August 12, 2009 by

Adult Stem Cells Treat Heart Ailments

Researchers at Northwestern Memorial Hospital in Chicago have announced positive results from the use of autologous adult stem cells in the treatment of heart damage. Led by Dr. Douglas Losordo, the FDA-approved, Phase II clinical trial is testing both the efficacy and safety of mesenchymal stem cells (MSCs) that are derived from each patient’s own peripheral blood. Results of the study are expected to be presented in September.

In the study, treatment involves the use of CD34+ cells which are harvested from each patient’s own blood, purified, expanded and administered to the patient by injection directly into the damaged heart muscle. Since CD34+ cells are capable of angiogenesis, which is the formation of new blood vessels, the treatment is expected not only to regenerate new heart muscle but also to prevent the formation of scar tissue. Sponsored by Baxter, Inc., the study is currently the largest adult stem cell study for heart disease that is being conducted in the United States.

As Dr. Losordo explains, "It’s important to point out that this is a use of a patient’s own body repair capabilities." In other words, there is no risk of immune rejection, nor are there any ethical controversies surrounding the source of the stem cells, which are strictly autologous (in which the donor and recipient are the same person) adult stem cells, not embryonic stem cells.

GCSF (granulocyte colony-stimulating factor) is used in conjunction with the autologous adult stem cell therapy, to increase stem cell mobilization and migration from the bone marrow into the peripheral blood. Thus far, no side effects have been observed.

According to Dr. Jeffrey Karp of Harvard University, who is collaborating with Dr. Te Chung Lee at the State University of New York at Buffalo on similar studies, "Essentially if we know the zip code of vessels within a certain tissue, we can program the address on the surface of the cell." Speaking metaphorically, in reference to the "homing" ability of adult stem cells to target and repair damaged tissue, Dr. Karp is referring to the specific properties of mesenchymal stem cells (MSCs), which he and Dr. Lee have used in a number of related studies.

Dr. Losordo believes that the treatment could be made widely available in approximately 4 years, with a target patient population that consists of end-stage cardiac patients who have unsuccessfully exhausted all other available therapies – a population that has been estimated to consist of anywhere from one-third of a million to a million people. Dr. Lee, however, believes it could take as long as 5 years for such a treatment to become widely available, while Dr. Karp believes it could take as long as ten years. In any case, As Dr. Ronald Crystal, chief of the Division of Pulomary and Critical Care Medicine at New York Presbyterian Hospital/Weill Cornell Medical Center, adds, "This is a good idea, but patients and families should not expect immediate results."

Filed Under: Heart Failure, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
August 11, 2009 by

NIH Awards Stem Cell Grant for the Study of Autism

The National Institutes of Health (NIH) have announced the awarding of a $3 million grant to Children’s Hospital of Orange County (CHOC) in California to study stem cells in autistic children.

More specifically, ordinary fibroblasts (skin cells) will be derived from the autistic children, which will then be reprogrammed and de-differentiated into iPS (induced pluripotent stem) cells which in turn will be re-differentiated into neural cells. Scientists hope that properties of the neural cells will shed some light on the unique characteristics of autism, thereby ultimately leading to more efficacious forms of treatment for the disorder.

Funding from the NIH grant is to be distributed over 5 years and will result in the first repository of neural cells derived from living patients. Prior to the recent development of iPS technology, the only way to derived neural cells from someone was via autopsy after death. Now, however, any living person can easily donate an ordinary skin cell, which, from the intermediate stage of an iPS cell, can be reprogrammed into virtually any type of cell found within the human body.

The grant was awarded through the Eunice Kennedy Shriver National Institute of Child Health and Human Development. The specific recipient of the grant at CHOC is the National Human Neural Stem Cell Resource program, and the award constitutes the largest federally funded basic science research grant that any department within CHOC has ever received.

According to Dr. Philip Schwartz, principal investigator and founding director of the National Human Neural Stem Cell Resource at CHOC, "This is a completely novel approach to studying the neurobiology of autism and the first time we’ll be able to do so with neural cells actually derived from large numbers of children living with the condition. We hope to confirm over the next several years that neural cells generated from these donated fibroblasts can provide a viable experimental model that will yield insights about the origins, diagnosis, and treatment of autism."

