Tuesday, September 23, 2008
Stem cells open door for studying human disease By Julie Steenhuysen
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.
Monday, September 22, 2008
Sunday, September 21, 2008
whoever wrote this-please email me
please email me at
schemera@yahoo.com
Friday, September 19, 2008
Wednesday, September 17, 2008
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
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
Monday, September 15, 2008
Saturday, September 13, 2008
Japan university gets patent for stem cell breakthrough Fri Sep 12, 12:02 PM ET
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
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
Tuesday, September 9, 2008
ALS patient denied access to drug/GREAT ARTICLE !!!!!!
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
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
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.
Copyright 2008 The Associated Press. All rights reserved.This material may not be published, broadcast, rewritten, or redistributed.
All AboutDiabetes • Stem Cell Research
Wednesday, August 27, 2008
Tuesday, August 26, 2008
The ALS Association to Support Trial of Lithium
CALABASAS HILLS, Calif. (August 26, 2008) -- 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 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 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 collaboration with Canadian and U.S. 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 said, "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."
ALS and The ALS Association Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost, leading to progressive paralysis.
The ALS Association is the only national, not-for-profit voluntary health organization devoted solely to fighting ALS through research, patient services, advocacy and public education and information. TREAT ALS is a drug discovery program and clinical trials process created by The Association that accelerates discovery and testing of clinical candidates.
###
The ALS Association National Office
27001 Agoura Road, Suite 150 Calabasas Hills, CA 91301-5104 (818) 880-9007 FAX (818) 880-9006
Contact: The ALS Association Gary Wosk (818) 587-2241 gwosk@alsa-national.orggwosk@alsa-
Friday, August 22, 2008
video
I ONLY MADE IT TO THE 3:17 min. mark. BEFORE I HAD TO SHUT IT OFF
.http://www.youtube.com/watch?v=yZeWWJHTV1g&feature=email
IT MIRRORS MY LIFE. except for the cancer
Monday, August 18, 2008
A POEM BY A PALS-people w/ALS
I face a giant
It's not one you can see
Yet, it is not invisible
The giant insists on winning
It's hold reaches deep
I am trapped within its grasp
The giant holds me tight, yet
I am not captured.
We live as one
The giant feeds on motor neurons
Slowly, it disables my limbs
The giant wants all of me
Yet, not all is his to take
For my soul belongs to Jesus
I am not captured.
Peace prevails
The giant has taken my voice
It has taken my throat too
Yet, my spirit remains free
What can be seen belies truth
There is more to me unseen
I am not captured.
There is strength
A deep well abides within
I live, even though I die
Yet, the giant demands more
With hands clasped, I pray
Thank you Lord for this day
The giant will not prevail
I am not captured.
I am alive
A wellspring of hope abides
Though my body slowly dies
My soul grows stronger still
Yet, the giant knows this not
No matter, God is on my side
The angels attend to me
I am not captured.
(© copyright, Jan 2008, Butterfly)
Saturday, August 16, 2008
ALS Association Research Grants Announced
ALS Association Research Grants Announced
New research grants by The ALS Association will support investigations of new disease models, pathogenesis of ALS, biomarkers of disease progression, and development of new treatments. The research portfolio supported by the Association is designed to speed therapy development by attacking ALS on all fronts, from basic research to clinical trials. Each year the Association funds the most promising research from hundreds of investigator-initiated proposals. In addition, it actively seeks researchers to undertake studies identified by the Association as critical for filling gaps in knowledge of ALS. The Association also gives the Milton Safenowitz post-doctoral fellowship award for ALS research to encourage young scientists to focus their research efforts on ALS.
Newly funded investigator-initiated research will study the following important questions:
Why do motor neurons die in ALS?
The cause of ALS is death of motor neurons, nerve cells that control muscle movements. A central question in ALS is why, and how, these neurons die. Nine new projects examine different aspects of that question.
