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How Injured Racehorses Might Save Your Knees
http://www.technologyreview.com/biomedicine/22998/
Orthopedic stem-cell therapies are moving into human trials.
By Emily Singer
A runner with a torn tendon has reason to envy a racehorse with the same affliction: horses have treatment options not available to human patients--most notably, injections of adult stem cells that appear to spur healing in these animals with shorter recovery time than surgical treatments. Now the same stem-cell therapies used routinely in competitive horses and increasingly in dogs are beginning to make their way into human testing.
Human stem-cell treatments are advancing quickly in many areas: therapies using adult stem cells derived from both fat and bone marrow are currently being tested for a variety of ailments, including Crohn's disease, heart disease, and diabetes. (Bone-marrow-derived stem-cell transplants have been used for decades to treat blood diseases and some cancers.) But when it comes to orthopedic injuries, such as torn tendons, fractures, and degenerating cartilage, veterinary medicine has outpaced human care.
Veterinarians and private companies have aggressively tested new treatments for the most common injuries in racehorses, in large part because these animals are so valuable and can be so severely incapacitated by these wounds. "Soft-tissue injury is the number-one injury competitive horses will suffer and can end a thoroughbred horse's career," says Sean Owens, a veterinarian and director of the Regenerative Medicine Laboratory, at the University of California, Davis. Veterinary medicine also has much more lax regulations when it comes to treating animals with experimental therapies, allowing these treatments to move rapidly into routine clinical use without clinical trials. "Regulatory oversight of veterinary medicine is minimal," says Owens. "For the most part, the USDA [U.S. Department of Agriculture] and the FDA [Food and Drug Administration] have not waded into the regulatory arena for us."
Owens's newly created research center aims to move both animal and human stem-cell medicine forward by conducting well-controlled trials not often performed elsewhere. "Part of our mission is to do basic science and clinical trials and also improve ways of processing cells," says Owens. The center has a number of ongoing clinical trials in horses--one for tendon tears and one for fractured bone chips in the knee--that are run in a similar way to human clinical trials. The goal is to develop better treatments for horses, as well as to leverage the results to support human studies of the same treatments. Owens is partnering with Jan Nolta,director of the Stem Cell Program, at UC Davis, who will ultimately oversee human testing.
A handful of studies in animals have shown that these stem-cell therapies are effective, allowing more animals to return to racing, reducing reinjury rates, and cutting healing times. VetCell, a company based in the United Kingdom that derives stem cells from bone marrow, has used its therapy on approximately 1,700 horses to date. In a study of 170 jumping horses tracked through both treatment and rehabilitation, researchers found that nearly 80 percent of them could return to racing, compared with previously published data showing that about 30 percent of horses given traditional therapies could return to racing. After three years, the reinjury rate was much lower in stem-cell-treated animals--about 23 percent compared with the published average of 56 percent, says David Mountford, a veterinary surgeon and chief operating officer at VetCell.
While scientists still don't know exactly how the cells aid repair of the different types of injuries, for tendon tears, initial studies show that stem cells appear to help the tissue regenerate without forming scar tissue.
Mountford says that the company chose to focus on tendon injuries in horses in part because they so closely resemble injuries in humans, such as damage to the Achilles tendon and rotator cuff. For both people and horses, tendon tears trigger the formation of scar tissue, which has much less tensile strength and elasticity than a healthy tendon. "It becomes a weak spot and prone to injury," says Owens.
Next year, VetCell plans to start a human clinical trial of its stem-cell treatment for patients with degeneration or damage of the fibers of the Achilles tendon. As in the horse therapy, stem cells will be isolated from a sample of the patient's bone marrow, then cultured and resuspended in a growth medium also derived from the patient. Surgeons will then inject the solution into the area of damage, using ultrasound imaging to guide the needle to the correct location. "Our long-term goal is to use it to treat a number of tendon injuries," says Mountford.
Stem-cell therapies also show promise for arthritis. Vet-Stem, a California-based company that uses stem cells isolated from fat rather than bone marrow, has shown in a placebo-controlled trial that the treatment can help arthritic dogs. "About 200,000 hip replacements are done every year in humans," says Robert Harman, a veterinarian and founder of the company. "That's a very good target for someone to look at cell therapy."
For osteoarthritis, the stem cells seem to work not by regenerating the joint, but by reducing inflammation. "But in the last couple of years, evidence has come out that the cells we use reduce inflammation and pain, and help lubricate the joint," says Harman.
While Vet-Stem does not plan to move into human testing, Cytori, a company based in San Diego, has developed a device for isolating stem cells from fat in the operating room. (Vet-Stem does the procedure manually: veterinarians collect a fat sample from the animal and then send it to the company for processing.) Cytori's device is currently approved for use for reconstructive surgeries in Japan but not yet in the United States.
Stem cell news, 7-7-09, 04:00 PDT Washington -
"Feds ease restrictions on use of stem cells"
http://www.sfgate.com/cgi-bin/article.cgi?f=/c/a/2009/07/07/MN8F18JS7Q.DTL&type=science
In muscle stem cells, age matters: study
http://www.reuters.com/article/scienceNews/idUSTRE55O2ML20090625?sp=true
CHICAGO (Reuters) - A new understanding of the genes that make muscle cells may change the way researchers think about stem cell transplants for muscular dystrophy and muscle injuries, U.S. researchers said on Wednesday.
In a surprise finding, they said genes important for forming muscle cells in embryos and newborns are not normally active in adult stem cells.
And researchers hoping to use muscle stem cells in stem-cell transplant therapies should not assume genes that control early muscle development serve the same purpose in repairing adult muscle, Christoph Lepper and colleagues at the Carnegie Institution in Baltimore reported in the journal Nature.
Earlier studies have shown that two genes -- Pax3 and Pax7 -- control cells that give rise muscle in embryos, and Pax7 also helps build muscle in newborn mice.
To get a better understanding of their function, Lepper and colleagues studied these genes at various stages of development in live mice.
"I thought that if they are so important in the embryo, they must be important for adult muscle stem cells," Lepper said in a statement.
The team used genetic engineering to suppress both the Pax3 and Pax7 genes in adult muscle stem cells, and they found that adult stem cells were still able to function normally.
"I was totally surprised to find that the muscle stem cells are normal without them," Lepper said.
The researchers then looked at whether the same was true in injured muscles, when muscle stem cells go to work making new muscle tissue.
To study this, they injured mouse leg muscles between the knee and ankle, and found the muscle stem cells were able to make new muscle, even without the two key embryonic muscle stem cell genes.
The team said the embryonic muscle cell genes appear to only be active in mice within the first three weeks after birth. After that, they believe the genes go quiet and allow a different set of genes to take over.
Finding those genes will be important as scientists pursue new treatments for diseases like muscular dystrophy, a genetic, degenerative disease that affects voluntary muscles, they said.
And they said teams should look at other types of stem cells to see how age might affect their properties, and they should take age of stem cells into account in transplant-based treatments.
)))PL1(((
Contact: Leslie Orr
Leslie_Orr@urmc.rochester.edu
585-275-5774
University of Rochester Medical Center
Controversial cancer stem cells offer new direction for treatment
In a review in Science, a University of Rochester Medical Center researcher sorts out the controversy and promise around a dangerous subtype of cancer cells, known as cancer stem cells, which seem capable of resisting many modern treatments.
The article proposes that this subpopulation of malignant cells may one day provide an important avenue for controlling cancer, especially if new treatments that target the cancer stem cell are developed and combined with traditional chemotherapy and/or radiation.
"The fact that these concepts are steadily making their way into the clinic is exciting, and suggests that the recent interest in cancer stem cells may yield beneficial outcomes in potentially unexpected ways," wrote co-authors Craig T. Jordan, Ph.D., professor of Medicine at URMC and director of the James P. Wilmot Cancer Center Translational Research for Hematologic Malignancies program; and Jeffrey Rosen, Ph.D., the C.C. Bell Professor of Molecular and Cellular Biology and Medicine at Baylor College of Medicine.
Cancer stem cells (CSCs) are a hot topic in the scientific community. First identified in 1994 in relation to acute myeloid leukemia, CSCs have now been identified in several solid tumors in mice as well. Scientists who study CSCs believe they have distinct properties from other cancer cells, and may be the first cells to undergo mutations.
Research from the past 10 years suggests that because CSCs may be the root of cancer, they also might provide a new opportunity for a treatment. Jordan and a group of collaborators, for example, are testing a new drug compound based on the feverfew plant that demonstrates great potential in the laboratory for causing leukemia CSCs to self destruct.
Another new approach, the authors said, is the use of chemical screens to search drug libraries for already approved agents that may target CSCs, or make resistant tumor cells more sensitive to chemotherapy and radiation.
Cancer stem cell biologists hypothesize that any treatment that targets the source of origin rather than simply killing all cells, healthy and malignant, would be an improvement over most conventional therapies.
Some scientists, however, are uncertain if CSCs have unique biological properties or any relevance to treatment, the authors noted. What is more likely to fuel cancer, other studies have found, are unfavorable factors in the neighboring cells surrounding the tumor, such as mutated genes, proteins that encourage cell growth, and a poor immune system, for instance.
The most challenging issue facing CSC biologists is that the number and type of cancer stem cells can vary from patient to patient. In some tumor samples, for example, CSCs are rare while in others they constitute a large portion of the tumor mass, the authors said.
To understand why CSCs are so variable, investigators are trying to determine what genes and pathways are responsible for activating cancers that have a poor prognosis, and whether these cancers also have a higher frequency of CSCs.
"Whether the cancer stem cell model is relevant to all cancers or not," they wrote, "it is clear that we need new approaches to target tumor cells that are resistant to current therapies and give rise to recurrence and treatment failure."
An unexpected benefit of so much attention on normal stem cells is that it has stimulated research in areas not previously the focus of cancer therapies, Jordan and Rosen said.
For example, pathways known to be important for normal stem cell self-renewal, such as the Wnt, Notch and Hedgehog(Hh) pathways, are now of increased interest due to their potential role in CSCs. The first clinical trial using an agent to block the Notch pathway in combination with chemotherapy for breast cancer has begun.
The authors conclude by spotlighting the pressing need for preclinical models to test appropriate doses and combinations of CSC therapies before they can move into human clinical trials.
Aastrom says FDA lifts stem cell trial halt
http://finance.yahoo.com/news/Aastrom-says-FDA-lifts-stem-apf-3848570636.html?x=0&.v=1
Aastrom Biosciences jumps after FDA ends suspension of stem cells to treat heart disease
* On Thursday June 18, 2009, 1:07 pm EDT
NEW YORK (AP) -- Aastrom Biosciences Inc., which develops adult stem cells to treat heart diseases, said Thursday the Food and Drug Administration has lifted a halt on a clinical trial.
Aastrom said it will resume enrolling patients in the midstage trial, called IMPACT-DCM, because the FDA has completed an investigation into the death of a patient. The FDA concluded that the patient died of dilated cardiomyopathy and not from treatment, the company said.
In afternoon trading, shares of the Ann Arbor, Mich., company climbed 8 cents, or 21.8 percent, to 46 cents.
In the IMPACT-DCM study, Aastrom is testing the ability of autologous stem cells, or stem cells from the patient's own body, to treat dilated cardiomyopathy, which is a severe form of chronic heart failure that causes the heart to swell and impairs circulation.
Aastrom said the study was suspended May 22. The company said it still expects to finish enrolling patients by the end of the year. The company has enrolled 14 patients in the trial, out of a planned goal of 40.
Stem-cell clarity
http://www.nature.com/nature/journal/v459/n7247/full/459615b.html
The draft NIH guidelines on stem-cell research are a good first step, but some revision is needed.
The proposed guidelines on federal funding for stem-cell research issued in April by the US National Institutes of Health (NIH) are a welcome effort to assert ethical and regulatory leadership over this field — especially given the vacuum in oversight left by the previous US administration. Yet concerns aired by the scientific community during the public comment period that closed last week have underscored the need for the NIH to revise the guidelines to allow the responsible progress of research.
For example, the NIH has acted admirably in setting forth nine strict informed-consent provisions regarding the source material for stem-cell lines that are eligible for federal funding. As currently written, however, the provisions would probably exclude funding for most embryonic stem cells now in use, because the cells were derived from leftover embryos at fertility clinics under rules less stringent than the ones now called for. In particular, one provision requires that embryo donors affirm that they are donating "without any restriction or direction" regarding the patients who may benefit from the research. Few of the consent forms currently in use by fertility clinics ask for that affirmation. Also absent from many forms is the stipulation that the donors "would not receive financial or any other benefits" from commercial development of the research.
It is doubtful that the guidelines are intended to bar future NIH funding from stem-cell lines that are currently eligible — or, indeed, from the hundreds of lines that are now in use but are not among the score of US-approved lines. That would contradict US President Barack Obama's intention, stated on 9 March, to "expand NIH support for the exploration of human stem cell research". The NIH should explicitly state that the informed-consent provisions apply only to newly created lines. All previously eligible lines should continue to be eligible, and existing non-eligible lines should become so — provided that the latter were created in accordance with guidelines issued by the US National Academies or the International Society for Stem Cell Research.
A much less clear-cut issue is whether federal funding should support work on lines created from sources other than leftover embryos. The NIH's draft guidelines currently exclude support for research on lines created through somatic-cell nuclear transfer (SCNT), also known as therapeutic cloning. And they prohibit funding for lines created through the generation of an embryo from an unfertilized egg cell.
Some investigators have protested this provision of the guidelines, arguing that the NIH should not cut off any avenues of research. Their contention is somewhat hypothetical, however, because no one has yet shown conclusive evidence that SCNT can successfully create a human embryo. Regardless of this, the ethical issues involved are extremely sensitive. Polls consistently show that a majority of the American public is willing to pay for research on stem cells derived from embryos that would be discarded otherwise. But it is not clear that a majority would support the use of taxpayers' money to study stem cells from embryos created and destroyed for research purposes alone. So unless the scientists arguing for federal funding of research on SCNT-derived stem cells can make a much stronger case, by spelling out the specific situations in which the research might be warranted and explaining how they will ensure proper oversight of the work, the NIH's proposed exclusion should stand.
At the same time, however, the NIH should affirm that it will revisit its draft guidelines as the science progresses. The past decade shows us that basing research policy on arbitrary cut-off dates does not serve science or the public interest well.
Stem cell breakthrough gets closer to the clinic
http://news.yahoo.com/s/afp/20090528/ts_alt_afp/healthbiotechstemcellslead_20090528174115
AFP/File – Embryonic stem cells are pictured through a microscope viewfinder in a laboratory. The quest for versatile, … by Mira Oberman Mira Oberman – Thu May 28, 1:40 pm ET
CHICAGO (AFP) – The technology for versatile, grow-in-a-dish transplant tissue took a step toward clinical use Thursday when researchers announced they have found a safe way to turn skin cells into stem cells.
Researchers say the method is so promising they hope to apply for approval to begin clinic trials by the middle of next year.
"This is the first safe method of generating patient specific stem cells," said study author Robert Lanza, the chief scientific officer at Stem Cell & Regenerative Medicine International.
"This technology will soon allow us to expand the range of possible stem cell therapies for the entire human body," Lanza told AFP.
"This allows us to generate the raw material to solve the problem of rejection (by the immune system) so this is really going to accelerate the field of regenerative medicine."
The research builds on an award-winning breakthrough in 2007 by Shinya Yamanaka of Kyoto University.
Yamanaka and his team introduced four genes into skin cells, reprogramming them so that they became indistinguishable from embryonic stem cells.
That achievement conjured the distant vision of an almost limitless source of transplant material that would be free of controversy, as it would entail no cells derived from embryos.
But the downside of the technique for creating these so-called induced pluripotent stem cells (iPS) is that the genes are delivered by a "Trojan horse" virus.
Reprogramming cells using a virus modifies their DNA in such a way that they cannot be given to patients without boosting the risk of cancer and genetic mutation.
Other researchers have succeeded in delivering the genes with a method called DNA transfection or using a chemical wash, but these techniques also posed health risks.
Lanza and the team led by Kwang Soo Kim of Harvard University succeeded in delivering the genes by fusing them with a cell penetrating peptide which does not pose the risk of genetic mutation.
While this method took twice as long to generate pluripotent stem cells, Lanza said he believes his team can increase the efficiency of the transmission by purifying the protein.
The study was published in the online edition of Cell Stem Cell.
Stem cells have excited huge interest over the past decade.
Promoters say this material could reverse cancer, diabetes, Alzheimer's and other diseases and also allow researchers to grow patient-specific organ and tissue transplants which will not require harmful anti-rejection drugs.