Autism spectrum disorders (ASD) constitute the fastest growing developmental disability in the United States, with approximately one child in every 150 being diagnosed with some form of ASD. According to the Autism Society of America, the rate of diagnosis increases between 10 and 17% every year. Although an indisputable, definitive etiology has not yet been proven, a certain amount of controversy continues to surround ASD, as a growing amount of evidence seems to implicate environmental causes as the initiating trigger of ASD, especially heavy metals such as the mercury that is traceable directly to thimerosal. Also known as sodium ethylmercurithiosalicylate (C9H9HgNaO2S), thimerosal was originally developed as an antiseptic and antifungal agent but continues to be routinely used as a preservative in childhood vaccines. The controversy surrounding thimerosal, however, and its possible link to autism, has resulted in the gradual phasing out of thimerosal in the U.S., the European Union and a few other countries. Nevertheless, by 2007 it was estimated that more than 5,000 families in the U.S. have filed claims in a federal vaccine court alleging that their children became autistic as a result of having been vaccinated with thimerosal-containing vaccines. Although most of the cases are still being adjudicated, the U.S. federal government did award damages in one case, to the family of a young girl with the pre-existing condition of mitochondrial enzyme deficiency, who subsequently developed autism after receiving a series of thimerosal-containing vaccines. Many parents regard this ruling as confirmation that thimerosal is indeed a cause of autism.

A number of prominent scientists have researched the autism-vaccine connection extensively, perhaps the most notable of whom was Bernard Rimland, Ph.D., of San Diego, who founded the Autism Society of America in 1965 and who also founded the Autism Research Institute (ARI) in 1967, which he directed until his death from cancer in 2006. Inspired by his autistic son, Mark, who was born in 1956 when autism was quite rare, Dr. Rimland dedicated the remainder of his life to finding a scientific explanation and cure for the disorder. After more than 40 years of research, he became increasingly convinced that the causes of autism can be traced directly to the increasing number of vaccines that are prescribed in childhood. Dr. Rimland was one of the first scientists to correlate the increasing number of autism diagnoses per year to the presence of mercury in vaccines, pointing out that in 1990 autism was diagnosed at a frequency of one child per every 10,000 (ten thousand) children, whereas by 2004 the frequency had risen to one child per every 150 children, which represents an increase of 6,670%. A prolific author and energetic lecturer on autism, Dr. Rimland garnered the attention of Hollywood who invited him to serve as primary technical advisor to the 1988 movie "Rain Man", in which Dustin Hoffman’s character portrayed the disorder, thereby initiating a global awareness of autism at a time when it was not nearly as prevalent as it is today. Although Dr. Rimland was often at odds with the conventional medical establishment, to this day the ARI data bank, which Dr. Rimland created, remains the world’s largest data bank on autism, containing over 40,000 detailed case histories of autistic children from more than 60 countries. Dr. Rimland’s 1964 book, "Infantile Autism: The Syndrome and Its Implications for a Neural Theory of Behavior", remains a seminal, ground-breaking book in the field, and is credited by many with having changed the prevailing medical view of autism at that time from a disorder that was regarded as purely psychiatric in nature, to one that is now recognized as being distinctly biological.

Families of autistic individuals who are interested in donating cells to the CHOC for study under the NIH grant should contact CHOC directly for further information.

Filed Under: Autism, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
August 11, 2009 by

Cord Blood America Announces Opening of New Laboratory

Cord Blood America, one of the leading companies in the cryopreservation and storage of adult stem cells derived from human umbilical cord blood, today announced the scheduled opening of a major new facility later in the year. To be located in Las Vegas, the new 17,000 square foot laboratory will involve the conversion of an already existing structure and will include state-of-the-art technology for the storage of stem cells as well as further stem cell R&D.

According to Matthew Schissler, founding CEO of Cord Blood America, "Our target for having a cord blood processing lab in operation, a testing lab, and a cryogenic storage area, is the fourth quarter of 2009. This will complete Phase One of the construction. Currently architectural plans are completed, and needed demolition of parts of the interior building is underway. Phase Two will involve building out of the largest cryogenic facilities in the country, as well as a research laboratory, additional processing labs and a clean room. Completion is expected in 2010."

Founded in 2002 and headquartered in Santa Monica, California, Cord Blood America is one of the largest companies in the world in the field of stem cell preservation. In addition to the storage of adult stem cells derived from human umbilical cord blood, Cord Blood America is also expanding its R&D into the processing and storage of adult stem cells derived from other sources, such as human peripheral blood and adipose (fat) tissue.

Dr. Geoffrey O’Neill will serve as laboratory director of the new facility, while Brian Pockett will remain vice president of laboratory operations. Dr. O’Neill has coauthored more than 75 publications about stem cells, and was formerly the laboratory and scientific/medical director for Cryo-Cell International.

As described on their website, Cord Blood America is the parent company of CorCell, "which facilitates umbilical cord blood stem cell preservation for expectant parents and their children." As further described on their website, "Collected through a safe and non-invasive process, cord blood stem cells offer a powerful and potentially life-saving resource for treating a growing number of ailments, including cancer, leukemia, blood and immune disorders."

