Saturday, January 31, 2009
Byline: Elizabeth Cooney When Dr. Robert H. Brown Jr. says it, it sounds so simple. "These diseases will yield," he said. "We just can't take no for an answer." The new head of neurology at University of Massachusetts Medical School UMMS is ranked fourth in primary care education among the nation’s 125 medical schools in the 2006 U.S.News & World Report annual guide, “America’s Best Graduate Schools”. UMMS is also a major center for research. and UMass Memorial Medical Center is single-minded in his determination to find better treatments for such devastatingdev·as·tate tr.v. dev·as·tat·ed, dev·as·tat·ing, dev·as·tates1. To lay waste; destroy.2. To overwhelm; confound; stun: was devastated by the rude remark. ..... Click the link for more information. neuromuscular diseases as amyotrophic lateral sclerosis amyotrophic lateral sclerosis (ALS) (ā'mīətrōf`ik, sklĭrō`sĭs) or motor neuron disease, , better known as Lou Gehrig's disease Lou Geh·rig's diseasen.See amyotrophic lateral sclerosis. . In October he left Massachusetts General HospitalMassachusetts General Hospital Health care The major teaching hospital for Harvard Medical School, widely regarded as one of the best health care centers in the world ..... Click the link for more information. and Harvard Medical SchoolHarvard Medical School (HMS) is one of the graduate schools of Harvard University. It is a prestigious American medical school located in the Longwood Medical Area of the Mission Hill neighborhood of Boston, Massachusetts. ..... Click the link for more information. to find a faster pathway to treatments at the state's medical school The lure was RNA interferenceRNA interferencen.A process in which the introduction of double-stranded RNA into a cell inhibits the expression of genes. ..... Click the link for more information., or RNAi for short, the mechanism that can silence genes. UMass professor and Nobel laureate Craig C. Mello was one of two scientists to discover the biological process in 1998. Since then, the medical school has added to its cadre of RNAi researchers by recruiting distinguished scientists such as Dr. Brown, who was already collaborating with UMass experts. The school's proposed Advanced Therapeutics Cluster will include a center devoted to RNAi, part of the state's $1 billion life sciences initiative. RNAi holds promise for treating diseases by shutting off defective genes that make harmful proteins. In ALSAls (äls), Ger. Alsen, island, 121 sq mi (313 sq km), Sønderjylland co., S Denmark, in the Lille Bælt, separated from the mainland by the narrow Alensund. ..... Click the link for more information., mutant genes produce proteins that kill nerve cells needed to move muscles. Most people die within three to four years after first developing muscle weakness and then paralysis. "I spent 30 years at the Mass. General Hospital, which is a wonderful institution," Dr. Brown, 61, said last month in his UMass Medical School office, already adorned by images of Lou Gehrig and photos of Red Sox players at ALS fundraisers. "But it seemed to me that I would probably make more progress here by joining forces with the people who made the basic discoveries in this (RNAi) technology. I just thought the route would be more direct here." Dr. Brown, both a research scientists and a neurologist who sees patients, began studying ALS in 1979. It was through his clinical work that he began to investigate the genetic underpinnings of ALS. One of his patients, Oscar Horvitz, was the father of H. Robert Horvitz H. Robert Horvitz (born May 8, 1947) is an American biologist best known for his research on the nematode worm Caenorhabditis elegans. He is currently at the Massachusetts Institute of Technology where he is Professor of Biology and a member of the McGovern Institute for , a scientist at MIT MIT - Massachusetts Institute of Technology who later won a Nobel Prize Nobel Prize, award given for outstanding achievement in physics, chemistry, physiology or medicine, peace, or literature. The awards were established by the will of Alfred Nobel, who left a fund to provide annual prizes in the five areas listed above. for his work on the genetic regulation of organ development and programmed cell death pro·grammed cell deathn.See apoptosis.
programmed cell deathproposed system of cell death, often including poly(ADP)-ribosylation, ensures that a cell will not survive if it is so badly damaged that its recovery would harm the . After Dr. Brown had the sad task of confirming a diagnosis of ALS in his patient, he began discussing research with his patient's son. At the time, the technology to find genes and map them was just entering laboratories, where scientists were trying to divine what they did. "Bob and I started talking about this horrific disease, and what we might do about it," Dr. Horvitz said in an e-mail interview about Dr. Brown. "At that point we embarked upon collaborative studies.... We agreed that the emerging technologies of human genetics Human geneticsA discipline concerned with genetically determined resemblances and differences among human beings. Technological advances in the visualization of human chromosomes have shown that abnormalities of chromosome number or structure are surprisingly offered new approaches, and it was this work that led to the identification of the first ALS gene, known as SOD1. Since then Bob has continued to pioneer the application of ever-more sophisticated genetic methods to ALS research." SOD1 was identified in 1993, breaking open the world of ALS research. The gene produces superoxide dismutase, an enzyme that protects cells from damage wrought by toxic free-radical molecules. Defective forms of the gene were found in families who had ALS. Most cases of ALS are sporadic, meaning they strike people who have no family history of the disorder, but finding the gene implicated im·pli·cate tr.v. im·pli·cat·ed, im·pli·cat·ing, im·pli·cates1. To involve or connect intimately or incriminatingly: evidence that implicates others in the plot.2. in the inherited forms offers a keyhole through which to learn about all forms of the disease. The scientific paper reporting the breakthrough was dedicated to Oscar Horvitz. Once the gene was identified, the next step was to study it in specially bred laboratory mice and then devise ways to fix it. "It's been a long slog. It became painfully clear that the treatments would be very difficult to find. This was a very hard nut to crack, and there were no easy fixes," Dr. Brown said. "Because of the frustration of working for 10 years with conventional therapies without success, it seemed to me we needed to look for something new and to go as far upstream as one could to shut off the disease process right at the start." That's when Dr. Brown and his colleagues began to get very interested in gene silencing through RNAi. "There are several lines of study suggesting that the technology actually works in mice," he said. "The question is how best to get it in people." One way would be to inject active RNAi molecules into the spinal fluid of patients that would then thwart the mutant gene, but there are doubts about how effective that would be, he said. Another approach is more complicated: using gene therapy to deliver into the spinal cord a gene that uses the machinery of the cell to continuously make gene-silencing RNA RNA: see nucleic acid.
RNA in full ribonucleic acidOne of the two main types of nucleic acid (the other being DNA), which functions in cellular protein synthesis in all living cells and replaces DNA as the carrier of genetic . Both methods pose challenges. Of the two, Dr. Brown thinks the gene therapy-RNAi combination holds the most promise for achieving a sustained effect. It's no coincidence that the planned UMass therapeutics cluster includes a gene therapy center as well as an RNAi center. (The third is devoted to stem cell science.) "The new dean is a gene therapist, so suddenly it's like a perfect storm," Dr. Brown said. "This isn't just a laboratory exercise. He's extremely serious about seeing this happen and implementing programs. That feeds in perfectly to the idea of a more effective way to deliver RNAi therapy." Dr. Brown's sense of urgency comes from the patients he hopes to help. They don't have a lot of time. "Bob is somebody who is a very caring and compassionate physician," said Dr. Terence R. Flotte, dean of the medical school since April 2007. "His motivation is heavily centered around coming up with solutions for the patients who are so tragically affected. People say stuff like that a lot, but you can see in his eyes - that's what really matters to him." Dr. Brown's optimism about finding a cure was clear last month, two days after the presidential election. "What did someone say the night before last?" he asked. "`Yes, we can.'" CUTLINE
Friday, January 30, 2009
Fri Jan 30, 11:30 am ET
Scientific inspiration can come from anywhere - a person, an event, even an experiment gone awry. But perhaps nothing can drive innovation more powerfully than the passion born of tragedy. Or, in Douglas Melton's case, near tragedy. The co-director of the Harvard Stem Cell Institute (HSCI) is one of the leading figures in the search for cures for presently incurable diseases, and his breakthrough work is challenging many long-held beliefs about the ways biology and human development work.
But it was a very personal experience that brought Melton to stem cells, one that 17 years later he still finds difficult to discuss. When his son Sam was 6 months old, he became ill with what his parents thought was a cold. He woke up with projectile vomiting and before long began taking short, shallow breaths. After several hours, he started to turn gray, and Melton and his wife Gail brought the baby to the emergency room. For the rest of that afternoon, doctors performed test after test, trying to figure out what was wrong. "It was a horrific day," says Melton. (See the top 10 medical breakthroughs of 2008.)