Researchers have found that some motor neurons are more susceptible than others to the disease process. Now, they will examine how these different cells vary in the genes they express during the disease, looking for clues that influence vulnerability to ALS. Researchers will also investigate the details of the pathways by which motor neurons die, in order to improve prospects for interrupting those processes. [Aebischer, Tsunemi]
While it is motor neurons that die in ALS, evidence has accumulated to suggest that other cell types contribute to the disease. Researchers will use mice that express the disease causing SOD1 gene in multiple different cell types, to examine the role each plays. Researchers will also investigate changes at the junction between nerve and muscle to determine how muscle contributes to the disease. Finally, they will ask whether new compounds that aid the cell’s powerhouses can reduce the harmful effects of one type of cell. [Roos, Manuel, Cassina]
A newly discovered, pervasive form of gene regulation involves so-called “micro-RNAs,” which act at many different levels to control cell function. Recently researchers have leaned that micro-RNAs are altered in models of ALS, suggesting they may be a key to understanding the disease process. Researchers will extend these studies to further explore this phenomenon. [Moeller]
Researchers will further their investigations of a newly discovered cause of ALS, the protein TDP-43, to determine how the abnormal protein accumulates and why it causes disease. Understanding this may help explain how different causes can lead to the same disease, and whether there are common “downstream” targets in each form of ALS that would be appropriate therapeutic targets. [Petrucelli, Sanelli, Morton]
Why is mutant SOD1 toxic?
Mutations in the superoxide dismutase (SOD1) gene cause about 2% of all cases of ALS, but it is not yet clear how this mutation leads to the disease. Two projects examine that question, which should help in understanding not only SOD1-caused ALS, but possibly other forms as well.
Researchers know that mutant SOD1 protein forms insoluble clumps in ALS motor neurons, but it is possible that soluble forms of the mutant protein are actually more toxic. Now, researchers will study these soluble forms to understand how they affect motor neurons. [Marklund]
Researchers will also examine whether and how the cell environment affects the toxicity of SOD1, an important step in determining whether altering that environment might potentially be beneficial. [Carri]
What can we learn from new animal models of ALS?
Animal models are vital to understanding ALS and searching for treatments. A new model uses the fruit fly, whose advantages include its rapid generation and extensive understanding of its genetics. This model will be used to explore a new ALS gene to investigate how this gene causes the disease, and how other factors contribute to the disease. Another model will place a newly discovered ALS gene into mice to investigate how this gene causes the disease. [Tsuda, Dupuis]
Can imaging be used to track disease progression?
Researchers will build on previous studies to determine if magnetic resonance spectroscopy can be used to discover biomarkers of disease progression in ALS patients. This non-invasive technique is related to the more common MRI (magnetic resonance imaging), but provides additional information about chemical compounds in the body. Researchers hope that this technique will reveal one or more compounds that change in step with the disease, allowing it to be used to accurately track disease progression. [Benatar]
How can transplanted motor neurons be optimized for therapy?
One potential therapy for ALS would be to transplant healthy motor neurons into the spinal cord. However, functional motor neurons need long extensions, called axons, to reach their targets, and growing these axons in transplanted cells is an unmet challenge. Researchers will examine whether genetically modifying motor neurons can help them to better grow axons after transplantation. [Strittmatter]
Friday, August 15, 2008
Thursday, August 14, 2008
this is why i have truly amazing friends and the reason i write this blog
what's up man? Long time no talk. I haven't seen you in awhile, but read your blog daily and am truly inspired by your courage and fight. It's very humbling when I think about the many things I used to stress about at work and in my personal life and then compare it to the challenges you experience every day. Sadly I have a colleague who I've worked with for years who was recently diagnosed with brain and lung cancer. Just as I told her, God is just testing you to see how strong you are. Make sure you continue to fight and show him how serious you are about beating this sucker! Only the strong survive and I have no doubt you have the strength to keep on fighting and ultimately win. You are constantly in my thoughts and prayers. As evident by the comments on your blog, you have an amazing supporting cast and we all look forward to seeing you get through this.
As Jimmy Valvano one said "Cancer (ALS) can take away all my physical ability. It cannot touch my mind; it cannot touch my heart; and it cannot touch my soul. And those three things are going to carry on forever. Don't give up, don't ever give up".
All the best!-Chris
Wednesday, August 13, 2008
BEIKE IN THE NEWS
The Repair Stem Cell Institute would like to congratulate Beike for the excellent work they are doing in the field of stem cell treatments. Throughout history, experimental treatments have been performed in academic settings and huge advancements in knowledge have been the result that all of mankind has benefited from. Beike is following this trail blazing attitude and doing so while maintaining an extremely high level of quality and standard of care and placing the needs of the patient at the forefront of their focus. We would like to join the academic institutions, their government and their world-wide patient base in voicing our support of their efforts in innovative technologies in general and stem cells specifically. We hope their example can be matched by many other treatment centers; to ultimately provide great benefits to science, medicine and the patients who are without significant treatment options and may be helped by stem cell treatments today.Visit our website, click on "Treatment Centers" and see what the Repair Stem Cell Institute was saying about Beike even before their P.R. was released. Without a doubt, they are the World’s #1 stem cell treatment center.