Filed Under: News, Stem Cells, Uncategorized Tagged With:
August 10, 2009 by

More Veterinary Success Seen With Adult Stem Cells

Even at five years of age, Lucy the Labradoodle was suffering from rheumatoid arthritis in her hind legs. But now, after receiving autologous adult stem cell therapy, she is showing significant improvement. According to her owner, Carol Fischman of Vero Beach, Florida, "We didn’t think she’d live anywhere near this long, and I know it’s because of the stem cells."

According to Dr. Kristin Kirkby, the veterinarian who performed the procedure, "I think it’s an exciting field. Undoubtedly the future of scientific research is going this way. It’s early on, especially on the small-animal side, to know what the results can be." But nevertheless, the results are consistently, dramatically, positve.

As previously reported a number of times on this website, an increasing number of veterinarians are finding success in the use of autologous (in which the donor and recipient are the same animal) adult stem cells for the treatment of a wide range of conditions in animals. Companies such as Vet-Stem in the U.S. and VetCell in the U.K. have developed procedures that are easily utilized by veterinarians and which are becoming increasingly popular as news of the success of such a technique continues to spread. The procedure that Dr. Kirkby used on Lucy the Labradoodle involved harvesting a small sample of the dog’s adipose (fat) tissue, which was then shipped to Vet-Stem’s laboratories in California where the dog’s own adult stem cells were isolated, purified and returned within 48 hours to Dr. Kirkby who administered the stem cells directly to the dog. So far Lucy has received 3 treatments with her own stem cells, and has shown such improvement that her owners are considering a fourth treatment.

Vet-Stem began treating animals in 2003, primarily horses for injuries and dogs for age-related osteoarthritis. To date Vet-Stem has now treated over 3,500 horses and 2,000 dogs, and the treatment of cats is planned for later this year. Approximately 1,500 vets throughout the U.S. are licensed by Vet-Stem to conduct the procedure. In all cases, adult stem cells are harvested from the animal’s own adipose tissue, which is a rich source of the highly potent adult stem cells known as mesenchymal stem cells. In no case are embryonic stem cells ever used, since embryonic stem cells remain highly problematic and are known to cause teratomas (tumors), among other problems, which therefore disqualifies embryonic stem cells as a treatment for animals as well as for people.

According to Dr. Bob Harman, a veterinarian and founding CEO of Vet-Stem, "Really, all we’re doing is harnessing the existing repair machinery in the body, concentrating it, and putting it right where an injury occurs, where healing is needed, to heal naturally."

Adipose-derived stem cells have been shown in a number of studies to exhibit highly beneficial immunomodulatory properties – which reduce inflammation, among other benefits – in addition to stimulating the regeneration of cartilage and other tissue, and such properties are well documented in the medical literature. (E.g., "Non-expanded adipose stromal vascular fraction cell therapy for multiple sclerosis", by N.H. Riordan et al., published in the Journal of Translational Medicine in April of 2009, of which Dr. Harman is a coauthor). As Dr. Harman further explains, "In the last couple of years, evidence has come out that the cells we use reduce inflammation and pain, and help lubricate the joint." Ordinarily, injuries of the bones, joints, tendons and ligaments result in scarring of the tissue, which not only prevents full healing but also often leads to further injuries at a later time. Conventional medical therapies do nothing to address the problem of scar tissue directly, and surgical procedures actually make the problem worse by increasing the severity of tissue scarring which in turn merely exacerbates later complications that will inevitably result from the scar tissue, since such tissue can never be fully rehabilitated. Stem cell therapy, however, allows for the full and complete healing of tissue without scarring, which not only reduces the risk of re-injury of the same tissue at a later date but also restores full physical performance and function, usually very quickly and dramatically. Such is the case in humans as well as in animals. As Dr. Harman succinctly states, "Our success in animals is directly translatable to humans, and we wish to share our evidence that stem cells are safe and effective."

Although Vet-Stem was the first company to commercialize the process in the U.S., and VetCell was the first to do so in the U.K., a number of other companies throughout the world are now also utilizing similar types of technology in which adult stem cells are derived from each animal’s own tissue and readministered to the animal as a clinical therapy for the particular medical condition from which the animal suffers. Autologous adult stem cell therapy has proven to be a highly preferable alternative treatment for many animals, especially those whose conditions require surgery or anti-inflammatory drugs, both of which can often be avoided with the stem cell therapy.

Dr. Adam Gassel, a veterinarian in Irvine, California, has treated nearly 40 dogs with Vet-Stem’s procedure and is now a strong believer in the therapy, despite his initial skepticism. As Dr. Gassel explains, "I was pretty skeptical. I was hoping that dogs would just be more comfortable." But of all of his canine patients who have received the adult stem cell treatment, 80% have shown significant improvement, approximately half of whom have been able to stop taking medication, and approximately a fourth of whom have completely returned to their normal activities.