It was not until that evening that a nurse thought to dip a testing strip into Sam's urine and they finally got a diagnosis. The boy's body was flooded with sugar; he had Type 1 diabetes. Then, as now, the disease had no cure, and patients like Sam need to perform for themselves the duties their pancreas cannot - keeping track of how much glucose they consume and relying on an insulin pump to break down the sugars when their levels climb too high. The diagnosis changed not only Sam's life but the lives of his parents and older sister Emma as well. Throughout Sam's childhood, Gail would wake every few hours during the night to check his blood sugar and feed him sugar if his concentration fell too low or give him insulin if it was too high. "I thought, This is no way to live," says Melton. "I decided I was not just going to sit around. I decided I was going to do something."
Trained as a molecular biologist in amphibian development, Melton began the work he pursues today: trying to find a way to make insulin-producing cells by using stem cells. "It was a courageous thing to do because he was at the pinnacle of his career," says Gail. "He brought home textbooks on the pancreas to figure it all out." Nearly two decades later, Melton is convinced that stem cells will be a critical part of new therapies that will treat and maybe cure not only diabetes but also other diseases for which there are no answers today.
Melton's confidence is testament to the extraordinary advances in stem-cell science, some of which have brought the promise of breakthrough therapies for conditions like diabetes, Parkinson's and heart disease closer than ever before. The cells filling petri dishes in freezers and incubators in Melton's lab and others around the world are so vastly different - in provenance, programming and potential - from the stem cells of just two years ago that even the scientists leading this biological revolution marvel at the pace at which they are learning, and in some cases relearning, rules of development. Until recently, the field has revolved around either embryonic stem cells - a remarkably plastic class of cells extracted from an embryo that could turn into any of the body's 200 tissue types - or their more restricted adult cousins, cells taken from mature organs or skin that were limited to becoming only specific types of tissue. On Jan. 23, after nearly a decade of preparation, the Food and Drug Administration approved the first trial of an embryonic- stem-cell therapy for a handful of patients paralyzed by spinal-cord injuries.
But today the field encompasses far more than just embryonic and adult stem cells; it has expanded into the broader field of regenerative medicine, and Melton's lab at Harvard is at the vanguard, bringing the newest type of stem cells, which do not rely on embryos at all, closer to the clinic, where patients will actually benefit. Last summer, Melton stunned the scientific community with yet another twist, finding a way to generate new populations of cells by reprogramming one type of fully mature cell so it simply became another, bypassing stem cells altogether. "If I were in high school, I can't imagine anything more interesting than stem cells," says Melton. "This is so cool. It's so amazing that cells in the body have this potential that we can now unlock by asking question after question."
A Battle Joined That hidden power in each of us did not become obvious until 1963, when Canadian researchers Ernest McCulloch and James Till first proved the existence of stem cells, in the blood. These cells possess the ability to divide and create progeny - some of which will eventually expire, others that are self-renewing. The pair irradiated mice, destroying their immune cells. They then injected versatile bone-marrow cells into the animals' spleens and were surprised to see a ball of cells grow from each injection site. Each mass turned out to have emerged from a single stem cell, which in turn generated new blood cells.
That discovery led, 35 years later, to James Thomson's isolation of the first human embryonic stem cells, at the University of Wisconsin in 1998. And that milestone in turn inspired researchers to think about directing these cellular blank slates to eventually replace cells that had been damaged or were depleted by disease. The key lay in finding just the right recipe of growth factors and nutrients to induce a stem cell to become a heart cell, a neuron, an insulin-making cell or something else. It would take decades, the researchers all knew, but new therapies were sure to come.
Then, in 2001, everything changed. The use of discarded embryos made embryonic-stem-cell research deeply controversial in the U.S. Citing moral concerns, then President Bush restricted federal funding for the study of human embryonic stem cells. Under the new policy, U.S. government funds could be used only to study the dozens of embryonic cell lines already in existence - many of which proved not to be viable.
Read Stem Cells: The Hope and The Hype.
See the Year in Health, from A to Z.
The decision sent some leading scientists abroad, to Britain, Singapore and China, where the governments were more receptive to their work. Others who stayed behind but lacked private funding shifted their attention from embryos to the less versatile adult stem cells. Federally backed scientists, like Melton, who continued embryonic work were forced to adopt a byzantine system of labeling and cataloging their cell cultures and equipment so that government money was not used to grow forbidden cells - and government microscopes were not even used to look at them.
Those days may soon be over. Barack Obama campaigned on a promise to lift the research ban and support "responsible oversight" of the stem-cell field. For scientists, that means "we can stop the silliness," says Melton.
As welcome as that change will be, it may be less urgent now - owing primarily to the work of scientists like Melton. While embryonic stem cells remain the gold standard for any treatments that find their way into the clinic, newer techniques using the next-generation stem cells may soon surpass the older ones.
The Fighter In looks and demeanor, Melton is the quintessential professor, soft-spoken and thoughtful, someone who appears more mentor than maverick. Born and raised on the South Side of Chicago, he developed an early fascination with animal development; that curiosity led to a bachelor's degree in biology at the University of Illinois in 1975, then a second undergraduate degree, in the history and philosophy of science, at Cambridge University on a Marshall Scholarship. Melton remained there for his Ph.D. work, studying under Sir John Gurdon - the first to clone a frog. At Harvard, Melton teaches a frequently oversubscribed undergraduate course on science and ethics, in which he uses his keen sense of logic to provoke. When the class discussed the morality of embryonic-stem-cell research, Melton invited Richard Doerflinger of the U.S. Conference of Catholic Bishops to present arguments against the field. Melton asked Doerflinger if he considered a day-old embryo and a 6-year-old to be moral equivalents; when Doerflinger responded yes, Melton countered by asking why society accepts the freezing of embryos but not the freezing of 6-year-olds.
Clearly, Melton does not shrink from a fight. As Washington's squeeze on stem-cell research tightened in the early part of this decade, he decided to take action, providing life support for what remained of the U.S. stem-cell community. Not convinced that an entire field could make much progress relying on a few dozen cell lines of questionable quality, in 2004 he used funds HSCI receives from the Juvenile Diabetes Research Foundation and the Howard Hughes Medical Institute, as well as from Harvard alumni, and developed a more streamlined method for generating stem-cell lines from embryos. He created more than 70 new ones and has since distributed 3,000 copies to scientists around the country for free.
"Doug drew a line in the sand," says Alan Trounson, president of the California Institute of Regenerative Medicine, the organization charged with dispensing state money for embryonic-stem-cell research. "He turned the tables on an Administration that was incredibly negative toward stem cells and showed [it] we are not going to tolerate being put out of this field by ideological views that we don't think are correct." Melton's motivation was, again, both professional and intensely personal. Two months after Bush announced his ban, Melton's daughter Emma, then 14, also received a diagnosis of Type 1 diabetes.
In part owing to the restrictive U.S. policy, the momentum in stem-cell research seemed to shift overseas. In 2004, South Korean researcher Hwang Woo Suk announced that he had generated the first human embryonic stem cells from healthy people - and in the following year, from afflicted patients themselves - using an abbreviated cloning method. The latter feat would mean that cardiac patients could essentially donate themselves a healthy new heart without fear of rejection.
The news was huge - but it was also a lie. In 2006, Hwang admitted he had falsified his results. (Melton's colleague at HSCI, Kevin Eggan, finally created embryonic stem cells from patients in 2008.) Although Hwang became a pariah, he had the right idea. Melton and others had been trying to do just what the Korean scientist claimed to have done - grow a new population of a patient's own cells. The key to the process is a supply of fresh, good-quality human eggs, which incubate skin cells taken from a patient. Building up such a stockpile, however, proved practically impossible. The egg-extraction process is invasive and carries certain risks; after the state of Massachusetts prevented donors from being compensated for their eggs, out of fear the women would feel coerced, HSCI ended up with only one volunteer out of more than two years of recruiting.
Melton faced mounting political pressure too. In 2004, voters in California approved a measure providing $3 billion in state funding to embryonic-stem-cell research. That threatened to draw scientists in the stem-cell community west, and Melton took pains to foster a "band of brothers" mentality. "I tried to create a cocoon here," he says, "and tell people that your job is to focus on the science. Don't worry what the politicians say." By then, Melton's team was one of only a handful in the country working on embryonic stem cells and was making headway in teasing apart the myriad critical steps needed to guide these impressionable cells into becoming insulin-generating cells. Both as a scientist and as a father, Melton remained convinced that the federal restrictions simply could not survive. He continued to insist that "the science is so significant that it will change the policy."