Keep up the good work!
David Granovsky
Senior Editor
Repair Stem Cell Institute
http://www.repairstemcells.org/
Friday, August 8, 2008
Scientists create stem cells for 10 disorders By STEPHANIE NANO, Associated Press Writer

By STEPHANIE NANO, Associated Press Writer1 hour, 3 minutes ago
Harvard scientists say they have created stems cells for 10 genetic disorders, which will allow researchers to watch the diseases develop in a lab dish.
This early step, using a new technique, could help speed up efforts to find treatments for some of the most confounding ailments, the scientists said.
The new work was reported online Thursday in the journal Cell, and the researchers said they plan to make the cell lines readily available to other scientists.
Dr. George Daley and his colleagues at the Harvard Stem Cell Institute used ordinary skin cells and bone marrow from people with a variety of diseases, including Parkinson's, Huntington's and Down syndrome to produce the stem cells.
The new cells will allow researchers to "watch the disease progress in a dish, that is, to watch what goes right or wrong," Doug Melton, co-director of the institute, said during a teleconference.
"I think we'll see in years ahead that this opens the door to a new way to treating degenerative diseases," he said.
The new technique reprograms cells, giving them the chameleon-like qualities of embryonic stem cells, which can morph into all kinds of tissue, such as heart, nerve and brain. As with embryonic stem cells, the hope is to speed medical research.
Research teams in Wisconsin and Japan were the first to report last November that they had reprogrammed skin cells, and that the cells had behaved like stem cells in a series of lab tests. Just last week, another Harvard team of scientists said they reprogrammed skin cells from two elderly patients with ALS, or Lou Gehrig's disease, and grew them into nerve cells.
Melton said the new disease-specific cell lines "represent a collection of degenerative diseases for which there are no good treatments and, more importantly, no good animal models for the most part in studying them."
A new laboratory has been created to serve as a repository for the cells, and to distribute them to other scientists researching the diseases, Melton said.
"The hope is that this will accelerate research and it will create a climate of openness," said Daley.
He expects stem cell lines to be developed for many more diseases, noting, "this is just the first wave of diseases." Other diseases for which they created stem cells are Type 1, or juvenile, diabetes; two types of muscular dystrophy, Gaucher disease and a rare genetic disorder known as the "bubble boy disease."
Daley stressed that the reprogrammed cells won't eliminate the need or value of studying embryonic stem cells.
"At least for the foreseeable future, and I would argue forever, they are going to be extremely valuable tools," he said.
The reprogramming work was funded by the National Institutes of Health and private contributions to the Harvard Stem Cell Institute.
___
On the Net:
Harvard Stem Cell Institute: http://www.hsci.harvard.edu
Thursday, August 7, 2008
Skin cells produce library of diseased stem cells By Maggie Fox, Health and Science Editor
By Maggie Fox, Health and Science EditorThu Aug 7, 12:01 PM ET
U.S. stem cell experts have produced a library of the powerful cells using ordinary skin and bone marrow cells from patients, and said on Thursday they would share them freely with other researchers.
They used a new method to re-program ordinary cells so they look and act like embryonic stem cells -- the master cells of the body with the ability to produce any type of tissue or blood cell.
The new cells come from patients with 10 incurable genetic diseases and conditions, including Parkinson's, the paralyzing disease amyotrophic lateral sclerosis, or ALS, juvenile diabetes and Down's Syndrome.
Writing in the journal Cell, the team at Harvard Medical School and Children's Hospital in Boston said the point is not yet to treat anyone, but to get as many researchers as possible experimenting with these cells in lab dishes to better understand the diseases.
"This is just the first wave of diseases," said Dr. George Daley, who is also a Howard Hughes Medical Institute investigator.
The new cells are called induced pluripotent stem cells, or iPS cells, and are made using four genes that reprogram an ordinary adult cell into a primitive stage resembling the first days of a human embryo -- a method pioneered by Shinya Yamanaka of Kyoto University in Japan.
