause or are caused by the disease.
HUMAN TISSUE
"Scientists have never had access to human tissue before," said James Thomson of the University of Wisconsin, who was first to derive embryonic stem cells from a frozen human embryo a decade ago.
Some people, including President George W. Bush, oppose experimenting on human embryos and Bush has allowed only restricted federal funding for this type of research.
Earlier this year, research teams in Japan and the United States found a way to coax ordinary skin cells into becoming induced pluripotent stem cells.
"We now have a way to genetically modify adult cells out of your body to have the same properties embryonic stem cells have," Thomson told the meeting.
"That will lead to better, faster cheaper drugs in a way that won't make the cover of The New York Times, but will very much affect patients," he said.
Scientists said both types of stem cells will offer a better way to understand human disease.
Goldstein's lab is studying diseases of the nervous system, such as Alzheimer's disease and amyotrophic lateral sclerosis or ALS, also known as Lou Gehrig's disease.
For Alzheimer's, Goldstein said there are very few approved drugs and a few in the discovery pipeline. "The reason is we fundamentally do not understand what is wrong in the nerve cells in that disease," he said.
His lab is gathering skin cells from people with a genetic form of Alzheimer's disease. "We've captured in these cells the quintessential aspects of the disease," Goldstein said. They want to use them to test new drugs.
Thomson said similar research will take place in cultures of heart cells and efforts are already underway to use stem cells to make human blood, which might be used to supplement the blood supply.
He said other stem cell therapies, including transplants to replace damaged or destroyed cells, are farther off, and will likely come within 5 to 10 years.

Sunday, September 21, 2008

whoever wrote this-please email me

Drew, we continue to be inspired by your strength and committment to fighting this horrific disease. Our daughter has a smiliar condition and will most likely not live past a year. We are interested in finding out more about your stem cell experience in China. Please email me at your earliest convenience.

please email me at
schemera@yahoo.com
Possibilities and miracles are one and the same.--Anonymous

Wednesday, September 17, 2008

UW scientists slow ALS using stem cells

UW scientists slow ALS using stem cells
Todd Finkelmeyer — 9/16/2008 11:53 am
Using engineered adult stem cells from bone marrow to deliver a growth factor directly to atrophied muscles, scientists at UW-Madison have successfully slowed the progression of amyotrophic lateral sclerosis (ALS) -- also known as Lou Gehrig's disease -- in rats.
The finding was published Tuesday in the journal Molecular Therapy.
Although it's at a relatively early stage, the research offers hope that the process might someday provide a new therapy for people who suffer from the debilitating and fatal disease, which is caused by the progressive loss of motor neurons and their connections to muscles.
There currently are no effective treatments for ALS.
"I don't want to give patients too much expectations," said Masatoshi Suzuki, the UW-Madison associate scientist who led the study out of the Waisman Center . "But so far the application is showing a lot of promise."
The study builds on previous research that showed motor neurons, the key cells that connect muscles to the central nervous system, could be protected by stem cells that carried a key growth component, glial cell line-derived neurotrophic factor (GDNF).
Past work by Suzuki and Clive Svendsen, a UW-Madison professor of neurology, showed that transplanting neural stem cells into the spinal cord could protect motor neurons that degenerate in an ALS rat model. But the motor neurons still didn't effectively connect with the muscles that atrophy due to ALS.
In the new study, Suzuki and his colleagues used adult bone marrow stem cells that were engineered to release GDNF directly to the muscle as sort of "Trojan horses" to deliver a growth factor. In the past, it was shown that bone marrow stem cells on their own had a modest effect, possibly by releasing their own protective factory.
But in the new research, the scientists delayed the progression of the disease and extended the lifespan of the afflicted animals. The Wisconsin group reported that the engineered cells survive well when introduced to muscle and significantly increased the number of neuromuscular connections and motor neurons in the spinal cord at mid stages of the disease.
"The positive effects that we reported in the paper are not so big," said Suzuki. "But they are important. So now we are working on modifying our method and enhancing the positive effects."
Although the study could someday lead to treatments for the disease, Suzuki cautioned that work remains before it could be attempted in humans.
Co-authors of the new Molecular Therapy report include Jacalyn McHugh, Craig Tork, Brandon Shelley and Antonio Hayes, all of UW-Madison; Ilaria Bellantuono of the Royal Manchester Children's Hospital, United Kingdom; and Patrick Aebischer of the Ecole Polytechnique Fdrale de Lausanne, Switzerland.
The new study was supported by grants from the ALS Association, the National Institutes of Health, the University of Wisconsin Foundation and the Les Turner ALS Foundation.