But the dynamic has been sapped by opposition from religious conservatives, who argue that research on embryos -- the prime source of stem cells so far -- destroys human life.
Generating stem cells from skin cells bypasses the controversy and also dramatically increases the availability of patient-specific stem cells
PL1
Hey all, since I'm in Houston I dropped by the Stem Cell Innovations (SCLL) offices last week and asked a bunch of questions. Figured the wider stem community might be interested...
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=37473076
Possible treatment of oral diseases
Human embryonic stem cells
http://www.eurekalert.org/pub_releases/2009-04/iaa-hes032409.php
Human embryonic stem cells (hESC) provide a potentially unlimited source of oral mucosal tissues that may revolutionize the treatment of oral diseases. When fully exploited in the future, this source of cells will be able to produce functional tissues to treat a broad variety of oral diseases. However, little is known about how hESC can be developed into complex, multilayer oral tissues that line the gums, cheeks, lips, and other intra-oral sites. However, the use of hES cells for oral application faces numerous obstacles that must be overcome before their therapeutic potential can be realized.
Today, during the 87th General Session of the International Association for Dental Research, investigators from Tufts University in Boston report on their research to optimize the potential of hESC cells to generate complex, functional multilayer tissues, such as the oral mucosa and skin, and to understand how tissue fabrication is controlled and directed.
The Garlick lab has used tissue engineering principles to produce complex oral-lining tissues that mimic many features of their counterparts found in the oral cavity. Making these tissues was a two-step process. With a combination of chemical signals and specialized surfaces on which these cells attach, an hESC cell line (H9) was directed toward two divergent cell populations. The first population comprises the surface layer (epithelial cells) of complex tissues, while the other is found beneath these cells (mesenchymal cells). Following their isolation and characterization, the team incorporated these two distinct cell populations into the two tissue compartments that comprise these tissue types. The populations were then grown at an air-liquid interface to mimic their growth environment in the oral cavity. Within two weeks, tissues developed that shared many features in common with normal tissues that were constructed with mature cells that are the "gold standard" of normal tissue generation in our lab.
For the first time, researchers have established proof of concept that a single, common source of pluripotent hESC could provide the multiple cell types needed to be recombined within different, but interactive, tissue compartments to generate complex, multilayer tissues. In addition to providing oral mucosal tissues for future transplantation, the tissues generated in these studies can now be used to answer questions regarding the stability and safety of hESC-derived cells and tissues by providing information that will predict how they will respond after therapeutic transplantation in the future.
ThermoGenesis Corp. (Nasdaq: KOOL), a leading supplier of innovative products and services that process and store adult stem cells, said that its devices will be the subject of several scientific abstracts to be presented...
http://www.medicalnewstoday.com/articles/144896.php
Scientists Discover That Heart Muscle Cells Are Renewed
03 April 2009
Research led by scientists in Sweden found that our heart muscle cells are renewed over our lifetime and we are not limited to those we are born with. They believe the discovery opens a door to treatments that could...
http://www.medicalnewstoday.com/articles/144917.php
Heart Hospital Of Austin First Site In World To Test Efficacy Of New Adult Stem Cell Therapy To Treat Damaged Heart Tissue In Phase II Trial:
http://www.medicalnewstoday.com/articles/144472.php
That's great, PL1. Your board link is there. afxm
This is a Stem Cells website developed by axfm:
http://afxm.com/StemCellStocks.html
PL1
Absolutely...consider it done and thanx for your link and work....PL1
Preciouslife1, I built a stem cell stocks research page and would like to exchange links with your message board. I would place a descriptive link to your board in the box with the StemCell Inc, board link in exchange for a link in the ibox of your board or in the sticky. Please take a look and let me know if you would like to do that. My website is not for profit.
thanks, afxm
http://afxm.com/StemCellStocks.html
Stem Cell Research: New Way To Make Stem Cells Avoids Risk Of Cancer
http://www.sciencedaily.com/releases/2009/03/090326141547.htm
Scientists at the University of Wisconsin-Madison have found a way to endow human skin cells with embryonic stem cell-like properties without inserting potentially problematic new genes into their DNA.
The team of researchers reports that it has created induced human pluripotent stem (iPS) cells completely free of viral vectors and exotic genes.
By reprogramming skin cells to an embryonic state using a plasmid rather than a virus to ferry reprogramming genes into adult cells, the Wisconsin group's work removes a key safety concern about the potential use of iPS cells in therapeutic settings.
The new method, which is reported in March 26 in the journal Science, also removes the exotic reprogramming genes from the iPS equation, as the plasmid and the genes it carries do not integrate into an induced cell's genome and can be screened out of subsequent generations of cells. Thus, cells made using the new method are completely free of any genetic artifacts that could compromise therapeutic safety or skew research results, according to the Science report.
The new work was conducted in the laboratory of James Thomson, the UW-Madison scientist who was the first to successfully culture human embryonic stem cells in 1998 and, in 2007, co-discovered a way to make human-induced pluripotent stem cells. Thomson, a professor in the UW-Madison School of Medicine and Public Health, is also the director of regenerative biology for the Morgridge Institute for Research, the private, nonprofit side of the new Wisconsin Institutes for Discovery at UW-Madison.
"We believe this is the first time human-induced pluripotent stem cells have been created that are completely free of vector and transgene sequences," says Thomson.
The new study was led by geneticist Junying Yu, the Wisconsin researcher who, with Thomson, co-discovered a method for reprogramming adult skin cells to behave like embryonic stem cells, the master cells that arise at the earliest stages of development and that ultimately develop into all 220 cell types in the human body.
While the methods first devised for reprogramming adult cells yielded embryonic-like cells, the process resulted in the permanent integration of both viral genes and the genes used for reprogramming into the genomes of the newly induced cells. Such genetic baggage posed safety concerns for potential therapies like cell transplants, and confounded work in the lab, as the introduced genes sometimes spurred mutations that interfered with the normal function of induced cells.
The new work was accomplished using a plasmid, a circle of DNA, and cells from the foreskins of newborns. "The plasmids carry all the needed transgenes, but don't integrate into the host DNA, they just float around as episomes" in the cell, Thomson says.
The plasmids replicate, but they do so somewhat inefficiently, Thomson explains, so that after they perform the job of reprogramming, they can subsequently be weeded out, leaving the induced cells free of any exotic genetic material. "Once the transgenes have done their job and are no longer needed, one can merely recover induced pluripotent stem cells that have lost their episomes."
The resulting cells, says Thomson, are remarkably similar to embryonic stem cells and show the same capacity to proliferate indefinitely in culture and diversify into all the cell types of the human body.
"The recent discovery that adult cells could be reprogrammed to iPS cells that resemble embryonic stem cells opened up tremendous potential for regenerative medicine," says Marion Zatz of the National Institute of Health's National Institute of General Medical Sciences, which partially funded the new work. "However, the early methods posed significant risks in using iPS cells in a clinical setting. This latest discovery by Thomson's group of a new method for generating iPS cells without inserting viral vectors into the cells' genetic material is a major advance toward safely reprogramming cells for clinical use."
Thomson notes that researchers have developed other promising approaches using mouse cells, and previously had removed most of the vector and exogenous gene sequences from human-induced pluripotent stem cells. However, those efforts did not succeed in removing all of the genetic artifacts of reprogramming, which could still result in mutations in induced cells.
"Given the rapid pace of the field, it won't be surprising if there are several alternative methods for producing vector and transgene free cells very soon," says Thomson. "However, it will be essential to determine which of these methods most consistently produces induced pluripotent stem cells with the fewest genetic abnormalities. Any problems would impact research, drug development and possible transplantation therapies."
Junying Yu, Kejin Hu, Kim Smuga-Otto, Shulan Tian, Ron Stewart, Igor I. Slukvin and James A. Thomson. Human Induced Pluripotent Stem Cells Free of Vector and Transgene Sequences. Science, March 26, 2009
Journal reference:
Junying Yu, Kejin Hu, Kim Smuga-Otto, Shulan Tian, Ron Stewart, Igor I. Slukvin and James A. Thomson. Human Induced Pluripotent Stem Cells Free of Vector and Transgene Sequences. Science, March 26, 2009
From the same group that also published the work posted in #msg-26127651
USC researchers uncover mechanism that regulates movement of blood-forming stem cells in the body
http://www.eurekalert.org/pub_releases/2009-03/uosc-uru032409.php
Findings may lead to improved efficiency of bone marrow transplants
Researchers at the Keck School of Medicine of the University of Southern California (USC) have identified a signaling pathway that helps regulate the movement of blood-forming stem cells in the body—a finding that provides important new insight into how stem cells move around the body and which may lead to improvements in the efficiency of bone marrow transplants.
The study will appear in the journal Nature, and is available online March 25th.
"By identifying the key mechanism by which these stem cells home and engraft to the bone marrow, it may be possible to pharmacologically treat the cells to activate this pathway and thus increase the effectiveness of bone marrow transplants," says lead author Gregor Adams, Ph.D., assistant professor of cell and neurobiology at the Keck School and a researcher at the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC.
Hematopoietic stem cells are blood-forming cells that circulate through the body shifting back and forth between the bloodstream and bone marrow, Adams explains. When patients receive a bone marrow transplant, healthy blood stem cells are injected into their veins. Unless those stem cells can find their way into a specific site known as the stem cell niche, they cannot develop properly to replenish the white cells, red cells and platelets in the patient's blood.
The mechanisms that guide the cells during this migration have not been well understood. However, in this study the researchers found that blood-forming stem cells that lacked a specific signaling molecule, called GalphaS, did not home to or engraft in the bone marrow of adult mice, Adams says.
"Here we show that the GalphaS pathway is a critical intracellular pathway involved in this process," he says. "Currently, large numbers of blood-forming stem cells are required in bone marrow transplantation due to the limited efficiency of the homing process. This study opens up the possibility of treating bone marrow cells with GalphaS pathway activators as a means to increase the effectiveness of bone marrow transplants."
Improving the efficiency with which stem cells colonize the bone marrow following transplantation could have far-reaching implications for disease treatment, says Martin Pera, Ph.D., director of the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC.
"For example, such a discovery might enhance the utility of umbilical cord blood, which contains only limited numbers of stem cells, for the treatment of cancer and blood disorders in children and adults," Pera says.
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Official: Source: Obama to reverse limits on stem cell work
By BEN FELLER and LAURAN NEERGAARD, Associated Press Writers Ben Feller And Lauran Neergaard, Associated Press Writers
2 mins ago
WASHINGTON – Reversing an eight-year-old limit on potentially life-saving science, President Barack Obama plans to lift restrictions Monday on taxpayer-funded research using embryonic stem cells.
The long-promised move will allow a rush of research aimed at one day better treating, if not curing, ailments from diabetes to paralysis — research that crosses partisan lines, backed by such notables as Nancy Reagan and the late Christopher Reeve. But it stirs intense controversy over whether government crosses a moral line with such research.
Obama will hold an event at the White House to announce the move, a senior administration official said Friday. The official spoke on condition of anonymity because the policy had not yet been publicly announced.
Embryonic stem cells are master cells that can morph into any cell of the body. Scientists hope to harness them so they can create replacement tissues to treat a variety of diseases — such as new insulin-producing cells for diabetics, cells that could help those with Parkinson's disease or maybe even Alzheimer's, or new nerve connections to restore movement after spinal injury.
"I feel vindicated after eight years of struggle, and I know it's going to energize my research team," said Dr. George Daley of the Harvard Stem Cell Institute and Children's Hospital of Boston, a leading stem cell researcher.
But the research is controversial because days-old embryos must be destroyed to obtain the cells. They typically are culled from fertility-clinic leftovers otherwise destined to be thrown away.
Under President George W. Bush, taxpayer money for that research was limited to a small number of stem cell lines that were created before Aug. 9, 2001, lines that in many cases had some drawbacks that limited their potential usability.
But hundreds more of such lines — groups of cells that can continue to propagate in lab dishes — have been created since then, ones that scientists say are healthier, better suited to creating treatments for people rather than doing basic laboratory science.
Work didn't stop. Indeed, it advanced enough that this summer, the private Geron Corp. will begin the world's first study of a treatment using human embryonic stem cells, in people who recently suffered a spinal cord injury.
Nor does Obama's change fund creation of new lines. But it means that scientists who until now have had to rely on private donations to work with these newer stem cell lines can apply for government money for the research, just like they do for studies of gene therapy or other treatment approaches.
The aim of the policy is to restore "scientific integrity" to the process, the administration official said.
"America's biomedical research enterprise experienced steady decline over the past eight years, with shrinking budgets and policies that elevated ideology over science. This slowed the pace of discovery and the search for cures," said Sean Morrison, director of the University of Michigan's Center for Stem Cell Biology.
Critics immediately denounced the move.
"Taxpayers should not have to foot the bill for experiments that require the destruction of human life," said Tony Perkins of the conservative Family Research Council. "President Obama's policy change is especially troubling given the significant adult stem cell advances that are being used to treat patients now without harming or destroying human embryos."
Indeed, there are different types of stem cells: So-called adult stem cells that produce a specific type of tissue; younger stem cells found floating in amniotic fluid or the placenta. Scientists even have learned to reprogram certain cells to behave like stem cells.
But even researchers who work with varying types consider embryonic stem cells the most flexible and thus most promising form — and say that science, not politics, should ultimately judge.
"Science works best and patients are served best by having all the tools at our disposal," Daley said.
Obama made it clear during the campaign he would overturn Bush's directive.
During the campaign, Obama said, "I strongly support expanding research on stem cells. I believe that the restrictions that President Bush has placed on funding of human embryonic stem cell research have handcuffed our scientists and hindered our ability to compete with other nations."
He said he would lift Bush's ban and "ensure that all research on stem cells is conducted ethically and with rigorous oversight."
"Patients and people who've been patient advocates are going to be really happy," said Amy Comstock Rick of the Coalition for the Advancement of Medical Research.
The ruling will bring one immediate change: As of Monday, scientists who've had to meticulously keep separate their federally funded research and their privately funded stem cell work — from buying separate microscopes to even setting up labs in different buildings — won't have that expensive hurdle anymore.
Next, scientists can start applying for research grants from the National Institutes of Health. The NIH already has begun writing guidelines that, among other things, are expected to demand that the cells being used were derived with proper informed consent from the woman or couple who donated the original embryo.
The 2 manuscripts discussed in the article are really good and I think these cells bring us closer to the intended target of regenerative medicine. They will also capture the major attention at the international stem cell meeting in Barcelona this summer.
Virus-free pluripotency for human cells
Stem-cell advance could bring tailored treatments closer.
Erika Check Hayden & Monya Baker
http://www.nature.com/news/2009/090227/full/458019a.html
Published online 1 March 2009 | 458, 19 (2009) | doi:10.1038/458019a
Researchers are close to making safer stem cells.
K. Woltjen et al.
For the first time, specialized human cells have been transformed into a state similar to that seen in embryonic stem cells, without using viruses. The advance edges stem-cell biologists closer to clearing a barrier to using reprogrammed cells for therapies and drug screening.
"The field has been waiting for these papers," says Marie Csete, chief scientific officer at the California Institute for Regenerative Medicine in San Francisco.
Embryonic stem cells are pluripotent — capable of generating all the body's specialized cell types — and creating tailor-made cell lines might allow scientists to better study human diseases and test possible treatments. That looked difficult until 2006, when Shinya Yamanaka and his colleagues at Kyoto University in Japan reported that they had reprogrammed mouse skin cells into an embryonic-like state by infecting them with a virus containing four genetic factors. Yamanaka called the reprogrammed cells induced pluripotent stem (iPS) cells.
Since then, scientists have used various viral vectors to reprogram human cells with the 'Yamanaka factors', and have used non-viral methods to reprogram mouse cells.
No one had been able to reprogram human cells without using viruses — which integrate unpredictably into the genome — until now.
Stem-cell researchers led by Andreas Nagy, of the Samuel Lunenfeld Research Institute at Mount Sinai Hospital in Toronto, Canada, and Keisuke Kaji, of the University of Edinburgh, UK, inserted genes encoding Yamanaka's factors into a piece of DNA, or cassette, that also contained a jumping gene known as piggyBAC. The teams showed that this cassette could be inserted into the DNA of mouse and human skin cells and could reprogram them back to an embryonic-like state (K. Kaji et al. Nature doi:10.1038/nature07864; 2009, K. Woltjen et al.
Nature doi:10.1038/nature07863; 2009).