The procedure that Vet-Stem has literally distilled to a science is quick, simple, minimally invasive, safe, highly effective, and while it is not cheap, it is less expensive than conventional surgical and pharmaceutical therapies which may not be effective at all. The first stem cell extraction and transplant for a dog typically run between $1,500 and $2,500, although subsequent transplants will often cost much less since extraction is only necessary once. The entire stem cell extraction procedure consists of the approximate equivalent of 2 to 3 tablespoons of the animal’s own adipose (fat) tissue which is surgically removed under anesthesia and shipped overnight to Vet-Stem’s laboratories in southern California where the stem cells are processed and returned two days later to the veterinarian who injects the stem cells back into the animal at the site of injury. Not only do the stem cells automatically target the injured tissue, but they also stimulate the animal’s other endogenous stem cells which in turn are mobilized into action and participate in the healing and repair process. Although improvements are usually dramatic and immediate, even after the first injection, additional injections may be necessary, depending upon the age and particular condition of the animal. Very few animals ever need more than a total or 2 or 3 treatments, however, before they are fully restored to their natural, pain-free state of mobility – which contrasts sharply with conventional therapies such as most prescription medications which may need to be taken indefinitely, without ever producing any tangible signs of improvement and while even possibly causing further damage to the animal through dangerous side effects and other associated risks.

It is worth emphasizing the point that Vet-Stem uses exclusively adult stem cells, derived from each animal’s own tissue. Since the cells are autologous (in which the donor and recipient are the same animal), there is no risk of immune rejection. More specifically, the stem cells that are harvested in Vet-Stem’s procedure are mesenchymal stem cells, which are highly potent adult stem cells that are also found in bone marrow and umbilical cord blood. Numerous scientific and clinical studies have been published in the peer-reviewed medical literature detailing the regenerative properties of mesenchymal stem cells.

No embryonic stem cells are ever used in Vet-Stem’s therapies, since embryonic stem cells are highly problematic in the laboratory, whether they are of human or non-human origin. Among other problems, the risk of teratoma (tumor) formation disqualifies embryonic stem cells for use as a clinical therapy, whether for humans or animals. Adult stem cells, however, do not pose such risks and are therefore rapidly accumulating a consistent history of successful clinical treatments in veterinary, as well as in human, medicine.

A number of companies throughout the world are replicating the procedure pioneered by Vet-Stem. It is fortunate that such companies are able to conduct their business without the same burdensome federal legislation that continues to impede progress in human medical adult stem cell therapies in the United States. Consequently, veterinary stem cell therapy has been applied very aggressively to animals, especially to the expensive, large animals such as competitive horses whose lives and careers have literally been saved by such therapies. Even for dogs who do not earn large salaries in high-profile competitions but who are merely beloved pets, autologous adult stem cell therapy has also proven to be life-saving. Meanwhile, in human medicine, however, nothing whatsoever has been allowed to happen in U.S. clinics outside of a small number of government-approved clinical trials, thanks to an outdated, lengthy, lethargic and prohibitively expensive FDA approval process. It is hardly surprising, therefore, that veterinary stem cell medicine has quickly outpaced human stem cell medicine – but now, at last, humans are beginning to learn something from their canine and equine friends.

And the market is huge. Both for humans as well as for animals, the potential commercial market is virtually limitless and thus far untapped. According to Dr. Charles Fischman, an immunologist and one of the owners of Lucy the Labradoodle, "I like the dog as much as I like my kids. People will spend more on their dogs than they will on themselves."

Filed Under: Adult Stem Cells, Multiple Sclerosis, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , , ,
August 10, 2009 by

Clinical Trial Uses Adult Stem Cells to Treat Diabetes

An official FDA-approved clinical trial is currently underway at the School of Medicine and Public Health at the University of Wisconsin at Madison, for the use of adult stem cells in the treatment of Type 1 diabetes. Specifically, the Phase II study is testing both the safety and efficacy of Prochymal, a proprietary mesenchymal stem cell (MSC) formulation manufactured by the company Osiris Therapeutics.

The Phase II, randomized, double-blind, placebo controlled, parallel assignment, multi-center study is being concurrently conducted at 20 medical centers nationally. The study has a target enrollment of 60 newly diagnosed patients between the ages of 18 and 30. Patient monitoring will be conducted during a follow-up period lasting two years. The study is co-sponsored jointly by Osiris Therapeutics and the Juvenile Diabetes Research Foundation.

John Markwardt, a 20-year-old UW student who was just diagnosed with Type 1 diabetes in the spring of this year, became the first patient to be treated with Prochymal in the study. His first infusion took place in July, and the second infusion is scheduled for later this month. The study is specifically targetted at newly-diagnosed Type 1 diabetes patients, in whom the pancreatic beta cells have not yet been completely destroyed.