http://www.madison. com/tct/mad/ breaking_ news/305115

Using engineered adult w ALS using stem cells
Todd Finkelmeyer — 9/16/2008 11:53 am stem cells from bone marrow to deliver a growth factor directly to atrophied muscles, scientists at UW-Madison have successfully slowed the progression of amyotrophic lateral sclerosis (ALS) -- also known as Lou Gehrig's disease -- in rats.
The finding was published Tuesday in the journal Molecular Therapy.
Although it's at a relatively early stage, the research offers hope that the process might someday provide a new therapy for people who suffer from the debilitating and fatal disease, which is caused by the progressive loss of motor neurons and their connections to muscles.
There currently are no effective treatments for ALS.
"I don't want to give patients too much expectations," said Masatoshi Suzuki, the UW-Madison associate scientist who led the study out of the Waisman Center . "But so far the application is showing a lot of promise."
The study builds on previous research that showed motor neurons, the key cells that connect muscles to the central nervous system, could be protected by stem cells that carried a key growth component, glial cell line-derived neurotrophic factor (GDNF).
Past work by Suzuki and Clive Svendsen, a UW-Madison professor of neurology, showed that transplanting neural stem cells into the spinal cord could protect motor neurons that degenerate in an ALS rat model. But the motor neurons still didn't effectively connect with the muscles that atrophy due to ALS.
In the new study, Suzuki and his colleagues used adult bone marrow stem cells that were engineered to release GDNF directly to the muscle as sort of "Trojan horses" to deliver a growth factor. In the past, it was shown that bone marrow stem cells on their own had a modest effect, possibly by releasing their own protective factory.
But in the new research, the scientists delayed the progression of the disease and extended the lifespan of the afflicted animals. The Wisconsin group reported that the engineered cells survive well when introduced to muscle and significantly increased the number of neuromuscular connections and motor neurons in the spinal cord at mid stages of the disease.
"The positive effects that we reported in the paper are not so big," said Suzuki. "But they are important. So now we are working on modifying our method and enhancing the positive effects."
Although the study could someday lead to treatments for the disease, Suzuki cautioned that work remains before it could be attempted in humans.
Co-authors of the new Molecular Therapy report include Jacalyn McHugh, Craig Tork, Brandon Shelley and Antonio Hayes, all of UW-Madison; Ilaria Bellantuono of the Royal Manchester Children's Hospital, United Kingdom; and Patrick Aebischer of the Ecole Polytechnique Fdrale de Lausanne, Switzerland.
The new study was supported by grants from the ALS Association, the National Institutes of Health, the University of Wisconsin Foundation and the Les Turner ALS Foundation.

http://www.madison. com/tct/mad/ breaking_ news/305115

Global Stem Cell Forum in China Showcases Cutting Edge Research, Focuses on New Findings for iPS Cel