And then, astonishingly, it did. In June 2006 a modest researcher from Japan made a startling announcement at the International Society for Stem Cell Research conference in Toronto. Shinya Yamanaka quietly described a study in which he took skin cells from a mouse and stirred them in with varying genetic cocktails made from a recipe list of 30 genes known to be important in development. When he hit on the right four genes and inserted them into the cells aboard retroviruses, he wiped the cells clean, reprogramming them and returning them to an embryo-like state without ever creating the embryo. Four genes, he told his audience, was all it took to undo a lifetime's worth of delicate genetic tapestry. No need for eggs, no need for embryos. Could it be that easy? Were the debate and controversy over embryonic stem cells now rendered moot? "It was unquestionably unexpected," says Melton of the breakthrough.
Read a TIME cover story on Stem Cells.
See the Year in Health, from A to Z.
A year later, Yamanaka followed up his work by reporting success with the same four factors in turning back the clock on human skin cells. At about the same time, in Wisconsin, Thomson achieved the same feat using a different cocktail of genes. With those studies, what became known as induced pluripotent stem cells (iPS cells) were suddenly a reality. Never mind the frustratingly fickle process needed to create embryonic stem cells; this was something any molecular-biology graduate student could do. "We figured somebody would have success with reprogramming. We just thought that somebody would come along a generation from now," says Dr. David Scadden, Melton's co-director at HSCI. "Yamanaka threw a grenade at all of that, and now all of the doors are open."
Beyond Stem Cells Melton, for one, isn't wasting any time before running through those doors. The iPS technology is the ultimate manufacturing process for cells; it is now possible for researchers to churn out unlimited quantities of a patient's stem cells, which can then be turned into any of the cells that the body might need to repair or replace.
Before that can happen, however, Melton wants to learn more about how diseases develop. And iPS cells make that possible too. For the very first time, he can watch Type 1 diabetes unfold in a petri dish as a patient's cells develop from their embryonic state into mature pancreatic cells. The same will be true for other diseases as well. "There is a good reason we don't have treatments for diseases like Parkinson's," says Melton. "That's because the only way science can study them is to wait until a patient appears in the office with symptoms. The cause could be long gone by then, and you're just seeing the end stages." No longer. Now the major steps in the disease process will be exposed, with each one a potential target for new drugs to treat what goes wrong. "This is a sea change in our thinking about developmental biology," says Dr. Arnold Kriegstein, director of the Institute for Regeneration Medicine at the University of California, San Francisco. "I consider it a real transformative moment in medicine."
The true power of reprogramming, however, does not stop with the stem cell. This summer, Melton flirted with the rules of biology once again when he generated another batch of history-making cells, switching one type of adult pancreatic cell, which does not produce insulin, to a type that does - without using stem cells at all. Why, he thought, do we need to erase a mature cell's entire genetic memory? If it's possible to reprogram cells back to the embryo, wouldn't it be more efficient in some cases to go back only part of the way and simply give them an extreme makeover? Using mouse cells, Melton did just that, creating the insulin-producing pancreatic cells known as islets. "The idea now is that you can view all cells, not just stem cells, as a potential therapeutic opportunity," says Scadden. "Every cell can be your source."
Realizing that potential - and with it, the prospect of successful treatments for conditions like Parkinson's or diabetes - may still be a few years away. Even iPS cells have yet to prove that they are a safe and suitable substitute for the diseased cells they might eventually replace in a patient. Ensuring their safety would require doing away with dangerous genes that can also cause cancer, as well as the retroviral carriers that Yamanaka originally used. Melton's team has already replaced two of the genes with chemicals, and he anticipates that the remaining ones will be swapped out in a few years. There are also hints that the iPS cells' short-circuited development makes them different in some ways from their embryonic counterparts. In mice, embryonic stem cells can generate a new mouse clone; iPS cells from the animals have so far stopped short of the same feat, aborting in midgestation, suggesting that some development cues may be missing. "It certainly makes me cautious," says Eggan.
Even if iPS cells do not prove as stable and as versatile as embryonic stem cells when they're transplanted into patients, they remain a powerful research tool. And if nothing else, they will have opened our eyes to the remarkable plasticity of biology and made possible new ways of thinking about repairing and replacing damaged tissues so we may consider not only treating but also curing disease. "It's a wonderful time," says Scadden. "Keep your seat belt on, because this ride is going to be wild."
For patients like Sam and Emma Melton, that ride carries with it the possibility of being free of the insulin pumps and injections they endure to keep their blood sugar under control. "I definitely think about how my life would be different if there is a cure," says Sam. His father is keenly aware that the ability of stem cells and reprogramming science to provide that cure is far from guaranteed. But his initial confidence in the power of the technology hasn't waned. "Everything we learned about stem cells tells us this was a really powerful approach," he says. "It would be a great shame if we let it wither and just go away." Melton, for one, is determined not to let that happen.
Science in Steps
A decade of conflicts and breakthroughs
1998 James Thomson, U of Wisconsin, isolates human embryonic stem cells
2001 President Bush restricts federal funding for research on human embryonic stem cells
2004 Douglas Melton of Harvard creates more than 70 embryonic-stem-cell lines using private funding and distributes free copies of the cells to researchers around the world
2006 Shinya Yamanaka, Kyoto University, turns back the clock on mouse skin cells to create the first induced pluripotent stem (iPS) cells, or stem cells made without the use of embryos. He uses only four genes, which are inserted into a skin cell's genome using retrovirus vectors
2007 Yamanaka and Thomson separately create the first human iPS cells
2008 July Kevin Eggan at Harvard generates the first patient-specific cells from iPS cells - motor neurons from two elderly women with ALS
August Melton bypasses stem cells altogether and transforms a type of mouse pancreatic cell that does not produce insulin into one that does
September Konrad Hochedlinger at Harvard creates iPS cells in mice using the common-cold virus rather than retrovirus vectors - an important step in making the technology safer for human use
October Melton's team makes human iPS cells by replacing two of the four genes, known to cause cancer, with chemicals. All four must be swapped out before iPS-generated cells can be transplanted into people
October Yamanaka creates mouse iPS cells using safer plasmids of DNA instead of retrovirus vectors
Read Stem Cells: The Hope and The Hype.
See the Year in Health, from A to Z.
View this article on Time.com
Related articles on Time.com:
A Leap Forward for Stem Cells
Cautious Optimism for the First Stem Cell Human Trial Approved by the FDA
Why Harvard Is Recruiting Egg Donors for Stem Cell Studies
A Breakthrough on Stem Cells
One Small, Small Step Toward Stem-Cell Nirvana
'Life is too short to wake up in the morning with regrets, so love
the people who treat you right,
forget about the ones who don't
and believe that everything
happens for a reason. If you get a
chance, take it.
If it changesyour life, let it.
Nobody said life would be easy, they just promised it would be worth it
Thursday, January 29, 2009
If you are trusting the FDA to protect you then you are following blindly. The FDA recalls several medicions every year secondary to adverse effects such as death that it had at some time in the past approved. The FDA approves unsafe drugs, products and such every year and people are injuried and die every year because of thier mistakes.Last year the FDA finally addmitted on record that they have no idea how to handle products involving stem cells. They finally came clean that thier guidelines are outdated and not appropriate when involving stem cells and that they need to set up all new polices to govern stem cells. They also admitted that they did not even have anyone within the FDA to guide them. They then annouced that they would hire new experts and set up a brand new panel to create stem cell policies and guidelines. Since this anouncement last year they have not publically made a statement as to what if any progress they have made.The FDA is far from perfect and only a fool would think that they are not subject to the making of mistakes. Do not trust blindly and do not be that fool. Be smart, be educated and look not only at what your goverment tells you as we all know our goverment is far from perfect, honest and angelic.BTW everything I have said above is fact not theroy and all of it is a matter of public record. Goggle and yahoo searches can reveal all of these things.
That info was sent to me by Don Margolis. I have known him for some time and he often sends me info. Don can be a little soap boxy but he is also very good at uncovering some interesting and often overlooked information. The "theroy" may or may not be correct but history does show us some very interesting facts as does sceince. Embryonic stem cells have been proven to be unsafe. That is just a fact. Another fact is that studies involving humans and embryonic stem cells have been approved before and then pulled. This has happened more than once. To my knowledge only one has gone through for Batten's.The world of stem cells is very nasty and cut throat so do not be fooled by what you think you know as there is a very unpleasent underworld to it that you do not see or read about everyday. Stem cells are big big money and people will do some very strange and often inapprpriate things when bilions of dollars are involved.