According to Dr. Melissa Meredith, associate professor of endocrinology at UW and the principal investigator of the study, "The reason we have focused on people recently diagnosed is they still have some beta cells capable of making insulin. If we can stop that immune destruction, they still have the ability to make some insulin. We also know beta cells have the ability to regenerate. Even if they aren’t totally off insulin, it’s a better way to control the disease when they aren’t reliant on injected insulin and are making more of it themselves. The reason I am excited about this is that it is the heart of what causes diabetes. The hope is it will preserve the ability that is left."

Also collaborating with Dr. Meredith in the study is Dr. Peiman Hermatti, assistant professor of hematology and oncology at UW, who has previously used bone-marrow-derived MSCs in the treatment of graft-versus-host disease.

According to the website of the National Institutes of Health (NIH), "The objective of the present study is to establish the safety and efficacy of multiple administrations of Prochymal in subjects recently diagnosed with Type 1 diabetes mellitus." As Dr. Paul Strumph, chief medical officer of the Juvenile Diabetes Research Foundation, adds, "This therapy is aimed at decreasing the immune response, which we know is involved in the progression of Type 1 diabetes. It’s very promising."

As further described on the website of NIH, this study utilizes an "intravenous infusion of ex vivo cultured adult human mesenchymal stem cells" (MSCs). As described on the website of Osiris Therapeutics, Prochymal consists of a proprietary formulation of human MSCs that "are derived from bone marrow aspirate obtained from qualified donors. … Upon arrival at Osiris, the MSCs are isolated and selectively removed from the bone marrow aspirate. These cells are then expanded, harvested, packaged and cryopreserved as an in-process intermediate, where a second series of tests ensure the highest level of quality and safety. … All manufacturing activities are performed in compliance with the Food and Drug Administration’s current Good Manufacturing Practices (cGMP) standards… Osiris Therapeutics, Inc., adheres to strict FDA regulations in the production and formulation of our stem cell treatments to ensure that our products are of the highest quality and safety."

Often featured in the news, Osiris Therapeutics is a prominent leader in the field of adult stem cell therapies and is focused on the development of products for the treatment of inflammatory, orthopedic and cardiovascular diseases. Osiris was founded in 1992 for the specific purpose of developing and commercializing the innovative technology for isolating and expanding bone-marrow-derived MSCs, a process originally pioneered by Dr. Arnold Caplan and his colleagues of Case Western Reserve University. Since MSCs are "immune privileged", "universal donor" cells, they do not need to be administered autologously (in which the donor and recipient are the same person), since even allogeneically (in which the donor and recipient are not the same person) these highly potent adult stem cells do not pose any risk of immune rejection. In addition to other products in its pipeline, Osiris currently has a number of clinical trials under way for both Prochymal and its other leading adult stem cell product, Chondrogen.

In November of last year, Osiris formed a strategic alliance with the biotech company Genzyme that was valued at over $1.3 billion. In 2007, the two companies were awarded a $224.7 million contract from the U.S. Department of Defense for the development of Prochymal in the treatment of radiation sickness.

As Dr. Hans Klingemann of Tufts Medical Center explains, "MSCs have the ability to interpret injury within the body and respond accordingly, giving them exceptional therapeutic potential to treat a variety of disorders. And, they can be administered quickly, where and when patients need them."

Formerly known as juvenile diabetes or insulin-dependent diabetes, Type 1 diabetes is a chronic, life-threatening condition for which conventional medicine has no known cure. Though not as common as Type 2 diabetes, Type 1 diabetes is characterized by the autoimmune destruction of the beta islet cells of the pancreas, which are required to produce insulin. Left untreated, both types of diabetes can lead to serious complications such as stroke, blindness, heart disease and peripheral neuropathy which in its most advanced form is treatable only by amputation. Together, Types 1 and 2 diabetes constitute the seventh most prevalent cause of death in the United States.

Now, for the first time, adult stem cell therapy may offer the first type of treatment which not only reverses the course of the disease but also regenerates damaged pancreatic tissue.

This FDA-approved clinical trial is currently still recruiting patients. Interested parties should contact Osiris Therapeutics directly for eligibility information.

Filed Under: Heart Failure, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
August 10, 2009 by

BioTime Appoints New V.P. of Stem Cell Research

The California-based biotech company, BioTime Inc., has announced the appointment of Dr. Walter Funk as Vice President for Stem Cell Research. Dr. Funk joins the upper management team of BioTime’s Human Embryonic Stem Cell Research and Product Development section, where he will participate in the formulation of policies that govern the scientific R&D of the company.