Global Stem Cell Forum in China Showcases Cutting Edge Research, Focuses on New Findings for iPS Cel l
RSS feed for Beike Biotechnology Company Limited:
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USA (SANEPR.com) PRNewswire/ -- Over 300 of China’s top stem cell biologists and researchers from around the globe shared their latest results and held China’s first ever symposium on advanced induced pluripotent stem (iPS) cell research during the just concluded first annual 2008 China Stem Cell Technology Forum at the China Medical City complex in Taizhou, China. At the Forum, which was chaired by Dr. Sean Hu, Chairman of Beike Biotechnology Co., Ltd., ( http://www.beikebiotech.com ) some of the world’s most respected researchers presented their latest laboratory findings and clinical trial results in using stem cells to treat common ailments like heart disease and nervous system diseases such as cerebral palsy, spinal cord injury, muscular dystrophy, and optic nerve hypoplasia (ONH). Biologists attending the Mandarin Chinese language Forum came from leading research centers in nearly all of China’s major cities, and as far away as the United States, Canada, India, Australia, and Malaysia.A special symposium entitled Advanced iPS Cell Technology, convened as part of the Forum, brought together for the first time over a dozen scientists involved in cutting edge stem cell research at some of America and China’s most respected science universities and research labs. A potential substitute for embryonic stem cells, genetically programmable iPS cells are widely believed to be at the very forefront of the field’s most exciting research frontier as iPS cells theoretically could deliver the greatest therapeutic benefit. Since iPS cells can be produced from the building blocks in any individual’s own body, they bring the potential that people may someday be able to heal a range of sicknesses from tissue cells they have banked themselves. At the symposium, renowned stem cell scientists like Dr. Hu Jifan of the Palo Alto VA Health Care System under the Stanford University Medical School, Dr. Xiang Peng of Sun Yat Sen University, Dr. Zhang Yaou of Tsing Hua University, Dr. Kong Hsiang-Fu of the Chinese University of Hong Kong, Dr. Zhou Xiangjun of Shanghai Jiaotong University, and Dr. Yong-Jian Geng of the University of Texas Health Science Center at Houston and the Texas Heart Institute, discussed their own latest advancements since recent breakthroughs at the University of Wisconsin and Kyoto University in Japan. The scientists also discussed mutual cooperation and research oversight opportunities, creation of ethical research guidelines in China, and future iPS cell research directions. At the symposium, a special subgroup of China’s leading cardiovascular scientists like Dr. Chen Haozhu of Fudan University, Dr. Wang Shiwen of the Chinese PLA General Hospital, and Dr. Zhang Fumin of Nanjing Medical University conferred on the use of stem cells in heart disease treatment, as well as new research methods and ethical responsibilities in that field.Dr. Yong-Jian Geng, M.D., Ph.D, Director of the Heart Failure and Stem Cell Research Lab at the Texas Heart Institute and Professor of Medicine at the University of Texas Medical School in Houston, said, "Scientists and clinicians have been doing incredible research on stem cells, and generating many exciting results. I feel confident more breakthroughs will be coming soon. I was very pleased and honored to be invited to participate in this discussion, and I am willing to help Chinese investigators establish guidelines that will, in my opinion, help develop a global standard for the stem cell therapies."Dr. Hu Jifan, noted senior research scientist at the Palo Alto VA Health Care System under the Stanford University Medical School said, "Leading scientists in China are constantly finding ways to create more effective iPS cells. Through this forum we agreed to help verify each other’s work, share experiences, and ensure safe, high-standard research takes place so that we may someday create the conditions for effective clinical treatments and industrial scale production of iPS cells. While no one knows yet when these conditions might be met, this Forum certainly helped speed up the process by bringing together some of the world’s best minds working towards these goals."Presentations at the 2008 China Stem Cell Stem Cell Technology Forum covered a wide range of new biological advancements as Chinese scientists hone in on the global goal of bringing practical stem cell treatments to market. Other topics covered included findings on the most potent combinations of stem cells, effective cell processing methods, the latest clinical trials targeting diseases like heart conditions, muscular dystrophy and optic nerve hypoplasia (ONH), and much more yet to be published stem cell research.Beike Biotechnology Co., Ltd., a worldwide leader in researching and providing safe and effective stem cell applications for medical treatment, hosted the event along with the China National Center for Biotechnology Development (CNCBD) under China’s Ministry of Science and Technology (MOST), the Jiangsu Provincial Department for Science and Technology, and the Taizhou Government. The 2008 China Stem Cell Stem Cell Technology Forum was attended by MOST officials, the Vice Governor of Jiangsu Province Mr. Zhang Taolin, and Taizhou Mayor Mr. Yao Jianhua. Dr. Sean Hu, the Chairman of Beike Biotech said, "Chinese science, and indeed global stem cell research, achieved a great deal through this forum. Beike is proud to work with China to bring together so many globally known Mandarin speaking stem cell scientists to better communicate and share resources regarding what may very well become the future of healthcare."About the China Medical City (CMC)Jiangsu Province is considered the number one location for China’s medical industry based on revenue generated over the past 5 years. The city of Taizhou in Jiangsu is not only the hometown of China’s President Hu Jintao, but is considered the fastest growing medical industry location in Jiangsu, with over 35 % annual growth in that time. Established by the Chinese Government in 2005 and consisting of 20-25 square Kilometers in the heart of Taizhou City, China Medical City (CMC) is fully supported by China’s local and national governments. CMC is emerging as a strong leader in China’s efforts to develop a streamlined pharmaceutical and medical materials industry that concentrates all medical services and support in one location. Businesses located in CMC carry out a range of manufacturing and support services including research and development, creation and processing of medical materials, distribution, comprehensive healthcare delivery solutions, and patent filing support. About Beike Biotechnology Company Limited Beike is a biotechnology company that was founded in July 2005 with capital from Beijing University, Hong Kong University of Science and Technology and Shenzhen City Hall when it commercialized stem cell technology that had been in research since 1999. The research and clinical work comes from collaborations with leading institutions in China including of Tsinghua University, Beijing University, Hong Kong University of Science and Technology, No. 3 Army Medical University, Zhongshan Medical University, Guiyang Medical College and Zhengzhou University. Over 250 patients every month are treated with Beike’s stem cells in leading hospitals throughout China. Patient experiences from treatments can be found at Stem Cell China News (http://www.stemcellschina.com ).###SOURCE Beike Biotechnology Company Limited-0- 07/25/2008/CONTACT: Jonathan Hakim of Beike Biotech Company, Ltd., +86 755-2637-7020, or info@beikebiotech.com/ /Web site: http://www.beikebiotech.com /