The teams then used an enzyme called transposase to remove the cassette from the mouse cells. But some scientists say that until the cassette is removed from human cells, the technique is not a major advance over viral methods.
Nagy, however, is confident that he will be able to use transposase to remove the cassette from human cells. He is currently trying to use his method to reprogram cat and dog cells.
The Washington Post piece on that:
Researchers Find Safer Way to Produce Stem Cell Alternative
Skin Cells Transformed Without Worrisome Use of Viruses
http://www.washingtonpost.com/wp-dyn/content/article/2009/03/01/AR2009030101741_2.html
By Rob Stein Monday, March 2, 2009; A05
Scientists have developed what appears to be a safer way to create a promising alternative to embryonic stem cells, boosting hopes that such cells could sidestep the moral and political quagmire that has hindered the development of a new generation of cures.
The researchers produced the cells by using strands of genetic material, instead of potentially dangerous genetically engineered viruses, to coax skin cells into a state that appears biologically identical to embryonic stem cells.
"It's a leap forward in the safe application of these cells," said Andras Nagy of Mount Sinai Hospital in Toronto, who helped lead the international team of researchers that described the work in two papers being published online today by the journal Nature. "We expect this to have a massive impact on this field."
In addition to the scientific implications, the work comes at a politically sensitive moment. Scientists are anxiously waiting for President Obama to follow through on his promise to lift restrictions on federal funding for research on human embryonic stem cells. Critics of such a move immediately pointed to the work as the latest evidence that the alternative cells make such research unnecessary.
"Stem cell research that requires destroying embryos is going the way of the Model T," Richard M. Doerflinger of the U.S. Conference of Catholic Bishops said. "No administration that values science and medical progress over politics will want to divert funds now toward that increasingly obsolete and needlessly divisive approach."
Scientists, however, while praising the work as a potentially important advance, said it remains crucial to work on both types of cells because it is far from clear which will turn out to be more useful.
"The point is, we don't know yet what the end potential of either of these approaches will be," said Mark A. Kay of Stanford University. "No one has cured any disease in people with any of these approaches yet. We don't know enough yet to know which approach will be better."
Because embryonic stem cells are believed capable of becoming any kind of tissue in the body, scientists believe they could eventually lead to treatments or even cures for a host of ailments, including heart disease, diabetes, and Alzheimer's and Parkinson's diseases. In 2001, President George W. Bush restricted federal funding for human embryonic stem cell research to prevent taxpayer money from encouraging the destruction of human embryos, which is necessary to obtain the cells.
The alternative cells, known as induced pluripotent stem cells, or iPS cells, appear to have many of the same characteristics as embryonic stem cells but are produced by activating genes in adult cells to "reprogram" them into a more primitive state, bypassing the moral, political and ethical issues surrounding embryonic cells. Until now, however, their use has been limited because the genetic manipulation required the use of viruses, raising concerns the cells could cause cancer if placed in a patient. That has triggered a race to develop alternative approaches.
"These viral insertions are quite dangerous," Nagy said.
In the new work, Nagy and his colleagues in Toronto and at the University of Edinburgh in Scotland instead used a sequence of DNA known as a transposon, which can insert itself into the genetic machinery of a cell. In this case, the researchers used a transposon called "piggyBac" to carry four genes that can transform mouse and human embryonic skin cells into iPS cells. After the conversion took place, the researchers removed the added DNA from the transformed cells using a specific enzyme.
"PiggyBac carries the four genes into the cells and reprograms the cells into stem cells. After they have reprogrammed the cells, they are no longer required, and in fact they are dangerous," Nagy said. "After they do their job they can be removed seamlessly, with no trace left behind. The ability for seamless removal opens up a huge possibility."
A series of tests showed that the transformed cells had many of the properties of embryonic stem cells, Nagy said.
The researchers did their initial work on skin cells from embryos but say the approach should work just as efficiently in adult cells, and they plan to start those experiments.
"We do not expect that adult cells would behave significantly differently than the ones we are using currently," Nagy said.
In addition to producing safer cell lines that would be less likely to cause cancer in patients, the advance will enable many more scientists to begin working on such cells because they require no expertise or special laboratories necessary for working with viruses, he said.
"This opens up the possibility of working in this field for laboratories that don't have viral labs attached to them. A much larger number of laboratories will be able to push this field forward," Nagy said.
Other researchers praised the work.
"It's very significant," said George Q. Daley, a stem cell researcher at Children's Hospital in Boston. "I think it's a major step forward in realizing the value of these cells for medical research."
"It's very exciting work," agreed Robert Lanza, a stem cell researcher at Advanced Cell Technology in Worcester, Mass. "With the new work, we're only a hair's breadth away from the biggest prize in regenerative medicine -- a way to create patient-specific cells that are safe enough to use clinically."
Kay agreed that the work is promising but cautioned that much more research will be needed to prove that cells produced this way are safe. Many scientists are working on other approaches that may turn out to be safer and more efficient, he said.
"This is a step forward. The research is heading in the right direction. But there still may be room for improvement," he said.
Stem Cell Breakthrough: New Method For Creating Stem Cells
(Mar. 2, 2009) — Mount Sinai Hospital's Dr. Andras Nagy discovered a new method of creating stem cells that could lead to possible cures for devastating diseases including spinal cord injury, macular degeneration, diabetes and Parkinson's disease. The study, published by Nature, accelerates stem cell technology and provides a road map for new clinical approaches to regenerative medicine.
"We hope that these stem cells will form the basis for treatment for many diseases and conditions that are currently considered incurable," said Dr. Nagy, Senior Investigator at the Samuel Lunenfeld Research Institute of Mount Sinai Hospital, Investigator at the McEwen Centre for Regenerative Medicine, and Canada Research Chair in Stem Cells and Regeneration. "This new method of generating stem cells does not require embryos as starting points and could be used to generate cells from many adult tissues such as a patient's own skin cells."
Dr. Nagy discovered a new method to create pluripotent stem cells (cells that can develop into most other cell types) without disrupting healthy genes. Dr. Nagy's method uses a novel wrapping procedure to deliver specific genes to reprogram cells into stem cells. Previous approaches required the use of viruses to deliver the required genes, a method that carries the risk of damaging the DNA. Dr. Nagy's method does not require viruses, and so overcomes a major hurdle for the future of safe, personalized stem cell therapies in humans.
"This research is a huge step forward on the path to new stem cell-based therapies and indicates that researchers at the Lunenfeld are at the leading edge of regenerative medicine," said Dr. Jim Woodgett, Director of Research for the Samuel Lunenfeld Research Institute of Mount Sinai Hospital. Regenerative medicine refers to enabling the human body to repair, replace, restore and regenerate its own damaged or diseased cells, tissues and organs.
The research was funded by the Canadian Stem Cell Network and the Juvenile Diabetes Research Foundation (United States).
Dr. Nagy joined Mount Sinai Hospital as a Principal investigator in 1994. In 2005, he created Canada's first embryonic stem cell lines from donated embryos no longer required for reproduction by couples undergoing fertility treatment. That research played a pivotal role in Dr. Nagy's current discovery.
One of the critical components reported in Nagy's paper was developed in the laboratory of Dr. Keisuke Kaji from the Medical Research Council (MRC) Centre for Regenerative Medicine at the University of Edinburgh. Dr. Kaji's findings are also published in the March 1, 2009 issue of Nature. The two papers are highly complementary and further extend Nagy's findings.
"I was very excited when I found stem cell-like cells in my culture dishes. Nobody, including me, thought it was really possible," said Dr. Kaji. "It is a step towards the practical use of reprogrammed cells in medicine."
Contact: Nikki Luscombe
luscombe@lunenfeld.ca
416-586-4800 x2046
Samuel Lunenfeld Research Institute
Mount Sinai Hospital researcher makes stem cell breakthrough
(Toronto, ON, February 25, 2009) – In a study to be released on March 1, 2009, Mount Sinai Hospital's Dr. Andras Nagy discovered a new method of creating stem cells that could lead to possible cures for devastating diseases including spinal cord injury, macular degeneration, diabetes and Parkinson's disease. The study, to be published by Nature online, accelerates stem cell technology and provides a road map for new clinical approaches to regenerative medicine.
"We hope that these stem cells will form the basis for treatment for many diseases and conditions that are currently considered incurable," said Dr. Nagy, Senior Investigator at the Samuel Lunenfeld Research Institute of Mount Sinai Hospital, Investigator at the McEwen Centre for Regenerative Medicine, and Canada Research Chair in Stem Cells and Regeneration. "This new method of generating stem cells does not require embryos as starting points and could be used to generate cells from many adult tissues such as a patient's own skin cells."
Dr. Nagy discovered a new method to create pluripotent stem cells (cells that can develop into most other cell types) without disrupting healthy genes. Dr. Nagy's method uses a novel wrapping procedure to deliver specific genes to reprogram cells into stem cells. Previous approaches required the use of viruses to deliver the required genes, a method that carries the risk of damaging the DNA. Dr. Nagy's method does not require viruses, and so overcomes a major hurdle for the future of safe, personalized stem cell therapies in humans.
"This research is a huge step forward on the path to new stem cell-based therapies and indicates that researchers at the Lunenfeld are at the leading edge of regenerative medicine," said Dr. Jim Woodgett, Director of Research for the Samuel Lunenfeld Research Institute of Mount Sinai Hospital. Regenerative medicine refers to enabling the human body to repair, replace, restore and regenerate its own damaged or diseased cells, tissues and organs.
The research was funded by the Canadian Stem Cell Network and the Juvenile Diabetes Research Foundation (United States).
Dr. Nagy joined Mount Sinai Hospital as a Principal investigator in 1994. In 2005, he created Canada's first embryonic stem cell lines from donated embryos no longer required for reproduction by couples undergoing fertility treatment. That research played a pivotal role in Dr. Nagy's current discovery.
One of the critical components reported in Nagy's paper was developed in the laboratory of Dr. Keisuke Kaji from the Medical Research Council (MRC) Centre for Regenerative Medicine at the University of Edinburgh. Dr. Kaji's findings are also published in the March 1, 2009 issue of Nature. The two papers are highly complementary and further extend Nagy's findings.
"I was very excited when I found stem cell-like cells in my culture dishes. Nobody, including me, thought it was really possible," said Dr. Kaji. "It is a step towards the practical use of reprogrammed cells in medicine."
Pluristem Therapeutics Receives FDA Clearance to Begin "First-In-Human" Placenta-Derived Stem Cell Clinical Trial
Clearance of IND Filing Paves Way for US-based Trial That Will Enroll Patients with Critical Limb Ischemia Who Are Facing Amputation
Company to Hold Conference Call at 10:30 am Eastern Time to Discuss Development
http://finance.yahoo.com/news/Pluristem-Therapeutics-bw-14506882.html/print
Researchers find safer way to make stem cells
LONDON (Reuters) – Researchers said on Sunday they had found a safer way to transform ordinary skin cells into powerful stem cells in a move that could eventually remove the need to use human embryos.
It is the first time that scientists have turned skin cells into induced pluripotent stem cells or iPS cells -- which look and act like embryonic stem cells -- without having to use viruses in the process.
The new method also allows for genes that are inserted to trigger cell reprogramming to be removed afterwards.
Stem cells are the body's master cells, producing all the body's tissues and organs.
Embryonic stem cells are the most powerful kind, as they have the potential to give rise to any tissue type. However, many people object to their use, making iPS cells an attractive alternative, provided they can be made safely.
Researchers have known for some time that ordinary skin cells can be transformed into iPS cells using a handful of genes.
But to get these genes into the cells they have had to use viruses, which integrate their own genetic material into the cells they infect. This can cause cancer.
The alternative approach, described in the online edition of the journal Nature by two teams of researchers from Britain and Canada, appears to avoid the risk of such abnormalities.
Obama to lift ban on stem cell research soon: aide
http://news.yahoo.com/s/nm/20090216/hl_nm/us_usa_obama_stemcells;_ylt=AtNyQii4yLiHw2G9S_2Y7VyJhMgF
WASHINGTON (Reuters) – President Barack Obama will soon issue an executive order lifting an eight-year ban embryonic stem cell research imposed by his predecessor, President George W. Bush, a senior adviser said on Sunday.
"We're going to be doing something on that soon, I think. The president is considering that right now," Obama adviser David Axelrod said on "Fox News Sunday."
In 2001, Bush limited federal funding for stem cell research only to human embryonic stem cell lines that already existed. It was a gesture to his conservative Christian supporters who regard embryonic stem cell research as destroying potential life, because the cells must be extracted from human embryos.
Embryonic stem cells are the most basic human cells which can develop into any type of cell in the body.
Scientists believe the research could eventually produce cures for a variety of diseases, including Parkinson's disease, diabetes, heart disease and spinal cord injuries.
Obama vowed to reverse Bush's ban during his presidential campaign and in his inaugural address last month promised to return science to its proper place in the United States.
The U.S. Food and Drug Administration last month cleared the way for the first trial to see if human embryonic stem cells could treat people safely.
The trial will try to use stem cells from already existing lines to regrow nerve tissue in patients with crushed spinal cords.
Stem cells are the body's master cells, giving rise to all the tissues, organs and blood. Embryonic stem cells are considered the most powerful kinds of stem cells, as they have the potential to give rise to any type of tissue.
A Possible Cure for Diabetes Ignored by Big Pharma
http://articles.mercola.com/sites/articles/archive/2009/02/14/A-Possible-Cure-for-Diabetes-Ignored-by-Big-Pharma.aspx
Twelve years ago, Professor Irving Weissman discovered a treatment that could have saved the lives of thousands of women with advanced breast cancer. Pharmaceutical companies weren’t interested in developing the therapy at the time.
Though interest in his methods are finally being ignited, Weissman regrets the wasted time. In a set of lectures, Weissman repeatedly expressed frustration that while many of his discoveries in the field of stem cell research seemed to hold remarkable potential for life-saving treatments, commercial or regulatory hurdles have prevented his scientific findings from benefiting patients.
One example is Weissman’s research on type I diabetes, in which he demonstrated the ability to fully cure type I diabetes in mice using stem cells. But even though his experiments avoided political controversy by using adult stem cells, which do not come from embryos, Weissman ran into a road block when pharmaceutical companies refused to sponsor clinical trials.
Weissman believes that the pharmaceutical companies put profit over principle, preferring to keep diabetes sufferers dependent on costly insulin than to cure them once and for all.
Sources: Columbia Spectator January 23, 2009
Dr. Mercola's Comments:
Type 1 diabetes, or insulin dependent diabetes, is a really sad chronic condition. This is in stark contrast to type 2, which is caused by insulin resistance and faulty leptin signaling, due to inappropriate diet and lack of exercise – an entirely preventable, and nearly 100 percent reversible condition through lifestyle modifications.
Type 1 diabetics, however, do not produce insulin and must therefore inject insulin several times a day. The disease tends to progress rather quickly and therefore needs to be diagnosed early, as it can result in serious long-term complications including blindness, kidney failure, heart disease and stroke.
Tragically those with type 1 diabetes can have the healthiest lifestyle possible yet still suffer many diseases, as current technology is a poor substitute for a fully functioning pancreas. The sad thing about type 1 diabetes is that it now appears nearly completely preventable. New research suggests if a pregnant woman has optimal vitamin D levels during her pregnancy and then maintains them in her baby once delivered, this should radically reduce if not virtually eliminate type 1 diabetes.
Obviously finding out this information after a person has had their pancreatic islet cells destroyed and is insulin dependent doesn’t help much, but the good news is that within your lifetime we will have developed the ability to safely replace these cells, most likely with similar approaches to the one described in the article above.
If this sounds farfetched, you should know that Dr. Weissman is not the only scientist to lay claim to the power of stem cells to cure type 1 diabetes.
In 2007 the British Times Online reported on the first clinical evidence for the efficacy of Hematopoietic stem cells (stem cells derived from bone marrow) in type 1 diabetes.
The Brazilian study, published in Journal of the American Medical Association (JAMA), found that treatment-related toxicity was low, with zero mortality. All but two of the volunteers (93 percent) in the trial did not require daily insulin injections for up to three years after the end of their treatment, as the therapy allowed their bodies to start producing the hormone naturally again.
The main drawback with the particular therapy used in that study, from my standpoint, is that drugs were used to first suppress the patients’ immune system before receiving transfusions of stem cells drawn from their own blood. Suppression of your immune system could lead to all types of health problems – after all, your immune system is your primary line of defense – however, the theory of using stem cells to reactivate natural insulin production is an intriguing and hopeful one.
Does Big Pharma Want to Find a Cure for Diabetes?