WHAT GOES WRONG
"They allow researchers ... to watch the disease progress in a dish, to watch what goes right or wrong," said Harvard's Dr. Doug Melton, who will head up the distribution of the cells. "I think we'll see in the years ahead that this opens the door to a new way of treating degenerative diseases."
Every cell in the human body contains the same genetic instructions, and in people with inherited genetic diseases, every cell carries the same mistakes, Daley and Melton said.
Stem cells -- and the new iPS cells -- will grow virtually immortal in the lab, and given the right conditions, can be made to form any desired tissue, from heart muscle to brain cells.
One day these might be used as tailor-made patches to fix diseased or damaged organs, but right now Melton said it is important to simply understand the diseases.
"We don't even know when a patient gets diabetes if each patient gets it the same way," Melton said. "There could be 50 different ways."
One thing his lab will do is try to make pancreatic beta cells -- the cells that make insulin and are destroyed in type 1 diabetes -- and study them to see what may be different among patients with the disease.
While the cells are an alternative to the more controversial embryonic stem cells, taken from a human embryo, Daley and Melton are adamant that they do not replace them.
For one thing, viruses are used to carry the transformative genes to make the iPS cells. Daley says cloning technology is still superior. "The egg does it faster and better," he said.
The federal government severely restricts embryonic stem cell research because of moral objections, but the Harvard lab works with private funding.
Melton said researchers can get batches of cells from the lab to grow on their own. Other cell types they have made to date include samples from people with Huntington's disease, a form of combined immunodeficiency commonly known as "bubble boy's disease," Lesch-Nyhan syndrome, Gaucher's disease, and two forms of muscular dystrophy.
(Reporting by Maggie Fox; editing by Julie Steenhuysen and Stacey Joyce)
Monday, August 4, 2008
Beike Uses Research Forum to Showcase its Stem Cell Therapies
Friday, August 01, 2008 9:53 PM
Recently, Shenzhen’s Beike Biotechnology held the first annual 2008 China Stem Cell Stem Cell Technology Forum, a gathering that was attended by over 300 of the world’s most renowned stem cell biologist and researchers. The Mandarin-language conference included many participants from China , while others attended from the United States , Canada , India , Australia , and Malaysia . The focus of the conference was on advanced induced pluripotent stem (iPS) cell research and therapeutic uses of stem cells. The event was held at the China Medical City complex in Taizhou , China and chaired by Dr. Sean Hu, who also serves as Chairman of Beike Biotechnology Co., Ltd. Beike is a controversial company because it has already begun treating patients with injections of stem cells, usually a combination of umbilical cord cells and stem cells derived from each patient. Beike claims to treat over 250 patients per month, a total of more than 3,000 so far. Approximately 70% to 80% of the patients report themselves satisfied with their treatments, and the treatments have the ability to produce at least some therapeutic effect in 86% of its patients, according to statistics provided by Beike. Beike has not submitted its techniques to the rigors of a double-blind clinical test. Nor has it done the otherwise necessary animal testing. Nevertheless, the company points to its success with the majority of its patients as sufficient proof that its stem cell therapies are a valuable addition to the medical arsenal. Beike charges its patients between $20,000 and $30,000 for several rounds of stem cells injections, which it says can help cure diseases such as Alzheimer’s, atoxia, multiple sclerosis, optic nerve hypoplasia, and spinal muscular atrophy . Although most patients come from China , its therapies draw significant numbers of patients from the West. The former Lord Mayor of Manchester , Audrey Jones, for example, flew to China for a round of stem cell injections to treat her cerebellar atrophy and came back to England proclaiming herself much improved. Before the Beike therapy, she was confined to a wheelchair. Now she plans to walk into Manchester City Hall without aid. Beike’s web pages are replete with similar success stories for a wide variety of diseases (www.stemcellschina. com). Stem cells are commonly used for bone marrow transplants in the West, but experts say that bone marrow, which constantly undergoes renewal, is a better candidate for basic stem cell injections than Beike’ specialties of nerve/heart/ spinal cord problems. Stem cells will not necessarily find the correct nerve to repair without coaxing, according to the experts. They believe a set of simple stem cell injections is unlikely to correct septo-optic dysplasia, for example. On the other hand, Dr. Hu does not claim to know the mechanism behind Beike’s treatments, but he points to the results. Adding to Beike’s credibility is the high quality of its academic affiliations. Beike was founded with capital from Beijing University , Hong Kong University of Science and Technology and