Tuesday, September 16, 2008

whoever wrote this-please email me

Hi I live in Sea Girt, NJ and I have not yet gotten a definitive dx.

please email me at

schemera@yahoo.com

Monday, September 15, 2008

Another turning point, a fork stuck in the road
Time grabs you by the wrist, directs you where to go
So make the best of this test, and don't ask why
It's not a question, but a lesson learned in time

Saturday, September 13, 2008

Japan university gets patent for stem cell breakthrough Fri Sep 12, 12:02 PM ET

Japan university gets patent for stem cell breakthrough
Fri Sep 12, 12:02 PM ET
Japan has given Kyoto University a patent for groundbreaking stem cell research in what is believed to be a world first for such scientific research, officials said Friday.
The move is aimed at preventing a pharmaceutical company from taking its own patent and then seeking money from researchers for their work, university officials said.
Teams at Kyoto University and at the University of Wisconsin at Madison in the United States last year discovered how to use skin to produce stem cells -- which can develop into various organs or nerves.
The finding was hailed by the Vatican and US President George W. Bush because it can circumvent an ethical row over conventional stem cell research using human embryos.
"It is important for the university to keep a patent so it can conduct medical research and treatment at low costs in the future," said Naoko Takasu, who is in charge of intellectual property issues for the university.
She said it was the first patent in the world for stem cells and that the university in western Japan would also seek patents in other major developed countries.
Shinya Yamanaka, the head of the research team, said he was "delighted" at the issuance of the patent.
"This is a first step," he said in a statement. "I will continue my utmost efforts in this research... so as to accelerate its practical application in clinical treatment."
Stem cell research is seen as having the potential to save lives by helping to find cures for diseases such as cancer and diabetes or to replace damaged cells, tissues and organs.
But religious conservatives argue that research on embryos destroys human life, albeit at its earliest stage of development.
Anticipating growing international competition in the field, Japan -- the largest spender on research after the United States -- in December announced a 10 billion-yen (92 million-dollar) plan to advance stem cell studies.
Another group of Japanese scientists said last month they had derived stem cells from wisdom teeth, opening another way to study deadly diseases without the ethical controversy of using embryos.