Wednesday, January 28, 2009
Mon Jan 26, 7:05 pm ET
It was nearly a decade in the making, but the first human trial using embryonic stem cells was approved on Friday.
The trial, which will test a stem-cell based treatment for spinal cord injury, will begin later this summer and will use cells generated by Geron Corporation. The approval marks the first time human stem cells, extracted and grown from embryos, will be transplanted into patients. Adult stem cells, which are present in many types of tissue, have been used in treatments for years - the most common being bone marrow transplants in cancer care - but an embryonic study is a whole new thing. There's good reason it's being greeted with so much excitement. (See the top 10 medical breakthroughs of 2008.)
Scientists believe that embryonic stem cells are more versatile than adult cells in generating the more than 200 different tissue types in the body. The need for healthy new cells is particularly acute in the case of spinal cord injury, because once central nervous system tissue is destroyed, it does not regenerate - not in any significant way at least. The Geron team began its work with what is known as a Presidential stem cell line - stem cells derived from discarded in vitro fertilization embryos that already existed in 2001 when former President Bush decided to prohibit the use of federal funds to pursue human embryonic stem cell work. At the time, fewer than two dozen of these stem cell lines were of good enough quality to use as a basis for human treatments.
The therapy revolves around coaxing the stem cells to develop into a type of cell known as an oligodendrocyte, which in turn gives rise to critical insulation and growth factors that can repair neurons damaged by a trauma to the spinal cord. Geron has named its version GRNPOPC1. By having more of these cells around the spinal cord, more nerves might be repaired and can then potentially re-establish proper connections. Simply by working with the GRNOPC1 cells, Geron scientists have learned more about how they operate, which will only expand their understanding of how the central nervous system might be healed. Says Geron's president and CEO Dr. Thomas Okarma: "They make dozens of factors that can stimulate nerve function, growth and regeneration." (Read "Scientists Reach Stem Cell Milestone.")
The trial will enroll patients with injury to the thoracic region, high in the spinal cord between the third and tenth vertebrae. Doctors will be trained to inject the cell treatment at specific locations, where the cells will remain to do their nerve-nurturing work. "I think it's incredibly exciting," says Dr. Susan Fisher, a stem cell scientist and a professor of obstetrics, gynecology and reproductive science at University of California San Francisco. "This really provides a blueprint for how to do these sorts of trials. It really proves the principle that these sorts of human embryonic stem cell therapies can survive the FDA approval process."
And that process wasn't such an easy hurdle to overcome. Because no one has used embryonic stem cells, or the cells derived from these stem cells, in people before, the FDA was particularly cautious. The trial that it did approve is what's known as a Phase 1. It will involve no more than a dozen patients and is not designed to test the effectiveness of the cells. Rather, it will simply monitor the safety of inserting them into people. The researchers will be looking for whether the cells cause tumors, trigger an immune response, or start to migrate away from the spinal cord area. "There are certainly unknowns that we can't predict," says Dr. David Scadden, co-director of the Harvard Stem Cell Institute. "We don't know whether or not these cells might grow abnormally in a person. We don't know if things might occur just by these cells being present that could result in an outcome we don't want. This really is a first go, with a lot on the line."
Dr. Ronald Crystal, chairman of the department of genetic medicine at New York-Presbyterian Hospital/Weill Cornell Medical College, knows those stakes all too well. He is a veteran of the last revolution in medical technology, gene therapy, which, after some hyped expectations in the 1990s, fell into disfavor after some unsuccessful trials. Crystal is cautiously optimistic about the potential for this trial to open the door to future stem cell therapies. "I think this is a very positive start, but the expectations and hype I see around stem cell therapies are the same that I saw around gene therapy. We just have to remember that these take a long time to develop. But you will never know if they work unless you study them in humans, so this is an important first step."
Stem Cells: The Hope and The Hype
See the Year in Health, from A to Z.
View this article on Time.com
Related articles on Time.com:
A Breakthrough on Stem Cells
Banking on Stem Cells
Finding a Master Heart Cell
Eeek! There's a Mouse Cell in My Stem Cells!
Scientists Reach Stem Cell Milestone
Tuesday, January 27, 2009
So glad you won!! Nasty cut...must have given you one whopper of a headache!I have been following your blog since June...thank you for providing so much up to date information.I have a family member who is very ill ...yet to be definitely diagnosed (symptoms all of ALS, of course, we continue to pray for a miracle that has any other name)Do you have an email address that I could contact you at to ask some questions?
Monday, January 26, 2009
Neuralstem wins key patent for stem cell work
Washington Business Journal - by Vandana Sinha Staff Reporter
Joanne S. Lawton
Richard Garr, CEO of Neuralstem.View Larger
Neuralstem Inc. said Monday it has nailed down a key patent for its core technology, which grows neural stem cells from the brain to replace damaged spinal cord cells.
The patent comes in time for Neuralstem’s first clinical trials, planned for this year if the Food and Drug Administration gives them the green light. The Rockville company intends to test the ability and safety of injecting fetal stem cells into the spinal cords of patients suffering from amyotrophic lateral sclerosis, more commonly known as Lou Gehrig’s Disease.
Under the new patent, Neuralstem (AMEX: CUR) can grow almost unlimited numbers of stem cells from all parts of the brain, all in a controlled and consistent manner. The company has been honing its technology for the past 13 years, relying on fetal stem cells rather than their more controversial counterpart, embryonic stem cells.
Neuralstem filed its request in December to launch its first human clinical trials this spring. It expects to hear the FDA’s decision by the middle of next month, but another competitor’s recent news has raised its hopes. On Friday, the FDA approved a request by Geron Corp. to test embryonic stem cells in human trials for the first time, illustrating to Neuralstem leaders a growing comfort level at the agency with using previously untested stem cells to treat non-fatal diseases.
“It removes a tremendous amount of uncertainty,” said Neuralstem CEO Richard Garr. “It makes it clearer what’s required to make it forward [to trials]. From what we’ve seen, we’ve done what’s required.”
While he said President Barack Obama’s entry in the White House is more coincidental than influential in the FDA’s decision for Geron, he does foresee it opening more doors for stem cell discovery and science.
“There’s no question that the tone will be different,” Garr said. “The tone from the top will always have an impact.”
Garr points out the difference between the two companies’ techniques, however, saying a green light for Geron doesn’t necessarily guarantee one for Neuralstem. Whereas Geron is working to essentially restrip the neural “wires” that connect spinal cells to help heal traumatic spinal cord injury, Neuralstem is opting to fully replace destroyed spinal cord cells to treat ALS.
Before the end of the year, Neuralstem also hopes to start clinical trials of its technology on improving traumatic spinal cord injury, the same disease area as Geron, potentially placing Neuralstem in direct competition with the Menlo Park, Calif., company.
The local company raised more than $1.7 million in a stock offering at the end of last year --enough, Garr said, to pay for the ALS trials on an anticipated 15 patients.
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Sunday, January 25, 2009
News came out this past Friday that the Food And Drug Administration (FDA) had okayed its first (ever) embryonic stem cell test. This test took several months to get through the review process because of the dogmatic roadblocks, the Bush administration put in place.
In an interview with CNN on January 18, Obama said: "I like the idea of the American people's representatives expressing their views on an issue like this." As President,,, it is Pres. Obama has the power to issue an Executive Order opening federal funding for stem cell research. ." Let's hold him to his campaign pledge!!
Only after this fruitless & obstructive red tape is eliminated and federal funding for stem cell research is allowed, can life saving research once again resume. . Although President Obama pledged to reverse the Bush administration's ban on federal funding of stem cell research during the campaign trail, he seems to be backing off on that pledge now.
If we leave this issue alone for any length of time, it will definitely be relegated to the back burner, and research for diseases like ALS, M.S., Parkinson's, and Alzheimer's, spinal cord injuries, and organ and tissue damage will definitely be set back.
President Obama must issue an Executive Order to open federal funding for stem cell research. ." Let's hold him to his campaign pledge!!
Right now, there is no bigger supporter than myself of Pres. Barack Obama, but if he starts passing the buck on such important issues as federal funding for stem cell research, there will be no greater critic. People's Lives are at stake here, let's do the right thing.
All family & friends of loved ones suffering from diseases that stem cell research funding could help, should take the time and get involved ?. We need your support
Eddie spaghetti) Esparza.
They came first for the Communists, And I didn't speak up because I wasn't a Communist;
And then they came for the trade unionists, And I didn't speak up because I wasn't a trade unionist;
And then they came for the Jews, And I didn't speak up because I wasn't a Jew;
And then . . . they came for me . . . And by that time there was no one left to speak up."