It certainly would not make financial sense, and I think it’s fairly safe to say that pharmaceutical companies are in business these days to make money – not to actually cure disease. So, when Dr. Weissman implies that he was unable to entice pharmaceutical sponsors because they prefer to keep diabetes sufferers dependent on costly insulin, he’s probably not far off.
There are numerous examples of well-educated, innovative doctors and scientists who have created alternative medical treatments that far supersede conventional drug treatments, and yet they are more frequently than not shunned, persecuted, or even prosecuted for their efforts.
At least Dr. Weissman did not suffer a fate similar to any of these doctors:
Gaston Naessens – Dr. Naessens cancer treatment is based on the theory that cancer is caused by a friendly microorganism called somatids ("little bodies") -- which are present in all cells -- that becomes unfriendly. His formula, 714X, provides nitrogen to the cancer cells, thus causing this microorganism to cease excreting their toxic compounds, and mobilize your immune system to kill the cancer cells. He was subsequently put on trial for his cancer discoveries.
Stanislaw Burzynski -- Dr. Burzynski, founder of the Houston-based Burzynski Institute, treats cancer patients with substances called antineoplastons. He was indicted by a grand jury in 1995 for his use of antineoplastons– his second trial that year. He was acquitted.
Ryke Geerd Hamer – Dr. Hamer’s “German New Medicine” (GNM), operates under the premise that every disease originates from an unexpected shock experience, and that all disease can be cured by resolving these underlying emotional traumas. Despite a 95 percent success rate, Dr. Hamer has spent time in prison for refusing to disavow his medical findings and stop treating his patients with his unorthodox techniques, and is currently living in exile, seeking asylum from persecution.
Adult Stem Cell Treatments are the Wave of the Future
I believe therapies using adult stem cells – as opposed to embryonic stem cells which are at the heart of the stem cell controversy – is and will continue to be a major, exciting part of the future of medicine.
There’s little risk of rejection when using your own adult stem cells, so you’re less likely to need dangerous immunosuppressive drugs. And besides certain ethical issues and regulatory barriers to embryonic stem cell therapy, adult stem cells may hold several technical advantages. For one, they may be more viable.
It’s a little known fact that only about 30 percent of conceptions advance to term. In other words, about 70 percent of embryos may be so defective that they naturally abort.
In addition to eventually helping restore internal organs, immune systems and more, adult stem cell therapies also hold the promise of restoring old skin and bald scalps. I am personally beta testing a new product for regrowing hair that has adult stem cell factors (primarily peptides, no DNA) and have been favorably impressed with the results to date.
One major player in the field is The Maximum Life Foundation. They have discovered an emerging technology that could fine tune stem cell treatments and make every adult stem cell therapy in the world much more safe and effective.
As you age, your stem cells diminish in quality and quantity, so just when you require strong stem cells the most, you’re becoming deficient. Hence your organs and tissues eventually wear out and need to be restored or replaced.
Cells become randomly damaged or mutated over time, but a small percentage may incur relatively little damage—or may escape damage altogether. So some of your stem cells may be as pristine as those that you enjoyed as a teenager, or even as an infant.
The technology discovered by The Maximum Life Foundation identifies, isolates, and amplifies those “pristine” stem cells from your own population for therapies. This selection process could supercharge stem cell treatments by using these “best of breed” cells instead of old cells.
Stem Cell Treatments Have Been Proven to Work
As a bridge to human therapies, a San Diego company has
already given us a peek into the future with their horse and dog treatments. Vet-Stem provides a quick-turnaround
laboratory service that lets veterinarians to use regenerative cells in animals.
The veterinarian simply collects a small fat sample from the patient and ships it overnight to the Vet-Stem laboratory. Then, Vet-Stem processes the sample, concentrates the cells which are shipped in ready-to-inject syringes. Finally, the veterinarian injects the cells directly into the injured site.
With more than 3,000 horses treated and multiple studies demonstrating the success and safety of their regenerative medicine, Vet-Stem currently helps horses and dogs with fractures, joint disease, or tendon or ligament injuries return to their full level of ability.
Just imagine what the prospects for aged humans might be if similar treatments used pristine human stem cells. Type 1 diabetes wouldn’t be the only chronic disease that could be effectively wiped out.
However, despite Dr. Weissman’s negative experiences in trying to bring his findings to the fore, it’s not completely out of the question yet. In an accompanying editorial in JAMA titled “Cellular therapy for type 1 diabetes: has the time come?” Dr Jay Skyler of the Diabetes Research Institute at the University of Miami, wrote:
“Research in this field is likely to explode in the next few years and should include randomized controlled trials, as well as mechanistic studies."
Stem Cells From Skin Cells Can Make Beating Heart Muscle Cells
http://www.sciencedaily.com/releases/2009/02/090212161808.htm
A little more than a year after University of Wisconsin-Madison scientists showed they could turn skin cells back into stem cells, they have pulsating proof that these "induced" stem cells can indeed form the specialized cells that make up heart muscle.
In a study published online Feb. 12 in Circulation Research, UW-Madison School of Medicine and Public Health professor of medicine Tim Kamp and his research team showed that they were able to grow working heart-muscle cells (cardiomyocytes) from induced pluripotent stem cells, known as iPS cells.
The heart cells were originally reprogrammed from human skin cells by James Thomson and Junying Yu, two of Kamp's co-authors on the study.
"It's an encouraging result because it shows that those cells will be useful for research and may someday be useful in therapy,'' said Kamp, who is also a cardiologist with UW Health. "If you have a heart failure patient who is in dire straits — and there are never enough donor hearts for transplantation — we may be able to make heart cells from the patient's skin cells, and use them to repair heart muscle. That's pretty exciting."
It's also a few more discoveries away. The researchers used a virus to insert four transcription factors into the genes of the skin cell, reprogramming it back to an embryo-like state. Because the virus is taken up by the new cell, there is a possibility it eventually could cause cancer, so therapies from reprogrammed skin cells will likely have to wait until new methods are perfected.
Still, the iPS cardiomyocytes should prove immediately useful for research. And Kamp said the speed at which knowledge is progressing is very encouraging.
Jianhua Zhang, lead author on the study, noted that it took 17 years, from when a mouse embryonic stem cells were first created in 1981, to 1998, when Thomson created the first human embryonic stem cells. In contrast, the first mouse iPS stem cells were created in 2006, and Thomson and Yu published their paper in November 2007, announcing the creation of human iPS stem cells that began as a skin cells.
While research on embryonic stem cells is controversial, because it destroys a human embryo, lessons learned through such research apply to current work with iPS cells made from adult cells.
"That's one of the important things that have come out of the research with embryonic stem cells, it taught us how human pluripotent stem cells behave and how to work with them,'' Kamp says. "Things are able to progress much more quickly thanks to all the research already done with embryonic stem cells."
Many types of heart disease have known genetic causes, so creating cardiomyocytes grown from patients who have those diseases will likely be some of the next steps in the research. One of Kamp's colleagues, Clive Svendsen, a UW-Madison School of Medicine and Public Health professor of neurology and anatomy, has grown the iPS cells into disease-specific neural cells. Kamp and Svendsen are also on the faculty of the Waisman Center and the Stem Cell and Regenerative Medicine Center.
Kamp's latest research, proving that iPS cells can become functional heart cells, is just one step along the way to better understanding and treatment of disease.
"We're excited about it, because it's the some of the first research to show it can be done, but in the future, we'll probably say, 'Well, of course it can be done,'" he says. "But you don't know until you do it. It's a very mysterious and complicated dance to get these cells to go from skin cells to stem cells to heart cells."
Stem cells: Modelling genetic diseases with iPS cells
By Monica Hoyos Flight
http://www.nature.com/nrd/journal/v8/n2/full/nrd2809.html
Pluripotent stem cells induced from skin fibroblasts have recently attracted considerable attention as an alternative to embryonic stem cells for potential applications in disease modelling, drug screening and regenerative medicine. Svendsen and colleagues now describe how induced pluripotent stem (iPS) cells from a child with spinal muscular atrophy (SMA) can be differentiated into motor neurons that maintain the disease genotype and phenotype, providing a pioneering demonstration of the potential of human iPS cells for modelling a genetic disorder and for drug screening.
SMA is an autosomal recessive genetic disorder that is caused by mutations in the survival motor neuron (SMN) genes, which lead to motor neuron degeneration and progressive paralysis. The authors generated iPS cells by infecting fibroblasts from the patient with SMA and his unaffected mother with viral vectors encoding the transcription factors OCT4 (also known as POU5F1), SOX2, NANOG and LIN28, which have previously been shown to reprogramme human somatic cells to a pluripotent state. Both cells from the patient (iPS-SMA cells) and cells from the unaffected control (iPS-WT cells) were able to generate teratomas harbouring tissue from the three primary embryonic germ layers (ectoderm, mesoderm and endoderm), but the iPS-SMA cells showed markedly lower levels of SMN1 than the iPS-WT cells.
Given that the lack of SMN proteins specifically affects the viability of motor neurons, the authors differentiated these iPS cells into motor neurons using various growth factors, including retinoic acid and sonic hedgehog. Differentiation was confirmed by checking for the expression of motor neuron transcription factors (HOXB4, OLIG2, ISL1 and HB9 (also known as MNX1)) and markers of mature motor neurons (SMI-32 and choline acetyltransferase). Although similar numbers of motor neurons were initially generated from the WT and SMA iPS cultures, after 6 weeks of differentiation there was a decrease in the size and number of the motor neurons differentiated from the iPS-SMA cultures. Furthermore, after 8 weeks in culture the iPS-SMA-derived cells did not exhibit punctate synapsin staining, suggesting that presynaptic maturation was impaired.
Finally, the authors examined whether compounds that are known to increase the levels of SMN proteins, namely valproic acid and tobramycin, were able to induce the formation of SMN gems (naturally forming aggregates of SMN proteins) in the cytoplasm and nucleus of the SMA-iPS-derived motor neurons. They found a significant increase in gems in the SMN-deficient cells after 2 days of drug treatment, confirming that these cells could be useful for drug screening.
Until now, studies of SMA have relied on animal models of the disease, which involve knockdown or overexpression strategies, or SMA patient fibroblasts, which do not show the same vulnerability as motor neurons. This paper validates the use of human iPS cells to model a genetically inherited neurological disease, highlighting its immediate potential for studying the mechanisms of SMA and drug screening in a more relevant setting.
References and links
1. Ebert, A. D. et al. Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature 21 Dec 2008 (doi: 10.1038/nature07677)
UC San Diego Engineers Develop Novel Method for Accelerated Bone Growth
http://www.jacobsschool.ucsd.edu/news/news_releases/release.sfe?id=813
San Diego, CA, January 29, 2009 --Engineers at the University of California at San Diego have come up with a way to help accelerate bone growth through the use of nanotubes and stem cells. This new finding could lead to quicker and better recovery, for example, for patients who undergo orthopedic surgery.
In recent years, stem cells have become a hot topic of investigation with studies suggesting revolutionary medical benefits due to their ability to be converted into selected types of newly generated cells. During their research, the group of UC San Diego bioengineers and material science experts used a nano-bio technology method of placing mesenchymal stem cells on top of very thin titanium oxide nanotubes in order to control the conversion paths, called differentiation, into osteoblasts or bone building cells. Mesenchymal stem cells, which are different from embryonic stem cells, can be extracted and directly supplied from a patient’s own bone marrow.
The researchers described their lab findings in a paper published this week in the Proceedings of the National Academy of Sciences (PNAS), “Stem Cell Fate Dictated Solely by Altered Nanotube Dimension.”
“If you break your knee or leg from skiing, for example, an orthopedic surgeon will implant a titanium rod, and you will be on crutches for about three months,” said Sungho Jin, co-author of the PNAS paper and a materials science professor at the Jacobs School of Engineering. “But what we anticipate through our research is that if the surgeon uses titanium oxide nanotubes with stem cells, the bone healing could be accelerated and a patient may be able to walk in one month instead of being on crunches for three months.
“Our in-vitro and in-vivo data indicate that such advantages can occur by using the titanium oxide nanotube treated implants, which can reduce the loosening of bones, one of the major orthopedic problems that necessitate re-surgery operations for hip and other implants for patients,” Jin added. “Such a major re-surgery, especially for older people, is a health risk and significant inconvenience, and is also undesirable from the cost point of view.”
This is the first study of its kind using stem cells attached to titanium oxide nanotube implants. Jin and his research team – which include Jacobs School bioengineering professors Shu Chien and Adam Engler, as well as post doctoral researcher Seunghan Oh and other graduate students and researchers –report that the precise change in nanotube diameter can be controlled to induce selective differentiation of stem cells into osteoblast (bone-forming) cells. Karla Brammer, a Jacobs School materials science graduate student, will also present these findings in a poster session during the Jacobs School of Engineering's Research Expo on February 19.
According to this breakthrough research, nanotubes with a larger diameter cause cells growing on their surface to elongate much more than those with a small diameter. The larger diameter nanotube promotes quicker and stronger bone growth. “The use of nano topography to induce preferred differentiation was reported in recent years by other groups, but such studies were done mostly on polymer surfaces, which are not desirable orthopedic implant materials,” Jin said.
It is common for physicians and surgeons to use chemicals for stem cell implants in order to control cell differentiation, a conversion into a certain desired type of cells, for example, to neural cells, heart cells, and bone cells. However, introducing chemicals into the human body can sometimes have undesirable side effects. “What we have accomplished here is a way to introduce desirable guided differentiation using only nanostructures instead of resorting to chemicals,” said Seunghan (Brian) Oh, who is the lead author of the PNAS article.
The next step for engineers will be to work with orthopedic surgeons and other colleagues at the UC San Diego School of Medicine to study ways to translate this breakthrough research to clinical application, said Shu Chien, a UC San Diego bioengineering professor and director of the university’s new Institute of Engineering in Medicine (IEM). Chien said this effort will be fostered by the IEM, whose goal is to bring together scientists, engineers and medical experts to come up with novel approaches to medicine.
“Our research in this area has pointed to a novel way by which we can modulate the stem cell differentiation, which is very important in regenerative medicine,” Chien said. “This will lead to a truly interdisciplinary approach between engineering and medicine to getting novel treatments to the clinic to benefit the patients.”
Stem cells ready for prime time
http://www.nature.com/news/2009/090128/full/457516a.html
US regulatory agency gives the go-ahead for first clinical trials of a human embryonic stem-cell treatment.
By Meredith Wadman
It was a triumph of science, not politics. Yet the approval for the world's first clinical trial of a therapy generated by human embryonic stem cells, announced on 23 January, certainly seemed to be deeply political.
It was just three days into US President Barack Obama's term of office when Geron, based in Menlo Park, California, told the world that the US Food and Drug Administration (FDA) had agreed to its phase I safety study of a stem-cell-derived therapy for spinal-cord injury.
But supporters say that the significance of the approval lies not in the ideology of the new administration, but in the considerable scientific hurdles that were overcome in reaching this milestone.
"There was a lot of scepticism as to whether we could reliably reproduce these manufactured products at levels of purity and identity sufficient to even allow the FDA to allow a phase I clinical trial," says Michael West, who founded Geron in 1990 and is now chief executive of BioTime in Alameda, California.
Geron, he says, "has convinced the FDA that those cells could be manufactured reliably enough for at least the first clinical trials. That is a milestone. A lot of the critics said it would be 30–50 years before we got there."
Financial benefit
The announcement boosted the price of shares in the company, which has an extensive patent portfolio relating to embryonic stem-cell research. As of 26 January, they were trading at US$8.15 a share — up 56% from the day before the announcement.
In the trial, up to ten individuals who have been left paralysed after spinal-cord injury will be injected at the point of injury with stem-cell-derived precursors of oligodendrocytes, which are key supportive cells in the central nervous system. The treatments will start within 7–14 days of their injury. The company hopes that the cells will lay down sheaths of myelin — an insulator essential for conducting nerve impulses — around injured neurons, as well as stimulating nerve cells to regenerate. The cells have demonstrated both capabilities in animals (H. Keirstead et al. J. Neurosci. 25, 4694–4705; 2005).
Geron says that it expects to begin enrolment early this summer at up to seven US medical centres. In a conference call with analysts and reporters, the firm's president and chief executive Thomas Okarma said that the trial "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's 22,000-page FDA application was first submitted in March 2008, at a time when President George W. Bush had placed tight restrictions on federal funding for embryonic stem-cell research. Although Obama has promised to reverse those restrictions, the company and the FDA deny that any politics were at play in the timing of the announcement.