Shenzhen City Hall . It is also supported with funds from the China State National Fund. Its research and clinical work benefits from collaborations with Beijing University , Hong Kong University of Science and Technology, No. 3 Army Medical University, Zhongshan Medical University , Guiyang Medical College and Zhengzhou University . Beike has over 60 PhD researchers focusing on stem cell research in its labs. In November 2007, Beike Biotechnology teamed up with the Szenzhen Graduate School of Tsinghua University to establish a laboratory that will research cell reprogramming and gene engineering. The lab will conduct research on stem cells, the nuclear transfer and reprogramming of cells, and monoclonal antibodies. The new lab was honored with the “China Key Laboratory” designation and given a grant worth $4 million for equipment from the Shenzhen Municipal Government. The grant was part of $12 million given out in 2007 by Shenzhen to encourage innovative technologies. In June of this year, Beike announce it began outfitting a 21,500 square foot comprehensive medical stem cell storage and processing facility in Jiangsu province. When completed, the Jiangsu Stem Cell Storage Facility will boast the technology to freeze, store, and process a broad range of human stem cell samples for clinical application. Initially, the facility will provide stem cell banking for human umbilical cord blood stem cells, placenta stem cells, amniotic membrane stem cells, bone marrow stem cells, and later it will also be able to house induced pluripotent stem cells (iPS). The facility is expected to begin operations later this year, adding another dimension to Beike’s manifold stem cell-related activities
http://www.istockan alyst.com/ article/viewarti cle+articleid_ 2466354&title=Beike_ Uses_Research. html
Thursday, July 31, 2008
Cell changes may help Lou Gehrig's research
Cell changes may help Lou Gehrig's research
YouNewsTV™
Story Published: Jul 31, 2008 at 11:22 AM PDT
By Associated Press WASHINGTON (AP) - Using a new technique to reprogram cells, scientists are growing neurons from people with Lou Gehrig's disease, a possible first step in understanding how the deadly illness develops.Technically known as amyotrophic lateral sclerosis, the disease damages the nerve cells in the brain and spinal cord, eventually leading to death. The ALS Association estimates that as many as 30,000 Americans may have the disease at any given time."What we now have in the culture dish is cells that have the same genetic makeup as the ALS patient and they are the same cells that are affected by the disease," said Dr. Chris Henderson, co-director of the Center for Motor Neuron Biology and Disease at Columbia University.That means that, for the first time, scientists hope to be able to observe the development of the disease in the cells and, from that, possibly begin studies of treatments."There is no way we could go to an ALS patient and take these cells," Henderson pointed out.Instead, they used skin cells from two patients, aged 82 and 89, and were able to reprogram the cells into a type of adult stem cell, and from that into nerve cells, the researchers report in Friday's edition of the journal Science.Co-author Dr. Kevin Eggan of the Harvard Stem Cell Institute said what they hope to do next is study the cells in the lab and compare them with cells of someone who doesn't have that disease.ALS is a slowly developing disease and the test cells have not yet begun to show illness, they noted. But in similar studies in mouse cells the cultured neurons did develop the disease.Dr. Lucie Bruijn, science director and vice president of the ALS Association, called the work a first step."This is the beginning," she said in a telephone interview. "It's a most important approach.""Now we have to figure out whether these motor neurons are able to mature," she added. "For these to be useful for drug development we need mature motor neurons."The researchers worked with a form of ALS that is caused by a defect in a single gene, a rare form of the disease.But Henderson said that they even though the vast majority of cases are caused by different triggers, they hope to learn about the mechanism of the disease, how it develops after being triggered."Up until now, it's been impossible to get access to the neurons affected by ALS and, although everyone was excited by the potential of the new technology, it was uncertain that we would be able to obtain them from patients' skin cells," Henderson said. "Our paper now shows that we can generate hundreds of millions of motor neurons that are genetically identical to a patient's own neurons. This will be an immense help as we try to uncover the mechanisms behind this disease and screen for drugs that can prolong life."The research was supported by the Harvard Stem Cell Institute, Project ALS, the SMA Foundation, MDA Wings Over Wall Street, the Spina and Bowen families, the New York Stem Cell Foundation, the National Science Foundation and the John D. and Catherine T. McArthur Foundation
Wednesday, July 30, 2008
World Stem Cell Summit
Here is a link to the program: http://www.worldstemcellsummit.com/
Thursday, July 24, 2008
these are some of the answers people gave to the question: What is your biggest ALS fear?