Thursday, September 11, 2008

Japan's stem-cell pioneer grapples with pressure of success by Guy Newey

Japan's stem-cell pioneer grapples with pressure of success
by Guy NeweyWed Sep 10, 12:33 PM ET
Pioneering stem-cell scientist Shinya Yamanaka, whose work could help transform medicine and find cures for a range of debilitating diseases, says he was a pretty awful doctor.
"I was a surgeon, but I found myself not good at surgery," said the Japanese professor, describing why he changed his career from medicine to stem cell research.
Yamanaka has been incredibly successful in his second career and was this week awarded a share of the Shaw Prize, a million-dollar science award set up by Hong Kong philanthropist and film mogul Run Run Shaw.
The award recognised Yamanaka's moment of genius, when he discovered how to turn skin cells into stem cells, the so-called master cells which can develop into any of hundreds of different cell types in the body, replacing those lost or damaged by disease.
Stem cell therapy has been touted as a promising intervention for neurodegenerative diseases such as Parkinson's or Alzheimer's, as well as helping create new drugs and improving research.
Although his technique is a long way from being perfected, it has created a potential alternative to stem cells harvested from embryos, a procedure that has provoked the ire of pro-life and religious groups across the world.
The new technique is so promising that one of the men who created the world's first cloned sheep, Dolly, and who shared the Shaw Prize with Yamanaka, has stopped cloning embryos to focus on studying stem cells derived from skin cells.
While Yamanaka is not against embryonic stem cell research -- he said he would use his wife's embryos if he needed to -- he does hope he has provided a potential, elegant solution.
"I was a physician, so the biggest concern for me was to help patients," said the Kyoto University professor.
"If embryo stem cell research is the only way to help patients, then I think that is what we should do.
"At the same time... as a natural feeling, I do want to avoid the usage of human embryos... Human embryos are not like skin cells, they can be babies if transplanted. That is why we are doing what we are doing."
Yamanaka is acutely aware his new science is creating new ethical dilemmas and is a supporter of firm regulation.
"We should limit the application of technology to treatment or what can make patients happier," he said.
"We may be able to generate new life (with this technique), so we are presenting another ethical issue with this problem."
He draws the line at creating a new life simply to grow new organs, theoretically possible using his technique.
"It is technically very difficult (but) organ shortage is a big problem right now in many countries. We need some kind of regulation," he said.
Born in 1962, Yamanaka was the only son of a factory owner, who produced parts for sewing machines.
When he was in his early teens, his father told him that he should not follow the traditional Japanese path and take over the business, but become a doctor.
"He said it was more useful," said the father of two, whose eldest daughter is at medical school.
While Yamanaka followed his father's wish, he found his niche when he turned away from medicine. But his new-found fame -- which has seen his work praised by US President George W. Bush and the Roman Catholic Church -- has created new pressure.
"Yes, it has been very stressful," he said, adding the pressure was why he avoided giving a timetable for the development life-saving treatments that could follow his work.
"It is quite dangerous to predict. If I say five years for some disease, patients suffering from that disease will expect it. After five years they may be very disappointed. I think we should avoid too much hype."

Wednesday, September 10, 2008

a quote from the book tuesday's w/ morrie that i get inspiration from-this came from my friend eric's als blog

“When I am taking, I feel like I’m dying but when I give of myself, I feel like I am living”. This is how I feel. I cannot sit back and let this disease take my life and only sit back and take from everyone. I need to have purpose by giving to others and sharing hope and the inspiration that only comes from God in ways that He has equipped me to feel like I have something to live for.

Tuesday, September 9, 2008

ALS patient denied access to drug/GREAT ARTICLE !!!!!!

ALS patient denied access to drug
Pearl man unable to get medication he thinks could combat disease
Shanderia K. Posey • sposey@jackson.gannett.com • September 9, 2008