"Science drives our decision-making," says Karen Riley, an FDA spokeswoman. "Political considerations have no role in this process." Riley adds that the process was prolonged by the time it took Geron to respond to additional questions the FDA asked the company last spring after receiving the application. Okarma says that the application included data from more than 24 studies, involving nearly 2,000 animals with injured spinal cords and requiring the production and injection of more than 5 billion cells.
Indeed, the Geron cells come from one of a score of lines approved for federal funding under the Bush policy.
Groups opposing such research say that the trial's risks include the growth of tumours. "The ethical concerns include both using human embryos for the experiment as the source material, but also concern for the patient," says David Prentice, senior fellow for life sciences at the Family Research Council, a Christian advocacy group in Washington DC. "This is not a life-threatening condition," he says. "Are you actually going to be shortening the patient's life?" He cites a 2006 study in which some neural support cells — derived from human embryonic stem cells — reverted to undifferentiated growth when injected in rat models of Parkinson's disease (N. S. Roy. et al. Nature Med. 12, 1259–1268; 2006).
Not perfect, but timely
Supporters of stem-cell research have praised the FDA approval, saying that there is no 'perfect' trial with which to begin. "What I care about, what investors care about, what people who are debilitated care about, is something that can happen in the near future — not a perfect product two generations from now," says Steve Brozak, president of investment company WBB Securities in Westfield, New Jersey. Brozak has tracked stem-cell research since James Thomson, a researcher at the University of Wisconsin in Madison, published his work on the first successful isolation of human embryonic stem-cell lines in 1998.
Other advocates say the trial should not become a test case on which the fortunes of the entire field rise or fall. "This is a trial of one particular application, not a trial of all embryonic stem cells," says Sean Tipton, immediate past-president of the Coalition for the Advancement of Medical Research in Washington DC.
Geron is a big fish in what is currently a very small pond. The other high-profile company in the field, Advanced Cell Technology in Los Angeles, California, was recently on the brink of bankruptcy. And Novocell in San Diego, is working to develop human embryonic stem cells into pancreatic β-cells, which produce insulin. Although diabetes provides a huge treatment target, Novocell's project is daunting because of the technical challenges involved in producing β-cells that safely mediate blood-sugar levels.
Stem-cell trial spotlights sector's prospects
Published: 1/24/2009 11:08 PM
http://www.dailyherald.com/story/?id=266918
NEW YORK -- Geron Corp. will enter uncharted territory when it begins the first federally approved human studies on an embryonic stem cell therapy, marking what some consider a major milestone in a field that's still a long way from commercialization.
The Menlo Park, Calif.-based company plans begin testing a treatment using embryonic stem cells that could fix major spinal cord injuries in people.
The market reacted swiftly and positively to clearance for the trials, pushing shares up $1.88, or 36 percent, to close at $7.09. The stock hit $8.38 earlier in the session, its highest point nearly two years.
The initial study is small and will mostly focus on safety, but the development process is expected to set a tone for how long such work will take, how the Food and Drug Administration will judge it -- and how much any drugs might cost.
"If Geron is successful, this will be the single most important health care advance we've seen," said Steve Brozak of WBB Securities.
The market potential of Geron's treatment is unclear, but it's targeting the right ailment from a commercial standpoint, Brozak said. "Geron is absolutely brilliant in that they picked an indication that has had no meaningful advances in the last generation."
Embryonic stem cells are unspecialized cells capable of turning into a wide variety of other cells. They are collected by cloning embryos in a laboratory, but the embryo is destroyed in the process. They were a hot-button issue throughout George W. Bush's presidency, with a ban on public research funding. That ban does not affect Geron, which does not rely on government financing.
Another publicly traded company developing embryonic stem-cell treatments is Alameda, Calif.-based Advanced Cell Technology Inc. But that company, like many others in the field, also focuses on less-controversial adult stem cells, which are gathered from a person's skin, for example. Its stock is traded over-the-counter.
Palo Alto, Calif.-based StemCells Inc. also focuses on adult stem cells and is already in early-stage clinical trials for a genetic disorder. Geron's news appeared to drive StemCells shares higher, up 38 cents, or 18 percent, to $2.53. The stock reached $2.89 earlier in the session, its highest point in nearly two years.
Rockville, Md.-based Neuralstem has been working for more than a decade on stem-cell therapies and expects to begin human studies using adult stem cells in 2009. Shares rose 5 cents, or 3.5 percent, to $1.48.
Others in the field include Columbia, Md.-based Osiris Therapeutics Inc. and San Diego-based Cytori Therapeutics Inc.
Large pharmaceutical companies also have been targeting stem cells. In October, Pfizer said it would form a Regenerative Medicine unit that would collaborate with researchers at drug developers and universities worldwide. GlaxoSmithKline PLC is in the beginning of a five-year collaborative deal with the Harvard Stem Cell Institute.
There are few privately held companies developing stem cells. South Africa-based Advanced Cell Therapeutics is among those. It develops therapies based on stem cells gained from umbilical cord blood.
Stem cells obtained from embryos, however, are considered potentially the most effective because they can turn into any cell in the body.
The FDA denied Geron's plan to conduct human studies in May and did not provide a reason. The clearance now comes three days after President Barack Obama took office. Geron Chief Executive Dr. Thomas Ikarma said Thursday that Obama's ascent to the White House had nothing to do with the FDA's turnaround.
Obama has previously said he planned to relax federal restrictions on public research funding.
Geron's headstart in development on its competitors further strengthens an already powerful intellectual property position. The company is considered the world's leading embryonic stem cell developer because of its exclusive rights on several cell types. The company helped finance researchers at the University of Wisconsin who first isolated human embryonic stem cells in 1998.
Though Geron is the most advanced, Brozak said investors excited by the attention Geron has received would have to keep a long-term outlook on the company. Analysts also typically say that investing in early-stage biotech companies can be risky because of the substantial chance the drug won't make it to market.
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First Embryonic Stem-Cell Trial Gets Approval From the FDA
http://online.wsj.com/article/SB123268485825709415.html?mod=yahoo_hs&ru=yahoo
By RON WINSLOW and ALICIA MUNDY
In a watershed moment for one of the most contentious areas of science and American politics, the U.S. Food and Drug Administration cleared the way for the first-ever human trial of a medical treatment derived from embryonic stem cells.
Geron Corp., a Menlo Park, Calif., biotechnology company, is expected to announce Friday that it received a green light from the agency to mount a study of its stem-cell treatment for spinal cord injuries in up to 10 patients. The announcement caps more than a decade of advances in the company's labs and comes on the cusp of a widely expected shift in U.S. policy toward support of embryonic stem-cell research after years of official opposition.
"This is the dawn of a new era in medical therapeutics," said Thomas B. Okarma, Geron's president and chief executive officer. The hope that stem-cell therapy will repair and regenerate diseased organs and tissue "goes beyond what pills and scalpels can ever do."
[stem cells]
Limits on stem-cell research, which prevented federal funding and were imposed by Congress and former President George W. Bush for ethical and religious reasons, have had a chilling effect on both academic and corporate research involving such cells. Proponents of stem-cell research say restrictions have delayed development of promising new treatments, while critics contend that harvesting stem cells from embryos destroys human life.
President Barack Obama said during his campaign that overturning research limits would be a top priority in his administration.
Both Geron and the FDA said the timing of the decision to approve the study was coincidental. "The FDA looks to the science on these types of issues, and we approve [such applications] based on a showing of safety," said Karen Riley, an FDA spokeswoman. "Political considerations have no role in this process."
Approval of the study is far from a guarantee that stem-cell treatments will work or make it to the market, but it is likely to be seen as an indication that opportunities for stem-cell research are poised to open and will fuel enthusiasm among academic and corporate researchers.
Mr. Obama's plans for acting on the current research restrictions haven't been finalized. Shortly after the election, Obama advisers thrilled biotech companies and investors when they suggested that the new president could use his executive authority to undo the Bush administration ban. But in a Jan. 18 interview on CNN, Mr. Obama said he might let Congress take the lead. "I like the idea of the American people's representatives expressing their views on an issue like this," he said.
Regulating stem-cell therapy is new turf for both industry and the FDA, a major reason why it took the agency nearly a year to review Geron's 21,000-page application for the trial, which it filed last March. Approval came in a phone call Wednesday afternoon, Dr. Okarma said.
The study will focus on the safety of the treatment. At an FDA hearing in April, several firms' executives and researchers complained that they were at a loss about what the FDA wanted in terms of clinical trials involving stem cells because the FDA itself wasn't sure.
Embryonic stem cells are the building-block cells that help drive prenatal development. Geron has developed banks of embryonic stem cells and found a way to coax them into differentiating as they do in nature into progenitors of specific cells that make spinal-cord tissue, heart muscle, cartilage and other organs and tissues.
Spinal-cord injury is one of medicine's most debilitating conditions, typically causing paralysis and other issues for which there are few, if any, effective treatments. The Geron study will enroll paralyzed patients who can be treated within 14 days of their injury. Patients will be evaluated for at least one year, after which, if the treatment proves safe, the company hopes to increase the dose and expand the potential candidates for the therapy.
In addition to safety, researchers will look for signs that the treatment is effective.
Neurologix Commences its Phase 2 Clinical Trial of Novel Gene Transfer Approach for Treatment of Parkinsons Disease
Wednesday January 21, 8:05 am ET
FORT LEE, N.J.--(BUSINESS WIRE)--Neurologix, Inc. (OTCBB:NRGX - News), a biotechnology company engaged in the development of innovative gene therapies for the brain and central nervous system, announced today that it has initiated its Phase 2 clinical trial for the treatment of advanced Parkinson’s disease. The first trial participants have undergone surgery at multiple institutions and additional subjects are currently being enrolled.
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The purpose of the trial is to validate the safety and efficacy of Neurologix’s gene transfer therapy, a novel non-dopaminergic approach to restore motor function in Parkinson’s patients who are sub-optimally responsive to available drug therapy. Neurologix’s approach is to reestablish the production of GABA (gamma-aminobutyric acid), the major brain inhibitory neurotransmitter that helps “quiet” excessive neuronal firing and has been determined to be deficient in patients in the advanced stages of Parkinson’s disease.
John E. Mordock, President and Chief Executive Officer of Neurologix, stated, “Initiating this Phase 2 clinical trial represents a significant milestone. We expect to enroll 40 subjects across six to eight leading U.S. academic research centers, with completion of enrollment expected during the second half of 2009.”
In Parkinson’s disease there is degeneration of many cells in the central nervous system including those that produce dopamine, which leads to a downstream deficiency in GABA signaling in areas of the brain that regulate movement. Most current therapies and research approaches target dopamine. Mr. Mordock commented, “In contrast, our preclinical and clinical research suggests that directly targeting GABA production rather than dopamine replacement may be a more effective way of improving brain function in late-stage Parkinson’s disease while also avoiding the known therapeutic limitations and complications associated with the over-production of dopamine.”
The Co-Chairmen of the trial Steering Committee are Dr. Andrew Feigin, Director of the Neuroscience Experimental Therapeutics Research Program at the Feinstein Institute of Medical Research of the North Shore-Long Island Jewish Health System, and Dr. Peter LeWitt, a neurologist who directs the Parkinson’s Disease and Movement Disorders Program at Henry Ford Hospital in Southfield, Michigan.
“Based on the encouraging functional and imaging results seen in the Phase 1 study of this innovative approach to improving Parkinson’s disease, we are extremely excited to be part of this study,” said Dr. Feigin.
Dr. LeWitt added, “The start of this clinical trial provides hope to the patient population which has had a longstanding need for new treatment options.”
The scientific underpinnings of Neurologix’s approach have undergone rigorous peer review resulting in highly cited articles in Nature Genetics and Science by the company’s co-founders, Drs. Matthew During and Michael Kaplitt. Moreover, the current trial follows a successful Phase 1 study, as published in the Lancet and Proceedings of the National Academy of Sciences, USA, in which 12 subjects completed the study showing no related serious adverse events and significant functional benefit with supportive imaging data.
Phase 2 Clinical Trial Design
As previously announced, 20 participants will receive an infusion of the gene-based treatment bilaterally via a catheter temporarily placed by stereotactic surgery in each participant’s subthalamic nucleus (STN), a deep brain structure that is the main target of surgery to treat Parkinson’s disease. The other 20 participants will receive sterile saline solution into a partial thickness burr hole made into the skull, with no brain infusion. Trial participants will be assessed for treatment effects by standardized Parkinson’s disease ratings at multiple time points post-procedure. The primary endpoint for the trial will be a clinical assessment of motor function at 6 months using the Unified Parkinson’s Disease Rating Scale (UPDRS). All participants in the trial will also be monitored for safety for 12 months following the gene transfer procedure. If the primary endpoint is met following the analysis of 6 month data, then the sham-control participants will be offered the opportunity to crossover into an open label study of the Neurologix gene transfer therapy if they continue to meet all entry, medical and surgical criteria.
For details about participating in the clinical trial, please visit the following link: http://clinicaltrials.gov/ct2/show/NCT00643890?term=neurologix&rank=1. Details about trial participation are also available at http://www.pdtrials.org/en/browse/all/view/241.
About the Neurologix Gene Transfer Approach to Parkinson’s Disease
In Parkinson’s disease, patients lose dopamine-producing brain cells, resulting in substantial reductions in the activity and amount of GABA (gamma-aminobutyric acid). This reduction in GABA causes a dysfunction in brain circuitry responsible for coordinating movement. GABA is made by a gene called glutamic acid decarboxylase, or GAD.
Neurologix’s gene transfer approach to Parkinson’s disease seeks to restore GABA -- and thus improve the patient’s motor control -- by inserting the GAD gene back into an area of the brain called the subthalamic nucleus, a key regulatory center for movement.
About Neurologix
Neurologix, Inc. (NRGX.OB) is a clinical-stage biotechnology company dedicated to the discovery, development, and commercialization of life-altering gene transfer therapies for serious disorders of the brain and central nervous system (CNS). Neurologix’s therapeutic approach is built upon the groundbreaking research of its scientific founders and advisors, whose accomplishments have formed the foundation of gene therapy for neurological illnesses. Current programs of the company address such conditions as Parkinson’s disease, epilepsy and Huntington’s chorea, all of which are large markets not adequately served by current therapeutic options. For more information, please visit the Neurologix website at http://www.neurologix.net.
Cautionary Statement Regarding Forward-looking Statements
This news release includes certain statements of the Company that may constitute “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and which are made pursuant to the Private Securities Litigation Reform Act of 1995. These forward-looking statements and other information relating to the Company are based upon the beliefs of management and assumptions made by and information currently available to the Company. Forward-looking statements include statements concerning plans, objectives, goals, strategies, future events, or performance, as well as underlying assumptions and statements that are other than statements of historical fact. When used in this document, the words “expects,” “promises,” “anticipates,” “estimates,” “plans,” “intends,” “projects,” “predicts,” “believes,” “may” or “should,” and similar expressions, are intended to identify forward-looking statements. These statements reflect the current view of the Company’s management with respect to future events. Many factors could cause the actual results, performance or achievements of the Company to be materially different from any future results, performance or achievements that may be expressed or implied by such forward-looking statements, including, but not limited to, the following:
The Company is still in the development stage and has not generated any revenues. From inception through September 30, 2008, it incurred net losses and negative cash flows from operating activities of approximately $32.9 million and $26.5 million, respectively. Management believes that the Company will continue to incur net losses and cash flow deficiencies from operating activities for the foreseeable future. Because it may take years to develop, test and obtain regulatory approval for a gene-based therapy product before it can be sold, the Company likely will continue to incur significant losses for the foreseeable future. Accordingly, the Company may never be profitable and, if it does become profitable, it may be unable to sustain profitability.
At September 30, 2008, the Company had cash and cash equivalents of approximately $20.1 million, which management believes will be sufficient to fund the Company’s operations through at least March 31, 2010. The Company does not know whether additional financing will be available when needed, or if available, will be on acceptable or favorable terms to it or its stockholders.
The Company will need to conduct future clinical trials for treatment of Parkinson’s disease using the Company’s NLX technology. If the trials prove unsuccessful, future operations and the potential for profitability will be materially adversely affected and the business may not succeed.
There is no assurance as to when, or if, the Company will be able to successfully receive approval from the Food and Drug Administration (FDA) on its Investigational New Drug Application to commence a Phase 1 clinical trial for the treatment of epilepsy
There is no assurance as to when, of if, the Company will be able to successfully complete the required preclinical testing of its gene therapy for the treatment of Huntington’s disease to enable it to file an Investigational New Drug Application with the FDA for permission to begin a Phase 1 clinical trial or that, if filed, such permission will be granted.