According to the official press release, "Dr. Funk is an experienced biotechnology professional, having held both senior technical and executive research positions over the past 15 years. He trained in biochemistry (Ph.D., University of British Columbia) and has published over 45 peer-reviewed journal articles. He was one of the first scientists to join Geron, and participated in the isolation of the telomerase gene which allows certain cells, such as embryonic stem cells, to proliferate without aging. Later at Geron, he worked with the world’s first human embryonic stem cell lines and provided the first detailed description of their gene expression patterns through a collaboration with Celera Corporation. Dr. Funk subsequently held the position of V.P. Research at Nuvelo, where he led a team developing human biotherapeutics. Most recently, Dr. Funk co-founded Parallax Venture Partners, a seed-stage venture capital group, and sits on the boards of Gene Oracle, a provider of synthetic gene technologies and services, and Phyllom, an agricultural biotech product development company."

As Dr. Funk formally stated, "I am excited to join the BioTime team at such an important time in the company’s development. The development of the ACTCellerate embryonic progenitor cell technology represents a fundamental advancement of cell therapeutic strategies, providing for the first time an elegantly simple means of scaling up commercial quantities of highly purified human cell types. I look forward to helping BioTime execute a strategic plan to push this technology towards commercialization. Our recent award of a prestigious CIRM grant will help BioTime and its collaborators make rapid progress toward this goal."

As the CEO of BioTime, Michael West, Ph.D., announced, "We couldn’t be more enthusiastic about Dr. Funk’s decision to join the BioTime management team. He has a track record of consistently providing critical scientific expertise and executive acumen. His expertise in genomics will help us reach our goal of leading the emerging industry of regenerative medicine by providing the first detailed gene expression map of BioTime’s hundreds of proprietary cell lines."

Headquartered in Alameda, California, BioTime describes itself as "a biotechnology company focused on regenerative medicine and blood plasma volume expanders" with applications in surgery and emergency trauma. Through its wholly owned subsidiary, Embryome Sciences, BioTime is focused on the research, development and ultimate commercialization of clinical therapies derived from human embryonic stem cells.

BioTime’s stock closed today at $3.35.

Filed Under: News, Stem Cells, Uncategorized Tagged With:
August 8, 2009 by

Adult Stem Cells Heal Arthritic Dog

Olli, a 12-year-old Gordon Setter in Ontario, was suffering from arthritis in his left knee and right hip. Within eleven days of receiving injections of his own adult stem cells, however, Olli is now showing significant improvement.

His vet, Dr. Melissa Boyle, is one of 26 veterinarians in Canada who have been qualified by the U.S. company Vet-Stem to administer the therapy, in which adult stem cells are derived from each animal’s own adipose (fat) tissue.

According to Dr. Deborah Boyd – Olli’s owner, a vet herself and the owner of the Grey Bruce Pet Hospital where Olli was treated – conventional veterinary therapies were ineffective in treating Olli and in fact his condition only worsened in response to such therapies. Now, after having tried adult stem cell therapy on Olli, Dr. Boyd is so pleased with the results that she asked her own physician why the same type of autologous adult stem cell therapy is not available for people. In response, as Dr. Boyd explains, "She just looked at me and said, ‘You veterinarians, you’re 10 years ahead of us.’"

Although the cost of the veterinary stem cell treatment runs between $1,500 and $2,500, conventional veterinary knee surgery for a dog of Olli’s size would have cost between $1,500 and $3,000 or more and also would have required expensive and dangerous medications for an indefinite period of time. Furthermore, the success rate of conventional surgeries and medications is much lower than that of adult stem cell therapy. Additionally, should the animal need future stem cell treatments, Vet-Stem’s fees also include banking services for 4 more doses of the animal’s stem cells at Vet-Stem’s laboratories in San Diego for the next year, after which time customers have the option of continuing to bank the cells at an annual fee of $120.

Dr. Thomas Koch, a researcher at the University of Guelph, recently received a 3-year post-doctoral fellowship worth more than $1 million for the research and development of adult stem cell therapies in the treatment of cartilage injuries in horses. (Please see the related news article on this website, entitled, "Canadian University Announces Major Adult Stem Cell Research Award on Horses", dated July 18, 2009). According to Dr. Koch, who was asked to comment on Vet-Stem’s therapy for dogs, "There doesn’t seem to be any adverse effects."

Indeed, as previously reported a number of times on this website, the company Vet-Stem continues to see consistently high success rates in both canine and equine clinical applications, with an 80% efficacy rate and a 100% safety rate in the animals that are treated with Vet-Stem’s autologous adult stem cell procedure. In other words, 80% of the animals treated are found to experience improvement in their condition with a reduction and often a full elimination of the need for medication, while adverse side effects have not been reported in any of the treated animals. Now, such applications are being repeated by other veterinarians and clinics around the world.