Richard John Neuhaus
Friday, January 23, 2009
By Steven ReinbergHealthDay Reporter by Steven Reinberghealthday Reporter 1 hr 56 mins ago
FRIDAY, Jan. 23 (HealthDay News) -- The first human trial using embryonic stem cells as a medical treatment has been approved by the U.S. Food and Drug Administration.
Geron Corp., a California-based biotech company, has been given the OK to implant embryonic stem cells in eight to 10 paraplegic patients who can use their arms but can't walk. Stem cell injections will be given within two weeks of the injury. The study will begin this summer, and will be conducted at up to seven different medical centers.
"This marks the dawn of a new era in medical therapeutics," Dr. Thomas B. Okarma, Geron's president and CEO said during a Friday morning teleconference. "This approach is one that reaches beyond pills and scalpels to achieve a new level of healing."
Ultimately, this type of therapy might have the power to restore permanent organ and tissue function, Okarma said. The goal of this first trial is to see if injecting embryonic stem cells into humans is safe. However, the researchers will also be looking for signs of improvement in the patients' ability to feel sensation in or move their legs.
Patients will receive injections at the site of the injury. It is hoped these cells will mature into cells that will repair damaged nerves and produce chemicals that nerve cells need to function and grow.
This phase I trial will be limited to patients whose injury is located in the middle of the spine. If the trial is successful, Okarma said, the hope is to extend the treatment to patients with cervical spine injuries who are paralyzed from the neck down.
Okarma said the injections must be given early after the injury, before scar tissue has developed that would prevent the cells from growing, but after the initial swelling has subsided.
In addition, patients will receive anti-rejection drugs for about two months, after which they should no longer need those drugs. Patients will be followed for at least one year, he said.
The treatment is not expected to restore full function to patients, but the researchers hope to see modest gains. "Any return of bladder or bowel function, a return of sensation, or a return of lower extremity locomotion would be a very exciting finding," Okarma said.
In experiments with rats, researchers found these cells were safe and did restore some function. "These cells insulate as well as stimulate nerve fibers, leading to restoration of function in animal models of spinal cord injury," Okarma said.
The cost of this therapy isn't known yet, but Okarma said it would be "affordable."
Embryonic stem cells are the most basic human cells. These cells are believed to be capable of growing into any type of cell.
The controversy surrounding the use of these cells has become a political issue, with some objecting that the use of these cells destroys potential life because they must be extracted from human embryos. This belief resulted in the Bush administration banning federal funding for embryonic stem cell research.
While the Obama administration has indicated that it will lift the ban, the stem cells used in this trial were obtained from one of the Bush administration's approved stem cell lines. However, no federal funds were used in the development of this treatment.
A decade has passed since the first embryonic stem cells were isolated at the University of Wisconsin, in groundbreaking research that was funded by Geron Corp.
Geron is also working on using embryonic stem cells to treat failing hearts and to create insulin-producing islets for type 1 diabetics, Okarma said.
"Embryonic stem cells are really nature's own way of making more of ourselves," Okarma said. "We are simply harnessing the biology of normal human development in our attempts to achieve permanent cures to chronic disease and injury."
Peter Kiernan, chairman of the Christopher And Dana Reeves Foundation, said he's excited about this latest development in stem cell research.
"This is not just a comet across our sky, this is really more like dawn," Kiernan said. "We are beginning a vast human experiment, and we have been waiting an extremely long time to get to this point. This is a very significant development."
At the same time, Kiernan said he does not overestimate what can be expected from this trial.
"Of the millions of people dealing with paralysis in our nation, they are all delighted with subtle increases in function," Kiernan said. "We eat, drink, sleep getting people out of wheelchairs, but the reality of the world we are in is if people get bowel function, some sexual function, some ability for movement, that is a wonderful outcome."
For more on embryonic stem cells, visit the U.S. National Institutes of Health.
By Maggie Fox, Health and Science Editor Maggie Fox, Health And Science Editor Fri Jan 23, 11:51 am ET
WASHINGTON (Reuters) – The U.S. Food and Drug Administration has cleared the way for the first trial to see if human embryonic stem cells can treat people safely, a company involved in the controversial research on Friday.
Geron Corp, a California biotechnology company, said it plans a clinical trial to try to use the stem cells to regrow nerve tissue in patients with crushed, but not severed, spinal cords.
The issue of human embryonic stem cell research has been a political touchstone, with anti-abortion forces backed by former president George W. Bush arguing the technique involves the destruction of human embryos. Advocates say it could transform medicine.
"For us, it marks the dawn of a new era in medical therapeutics. This approach is one that reaches beyond pills and scalpels to achieve a new level of healing," Geron Chief Executive Dr. Thomas Okarma said in a telephone briefing.
Shares of Geron rose more than 53 percent to $8 in mid-morning trading on Nasdaq after touching $8.38.
Geron will recruit eight to 10 recently injured patients and inject them with small numbers of human embryonic stem cells manipulated to become the oligodendrocyte cells that insulate nerves, and that produce compounds to stimulate the growth of nerve cells.
Okarma said there was no political significance to the announcement coming the same week as President Barack Obama took office. Obama has been widely expected to lift restrictions on federal funding and support of human embryonic stem cell research.
An FDA spokeswoman said, "Before FDA allowed the study to proceed, Geron worked with FDA to address important scientific questions."
CHEAP AND EASY
Okarma said the treatment should eventually become cheap and easy to mass produce because the cells can be grown in vats. He believes the cells may be useful for other diseases such as multiple sclerosis, in which nerve cells are stripped of their insulating sheaths, and perhaps strokes.
Financial analysts celebrated. Stephen Brozak and Daniel Mallin of WBB Securities LLC said it could "as important to drug therapy as the discovery of ... penicillin."
Dr. Robert Lanza of the rival Massachusetts-based Advanced Cell Technology, which is also seeking to create therapies using human embryonic stem cells, called the decision a "huge advance for the entire field". This sends a message that we're ready at last to start helping people," he said.
Stem cells are the body's master cells, giving rise to all the tissues, organs and blood. Embryonic stem cells are considered the most powerful kinds of stem cells, as they have the potential to give rise to any type of tissue.
Researchers are also trying to find ways to use so-called adult stem cells, taken from bone marrow and elsewhere in the body, and have learned how to transform ordinary skin cells into stem-like cells. But scientists argue that no one knows which route will work so all avenues must be pursued.
The Phase I trial will be designed to show that patients do not develop tumors, or damage to their nervous systems. But Okarma believes it will also indicate whether the stem cells might repair the damaged spinal cords.
"These are living cells that will divide, make more of themselves and migrate throughout the lesion after injection," he said.
While the patients will get low doses of immune-suppressing drugs for the first two months, Okarma is confident the cells will escape immune system recognition and patients will not have to endure the treatments that organ and tissue transplant recipients usually do. Treatment on the first patient should begin this summer.
Okarma said the company has a strong balance sheet, free of debt, and "ample to fund the company through this trial".
(Additional reporting by Susan Heavey, Doina Chiacu and Toni Clarke; Editing by Alan Elsner and Julie Steenhuysen)
Tuesday, January 20, 2009
Mon Jan 19, 11:29 am ET
LONDON (AFP) – Two separate trials are set to begin in Britain utilising cutting edge stem cell research in a bid to help treat victims of strokes and blindness, medical experts announced Monday.
Doctors are hoping to launch the world's first trial for a treatment that aims to improve the quality of life for thousands of stroke victims on patients in Glasgow in June, although the procedure must still be approved by an ethics committee.
The treatment, which uses cells taken from an aborted foetus that are to be injected into the brains of stroke victims to see if they can effectively regenerate damaged areas, was developed by Britain-based company ReNeuron.
"That single cell was expanded by means of technology so we can have something to treat many, many thousands of patients," said ReNeuron founder John Sindon, who is working with consultant doctor Keith Muir on the planned trial at Southern General Hospital in Glasgow.
"You could make the argument that it would have otherwise gone to waste. The reality is that we're trying to turn that into something with a lasting effect."
Separately, a two-year trial involving 20 patients with corneal blindness will begin this month at the Princess Alexandra Eye Pavilion in Edinburgh and the Gartnavel General Hospital in Glasgow.
The treatment being used involves using the stem cells of dead adult donors, rather than the more controversial research involving embryonic stem cells, and if successful could help millions of people around the world who suffer from corneal blindness, around 80 percent of whom are elderly.