I'M SHARING SOME OF THE MIND SETS OF PEOPLE WHO ARE FACING THE FUTURE W/ THIS DISEASE. THIS DISEASE HAS DEPLETED OUR PHYSICAL ABILITIES. AND IT DOES NOT STOP.
- Total paralysis, I'm scared of it
- losing the ability to walk.
- I'm with Sheila on this one. Total paralysis. I nearly drowned when I was a kid, and I've had a fear of being helpless ever since. I've become claustrophobic because of this. The idea of being trapped in my body, not even able to move my head, scares the crap out of me. I believe this will drive me insane.
- my mother having to watch me die.
- No cure.
- I can't spend time fearing what is ahead for me...I'll go nuts. I only focus on today and what I can still do. Everyday I pray for 3 things...strength, courage, and inner peace.
- I fear being alive when the body bag is zipped!
- A long drawn-out diminishing until I actually die. I have a slow form of MND - that is great in the earlier stages as I have longer to enjoy doing the things I can still do but that benefit is amply balanced by the fact that the later stages when total paralysis kicks in will last much longer too. I have no fear of death itself but I certainly fear the stage before when I am unable to do anything except just be there.
Wednesday, July 23, 2008
Jul 22 What is your biggest ALS fear?
What is your biggest ALS fear?
Created by TipaCow on www.patientslikeme.com
my answer:
it is not DYING that scares-i'm okay w/ that. it's GETTING dead that keeps me awake at night....
Monday, July 21, 2008
World’s First Genome-Wide Spinal Cord Atlas Unveiled
July 21, 2008
World’s First Genome-Wide Spinal Cord Atlas Unveiled
Co-Funded by The ALS Association
The ALS Association has joined a funding consortium of non-profit and private organizations to fund the Allen Spinal Cord Atlas, which was unveiled as the world’s first genome-wide map of the mouse spinal cord, key to the study of amyotrophic lateral sclerosis and other diseases, disorders and traumatic injuries of the spinal cord.
Created by the Seattle, Wash.-based Allen Institute for Brain Science, the atlas enables researchers to access the free online data to advance their research surrounding these conditions.
“The atlas enables scientists to determine the location of genes and their expression patterns at the cellular level in the spinal cord,” said Lucie Bruijn, Ph.D., science director and vice president of The Association. “This will provide an important reference when trying to understand gene changes and how these are linked to disease in mouse models of ALS.”
From Lou Gehrig’s Disease (ALS) to Spinal Muscular Atrophy, spinal cord related diseases and disorders affect people of all ages. As many as 30,000 Americans, including military veterans, suffer from ALS at any given time; and multiple sclerosis affects 2.5 million people worldwide, to name a few. Nearly one-quarter of a million Americans—including several thousand troops who have served in Iraq—have suffered or suffer from a spinal cord injury.
The Institute’s unique funding model, designed to transform public, private and foundation funds into breakthrough scientific discoveries, supported the Spinal Cord Atlas’ dedicated consortium of public and private entities—including The Association, PVA Research Foundation, Wyeth Research, PEMCO Insurance, National Multiple Sclerosis Society, International Spinal Research Trust and philanthropist and Institute founder Paul G. Allen, as well as numerous anonymous donors.
“We were so thrilled to work with such a diverse array of funders with a shared goal of supporting this important project—which the Institute would never have completed without their support,” said Elaine Jones, chief operating officer at the Allen Institute. “The Allen Spinal Cord Atlas serves as a successful example of how major scientific projects can be funded, and we are eternally grateful to our partners.”
The atlas will be completed in a 12-month time frame. While inaugural data—approximately 2,000 genes—from the Allen Spinal Cord Atlas is now available (http://mousespinal.brain-map.org), the Institute will continue to follow its founding mission and upload additional information until the projected completion by the end of 2008. It is estimated that hundreds of users from universities, research institutes, pharmaceutical companies and government organizations will use the atlas.