Companies such as Vet-Stem in the U.S. and VetCell in the U.K. have accumulated numerous documented cases of the benefits of autologous adult stem cell therapy in animals. To name just a few of the advantages, adult stem cell therapy yields faster healing and shorter recovery times than surgical treatments do, and adult stem cell therapy does not pose a risk of any side effects like medications do. Additionally, since the adult stem cells are autologous, there is no risk of immune injection. The U.K. company VetCell derives the autologous adult stem cells from the animal’s bone marrow, and to date has treated approximately 1,700 horses with an 80% success rate. By comparison, the U.S. company Vet-Stem derives the autologous adult stem cells from the animal’s adipose (fat) tissue, and to date has treated over 2,000 dogs and over 3,000 horses, also with an 80% success rate. With both companies, the procedure is quick, simple, and minimally invasive. Although the treatment is more expensive than conventional veterinary procedures, the adult stem cell treatment actually works, and noticeable improvement is seen almost immediately in all cases, not just in the 80% of cases that exhibit a complete recovery. By sharp contrast, however, conventional surgical and pharmacological therapies, which might initially be less expensive than stem cell therapy, only have a 30% success rate and therefore in the long-term are actually more expensive when repeated treatment is needed, or when improvements are not seen at all. Additionally, reinjury is significantly lower in animals who receive autologous adult stem cell therapy, due to the mechanism of action by which these stem cells activate the healing process. As Dr. David Mountford, a veterinary surgeon and chief operating officer at VetCell, explains, "After 3 years, the reinjury rate was much lower in stem-cell-treated animals: about 23% compared with the published average of 56%" for animals treated with conventional therapies.

Not only do the stem cells automatically target the injured tissue, but they also stimulate other endogenous stem cells which in turn are mobilized into action and participate in the healing and repair process. Although improvements are usually dramatic and immediate, even after the first injection, additional injections may be necessary, depending upon the age and condition of the animal. Very few animals ever need more than a total or 2 or 3 treatments, however, before they are fully restored to their natural, pain-free state of mobility – which contrasts sharply with conventional therapies such as most prescription medications which may need to be taken indefinitely, without ever producing any tangible signs of improvement and while even possibly causing further damage to the animal through dangerous side effects and other associated risks.

Ordinarily, injuries of the bones, joints, tendons and ligaments result in scarring of the tissue, which not only prevents full healing but also often leads to further injuries at a later time. Conventional medical therapies do nothing to address the problem of scar tissue directly, and surgical procedures actually make the problem worse by increasing the severity of tissue scarring which in turn merely exacerbates later complications that will inevitably result from the scar tissue, since such tissue can never be fully rehabilitated. Adult stem cell therapy, however, allows for the full and complete healing of tissue without scarring, which not only reduces the risk of re-injury of the same tissue at a later date but also restores full physical performance and function, usually very quickly and dramatically. Such is the case in humans as well as in animals. According to Dr. Robert Harman, veterinarian and founding CEO of Vet-Stem, "Our success in animals is directly translatable to humans, and we wish to share our evidence that stem cells are safe and effective." Additionally, adipose-derived stem cells have been shown in a number of studies to exhibit highly beneficial immunomodulatory properties – which reduce inflammation, among other benefits – in addition to stimulating the regeneration of cartilage and other tissue. (E.g., "Non-expanded adipose stromal vascular fraction cell therapy for multiple sclerosis", by N.H. Riordan et al., published in the Journal of Translational Medicine in April of 2009, of which Dr. Harman is a coauthor). As Dr. Harman further adds, "In the last couple of years, evidence has come out that the cells we use reduce inflammation and pain, and help lubricate the joint. About 200,000 hip replacements are done every year in humans. That’s a very good target for someone to look at cell therapy."

Vet-Stem uses exclusively adult stem cells, derived from each animal’s own tissue. Since the cells are autologous (in which the donor and recipient are the same animal), there is no risk of immune rejection. More specifically, the stem cells that are harvested in Vet-Stem’s procedure are mesenchymal stem cells, which are highly potent adult stem cells that are also found in bone marrow and umbilical cord blood. Numerous scientific and clinical studies have been published in the peer-reviewed medical literature detailing the regenerative properties of mesenchymal stem cells.

No embryonic stem cells are ever used in Vet-Stem’s therapies, since embryonic stem cells are highly problematic in the laboratory, whether they are of human or non-human origin. Among other problems, the risk of teratoma (tumor) formation disqualifies embryonic stem cells for use as a clinical therapy, even in animals. Adult stem cells, however, do not pose such risks and are therefore rapidly accumulating a consistent history of successful clinical treatments in veterinary, as well as in human, medicine.