As part of the process, adult stem cells are cultivated and then transplanted onto the cornea's surface.
"This study is the first of its kind anywhere in the world and it is exciting to be involved in such groundbreaking work," said Professor Bal Dhillon, who is heading the trial.
"I probably see two or three new cases of corneal disease every month. On a larger scale, it's a significant problem."
A similar study by the University of Pennsylvania in the United States last year found that people with inherited blindness saw dramatic improvements in their vision when a corrective gene was injected into their eyes.
Scientists believe stem cells, which are capable of developing into almost every tissue of the body, could prove key in finding a cure for a number of serious diseases, including also diabetes and cancer.
Sunday, January 18, 2009
Saturday, January 17, 2009
Fri Jan 16, 6:11 pm ET
WASHINGTON (AFP) – President-elect Barack Obama said Friday he wanted legislation in Congress to permit federal funding on stem cell research and overturn a ban imposed by President George W. Bush.
Obama, who favors medical research on stem cells derived from human embryos, told CNN he was still exploring an executive order to revoke Bush's ban.
"But I like the idea of the American people's representatives expressing their views on an issue like this," he said, lauding a "bipartisan" consensus in Congress that such research is ethical and potentially life-saving.
If the research could yield hope for victims of degenerative diseases such as Parkinson's and Alzheimer's, "I think that sends a powerful message," he said.
In blocking federal funding for stem cell research, Bush sided with religious conservatives who argue that research on embryos destroys human life, albeit at its earliest stage of development.
Stem cells are primitive cells from early-stage embryos capable of developing into almost every tissue of the body.
Scientists believe they could prove key in finding a cure for a number of serious diseases, including also diabetes and cancer.
Please review the attachment/article, well all I can say about yet another bogus, don't get out of line, hopeless message ... -- WELCOME TO THE 21st CENTURY !! -- The Old Way of Doing Things Are Almost over for you guys. As Stephen Byer articulately pointed out the Internet is king, no longer will people be blindly corralled like cattle to their doom, at least not progressive minded fighters .
Articles like this are just a last, desperate attempt by the ALS establishment to keep control . For too long they've been making us believe that, the end all gospel of medical evidence and facts, are in the hands of the holy Grail = "experts" and no one, especially mere mortals who surf the Internet, should ever dare try their hand at questioning /challenging the established order.
Let's face it we (PALS) have been at the mercy of waiting for unending research, big pharma interest, orphan disease politics, etc. They also think that calling something a "myth" will belittle the efforts of those who educate themselves & fight for their own health freedoms. Sorry, that may have been last century, but not this one.
I love this statement from the article/attachment (Red):
We need to do a better job of distributing information to PALS on the many benefits of attending specialized ALS clinics, on the many benefits of participating in the research studies offered there, and on the dangers of self-experimentation. We should refrain from off-label prescribing of unproven therapies. Most importantly, we need to educate PALS on how to distinguish myth from realityAllow Me to Sort This Ridiculous Paragraph into Sections
1. Do a better job of distributing information to PALS??? -- What information are they referring to? Oh, let me think of a classic "Mr. Johnson, your breathing capacity has dropped to dangerous levels, you may want to consider a trache" but then again only 10% of all patients at this neuromuscular center have ever taken that option. WOW! Could you repeat the second option again? Or what about this priceless tidbit (information distribution ) -- Mr. Johnson there are no available treatment options except Rilutek/Riluzole , which is highly recommended by this neuromuscular team, nevertheless you probably have anywhere from 2-5 years of your life left, I suggest you get your affairs in order.. would you like to speak to our an M D A representative?.
2. Many benefits of attending specialized ALS clinics -- Oh Really, Would You Care to Expand on What They Are? --I am not clear what are the benefits of specialized ALS clinics. Oh, I know, refills for Rilutek, prescription for XYZ depression, anxiety, order lab work to check if my liver is cooked by so much Rilutek, prescription for drooling, referral to ENT, prescription for a wheelchair, or just to tell me every three months that my disease is progressing and there are no options/is NOTHING out there. I call that the trip to HOPELESS LAND...it is more of a hussle. A typical day at a neuromuscular clinic includes... bathing time, dressing time, loading time, driving time, waiting time, (in between specialist's) and finally it's time to see the "specialist/neurologists" to hear a 25 minute no-hope message that includes "see you in three months." So, if I am missing something, I need these authors to tell me what other benefits are there at specialized ALS clinics!!!!
3. On the many benefits of participating in the research studies offered there -- Which one? The one that generates the most donations or the one where I share a cage with the mouse? Tell me Mr. authors, which of the research studies are moving fast enough to generate my interest? What I see mainly is tons of money wasted by big name university researchers to ensure job security for centuries to come. Or the real warrior scientists wanting to participate on research funds that get push aside because they are not affiliated with a big name university or organization. How about "creating false hope" dear authors!
4. And on the dangers of self-experimentation. I recently heard of two scientists who have been spending their OWN money working around the clock to find a cure for ALS, out of their OWN pocket! They know that rare diseases like ALS will not be a one size fits all cure. We all know that ALS is just an umbrella term for a number of different types of motor neuron diseases. And with the exception of the 5% familial type, treatments will need to be customized to the individual and the only way is to do it on trial and error basis. If I am willing to use my body for science sake, why should any article stop me from doing it?
5. We should refrain from off-label prescribing of unproven therapies ... What ? ? ? -- Do these authors know that new drugs are often not tested for safety and efficacy specifically in children. Therefore between 50 percent and 75 percent of all medications prescribed by pediatricians in the U.S. are for off-label applications. Are they proposing to tell these pediatrician not to continue this practice? Another example, the standard of care for a particular type or stage of cancer involves the off-label use of one or more drugs. Another example is the use of tricyclic antidepressants to treat neuropathic pain. According to medical literature, this old class of antidepressants is now rarely used for clinical depression due to its side effects but the tricyclic are often effective for treating pain on those patients with neuropathic conditions. Should we share the authors recommendations with the neuro community too? Oh forgot, how about Provigil an off label drug used for MS patients....the list goes on and one. Do we stop this practice all together for everyone or are the authors suggesting just IPLEX for PALS to be restricted and not prescribed off label. THAT will be discrimination BIG TIME!!!! Law suit, law suit....
6. Most importantly, we need to educate PALS on how to distinguish myth from reality - MAN!... Does anyone else feel insulted?... what about Rilutek/Riluzole? -- Do they honestly think that PALS are a bunch of dummies without brain? Actually, PALS brains are very active and perform best without the distractions of the other body parts it is called ENHANCED functioning....and last time I check Rilutek was really a MYTH....prove me wrong!
Summarizing: These gents need to accept the fact that the world has changed, and the game is almost up. Blessed be cyber technologies that allow people to break through special interests, share information (good or bad) and take over a cause and become engaged activists. Next step, We All Write Letters to Those Well Intended Authors who are just misguided and cannot see that the world has changed and remind them that whenever writing an article just directed to PALS, they must be reminded that, although PALS bodily functions are steadily declining, the one thing that's left intact is the brain . Should our (PALS) families, not have the right to any and all possible therapies including stem cells, IPLEX, or whatever other reasonable therapeutic that might have even marginal efficacy? -- save our lives-? Should it not be our doctors responsibility to adopt the attitude that, all reasonable treatments/therapies including stem cell should be considered , instead of the fatalistic “oh sorry, Mr. Charlie - . Life isn't fair, that’s just the way it is, there's nothing we can do -- SEE YOU IN THREE MONTHS!!
Friday, January 16, 2009
Thursday, January 15, 2009
Downtown Mannequin Torsos Symbolic Of Disease
By JOHN W. ALLMAN The Tampa Tribune
Published: January 15, 2009
More On Lou Gehrig's
More Health News
TAMPA - Most people know the name, but not the disease.
"Piece by Piece," a new campaign by the ALS Association Florida Chapter, hopes to change that by increasing public awareness of Amyotrophic Lateral Sclerosis, or Lou Gehrig's disease.
The campaign's official debut is today at Joe Chillura Courthouse Square Park in downtown Tampa.
Across the grass, volunteers worked in early morning cold to set up 150 mannequin torsos. Each torso bears a black T-shirt. Some have names of Lou Gehrig's patients, both those still living and some who have died.
The display will be open to the public until 6 p.m.
The mannequin bodies have no arms or legs, which is symbolic of the devastating impact of the disease, said Kamden Kuhn, spokeswoman for the Florida chapter.
Lou Gehrig's is a progressive neurodegenerative disease that causes victims to become completely paralyzed. The disease is gradual, however, and can slowly take away the ability to walk, speak and, finally, breathe.