When completed, the Allen Spinal Cord Atlas will detail approximately 20,000 genes including data from youth and adult developmental stages. It will also feature data across the full length of the spinal cord as well as anatomical reference sections.
The ALS Association is the only not-for-profit voluntary health organization dedicated solely to the fight against ALS through research, patient care, advocacy and public education. The mission of The Association is to lead the fight to cure and treat ALS through global, cutting-edge research, and to empower people with Lou Gehrig’s Disease and their families to live fuller lives by providing them with compassionate care and support.
Sunday, July 20, 2008
Thursday, July 17, 2008
If everyone loved and nobody lied
If everyone shared and swallowed their pride
Then we'd see the day when nobody died
When nobody died....We'd see the day,
we'd see the day
When nobody died
We'd see the day, we'd see the day
When nobody died
We'd see the day when nobody died
Wednesday, July 16, 2008
it's at the 9-12 month mark when will know if it works
Dear all,
You are receiving this message because you or your PALS are participating in or have participated in or requested information about the online worldwide ALS lithium study. We wanted to let you know that the preliminary three month study report is now available for download. Please go to http://alslithium.atspace.com/ and download the report from the latest "updates" link at the bottom of the page. Please note that this is a preliminary report which has not yet been edited or approved by PatientsLikeMe.We will continue to collected data through the 6 month point after which we will write up a final report. All data collection and analysis after that point will be done by PatientsLikeMe.Our basic results so far are, in summary:At the three month point:
Lithium does not affect ALS progression rates, at least for most PALS
Taking riluzole with lithium does not make a difference
Reaching a blood level of 0.4 does not make a difference
The ALSFRS-R score when lithium was started doesn't make a difference
Lithium may relieve cramps and fascics and spasticity for some at low doses (0.2 mmol/l blood concentration)Although not in the 3 month report, it seems that PALS who are taking an Omega 3 supplement are progressing a bit more slowly. There was also an interesting research report that came out yesterday that calcium supplements may be helpful (Medications and laboratory parameters as prognostic factors in amyotrophic lateral sclerosis. Qureshi M, Shui A, Dibernardo AB, Brown Jr RH, Schoenfeld DA, Cudkowicz ME.) The research abstract didn't give the dosage, but a quick search of PLM indicated that many PALS on calcium are taking ~1200 mg/day.
Thank you so, so much to everyone who has provided data for the lithium trial. So far our research results have been disappointing, but it is so much better to get results quickly so that we can move onto the next thing rather than to have to wait 2 to 3 years for the traditional research machinery to do its work. You have shown what a dedicated groups of PALS and CALS can do!Best wishes to everyone -- I'll be in touch,Karen
Posted: 15 Jul 2008 03:21 PM CDT
“Hope” is the thing with feathers
That perches in the soul
And sings the tune without the words
And never stops at all,
And sweetest in the gale is heard;
And sore must be the storm
That could abash the little bird
That kept so many warm.
I’ve heard it in the chillest land
And on the strangest sea,
Yet never, in extremity,
It asked a crumb of me.
By: Emily Dickinson
- Emily Dickinson
Monday, July 14, 2008
Saturday, July 12, 2008
a great friend sent this to me
I can see clearly now, the rain is gone,
I can see all obstacles in my way
Gone are the dark clouds that had me blind
It's gonna be a bright (bright), bright (bright)Sun-Shiny day. (I FUCKING HOPE SO!!)
Johnny Nash ~ I Can See Clearly Now
Friday, July 11, 2008
good news
By TIM MARTIN,
Associated Press WriterTue Jul 8, 4:06 AM ET
Supporters of a ballot measure that would loosen Michigan's restrictions on embryonic stem cell research took a big step toward placing it on the November ballot.
The Stem Cell Research Ballot Question Committee said Monday it turned in more than 570,000 voter signatures backing the measure. More than 380,000 of them must be ruled valid for the proposal to reach voters.
Backers say embryonic stem cell research holds the potential to help treat or cure diseases such as Alzheimer's, Parkinson's, cancer, sickle cell anemia and diabetes.
The politically diverse group that helped launch the campaign Monday included former Republican U.S. Rep. Joe Schwarz and former Democratic gubernatorial hopeful Larry Owen.
"It is research that we know has a high chance of curing many diseases and saving many lives," said Owen, the campaign chairman.