Other factors and assumptions not identified above could also cause the actual results to differ materially from those set forth in the forward-looking statements. Additional information regarding factors that could cause results to differ materially from management’s expectations is found in the section entitled “Risk Factors” in the Company’s 2007 Annual Report on Form 10-KSB. Although the Company believes these assumptions are reasonable, no assurance can be given that they will prove correct. Accordingly, you should not rely upon forward-looking statements as a prediction of actual results. Further, the Company undertakes no obligation to update forward-looking statements after the date they are made or to conform the statements to actual results or changes in the Company’s expectations.
Contact:
Neurologix, Inc.
Marc Panoff, 201-592-6451
Chief Financial Officer, Treasurer and Secretary
marcpanoff@neurologix.net
or
Kureczka/Martin Associates
Joan Kureczka, 415-821-2413
Jkureczka@comcast.net
--------------------------------------------------------------------------------
Source: Neurologix, Inc.
Green light for UK stem-cell trial
Stroke patients to be treated with tailor-made brain cells.
http://www.nature.com/news/2009/090119/full/news.2009.41.html
Helen Pilcher
Stem cells will be grafted into the brains of patients during the new trial.
ALAMYUK researchers have been given the go-ahead for a clinical trial to assess the use of stem-cell transplants for stroke. Twelve people will take part in the preliminary safety study, the first time that brain-derived stem cells have been used to treat stroke patients.
The trial, due to start later this year, will see different doses of cultured human neural stem cells grafted into the brains of patients who have had a stroke — often caused by a blood clot blocking one of the vessels leading to the brain. The study will assess how safe the procedure is, but will also monitor changes in mobility and brain function.
"I'm cautious but hopeful," says neurologist Keith Muir of the University of Glasgow, UK, who will coordinate the trial. "We've tried a lot of things in stroke over the years, most of which haven't been helpful."
Stroke is the leading cause of death and the single biggest cause of adult disability in the developed world. Around half of all stroke survivors are left with permanent disabilities. Although clot-busting treatments can help restore blood flow if given soon after the stroke, most treatment focuses on rehabilitation such as physiotherapy.
So researchers are searching for other therapies. Stem-cell transplants are an attractive option because the primitive cells have the potential to form many other cell types, and may be able to repair or replace damaged brain cells.
Human stem cells have been grafted into the brains of stroke patients before, but the results were inconclusive. Because those cells came from an embryonic tumour, scientists were concerned that they might become cancerous in the patients' brains.
Chemical switch
The cells in this study, developed by UK biotechnology firm ReNeuron, have been tailor-made for transplantation. Originally isolated from a human fetus, the cells have been modified by the addition of a gene that promotes cell growth. This helps them to divide in culture in the lab so they can be grown up into the vast numbers required for the trial.
Crucially, the gene's activity can be switched on and off by adding or removing a chemical in the culture dish. When the chemical is removed, the cells stop dividing and are ready for transplantation without the risk of tumour formation. The UK Medicines and Healthcare products Regulatory Agency has given the green light for the use of these cells in the study.
"It's exciting to see things that we've worked on for many years coming to trial," says Steve Dunnett from Cardiff University, UK, who studies cell transplantation.
ReNeuron researchers have already tested the therapy in stroke-damaged rat brains, in which the cells prompted new blood vessels and neurons to form. The animals also regained control of movement in their front paws.
The cells will be transplanted into patients around six months after their stroke, by which time any remaining brain damage is thought to be permanent. "But it's hard to imagine that once you've got an area of dead tissue, transplanting cells into it will make much difference," says Roger Barker from the University of Cambridge, UK, who studies transplantation therapies for neurodegenerative diseases. It may be that the cells will work better if transplanted earlier, he adds.
ReNeuron gains UK regulatory approval to start ground-breaking clinical trial with stem cell therapy for stroke . 19/01/09
Guildford, UK, 19 January 2009: ReNeuron Group plc (LSE: RENE.L) today announces that it has received approval from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) to commence a first-in-man clinical trial for the treatment of patients who have been left disabled by an ischaemic stroke, the most common form of the condition. Stroke is the third largest cause of death and the single largest cause of adult disability in the developed world.
In this ground-breaking Phase I trial, the first of its kind using expanded neural stem cells, stroke patients will be treated with ReNeuron's ReN001 stem cell therapy at the Institute of Neurological Sciences, Southern General Hospital, Greater Glasgow and Clyde NHS Board. The Principal Investigator for the trial is Dr. Keith Muir, Senior Lecturer in Neurology at the University of Glasgow. MHRA approval for the trial has been given subject to provision of data both from an ongoing pre-clinical study and from the long term follow-up of trial participants. Following successful completion of the ethics approval process which is currently underway for the trial, patient recruitment is expected to commence in the second quarter of this year.
The trial is designed primarily to test the safety profile of ReN001 in ischaemic stroke patients at a range of cell doses, but a number of efficacy measures will also be evaluated over the course of the trial. The ReN001 cells will be administered by direct injection into the affected region of the brain in a straightforward surgical procedure. Importantly, the nature of the procedure and the characteristics of the ReN001 cells mean that the patients will not require immunosuppression following treatment, thus eliminating the safety risks typically associated with immunosuppression regimens. Patients in the trial will be monitored for one year, with longer term follow-up procedures in place thereafter.
Dr. Keith Muir said:
"Stem cell treatment offers the potential to repair brain tissue lost as a result of stroke. We are very excited at the opportunity to undertake this, the first clinical trial involving neural stem cell therapy in stroke. At this stage, we are primarily seeking to establish the safety and feasibility of this form of treatment, and if successful, we hope that it will lead on to larger studies looking at the effects of the treatment on patient recovery".
Michael Hunt, Chief Executive Officer of ReNeuron, said:
"This regulatory approval marks the first step in the process of testing the safety and potency of our lead ReN001 stroke therapy at a clinical level. It is the most important milestone in ReNeuron's history thus far and also represents a significant development in the wider field as regards the translation of exciting stem cell science into clinical stage therapies. In many ways, ReNeuron has set the regulatory pathway in the UK for cell therapy trials of this type, and we are delighted to have been given the opportunity to move ReN001 into its clinical phase on home territory in the UK"
Enquiries:
ReNeuron
Michael Hunt, Chief Executive Officer +44 (0) 1483 302560
John Sinden, Chief Scientific Officer
David Yates, Susan Quigley; Financial Dynamics +44 (0) 20 7831 3113
Stewart Wallace, Adam Cowen; Collins Stewart +44 (0) 20 7523 8350
University of Glasgow
Martin Shannon, Media Relations Officer +44 (0) 141 330 8593
About stroke
Approximately 150,000 people suffer a stroke in the UK each year. The vast majority of these strokes are ischaemic in nature, caused by a blockage of blood flow in the brain (as opposed to a haemorrhagic or bleeding stroke).
Approximately one half of all stroke survivors are left with permanent disabilities as a result of the damage caused to brain tissue arising from the stroke. The annual health and social costs of caring for these patients is estimated to be in exc ess of £5 billion in the UK, with stroke patients estimated to be occupying at least 25 per cent of long term hospital beds.
The only current treatment for ischaemic stroke patients occurs in the acute phase of the condition (within several hours of the stroke), when anti-clotting agents are administered to dissolve the clot causing the blockage in blood flow to the brain. Only a small proportion of patients get to the hospital in time to be treated in this way.
Beyond the acute phase, there are no existing treatments, other than preventative or rehabilitation measures, to alleviate the disabilities suffered by stroke patients who have survived their stroke.
About ReNeuron's ReN001 stem cell therapy for stroke
ReNeuron's ReN001 cell therapy for stroke consists of a neural stem cell line, designated CTX, which has been generated using the Company's proprietary cell expansion and cell selection technologies and then taken through a full manufacturing scale-up and quality-testing process. As such, ReN001 is a standardised, clinical and commercial-grade cell therapy product capable of treating all eligible patients presenting.
ReN001 has been shown to reverse the functional deficits associated with stroke disability when administered several weeks after the stroke event in relevant pre-clinical models of the condition. Extensive pre-clinical testing also indicates that the therapy is safe, with the ReN001 cells eventually cleared from the body with no adverse safety effects arising.
In the first-in-man, Phase I trial, a total of twelve patients will receive the ReN001 therapy between 6 and 24 months after their stroke. If ultimately shown to be safe and effective clinically, ReN001 would therefore offer a significant new treatment option for stroke survivors. The therapy offers the potential for a degree of recovery of function in disabled stroke patients, resulting in greater independence and quality of life for these patients and reduced reliance on health and social care systems.
The ReN001 cells that are being used in the initial clinical trial are taken from the existing manufactured cell banks that will form the basis of the eventual marketed product. There will therefore be no need to re-derive and test new ReN001 cell lines for subsequent clinical trials or for the market; all such cells can simply be expanded from the existing banked and tested product.
About the Institute of Neurological Sciences at Glasgow University
The clinical Stroke Research Group of the Division of Clinical Neurosciences is based at the Institute of Neurological Sciences at Glasgow University, and has major collaborations, internally with the Glasgow Experimental MRI Centre, with SINAPSE (Scottish Imaging Network: A Platform for Scientific Excellence), and with the Translational Medicine Research Initiative (TMRI). Around 900 patients per year are admitted through the Acute Stroke Unit, which provides stroke services to the population of south Glasgow and specialist stroke treatments for the West of Scotland.
The unit is the highest user of acute clot-busting (thrombolytic) treatment in the UK at present, and has been extensively involved in clinical trials in stroke. Major research interests include evaluation of advanced brain imaging techniques in acute stroke, development of novel brain imaging techniques, improving the use of clot-busting drug treatments in stroke, and developing trial methodology for evaluation of regenerative treatments. The group has support from the Stroke Association, the Medical Research Council, and the TMRI. Further work with regenerative strategies include collaborations with groups developing both drug-based and stem cell therapies across Europe.
About ReNeuron
ReNeuron is a leading, UK-based stem cell company. Its primary objective is the development of stem cell therapies targeting areas of significant unmet or poorly met medical need.
ReNeuron expects to commence initial clinical studies with its lead ReN001 stem cell therapy for disabled stroke patients in the UK in the second quarter of 2009. In addition to its stroke programme, ReNeuron is developing stem cell therapies for a number of other conditions, including peripheral ischaemia, Type 1 diabetes and diseases of the retina.
ReNeuron has also developed a range of stem cell lines for non-therapeutic applications, its ReNcell® products for use in academic and commercial research. The Company's ReNcell®CX and ReNcell®VM neural cell lines are marketed worldwide under license by USA-based Millipore Corporation.
ReNeuron's shares are traded on the London AIM market under the symbol RENE.L. Further information on ReNeuron and its products can be found at www.reneuron.com.
Data source: UK Stroke Association.
http://www.reneuron.com/news__events/news/document_178_237.php
Pfizer's $100 million stem cell stake
Nayanah Siva, Nature Biotechnology 27, 10 (2009)
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.
Scientists call up stem cell troops to repair the body using new drug combinations
http://www.eurekalert.org/pub_releases/2009-01/icl-scu010709.php
Scientists have tricked bone marrow into releasing extra adult stem cells into the bloodstream, a technique that they hope could one day be used to repair heart damage or mend a broken bone, in a new study published today in the journal Cell Stem Cell.
When a person has a disease or an injury, the bone marrow mobilises different types of stem cells to help repair and regenerate tissue. The new research, by researchers from Imperial College London, shows that it may be possible to boost the body's ability to repair itself and speed up repair, by using different new drug combinations to put the bone marrow into a state of 'red alert' and send specific kinds of stem cells into action.
In the new study, researchers tricked the bone marrow of healthy mice into releasing two types of adult stem cells – mesenchymal stem cells, which can turn into bone and cartilage and that can also suppress the immune system, and endothelial progenitor cells, which can make blood vessels and therefore have the potential to repair damage in the heart.
This study, funded by the British Heart Foundation and the Wellcome Trust, is the first to selectively mobilise mesenchymal stem cells and endothelial progenitor cells from the bone marrow. Previous studies have only been able to mobilise the haematopoietic type of stem cell, which creates new blood cells. This technique is already used in bone marrow transplants in order to boost the numbers of haematopoietic stem cells in a donor's bloodstream.
The researchers were able to choose which groups of stem cells the bone marrow released, by using two different therapies. Ultimately, the researchers hope that their new technique could be used to repair and regenerate tissue, for example when a person has heart disease or a sports injury, by mobilising the necessary stem cells.
The researchers also hope that they could tackle autoimmune diseases such as rheumatoid arthritis, where the body is attacked by its own immune system, by kicking the mesenchymal stem cells into action. These stem cells are able to suppress the immune system.
Dr Sara Rankin, the corresponding author of the study from the National Heart & Lung Institute at Imperial College London, said: "The body repairs itself all the time. We know that the skin heals over when we cut ourselves and, similarly, inside the body there are stem cells patrolling around and carrying out repair where it's needed. However, when the damage is severe, there are limits to what the body can do of its own accord.
"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, in order to boost its ability to mend itself and accelerate the repair process. Further down the line, our work could lead to new treatments to fight various diseases and injuries which work by mobilising a person's own stem cells from within," added Dr Rankin.
The scientists reached their conclusions after treating healthy mice with one of two different 'growth factors' – proteins that occur naturally in the bone marrow – called VEGF and G-CSF. Following this treatment, the mice were given a new drug called Mozobil.
The researchers found that the bone marrow released around 100 times as many endothelial and mesenchymal stem cells into the bloodstream when the mice were treated with VEGF and Mozobil, compared with mice that received no treatment. Treating the mice with G-CSF and Mozobil mobilised the haematopoietic stem cells – this treatment is already used in bone marrow transplantation.
The researchers now want to investigate whether releasing repair stem cells into the blood really does accelerate the rate and degree of tissue regeneration in mice that have had a heart attack. Depending on the outcome of this work, they hope to conduct clinical trials of the new drug combinations in humans within the next ten years.
The researchers are also keen to explore whether ageing or having a disease affects the bone marrow's ability to produce different kinds of adult stem cells. They want to investigate if the new technique might help to reinvigorate the body's repair mechanisms in older people, to help them fight disease and injury.
'Scrawny' gene keeps stem cells healthy
http://www.eurekalert.org/pub_releases/2009-01/ci-gk010609.php
Baltimore, MD—Stem cells are the body's primal cells, retaining the youthful ability to develop into more specialized types of cells over many cycles of cell division. How do they do it? Scientists at the Carnegie Institution have identified a gene, named scrawny, that appears to be a key factor in keeping a variety of stem cells in their undifferentiated state. Understanding how stem cells maintain their potency has implications both for our knowledge of basic biology and also for medical applications. The results will be published in the January 9, 2009 print edition of Science.
"Our tissues and indeed our very lives depend on the continuous functioning of stem cells," says Allan C. Spradling, director of the Carnegie Institution's Department of Embryology. "Yet we know little about the genes and molecular pathways that keep stem cells from turning into regular tissue cells—a process known as differentiation."
In the study, Spradling, with colleagues Michael Buszczak and Shelley Paterno, determined that the fruit fly gene scrawny (so named because of the appearance of mutant adult flies) modifies a specific chromosomal protein, histone H2B, used by cells to package DNA into chromosomes. By controlling the proteins that wrap the genes, scrawny can silence genes that would otherwise cause a generalized cell to differentiate into a specific type of cell, such as a skin or intestinal cell.
The researchers observed the effects of scrawny on every major type of stem cell found in fruit flies. In the experiments, mutant flies without functioning copies of the scrawny prematurely lost their stem cells in reproductive tissue, skin, and intestinal tissue.
Stem cells function as a repair system for the body. They maintain healthy tissues and organs by producing new cells to replenish dying cells and rebuild damaged tissues. "Losing stem cells represents the cellular equivalent of eating the seed corn," says Spradling.
While the scrawny gene has so far only been identified in fruit flies, very similar genes that may carry out the same function are known to be present in all multicellular organisms, including humans. The results of this study are an important step forward in stem cell research. "This new understanding of the role played by scrawny may make it easier to expand stem cell populations in culture, and to direct stem cell differentiation in desired directions," says Spradling.
Scientists can now differentiate between healthy stem cells and cancer stem cells
http://www.eurekalert.org/pub_releases/2009-01/mu-scn010509.php
One of the current handicaps of cancer treatments is the difficulty of aiming these treatments at destroying malignant cells without killing healthy cells in the process. But a new study by McMaster University researchers has provided insight into how scientists might develop therapies and drugs that more carefully target cancer, while sparing normal healthy cells
Mick Bhatia, scientific director of the McMaster Stem Cell and Cancer Research Institute in the Michael G. DeGroote School of Medicine, and his team of investigators have demonstrated – for the first time – the difference between normal stem cells and cancer stem cells in humans.