"There are cancer survivors. There are heart attack survivors. There is no such thing as an ALS survivor," said Kuhn, who lost her grandfather to the disease prior to joining the association. "That's why we think this needs a lot of attention."
The "Piece by Piece" campaign was conceived by Tampa advertising agency Dunn & Co. It will travel throughout Florida this year with stops in seven cities.
The mannequins will be on display in Tampa through February. They will be in Lykes Gaslight Square Park next Thursday and Centennial Park in Ybor City on Saturday, Jan. 31.
The ALS Association Florida Chapter is accepting donations for research and patient care. People wishing to give money can have the name of a loved one put on a T-shirt used in the display.
Volunteers will be on hand at each stop of the campaign to provide information.
Steve Franks' name is on a T-shirt.
The Pinellas Park resident, diagnosed with Lou Gehrig's in 2003, considers himself "one of the lucky ones."
Most people die within five years of being diagnosed, Franks said. The disease has limited his ability to walk for long stretches, and affected his speech, but he is still able to drive. Franks, 50, is in charge of transporting the mannequins to each city during the campaign.
"My biggest frustration is that people don't know what ALS is and what it's doing to the people who have it," he said Thursday morning. "That's why I want to be part of this. It's going to educate so many people."
For More Information:To learn more about Amyotrophic Lateral Sclerosis, or Lou Gehrig's disease, and the "Piece by Piece" campaign, go to: http://www.stealingpieces.org/.
To donate to the ALS Association Florida Chapter and sponsor a T-shirt in the campaign, call 888-257-1717, ext. 107.
Reporter John W. Allman can be reached at firstname.lastname@example.org or (813) 259-7915.
Wednesday, January 14, 2009
Nature Biotechnology 27, 10 (2009)
Pfizer's $100 million stem cell stake
Pfizer has launched Pfizer Regenerative Medicine, an independent research unit focused exclusively on using stem cells to develop new medicines. The New York-based company will spend more than $100 million over the next 3-5 years on the new initiative, which will employ 70 researchers based at two facilities, in Cambridge, Massachusetts, and Cambridge, UK. The UK group will focus on neural and sensory disorders, whereas the US team will concentrate on endocrine and cardiac research. In-house researchers will work with both embryonic and adult stem cells, but significant collaborations are also planned. Chief Scientific Officer Ruth McKernan, who will head the UK site, says: "We are keen to take advantage of successful work done by other companies and academic labs. We will be working with several collaborators and these will be announced in the new year." In the past, big pharma has shied away from investing in stem cell research, but Pfizer's move confirms that attitudes are changing. London's GlaxoSmithKline recently signed a $25 million four-year deal with Harvard University, and the venture funds of Basel-based Novartis and Roche helped bankroll Cellerix, a Madrid company testing stem cells from fat to treat rare skin conditions. Stanford University, California, also recently announced the construction of the world's largest stem cell research building to house over 600 scientists by 2010.
Tuesday, January 13, 2009
Jan. 12, 2009
by Janet Kelly
The U.S. National Stem Cell Bank (NSCB) has announced that it has received deposits of two human embryonic stem cell lines from Cellartis AB, a biotechnology company based in Sweden. With the addition of the new lines, the National Stem Cell Bank now has received all 21 cell lines from the six providers listed on the National Institutes of Health (NIH) federal registry.
Currently, 16 of these lines have completed the NSCB's extensive quality control process and are available for distribution to research scientists around the world. The NSCB's initial testing process, which can take several months or longer to finalize, begins upon receipt of a new cell line and is carried out to ensure the identity of the cell line, cell characteristics and that the starting cell material is free from contaminants.
The NIH established the country's first National Stem Cell Bank at the WiCell Research Institute, a private, nonprofit supporting organization to the University of Wisconsin-Madison, in September 2005. Its mission is to obtain, characterize and distribute the 21 human embryonic stem cell lines that currently may be used in U.S. federally funded research. All six providers of the NIH-registry stem cell lines — WiCell at UW-Madison, University of California, San Francisco, and Novocell in the U.S.; ES Cell International (ESI) in Singapore; Technion in Israel; and Cellartis in Sweden — were invited to deposit their cells by the NSCB shortly after it was established.
Derek Hei, a UW-Madison researcher and leader of the NSCB, says the availability of a variety of human embryonic stem cell lines for study is critical to advancing the field. "The addition of the Cellartis lines to the National Stem Cell Bank is extremely important because now we'll be able to distribute these lines to the worldwide research community," he says. "We'll also be able to generate data unique to these lines that is valuable to the advancement of stem cell research."
Mats Lundwall, CEO of Cellartis, says, "We are delighted to have this collaboration with the U.S. National Stem Cell Bank that will increase the amount of NIH eligible lines readily available in the U.S. The Cellartis cell lines are among the most extensively characterized in the world and now their distribution within the U.S. has been further facilitated through this partnership."
In addition, the host organization for the NSCB, the WiCell Research Institute, recently began its own bank, the WiCell International Stem Cell (WISC) Bank, to study and distribute stem cells that currently cannot be offered through its current contract with the NIH. "Due to the NSCB's deposits of all 21 NIH-registry lines and WiCell's ability to offer induced pluripotent and other stem cells through our new WISC Bank, our facility provides a tested operation with experienced staff that is a unique source for stem cell researchers worldwide," says Erik Forsberg, executive director of WiCell.
WiCell researchers are working to characterize the cells in the National Stem Cell Bank to support the development of human embryonic stem cells for research and therapeutic applications. Their efforts include trying to understand how each cell line behaves under different conditions in the laboratory, testing the cells for potential pathogens and assessing their potential to become specific tissues, such as heart, neuronal or insulin-producing islet cells. The results of these studies and tests are made available to the scientific community on the National Stem Cell Bank Web site.
Scientists from nonprofit and academic institutions anywhere in the world can request the human embryonic stem cell lines deposited in the bank. The site provides research protocols and in-depth information on specific cell lines. The NIH Registry Lines available through the National Stem Cell Bank are priced at an affordable $500 per two vials, which contain approximately six million cells capable of establishing multiple new colonies.
The WiCell Research Institute, founded in 1999, is dedicated to expanding the frontiers of science and medicine by unlocking the potential of stem cells. As a private, nonprofit supporting organization of UW-Madison, WiCell conducts research, supports research at UW-Madison, hosts the National Stem Cell Bank and the WISC Bank, provides training for scientists and offers educational outreach programs for K-12 students and the community.
WiCell has provided free human embryonic stem cell licenses to more than 500 researchers in 32 countries and 42 states. WiCell offers an ongoing schedule of stem cell training classes and workshops customized for participants ranging from youth to non-technical adults to lab technicians to top researchers. Since 2002, WiCell has trained more than 600 individuals in stem cell technologies and methodologies.
Cellartis AB is a premier provider of human embryonic stem cell (hES)-derived products and technologies that will drive unprecedented innovation in drug discovery and regenerative medicine today and in the future. Today, alongside fully characterized, ethically derived human embryonic stem cell lines, the company offers custom-prepared human cardiomyocytes and mesenchymal progenitors derived from hES cells along with stem cell antibodies and tools. Cellartis is engaged in programs for the production of homogenous populations of hepatocytes suitable for use as bio-tools in industrial applications. In addition, Cellartis has built the world's first large-volume automated production facility for human ES cells and can provide human ES cells for screening campaigns. The company was founded in 2001, has more than 50 employees and is located in Gothenburg, Sweden and Dundee, Scotland/UK. For more information, e-mail email@example.com.
Friday, January 9, 2009
By Michael Kahn Michael Kahn
Thu Jan 8, 11:51 am ET
LONDON (Reuters) – A novel drug combination using Genzyme Corp's Mozobil shows it may be possible to spur bone marrow into releasing extra adult stem cells into the bloodstream to repair the heart and broken bones, researchers said on Thursday.
The study of mice raises hope that researchers could use the same technique to tackle autoimmune diseases such as rheumatoid arthritis in which the body confuses healthy tissues for foreign substances and attacks itself, they said.
"We hope that by releasing extra stem cells, as we were able to do in mice in our new study, we could potentially call up extra numbers of whichever stem cells the body needs," said Sara Rankin of Imperial College London, who led the study.
"Our work could lead to new treatments to fight various diseases and injuries which work by mobilizing a person's own stem cells from within."
Stem cells are the body's master cells, giving rise to various tissues and the blood. They are found throughout the organs, blood and tissue and are in immature form until they generate needed cell types.