Opponents raise ethical concerns because the research involves the use and destruction of human embryos. The Michigan Catholic Conference and Right to Life of Michigan oppose the proposal and an opposition group called Michigan Citizens Against Unrestricted Science and Experimentation is forming.
"The proposal is deliberately deceptive," group spokesman David Doyle said. "It's the confusing legalese that is the problem."
Ballot proposal supporters countered that their opponents are the ones misrepresenting the issues. Supporters are trying to make the ballot because their efforts to change state law have failed in the Legislature.
Some embryonic stem cell research is allowed in Michigan. But the state's laws related to the research are among the nation's most restrictive, allowing only the use of stem cell lines from California, Illinois or other states with less restrictive laws. Those lines sometimes are patented by other researchers.
Ballot proposal supporters say changing Michigan's law would help broaden the type of available stem cell lines, opening up new avenues for potential cures and eventually drawing more research money to the state.
The proposal would change Michigan law to allow research on donated embryos created during fertility treatments that otherwise would be discarded. It's now a state felony to use new embryonic stem cells for research.
Supporters of the proposal to amend the constitution say it protects and strengthens Michigan's ban on human cloning. The proposal says nothing in it "shall alter Michigan's current prohibition on human cloning."
Opponents say the proposal does not explicitly put a ban on human cloning in the state constitution, so cloning could be allowed if state law is ever changed to permit it.
Stem cells are rare cells in tissues that give rise to most other cells. While many scientists say embryonic stem cell research holds the most medical versatility and potential, critics are upset that stem cells are harvested from adults or umbilical cords.
___
On the Net:
Stem Cell Research Ballot Question Committee: http://www.curemichigan.com
Michigan Citizens Against Unrestricted Science and Experimentation: http://www.micause.com
Thursday, July 10, 2008
A POEM: — anon·y·mous·ly SENT TO ME
Three Little Letters
In Honor of Robert J Cram
A life altering change at a blink of an eye,
a debilitating disease can leave you wondering “Why”?
Weakness and Strength is the bodies constant fight,
no cure to the illness leaves a future un-bright.
Speaking with no words is a challenge to conquest,
faith and patience is what does it best.
Courage and Hope is your medication for pain,
time is the enemy with nothing to gain.
Day to day struggles makes it difficult to see,
that God has a plan and knows what’s best for me.
Education and awareness is priority no less,
with hopes to finding a cure and strike out ALS.
-Ashley Meadows
June 2008
Wednesday, July 9, 2008
ALS Association Wins Fight for Power Wheelchairs
Dear Drew,
Thanks to the outreach of ALS advocates in key states across the country, the U.S. Senate this afternoon passed legislation (HR 6331) that would exclude power wheelchairs from Medicare's new competitive bidding program. The bill, which passed by a 69-30 vote, also would delay implementation of the program for 18 months. This is a tremendous victory for people with ALS and their families and helps to ensure PALS will continue to have access to needed power wheelchairs!
For nearly two years, The ALS Association has worked with Congress to exclude high-end power chairs from the new program for fear that it would limit the types of chairs available to PALS, reduce services and force PALS to obtain chairs from suppliers who do not know the unique medical needs of people with ALS.
The Association held a breakout session on competitive bidding during our Advocacy Conference in May and worked closely with PALS and Chapters to educate Congress on the unique needs of people with ALS, making it clear that one-size-fits-all policies like competitive bidding are not appropriate when it comes to this disease. In fact, the participation of PALS at a press conference on competitive bidding last year helped to highlight the concerns of the ALS community and clearly demonstrated that the chairs needed by PALS are much different than those used by most Medicare beneficiaries.
With today's vote, we are one step closer to ensuring that competitive bidding will not limit the ability of PALS to access to the power chairs they need when they need them.
The House of Representatives already has passed HR 6331 with broad support so the bill now heads to the President for his signature. The President has signaled that he may veto the bill due to concerns about other unrelated provisions. However, both the Senate and House passed the bill with enough support to override a veto and enact HR 6331 into law.
We would like to thank all of the PALS, families and Chapters who have helped to reach out to Congress in support of this issue over the past two years. Your outreach has continued to make a difference!
If you have any questions about today's vote or would like additional information, please contact the Advocacy Department at advocacy@alsa-national.org or 1-877-444-ALSA.
Thank you!