What could be more disheartening than living with a terminal illness that daily diminishes your quality of life?
Answer: Being denied access to the one drug you think could improve your condition.
And when you've been diagnosed with amyotrophic lateral sclerosis, also known as Lou Gehrig's disease, time is not on your side. Most ALS patients die within years of the diagnosis. Something Vicki and Ricky Muirhead of Pearl are well aware of.
"ALS takes everything away from you, the only thing you're left with is your mind," Ricky said. "You can't walk, talk or breathe on your own. You're dependent on your caregiver."
For four years, Ricky, 48, lived with involuntary muscle twitches local neurologists diagnosed as benign fasciculation. Then in August 2007, Dr. Y. Harati at Baylor College of Medicine in Houston, Texas, diagnosed him with ALS. Vicki said Harati noticed the muscle between Ricky's thumb and index finger was sunken in - a sign to Harati that ALS was present.
Mayo Clinic defines ALS as a neurological disease that attacks nerve cells that control voluntary muscles. It may begin with muscle twitching or weakness in an arm or leg or with slurring of speech. Eventually, it affects the ability to control muscles needed to move, speak, eat and breathe.
Eighty percent of ALS patients die within three to five years of diagnosis, according to Leigh Langford, an instructor of neurology at the University of Mississippi Medical Center who has worked with ALS patients for 10 years. She said 20 percent die within six to 10 years and some patients' conditions may plateau.
The only drug available for treatment of ALS is Rilutek. Langford estimates the drug costs between $800 and $1,000 a month. It's main purpose is to prolong the life of ALS patients about three to six months.
But a few weeks ago, Ricky joined an ALS online forum and became aware of a drug called Iplex made by Insmed Inc. of Richmond, Va. The Food and Drug Administration approved Iplex Dec. 12, 2005 to treat growth failure in children. It is a man-made form of insulinlike growth factor-1 (IGF-1) normally produced in the body.
Ricky learned ALS patients in Italy were using the drug and reporting amazing results, so he wanted to try it, too.
"Testimonies online of those in Italy showed people who couldn't walk are now walking with a cane. People that couldn't talk are now talking or (people who) couldn't be on the phone are now picking up the phone," Ricky said.
However, as a result of a settlement agreement between Insmed and two biopharmaceutical companies called Tercica and Genentech, American ALS patients can not have access to the drug. Tercica and Genentech hold patent rights for the manufacture and use of IGF-1 products in the treatment of ALS. They sued Insmed for patent infringement, and the case was settled in March 2007. Before the settlement, an expanded access program was created for ALS patients in Italy to have access to Iplex. Tercica and Genentech agreed to allow the program to continue. Tercica and Genentech offer an alternative to Iplex called Increlex.
"As part of the agreement, Insmed is allowed a nonexclusive license with Tercica/Genentech to develop Iplex for the following named indications: MMD (myotonic muscular dystrophy), HARS (HIV-associated adipose redistribution syndrome), ROP (retinopathy of prematurity), recovery from burns and trauma, and recovery from hip fracture," said John Procter, a spokesman for Insmed Inc., in an e-mail to The Clarion-Ledger.
Vicki and Ricky Muirhead are now on a mission to raise awareness of the condition and get access of Iplex to all ALS patients.
"We are trying to get enough people to sign the petition (online) for ALS patients and burn patients to rebuild muscles," Ricky said. They also want senators and congressmen to propose legislative measures for their cause.
Some ALS experts point out a lack of documented evidence of Iplex's effectiveness.
"I don't know how much scientific proof there is," Langford said. She is focused on a year-long study that began in June using lithium to treat patients. Ten centers across the nation are involved.
"It's the biggest promise we've seen," Langford said, noting it's the first time in 25 years another drug could be approved for treatment.
Sharon Matland, vice president of patient services for the ALS Association national office in Calabasas Hills, Calif., isn't ready to give her stamp of approval to Iplex either.
"Right now it has not (been) tested in this country," Matland said, explaining her organization promotes and supports the drug safety processes in the United States, including research on lithium. "We are monitoring how that work is going on over there (in Italy)."
The Muirheads are doing the same and plan to continue to spread the word about their discovery. They attend a monthly ALS support group that meets at the Methodist Rehabilitation Center in Jackson.
Vicki, who is an insulin-dependent diabetic, quit her job as a travel agent to become the primary caregiver of her husband. He's been fortunate to continue working as an electrical engineer in Chicago and flies home weekly, but ALS eventually may make those trips unbearable.
Ricky's speech is now slow and slurred. Two fingers on his right hand feel like they are asleep 24/7, he said. However, he maintains a positive, even comical, attitude. He jokes about hoping others can understand his "ALS accent."
Langford said the disease is more common in men and strikes at the average age of 55. It is mostly considered sporadic and not genetic. Data indicate about 5,600 people are diagnosed with ALS yearly. There is no known cause though some environmental factors have been suggested. For example, the Muirheads wonder if chemicals Ricky used to spray and treat their lawn could have attributed to the onset of ALS.
UMC's ALS clinic takes a multidisciplinary approach to treatment including physical therapy and pulmonary monitoring, Langford said. Diagnosis is one of exclusion, meaning other diseases or conditions must be ruled out. Langford suggests those experiencing symptoms such as weakness of any extremity; tightness in the leg or arm; muscle twitching of the leg, arm or tongue; slurred speech; or difficulty swallowing see their primary physician and get a referral to the Muscular Dystrophy Clinic at the Jackson Medical Mall, which works in conjunction with UMC.
For now, the Muirheads treasure what time they have left. They will celebrate their first wedding anniversary Sept. 16.
"All we are asking for is a chance," Ricky said.
"A chance to live a normal life as long as you can," Vicki said. "In this situation, it's all about quality of life. And if this drug can give you more quality in the amount of time you have, why not?"
To comment on this story, call Shanderia K. Posey at (601) 961-7264.