Doctors hope to use them some day in a new field called regenerative medicine in which tailor-made transplants of tissues and perhaps organs can be grown from a patient's own cells.
Rankin and her team looked at mesenchymal stem cells -- immature cells than can give rise to bone, muscle or blood vessels -- and endothelial cells that help make blood vessels in the heart.
They treated healthy mice with one of two proteins that occur naturally in bone marrow called VEGF and G-CSF growth factor. Following this treatment the mice received Genzyme's stem-cell transplantation drug Mozobil.
Researchers know that G-CSF in combination with Mozobil mobilizes one kind of stem cell used in bone marrow transplantation known as hematopoetic stem cells, which give rise to blood cells. But Rankin and colleagues wanted to see if VEGF growth factor could stimulate other types of stem cells involved in building heart and bone tissue and blood vessels.
The team, which reported the findings in the journal Cell Stem Cell, found that mice given VEGF and Mozobil released around 100 times as many endothelial and mesenchymal stem cells into the bloodstream compared to mice that had no treatment.
While trials of the drug combinations in humans is years away, the researchers said the next step is determining in mice whether the technique actually helps repair damage, Rankin said.
"One of the exciting aspects is this would be a non-invasive treatment," she said in a telephone interview. "With this you are just giving a drug to promote what is a natural process."
(Reporting by Michael Kahn; editing by Maggie Fox and Elaine Hardcastle)
Thursday, January 8, 2009
The reason God said we’d flourish like a palm tree is because He knew there would be difficult times. He knew things would come against us to try to steal our joy and victory. God said, “You’re going to be like a palm tree because the storms of life will come, the winds will blow, but you are going to come right back up again stronger than before.” Nothing can hold you back! No weapon formed against you will ever prosper. No matter what’s happening in the world around you, keep standing. Keep praying. Keep believing. Your brightest days are right out in front of you, and God’s plan is to bring you blessing and victory all the days of your life.
Wednesday, January 7, 2009
January 5, 2009
Time Magazine Recognizes ALS Stem CellResearch as No. 1 Medical Breakthrough in 2008
Time magazine has named the creation of motor neurons using ordinary skins cells from people with ALS as the No. 1 medical breakthrough in 2008. The research was reported last summer by The ALS Association.
"Researchers at Harvard and Columbia reported a milestone experiment in July, using a new method - one that doesn't require embryos at all - to generate the first motor neurons from stem cells in two elderly women with Lou Gehrig's disease, or ALS," wrote Alice Park in the magazines Top 10 Everything of 2008 issue published in December.
"The technique, developed by Kyoto University scientist Shinya Yamanaka in 2006, involves reprogramming a patient's ordinary skin cells to behave like stem cells, then coaxing them into the desired tissue-specific cells," continued Park. "Using the motor neurons created from ALS patients, scientists can now study the progress of the disease as the affected cells develop, degenerate and die in a dish - something researchers could never do before for such slow-moving conditions. Once scientists understand the development of ALS, they may be able to create more effective treatments, or perhaps even a cure."
"The recognition by a major news organization of the importance of this research is gratifying," said Dr. Lucie Bruijn, science director and vice president of The ALS Association. "Advances such as this make it possible to achieve a more detailed understanding of the ALS disease process, which we need in order to design therapies."
In an article dated August 1, The Association described how stem cells had been generated from individual patients with ALS, and that the accomplishment was likely to lead to development of new models of ALS and new understanding of disease mechanisms. "They will also provide a potential resource for drug discovery and the development of new treatments for ALS," Bruijn said at the time.
"Model systems to date have focused on the SOD1 mutations linked to 2 percent of ALS. These findings enable the development of cell lines from ALS patients, even those for which the specific causative genes remain unknown," continued Bruijn. "The ability to generate human motor neurons from ALS patients carrying genes linked to the disease is a very exciting accomplishment building on novel technology and the work of several groups." Motor neurons are the nerve cells that die in ALS.
In the study recognized by Time magazine, researchers at Harvard University and Columbia University took skin cells from patients with a genetic form of ALS, caused by mutation in the superoxide dismutase (SOD1) gene. The skin cells were treated with a small set of genes that scientists have recently learned will reprogram adult cells to become stem cells capable of developing into many cell types.
The researchers showed that the genes "deprogrammed" the skin cells, reverting them to an earlier stage in their development, turning them into stem cells. Stem cells, which are formed normally during human development, have the ability to become many different kinds of cells. The researchers showed the new stem cells could transform into motor neurons.
Parallel Efforts Underway by The ALS Association
One important next step will be to make sure the motor neurons can mature, since it is mature motor neurons that are affected by ALS. Such cells could then be examined to determine what factors make them susceptible to the disease process. These studies will need to be done in cells derived from many different patients in the current work, but also in cells derived from other patients, to make sure the results are widely applicable. Another important step will be to generate stem cells from patients with non-genetic (sporadic) forms of the disease. Motor neurons derived from ALS patients will be compared with those derived from people that do not have ALS. These studies are currently underway in parallel efforts funded by The Association. See http://www.alsa.org/research/grants.cfm?grant_id=192
Mature motor neurons would likely be useful for drug discovery, looking for compounds that improve their survival. While much publicity has surrounded the possible use of stem cells to replace dying motor neurons in ALS, the potential of such a treatment is unknown and was not attempted in the study highlighted by Time magazine.
More information on stem cells in ALS can be found HERE (http://www.alsa.org/research/article.cfm?id=715).
Monday, January 5, 2009
There comes a point in your life when you realize who matters, who never did, who won't anymore... and who always will. Don't worry about people from your past, there's a reason why they didn't make it to your future.
'Be kinder than necessary because everyone you meet is fighting some kind of battle.'
For more information contact: Darryl Geddes, 315-464-4828
SUNY Upstate Medical University to Study If Lithium Can Slow Progress of ALS in Humans
Patients sought for clinical trial
SYRACUSE, NY (01/05/2009; 1040)(readMedia)-- SUNY Upstate Medical University is participating in a national clinical trial to determine if lithium, a mood stabilizer, can slow the disease progression of early stage Amyotrophic Lateral Sclerosis (ALS), in humans.
According to Jeremy Shefner, M.D., Ph.D., findings from a study conducted in Italy demonstrated that lithium showed neuroprotective properties in experimental animals and promising results in a small trial of ALS patients. Shefner is SUNY Upstate's lead investigator of the study and professor and chair of the university's Department of Neurology.
"To more fully study the effects of lithium in ALS, the National Institutes of Health /National Institute of Neurologic Diseases and Stroke, the ALS Association and the Canadian ALS Association has funded this important trial," Shefner said. "We will recruit volunteers to participate in the trial. Patient volunteers may be included if they are within three years of their diagnosis and are not already taking lithium. Treatment will be for up to one year."
Lithium is a simple chemical substance approved for use in humans and prescribed as a mood stabilizer. It protects neurons in the brain in animal models of neurodegenerative diseases, including Alzheimer's disease and Parkinson's disease, and has been recently shown to do the same in a mouse model of ALS. In that study, lithium prolonged survival and protected cells in both the brain and spinal cord. While the exact mechanism of lithium's effect is unknown, researchers have proposed it promotes clearance of toxic protein accumulation.
The multi-site investigation will be a double-blind, placebo-controlled trial with 84 patients who will be randomized to either lithium or placebo. A review of data will occur after the 84th person is enrolled, and then a decision will be made on whether to expand the study to 250 patients. The disease course and safety assessments will be measured at regular intervals over that time.
The trial is being supported through the ALS Association's TREAT ALS (Translational Research Advancing Therapies for ALS) initiative, a drug discovery program and clinical trials process created by the association that accelerates discovery and testing of clinical candidates. In addition to SUNY Upstate, investigators from Massachusetts General Hospital, the University of Toronto and Columbia University will participate in the trial.
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost, leading to progressive paralysis.
For more information about the study or to participate in the study, call 315-464-5004.
Sunday, January 4, 2009
Target: President-Elect Barack ObamaSponsored by: Care2
The Bush Administration's ban on federal funding for human embryonic stem cell research has set back medical research on cures for Parkinson's disease, cancer and other diseases. Fear of violating Bush's edict (even for something as minor as buying office supplies with federal funding) has caused many researchers and institutions logistical headaches. Lifting these restrictions would free researchers to spend less time being concerned about government red tape and more time focusing on research. President-elect Obama has pledged to reverse the restrictions, opening federal funding for medical research. Tell Obama now that one of his first acts as president should be to act on his commitment to stem cell research!