The discovery, published in the prestigious journal Nature Biotechnology today, could eventually help with the further customization and targeting of cancer treatments for the individual patient. It will immediately provide a model to discover drugs using robotic screening for available molecules that may have untapped potential to eradicate cancer.
"Normal stem cells and cancer stem cells are hard to tell apart, and many have misconstrued really good stem cells for cancer stem cells that have gone bad - we now can tell the ones masquerading as normal stem cells from the bad, cancerous ones," said Bhatia.
"This also allows us to compare normal versus cancer stem cells from humans in the laboratory - define the differences in terms of genes they express and drugs they respond to. Essentially, we can now use this to find the "magic bullet", a drug or set of drugs that kill cancer stem cells first, and spare the normal healthy ones," he said.
"McMaster is uniquely positioned for this discovery platform, and this was the missing ingredient - we have one of the best screening/robotic platforms, chemical libraries and expertise in professors Eric Brown and Gerry Wright, who have discovered molecules to combat infectious disease. Now we can combine it all. This team now aims to kill cancer."
Brain Birth Defects Successfully Reversed Through Stem Cell Therapy
http://www.sciencedaily.com/releases/2008/12/081228191056.htm
Scientists at the Hebrew University of Jerusalem have succeeded in reversing brain birth defects in animal models, using stem cells to replace defective brain cells.
The work of Prof. Joseph Yanai and his associates at the Hebrew University-Hadassah Medical School was presented at the Tel Aviv Stem Cells Conference last spring and is expected to be presented and published nest year at the seventh annual meeting of the International Society for Stem Cell Research in Barcelona, Spain.
Involved in the project with Prof. Yanai are Prof. Tamir Ben-Hur, head of the Department of Neurology at the Hebrew University-Hadassah Medical School, and his group, as well as Prof. Ted Slotkin at Duke University in North Carolina, where Prof. Yanai is an adjunct professor.
Neural and behavioral birth defects, such as learning disabilities, are particularly difficult to treat, compared to defects with known cause factors such as Parkinson’s or Alzheimer’s disease, because the prenatal teratogen – the substances that cause the abnormalities -- act diffusely in the fetal brain, resulting in multiple defects.
Prof. Yanai and his associates were able to overcome this obstacle in laboratory tests with mice by using mouse embryonic neural stem cells. These cells migrate in the brain, search for the deficiency that caused the defect, and then differentiate into becoming the cells needed to repair the damage.
Generally speaking, stem cells may develop into any type of cell in the body, however at a certain point they begin to commit to a general function, such as neural stem cells, destined to play a role in the brain/ nervous system. At more advanced developmental stages, the neural stem cells take on an even more specific role as neural or glial (supporting) cells within the brain/ nervous system.
In the researchers’ animal model, they were able to reverse learning deficits in the offspring of pregnant mice who were exposed to organophosphate (a pesticide) and heroin. This was done by direct neural stem cell transplantation into the brains of the offspring. The recovery was almost one hundred percent, as proved in behavioral tests in which the treated animals improved to normal behavior and learning scores after the transplantation. On the molecular level, brain chemistry of the treated animals was also restored to normal.
The researchers went one step further. Puzzled by the stem cells’ ability to work even in those cases where most of them died out in the host brain, the scientists went on to discover that the neural stem cells succeed before they die in inducing the host brain itself to produce large number of stem cells which repair the damage. This discovery, finally settling a major question in stem cell research, evoked great interest and was published earlier this year in one of the leading journals in the field, Molecular Psychiatry.
The scientists are now in the midst of developing procedures for the least invasive method for administering the neural stem cells, which is probably via blood vessels, thus making the therapy practical and clinically feasible.
Normally, stem cells are derived from individuals genetically different from the patient to be transplanted, and therefore the efficacy of the treatment suffers from immunological rejection. For this reason, another important avenue of the ongoing study, toward the same goals, will be to eliminate the immunological rejection of the transplant, which will become possible by taking cells from the patient’s own body -- from a place where they are easily obtained -- by manipulating them to return to their stem cell phase of development, and then transplanting them into the patient’s brain via the blood stream. One important advantage of this approach will be to eliminate the controversial ethical issues involved in the use of embryo stem cells.
The research on the project has been supported by the US National Institutes of Health, the US-Israel Binational Science Foundation and the Israel anti-drug authorities.
Cancer stem cells: Common as muck
Nature Reviews Cancer 9, 6-7 (January 2009) | doi:10.1038/nrc2563
http://www.nature.com/nrc/journal/v9/n1/full/nrc2563.html
Safia Ali Danovi
It's not easy being a cancer stem cell.
They have been blamed for single-handedly propelling tumour growth, along with mediating other evils such as chemoresistance and radioresistance.
It is thus contended that the key to conquering cancer lies in their annihilation. However, others have argued that the existence of cancer stem cells in solid tumours relies on results derived from artificial experimental conditions that bear little resemblance to the in vivo situation in a tumour. Recent data from Sean Morrison and colleagues provide ammunition for the naysayers.
A fundamental pillar of the cancer stem cell hypothesis is hierarchy, with cancer stem cells at the top of the pecking order — the biological equivalent of a hen's tooth. This dogma is based on the observation that only a minority of cells from a tumour have tumorigenic potential when xenotransplanted into non-obese diabetic, severe combined immunodeficient (NOD/SCID) mice. Based on this method, the frequency of melanoma-initiating cells has been estimated to be about 0.0001%. However, by modifying three xenotransplantation conditions — the strain of mouse used, the length of time over which the animals were followed, and mixing the cells with Matrigel prior to injection — Morrison's team could increase this frequency by several orders of magnitude. Importantly, the authors confirmed that these new experimental conditions were simply providing an environment that was more conducive for melanoma tumour formation, rather than inducing heritable changes in the cells themselves that could also have explained the observed increase in tumorigenicity. Furthermore, both xenografted human melanoma cells and freshly disassociated melanoma cells obtained directly from patients with either primary cutaneous or metastatic melanoma exhibited improved tumorigenicity under these new conditions, indicating again that the ability of cells to form tumours was more a function of environment than of cell type.
Taken together, these data indicate that tumour-initiating cells are far more common than previous reports have suggested, and the authors further reinforced this idea by injecting single cells obtained from xenografted melanomas into mice and showing that as many as 27% of injections resulted in tumour formation. Moreover, extensive analysis of over 50 surface markers — including the 'cancer stem cell marker' CD133 — failed to discriminate between tumorigenic and non-tumorigenic cells, suggesting that cells that have poor tumorigenic capacity under certain conditions may in fact readily form tumours in others.
...tumorigenic cells are far more common than previous reports have suggested...
The authors grant that no data thus far provide insight into the question of whether a specific population of cells contributes to tumour progression in patients, and admit that a distinct population of cells capable of propelling tumour growth might yet be identified. They show, however, that the commonly used NOD/SCID assay significantly underestimates the frequency of human cells with tumorigenic potential. Thus, some cancers that have only few tumorigenic cells in NOD/SCID mice, such as melanomas, actually have many tumorigenic cells under other assay conditions. So, whereas the cancer stem cell model probably holds true for certain cancers, other paradigms might be in play for others.
References and links
ORIGINAL RESEARCH PAPER
Quintana, E. et al. Efficient tumour formation by single human melanoma cells. Nature 456, 593–598 (2008)
A Joyous Christmas and Channukah to all here and
your families....
May the holidays bring joy, peace, funfilled family
gatherings as well as hope for a brand new year coming,
with the expectations of a new start, a new administration,
a new slate to work on. What I pray for all here is
abundant great health and peace. Without health, all the
money in the world does not matter.
May God's blessings in your life be magnified and
acknowledged with praiseful reverence, and no matter
what religion you aspire to follow, may you be a beacon
of light unto others...
*****PL1*****
Single Virus Used To Convert Adult Cells To Embryonic Stem Cell-like Cells
http://www.sciencedaily.com/releases/2008/12/081215184343.htm
(Dec. 19, 2008) — Whitehead Institute researchers have greatly simplified the creation of so-called induced pluripotent stem (iPS) cells, cutting the number of viruses used in the reprogramming process from four to one. Scientists hope that these embryonic stem-cell-like cells could eventually be used to treat such ailments as Parkinson's disease and diabetes.
The earliest reprogramming efforts relied on four separate viruses to transfer genes into the cells' DNA--one virus for each reprogramming gene (Oct4, Sox2, c-Myc and Klf4). Once activated, these genes convert the cells from their adult, differentiated status to an embryonic-like state.
However, this method poses significant risks for potential use in humans. The viruses used in reprogramming are associated with cancer because they may insert DNA anywhere in a cell's genome, thereby potentially triggering the expression of cancer-causing genes, or oncogenes. For iPS cells to be employed to treat human diseases, researchers must find safe alternatives to reprogramming with such viruses. This latest technique represents a significant advance in the quest to eliminate the potentially harmful viruses.
Bryce Carey, an MIT graduate student working in the lab of Whitehead Member Rudolf Jaenisch, spearheaded the effort by joining in tandem the four reprogramming genes through the use of bits of DNA that code for polymers known as 2A peptides. Working with others in the lab, he then manufactured a so-called polycistronic virus capable of expressing all four reprogramming genes once it is inserted into the genomes of mature mouse and human cells.
When the cells' protein-creating machinery reads the tandem genes' DNA, it begins making a protein. However, when it tries to read the 2A peptide DNA that resides between the genes, the machinery momentarily stops, allowing the first gene's protein to be released. The machinery then moves on to the second gene, creates that gene's protein, stalls when reaching another piece of 2A peptide DNA, and releases the second gene's protein. The process continues until the machinery has made the proteins for all four genes.
Using the tandem genes, Carey created iPS cells containing just a single copy of the polycistronic vector instead of multiple integrations of the viruses. This significant advancement indicates that the approach can become even safer if combined with technologies such as gene targeting, which allows a single transgene to be inserted at defined locations.
Interestingly, while Carey's single-virus method integrates all four genes into the same location, it has proven to be roughly 100 times less efficient than older approaches to reprogramming. This phenomenon remains under investigation.
"We were surprised by the lower efficiency," Carey says.
"We're not sure why, but we need to look what's going on
with expression levels of the polycistronic virus's proteins compared to separate viruses' proteins."
Although the one virus method is less efficient, Jaenisch maintains it represents an important advance in the field.
"This is an extremely useful tool for studying the mechanisms of reprogramming," says Jaenisch, who is also a professor of biology at MIT. "Using this one virus creates a single integration in the cells' DNA, which makes things much easier to handle."
Journal reference:
Bryce W. Carey et al. Reprogramming of murine and human somatic cells using a single polycistronic vector. PNAS, December 15, 2008
Mesenchymal Stem Cells and Neurodegenerative Disease
http://www.nature.com/clpt/journal/v85/n1/full/clpt2008205a.html
Clinical Pharmacology & Therapeutics (2009); 85, 1, 19–20 doi:10.1038/clpt.2008.205
AL Whone and NJ Scolding
Burden Neurological Institute, University of Bristol Institute of Clinical Neurosciences, Frenchay Hospital, Bristol, UK
Abstract
The prospect of cell therapy for incurable neurodegenerative disease excites scientists, the public, and patients alike. Clinical and scientific enthusiasm must, however, always be tempered by methodological rigor and by the overwhelming imperative of protecting vulnerable sufferers.
We tentatively suggest that, in the case of autologous mesenchymal stem cells (MSCs), the balance between our current understanding of their biology and an informed assessment of their probable safety allows a case to be made for cautious pilot clinical studies.
Quinn and colleagues, commenting on the recent safety and feasibility study of autologous MSC therapy in individuals with multiple system atrophy (MSA),1 make a number of invaluable and timely points concerning the premature introduction of stem cell therapies.2 They rightly draw attention to the need for very great caution in advancing or inferring efficacy from a single, small, uncontrolled phase I study; they allude to important questions about the potential hazards of intra-arterial cell delivery; and they hint that such clinical studies may be premature. We wholeheartedly endorse their conclusion that, at present, there is "little scientific justification for their clinical use [our emphasis] in neurodegenerative conditions."
They might have added that the uninhibited claims of clinical benefit made in such studies (not least following their appearance in a highly influential journal) will doubtless be exploited by the numerous outfits around the globe currently preying on vulnerable patients by the direct-to-sufferers sale of so-called stem cell therapies of no proven value at great price and profit.
But would it be right to conclude that such feasibility studies should not be performed at all, or only that they should be meticulously designed and properly interpreted? Although, as Quinn and colleagues suggest, larger comparable studies using intra-arterial delivery in MSA may not yet be justified, and certainly not widespread clinical use, we suggest that recent advances in our understanding of the biology of MSCs allow a case to be made for small-scale studies beginning to explore the potential of autologous, intravenously delivered MSCs in neurodegenerative disease.
Quinn et al. rightly point out that research five or more years ago showing that MSCs could fuse in vitro with other cell types was widely interpreted as indicating that the therapeutic potential of adult stem cells might be very limited.4 Others at that time, however, suggested the opposite—that fusion might represent a means by which MSCs could "rescue" damaged or effete cells and so help tissue repair.5 Over the next few years it became clear that cell fusion was indeed one mechanism by which MSCs could deliver therapeutic benefit6—but that in other experimental circumstances true transdifferentiation, not fusion, seemed a more likely explanation.
Furthermore, other mechanisms by which MSCs could contribute to tissue repair, including significant immunity-modulating properties, vasculogenic effects, and (especially important in the current context) the release of neurotrophic factors, were subsequently revealed.7 It has become clear that transdifferentiation is but one of many potentially therapeutic properties of adult stem cells8—and quite possibly not the most important. Furthermore, stem cells from developing tissue also act beneficially (in experimental neurodegenerative disease) through multiple mechanisms, with only a "small degree of neuronal replacement."9 The early, near-unanimous emphasis on transdifferentiation to replace cells as the key to cell therapy is, arguably, proving an insufficiently subtle approach to the complexities of both spontaneous and therapeutic tissue regeneration and repair.
More recently, fusion of adult stem cells has received further experimental attention. It has been confirmed that bone marrow–derived cells can contribute to differentiated cell populations in various tissues—including cerebellar Purkinje cells (of particular relevance, of course, to MSA)—through the formation of stable reprogrammed fusion hybrids.10,11 This is triggered by injury and, especially, inflammation, strongly suggesting a reparative (or "rejuvenating") effect of obvious therapeutic potential.12
Finally, autopsy studies of persons many years after receiving sex-mismatched bone marrow transplants (for blood disorders) reveal small numbers of apparently fully integrated and functional cells of highly specialized morphology in a variety of organs—again, including cerebellar Purkinje cells—whose nuclei are clearly of donor origin.13,14 These findings lend strong support to the reparative potential of bone marrow–derived cells (delivered intravenously in these instances, of course).
A glance outside neurological medicine to cardiology may also be informative. Here, a more accelerated pace of investigating the possible clinical benefits of MSCs has been apparent. A recent authoritative review considered more than 30 clinical trials in both acute and chronic heart disease, and the authors already felt able to conclude that "mesenchymal stem cells...can, under appropriate conditions in select patients, provide disease-ameliorating effects in...cardiovascular disorders."15 Significantly, however, the precise mechanism(s) by which the cells exerted this benefit remain unclear.
We therefore believe that there are, however limited, clear experimental reasons to believe that autologous MSCs could be of benefit in neurodegenerative disease. There are few, if any, reasons to fear that such cells are intrinsically likely to have adverse effects. Although the balance of evidence may arguably not be sufficient to overcome the hazards of intra-arterial or, more obviously, intracerebral implantation, intravenous delivery is very likely to be harmless, and indeed cardiological and extensive other clinical experience supports that likelihood. It is absolutely vital, of course, to continue the further investigation of MSCs and their basic and applied biological properties, but, in light of the above progress, we believe that, in progressive and untreatable neurodegenerative diseases such as MSA, a case can be made for cautious pilot clinical studies.
Top of pageConflict of Interest
The authors declared no conflict of interest.