Monday, September 8, 2008

The ALS Association to Support Trial of Lithium

The ALS Association to Support Trial of Lithium
By Richard Robinson
The ALS Association is funding a major clinical trial to determine if lithium can slow disease progression in patients in the early stages of ALS. The study builds on earlier promising results from preclinical research and a small, open-label investigation in ALS patients.
“Lithium has generated a lot of interest in the ALS community,” according to Lucie Bruijn, Ph.D., Senior Vice President, Research and Development of The ALS Association. “This trial is vital for testing the efficacy of lithium in a well-controlled way. It’s the crucial next step for investigating the potential of this drug.”
The trial is being supported through The Association’s TREAT ALS (Translational Research Advancing Therapies for ALS) initiative, and will use the TREAT ALS/NEALS Clinical Trials Network. Lead investigators for the study will be Merit Cudkowicz, M.D., and Swati Aggarwal, M.D., of Massachusetts General Hospital, Lorne Zinman, M.D., from the University of Toronto, Petra Kaufman, M.D., from Columbia University, and Jeremy Shefner, M.D., of SUNY Upstate Medical University.
In addition to The ALS Association, funding partners for the new study include the National Institute of Health/ National Institute of Neurologic Diseases and Stroke and ALS Society of Canada. Investigators worked together with the funding agencies to design the most appropriate study within the shortest possible time frame. “Collaboration among researchers and funding agencies makes this trial possible,” Dr. Bruijn said. “All of us are working together to support this important research to find a new treatment for ALS.”
Lithium is a simple chemical substance approved for use in humans and prescribed as a mood stabilizer. Lithium protects neurons in the brain in animal models of neurodegenerative diseases, including Alzheimer’s disease and Parkinson’s disease, and has been recently shown to do the same in a mouse model of ALS. In that study, lithium prolonged survival and protected cells in both the brain and spinal cord. While the exact mechanism of lithium’s effect is unknown, researchers have proposed it promotes clearance of toxic protein accumulation.
A study in a small number of ALS patients compared lithium plus riluzole (an approved treatment for ALS) to riluzole alone. After 15 months, no deaths occurred in the 16 participants treated with lithium plus riluzole, while 29% of those taking riluzole alone had died. Patients on lithium also had a markedly lower decline in disease-related disability. However, the trial was limited by the small numbers of patients and the absence of a placebo treatment—both the patient and the doctor knew whether the patient was taking lithium or not, which may have influenced the response to treatment or interpretation of disability. Due to diagnostic and prognostic variability in persons showing symptoms of ALS, the small numbers in this study were not sufficient to know whether lithium was indeed an effective treatment. Many apparently effective treatments in open trials (trials lacking a concurrent placebo group) fail in larger double-blind trials, in which neither doctor nor patient knows whether the patient is receiving the study drug.
The new trial will be a double-blind, placebo-controlled trial with 84 patients, who will be randomized to either lithium or placebo. A review of data will occur after the 84th person is enrolled, and then a decision will be made on whether to expand to 250 patients.
Patients may be included if they are within three years of their diagnosis and are not already taking lithium. Treatment will be for up to one year. The disease course and safety assessments will be measured at regular intervals over that time. Further details including trial sites, enrollment criteria and start dates will follow shortly. “We formed a collaboration with Canadian and US sites to perform a study that will provide an answer quickly on whether lithium is effective in the treatment of ALS. The study is designed to answer this key question definitively and with appropriate monitoring of safety,” commented Merit Cudkowicz, M.D., one of the lead investigators on the study.
Denise Figlewicz, Ph.D., Director of Research at the ALS Society of Canada says, “We welcome this opportunity to support the lithium clinical trial. The promising results from the Italian study data serve as the impetus for new research and new treatment strategies. This is very exciting news for the ALS community. We also welcome this opportunity to work together with our American colleagues. This collaborative approach between Canada and the United States will serve as a model for subsequent clinical trials.”

Thursday, September 4, 2008

Cells transformed in promising research

Cells transformed in promising research

NEW YORK (AP) -- Talk about an extreme makeover: Scientists have transformed one type of cell into another in living mice, a big step toward the goal of growing replacement tissues to treat a variety of diseases.
The cell identity switch turned ordinary pancreas cells into the rarer type that churns out insulin, essential for preventing diabetes. But its implications go beyond diabetes to a host of possibilities, scientists said.
It's the second advance in about a year that suggests that doctors might be able to use a patient's own cells to treat disease or injury without turning to stem cells taken from embryos.
The work is "a major leap" in reprogramming cells from one kind to another, said one expert not involved in the research, John Gearhart of the University of Pennsylvania.
That's because the feat was performed in living mice rather than a lab dish, the process was efficient, and it was achieved directly without going through a middleman like embryonic stem cells, he said.
The newly created cells made insulin in diabetic mice, though they were not cured. But if the experiment's approach proves viable, it might lead to treatments like growing new heart cells after a heart attack or nerve cells to treat disorders like ALS, also known as Lou Gehrig's disease.
Douglas Melton, co-director of the Harvard Stem Cell Institute and a researcher with the Howard Hughes Medical Institute, cautioned that the approach is not ready for people.
He and his colleagues report the research in a paper published online Wednesday by the journal Nature.
Basically, the identity switch comes about by a reprogramming process that changes the pattern of which genes are active and which are shut off.
Scientists have long hoped to find a way to reprogram a patient's cells to produce new ones. Research with stem cells, and similar entities called iPS cells that were announced last year, has aimed to achieve this in a two-step process.
The first step results in a primitive and highly versatile cell. This intermediary is then guided to mature into whatever cell type scientists want. That guiding process has proven difficult to do efficiently, especially for creating insulin-producing cells, Gearhart noted.
In contrast, the new method holds the promise of going directly from one mature cell type to another. It's like a scientist becoming a lawyer without having to go back to kindergarten and grow up again, Melton says.
So, he says, scientists may be able to replace dead nerve or heart cells in people by converting some neighboring cells. At the same time, he stressed that it's still important to study embryonic stem cells and iPS cells.
The Melton team started its work with pancreas cells that pump out gut enzymes used in digestion and turned them into pancreatic "beta" cells, which make insulin.
The researchers destroyed beta cells in mice with a poison, giving the mice diabetes. Then they injected the pancreas with viruses that slipped into the enzyme-making cells. These viruses delivered three genes that control the activity of other genes.
Just three days later, new insulin-secreting cells started to show up. By a week after that, more than a fifth of the virally infected cells started making insulin. That shows "an amazingly efficient effect," commented Richard Insel, executive vice president of research at the Juvenile Diabetes Research Foundation.
Scientists found evidence that the newcomers were converts from mature enzyme-making cells. They identified the new cells as beta cells by their detailed appearance and behavior, and Melton said they've continued functioning for months.
The new cells didn't fully replenish the insulin supply, but maybe there were too few of them or they were hampered by not forming clusters like ordinary beta cells do, researchers said.
The work brings "more excitement to the idea of using reprogramming as a way to treat diabetes," said researcher Mark Kay of Stanford University, who is studying the approach with liver cells.
Christopher Newgard, who studies beta cells at Duke University Medical Center, called the work convincing but cautioned that significant scientific questions remained about using the approach in treating disease.
Melton, who began his diabetes research in 1993 when his infant son was diagnosed with the illness, said he's obsessed with trying to find a new treatment or cure for Type 1 diabetes, in which beta cells are destroyed.
"I wake up every day thinking about how to make beta cells," he said.
Melton said he hopes drugs can replace the virus approach because of concern about injecting viruses into people.
As for converting other kinds of cells, scientists noted that the two cell types in the mouse experiment are closely related, and it remains to be shown whether the trick can be achieved with more distant combinations. In any case, scientists would have to deliver different reprogramming signals to other kinds of cells, Melton said.
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