Top of pageReferences
Lee, P.H. et al. Autologous mesenchymal stem cell therapy delays the progression of neurological deficits in patients with multiple system atrophy. Clin. Pharmacol. Ther. 83, 723|[ndash]|730 (2008). | Article | PubMed | ChemPort |
Quinn, N., Barker, R.A. & Wenning, G.K. Are trials of intravascular infusions of autologous mesenchymal stem cells in patients with multiple system atrophy currently justified, and are they effective? Clin. Pharmacol. Ther. 83, 663|[ndash]|665 (2008). | Article | PubMed | ChemPort |
Baker, M. Stem cell therapy or snake oil? Nat. Biotechnol. 23, 1467|[ndash]|1469 (2005). | Article | PubMed | ChemPort |
Pearson, H. Stem cells: articles of faith adulterated. Nature 420, 734|[ndash]|735 (2002). | Article | PubMed | ChemPort |
Blau, H.M. A twist of fate. Nature 419, 437 (2002). | Article | PubMed | ISI |
Wang, X. et al. Cell fusion is the principal source of bone-marrow-derived hepatocytes. Nature 422, 897|[ndash]|901 (2003). | Article | PubMed | ISI | ChemPort |
Korbling, M. & Estrov, Z. Adult stem cells for tissue repair. N. Engl. J. Med. 349, 570|[ndash]|582 (2003). | Article | PubMed | ISI |
Rice, C.M. & Scolding, N.J. Adult stem cells|[mdash]|reprogramming neurological repair? Lancet 364, 193|[ndash]|199 (2004). | Article | PubMed | ChemPort |
Lee, J.P. et al. Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease. Nat. Med. 13, 439|[ndash]|447 (2007). | Article | PubMed | ISI | ChemPort |
Johansson, C.B. et al. Extensive fusion of haematopoietic cells with Purkinje neurons in response to chronic inflammation. Nat. Cell Biol. 10, 575|[ndash]|583 (2008). | Article | PubMed | ChemPort |
Nygren, J.M. et al. Myeloid and lymphoid contribution to non-haematopoietic lineages through irradiation-induced heterotypic cell fusion. Nat. Cell Biol. 10, 584|[ndash]|592 (2008). | Article | PubMed | ChemPort |
Singec, I. & Snyder, E.Y. Inflammation as a matchmaker: revisiting cell fusion. Nat. Cell Biol. 10, 503|[ndash]|505 (2008). | Article | PubMed | ChemPort |
Cogle, C.R. et al. Bone marrow transdifferentiation in brain after transplantation: a retrospective study. Lancet 363, 1432|[ndash]|1437 (2004). | Article | PubMed | ISI | ChemPort |
Weimann, J.M., Charlton, C.A., Brazelton, T.R., Hackman, R.C. & Blau, H.M. Contribution of transplanted bone marrow cells to Purkinje neurons in human adult brains. Proc. Natl. Acad. Sci. USA 100, 2088|[ndash]|2093 (2003). | Article | PubMed | ChemPort |
Burt, R.K. et al. Clinical applications of blood-derived and marrow-derived stem cells for nonmalignant diseases. JAMA 299, 925|[ndash]|936 (2008).
StemCells, Inc. Receives FDA Approval to Initiate Clinical Trial of HuCNS-SC® Cells in a Myelin Disease
Thursday December 18, 7:30 am ET
PALO ALTO, Calif.--(BUSINESS WIRE)--StemCells, Inc. (NASDAQ:STEM - News) today announced that it has received approval from the U.S. Food and Drug Administration (FDA) to initiate a clinical trial of the Company’s proprietary HuCNS-SC product candidate (purified human neural stem cells) to treat Pelizaeus-Merzbacher Disease (PMD), a fatal brain disorder that mainly affects young children. This Phase I trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for PMD. Currently, there are no approved treatments for this disease.
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This is the Company’s second FDA-approved clinical trial to evaluate HuCNS-SC cells as a potential treatment for neurodegenerative diseases. The first such study was the Company’s Phase I clinical trial of HuCNS-SC cells to treat neuronal ceroid lipofuscinoses (NCL), or Batten disease. The Phase I NCL trial will be completed in January 2009.
Patients with PMD are born with a gene mutation that results in insufficient myelination of nerve fibers in the brain, neurological impairment and eventually death. Myelin, which is produced by special cells called oligodendrocytes, insulates nerve fibers to allow electrical signals to be conducted normally. Other, more common, myelination diseases include cerebral palsy, transverse myelitis and multiple sclerosis.
Preclinical studies performed by the Company and its collaborators provide a rationale for potential therapeutic use of HuCNS-SC cells in myelination diseases. The Company has demonstrated that, when transplanted into an animal model of hypomyelination (shiverer mouse), HuCNS-SC cells engraft and differentiate into mature oligodendrocytes and form myelin sheaths around host nerve fibers. The initial myelination data in the shiverer mouse was published in the Proceedings of the National Academy of Science (Cummings, et al. 2005) and the results of additional myelination studies were presented by Nobuko Uchida, Ph.D., the Company’s Vice President of Stem Cell Biology, at the International Society of Stem Cell Research annual meeting in Philadelphia earlier this year.
“PMD is one of the most severe myelin disorders known to man. Children afflicted with this disease suffer progressive neurodegeneration and an untimely death. Unfortunately, they have no option for restorative treatment,” said Stephen Huhn MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. “We are very pleased to be the first company to initiate a neural stem cell trial for a myelination disease. Establishing safety and efficacy of our cells in patients with PMD may pave the way for similar studies in patients with other myelin diseases.”
The Company has begun the process of seeking approval by the Investigational Review Board (IRB) of potential clinical trial sites in order to begin enrolling patients.
About Pelizaeus-Merzbacher Disease (PMD)
PMD is a rare, degenerative, central nervous system disorder and is one of a group of genetic disorders known as leukodystrophies. Leukodystrophies involve abnormal growth of the myelin sheath which is the fatty substance—or insulator—on nerve fibers in the brain and spinal cord. PMD is most commonly caused by a genetic mutation that affects an important protein found in myelin, proteolipid protein (PLP). PMD is most frequently diagnosed in early childhood and is associated with abnormal eye movements (nystagmus), abnormal muscle function, and in some cases seizures. The disease form in early infancy is referred to as connatal PMD and diagnosis in later childhood is most typically associated with the classic form. The neurological course of both forms is marked by progressive deterioration resulting in premature death.
About HuCNS-SC® Cells
StemCells’ lead product candidate, HuCNS-SC cells, is a purified composition of normal human neural stem cells that are expanded and stored as banks of cells. The Company’s preclinical research has shown that HuCNS-SC cells can be directly transplanted; they engraft, migrate, differentiate into neurons and glial cells; and they survive for as long as one year with no sign of tumor formation or adverse effects. These findings show that HuCNS-SC cells, when transplanted, act like normal stem cells, suggesting the possibility of a continual replenishment of normal human neural cells.
About StemCells, Inc.
StemCells, Inc. is a clinical-stage biotechnology company focused on the discovery, development and commercialization of cell-based therapeutics to treat diseases of the central nervous system and liver. The Company’s product development programs seek to repair or repopulate CNS and liver tissue that has been damaged or lost as a result of disease or injury. StemCells has pioneered the discovery and development of HuCNS-SC cells, its highly purified, expandable population of human neural stem cells. StemCells owns or has exclusive rights to more than 50 issued or allowed U.S. patents and more than 150 granted or allowed non-U.S. patents. Further information about the Company is available on its web site at: www.stemcellsinc.com.
Apart from statements of historical fact, the text of this press release constitutes forward-looking statements regarding, among other things, the future business operations of StemCells, Inc. (the “Company”) and its ability to conduct clinical trials as well as its research and product development efforts. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management’s current views and are based on certain assumptions that may or may not ultimately prove valid. The Company’s actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including uncertainty as to whether the FDA or other applicable regulatory agencies will permit the Company to continue clinical testing in NCL, PMD or in future clinical trials of proposed therapies for other diseases or conditions despite the novel and unproven nature of the Company’s technologies; uncertainties about whether the Company will receive the necessary support of a clinical study center and its ethics board to initiate a clinical trial in PMD; uncertainties regarding the Company’s ability to obtain the increased capital resources needed to continue its current research and development operations and to conduct the research, preclinical development and clinical trials necessary for regulatory approvals; uncertainty as to whether HuCNS-SC and any products that may be generated in the future in the Company’s cell-based programs will prove safe and clinically effective and not cause tumors or other adverse side effects; uncertainties regarding the Company’s manufacturing capabilities given its increasing preclinical and clinical commitments; and other factors that are described under the heading “Risk Factors” in Item 1A of Part II of the Company’s Quarterly Report on Form 10-Q.
Contact:
StemCells, Inc.
Rodney Young, 650-475-3100 ext. 105
Chief Financial Officer
irpr@stemcellsinc.com
Source: StemCells, Inc.
Unraveling Brain Tumors
http://www.sciencedaily.com/videos/2007/0910-unraveling_brain_tumors.htm
Molecular Biologists Devise Strategy To Starve Brain Tumors
Brain tumor researchers have found that brain tumors arise from cancer stem cells living within tiny protective areas formed by blood vessels in the brain. Killing those cells is a promising strategy to eliminate tumors and prevents them from re-growing. The researchers have found that drugs that block new blood vessel formation can destroy the protected areas and stop cancer from developing.
Brain tumors are often deadly. Figuring out a way to wipe them out has been a mystery for scientists. But now, a new discovery may offer clues and hope for those with even the most hard-to-treat tumors.
In the last two months, Will Pappas has had three surgeries, chemo and radiation.
"You hold out hope that well, it's just something little, and they can get it all. And then it wasn't. Then you think, well, at least it's not cancerous, and then it is," Cayce Pappas, Will's mom, says.
"It" is a brain tumor -- the stubborn kind that's hard to treat. In fact, doctors gave this seven-year-old only a 20 percent chance of surviving. Stories like Will's have molecular biologists determined to find a way to destroy brain tumors.
"It's what makes us all come to work in the morning," Richard Gilbertson, a molecular biologist from St. Jude Children's Hospital, says.
For years, researchers thought all cells inside a tumor were the same. But recently, they've discovered something different -- a small group of cancer stem cells.
"They give rise to all the cells that make up the cancer," Dr. Gilbertson explains.
Dr. Gilbertson's research shows those cancer stem cells live close to blood vessels, which fuel them. In lab experiments, he's proven drugs that target the blood vessels also destroy the cancer stem cells and can ultimately wipe out the tumor.
"So, if you can target those cells, you can have a devastating effect on the disease," Dr. Gilbertson says. Drugs like Avastin and Tarceva are now being tested in humans to see if they can target the cancer stem cells. "It's this tangible way of actually getting at the heart of the disease," Dr. Gilbertson says.
Will is taking the drug Tarceva. His mom is hoping it will work a miracle.
"That would be amazing. We would jump at the opportunity to increase our odds. He's still got a lot left to do," Cayce says.
Dr. Gilbertson says other cancers, like those of the blood, breast and colon, also contain cancer stem cells and may be treated in a similar way in the future.
BACKGROUND: Researchers at St. Jude Children's Hospital have found that brain tumors appear to arise from cancer stem cells that live inside tiny protective 'niches' formed by blood vessels in the brain. Breaking down these niches is a promising strategy for eliminating the tumors and preventing them from regrowing.
ABOUT CANCER STEM CELLS: Scientists previously believed that tumors are lumps of cancerous tissue that must be completely removed or destroyed to cure a patient.
But over the last five years, cancer researchers have learned that not all cancer cells are created equal. In the same way that normal tissue in the body is generated from stem cells, so is cancer. CSCs are the ultimate source of the tumor, consistently supplying it with new cells. Researchers have identified the CSCs for acute myeloma leukemia, four types of brain cancer, and breast cancer. So it is possible that we need not kill all cancer cells to rid a patient of the disease. Targeting the CSCs specifically might be much more efficient.
CANCER'S ACHILLES HEEL: To find a weakness for CSCs, neurobiologists at St. Jude compared them to noncancerous neural stem cells. These neural tissue generators are concentrated in regions rich in blood vessels. The vessels are lined with endothelial cells, which secrete chemical signals that help stem cells survive. CSCs, they discovered, required similar conditions to flourish: in over 70 human brain tumors, the CSCs were frequently located close to tiny vessels called capillaries. When the researchers injected mice with a mix of stem and endothelial cells from human brain tumors, those animals sprouted larger tumors than the mice that received stem cells alone.
NEW DRUG THERAPY: The new findings from St. Jude indicates that it is possible to kill the cancer by disrupting the shielded compartments in the small capillaries of the brain where CSCs reside. Anti-angiogenic drugs, such as Avastin, block the formation of new blood vessels. In tests with mice, those same drugs cause a significant drop in cancer stem cells and slow tumor growth. Human clinical trials are currently in progress at St. Jude to determine the effectiveness of Avastin and another anti-angiogenic drug in eliminating tumors and preventing their recurrence in children with brain cancers.
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This board is created for the discussion of any stem cell stock since the 'stem' sector is heating
up again, finally; for example: astm, stem, gern , ccel.ob, plrs.ob and others feel free to add
& join in the on-topic discussion.
Stocks:
Aastrom (ASTM):
http://finance.yahoo.com/q/ks?s=ASTM
http://finance.yahoo.com/q/pr?s=astm
http://www.investorshub.com/boards/board.asp?board_id=2080
Stem Cells (STEM):
http://finance.yahoo.com/q/ks?s=STEM
http://finance.yahoo.com/q/pr?s=STEM
http://www.investorshub.com/boards/board.asp?board_id=3023
Geron Corporation (GERN)
http://finance.yahoo.com/q/ks?s=gern
http://finance.yahoo.com/q/pr?s=gern
http://www.investorshub.com/boards/board.asp?board_id=1634
Advanced Cell Technology Inc. (ACTC.OB)
http://finance.yahoo.com/q?s=ACTC.OB
http://www.investorshub.com/boards/board.asp?board_id=5319
TISSERA INC (TSSR.OB):
http://finance.yahoo.com/q?s=TSSR.OB
http://finance.yahoo.com/q/h?s=TSSR.OB
Contact:
Tissera, Inc.
(Investor Relations)
Dr. Uri Elmaleh, 972-9-9561151
uri@tissera.com
Source: Tissera, Inc.
BioStem Inc.(BTEM.OB):
http://finance.yahoo.com/q/pr?s=BTEM.OB
http://finance.yahoo.com/q/ks?s=BTEM.OB
http://www.investorshub.com/boards/board.asp?board_id=7996
Stem Cell Innovations:
http://www.investorshub.com/boards/board.asp?board_id=5472
OSIR ~ Osiris Therapeutics (NasdaqGM:OSIR)
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=33470989
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=33471053
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Stem Cell Storage Stocks
Cryo-Cell International Inc. (CCEL.OB)
ccel>>http://finance.yahoo.com/q/ks?s=ccel.ob
http://finance.yahoo.com/q/pr?s=ccel.ob
http://www.investorshub.com/boards/board.asp?board_id=3965
Cord Blood America Inc. (CBAI.OB)
http://www.cordblood-america.com/
http://finance.yahoo.com/q?s=cbai.ob&d=t
http://finance.yahoo.com/q/pr?s=CBAI.OB
http://www.smallcapwatch.com/company.asp?TICKER=cbai
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Thermogenics board: KOOL
http://investorshub.advfn.com/boards/board.aspx?board_id=13388
RESEARCH ARTICLES:
http://news.google.com/news?q=Stem+cell+news&hl=en&um=1&sa=X&oi=news_group&resnum=4&ct=title
http://www.stemcellresearchnews.com/
http://science.bio.org/cloning.news.html
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Charts:
galleryview/stockcharts,http://stockcharts.com/charts/candleglance.php?STEM,KOOL,ASTM,ALNY,CLBE,CBAI,GERN,PSTI|B|L....
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Helpful Links:
http://www.nature.com/stemcells/index.html
http://www.stemcellresearchnews.com/stem_cell_lab__world.htm
http://www.nature.com/stemcells/index.html
http://stemcells.nih.gov/policy/legislation.asp
http://www.stemcellnews.com/
http://www.stemcell.com/
http://www.scirus.com/srsapp/search?
http://www.stemcellresearchnews.com/
Sticky Post: For news in the Stem Cell sector:
http://www.stemcellresearchnews.com/
http://www.stemcellnews.com/
http://www.sciencedaily.com/news/health_medicine/stem_cells/
PL1
http://stemcells.nih.gov/policy/taskForce/workingGroups/resourceaccess/raMembers.asp
http://novocell.com/
Medical World News:
http://investorshub.advfn.com/boards/board.asp?board_id=10777
Thank you for visiting the StemWare board here on IHUB, and please feel free to post information on Stem Cell stocks and Stem Cell news.....PL1
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