As numerous reports about more and more pioneering canine patients such as Olli continue to make their way into the veterinary literature, perhaps humans could also learn to benefit from such reports. After all, with enough patience, perseverance, and repetitive training, perhaps it might actually be possible for a few dogs to teach a few old humans some new stem cell tricks.

Filed Under: Adult Stem Cells, Multiple Sclerosis, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , , ,
August 7, 2009 by

Novartis Acquires Opexa’s Adult Stem Cell Technology

The biotech companies Novartis and Opexa Therapeutics have signed an agreement in which Novartis will pay $4 million for Opexa’s novel adult stem cell technology.

The deal includes $3 million paid up-front, with an additional $1 million in fees that will be distributed over 6 months. Additional future milestone payments are estimated to exceed $50 million, not including royalties.

Novartis will be responsible for the funding of all R&D as well as commercial activities of Opexa’s technology, which enables the production of monocyte-derived stem cells (MDSCs) from blood. Currently the technology is in preclinical development where it was been shown to generate MDSCs from peripheral blood mononuclear cells.

As described on their website, "Opexa is focused on the development of patient-specific cellular therapies for the treatment of autoimmune diseases including multiple sclerosis and diabetes. The company’s lead product is Tovaxin, a T-cell MS vaccine that is specifically tailored to each patient’s disease profile. In October 2008 the company reported positive top-line results from a Phase IIb study in early, relapsing MS patients."

Formed in 1996 through the merger of the two companies Ciba-Geigy and Sandoz, Novartis is one of the world’s largest multinational corporations and a leader in healthcare, offering products and services in pharmaceuticals, vaccines and diagnostics. In 2008, Novartis invested $7.2 billion in its R&D, and that same year the company was ranked number 2 in Fortune magazine’s "World’s Most Admired Companies" survey. Novartis currently employs approximately 100,000 people in 140 countries worldwide.

Filed Under: Adult Stem Cells, Multiple Sclerosis, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , , ,
August 6, 2009 by

Athersys Seeks Commercial Partners

The biopharmaceutical company Athersys is looking for commercial partners in the R&D of its various therapies, which include conventional drug therapies as well as some novel stem cell therapies.

According to Athersys chairman and CEO, Dr. Gil Van Bokkelen, who spoke to analysts during a company conference call, Athersys currently has the cash equivalents of $25.3 million, which is enough to keep the company operating through 2011. Nevertheless, the company would like to add to its capital base by partnering with other companies in the completion of the testing and development of therapeutic product lines.

The main product in the company’s stem cell line, known as MultiStem, is an off-the-shelf therapy that Athersys is currently testing for the treatment of heart attack, stroke, and graft-versus-host disease. As Dr. Van Bokkelan explained during the conference call, "We believe these and other capabilities will allow them to be relevant in a number of therapeutic areas, and we and our collaborators are excited about the potential utility of MultiStem for treating major conditions." Unlike many other types of stem cell therapies, which are designed "to achieve wholesale replacement of damaged tissue", Dr. Van Bokkelan described MultiStem as exhibiting "a more drug-like profile in which cells augment healing and exert a benefit in multiple ways, and then are cleared from the body over time." He added, however, that the wide range of applications for MultiStem "represent a development challenge, especially for an emerging biopharmaceutical company," which is why Athersys has decided to seek commercial partnerships. As Dr. Van Bokkelan further adds, "Accordingly, we believe that the optimal way to advance our MultiStem program is to find a significant commercial partner."

Under Dr. Van Bokkelan’s leadership, Athersys has been exploring partnership opportunities for months and is "greatly encouraged by the ongoing level of interest among potential partners." As he further describes, "We are confident that we will be able to secure strategic partnerships that will enable us to advance our MultiStem programs in a meaningful and substantial way."

Located in Cleveland, Ohio, Athersys is also currently developing two separate drug candidates for the treatment of obesisty and problems in cognitive attention, both of which deal with the chemical regulation and control of neuroreceptors. Even in this field, Dr. Van Bokkelan finds that, "Once again, we are greatly encouraged by the level of interest among potential partners in both areas, and believe we are in a strong position to construct relationships with leading companies that share our vision of developing best-in-class therapeutics."

As described on their website, "Athersys is a clinical stage biopharmaceutical company with a growing pipeline of highly differentiated, potential best-in-class therapeutics to treat significant and life-threatening diseases. … Our current product development portfolio consists of MultiStem, a patented and proprietary stem cell product that we are developing as a treatment for multiple disease indications, and that is currently being evaluated in two Phase I clinical trials. In addition, we are developing novel pharmaceuticals to treat indications such as obesity, as well as certain conditions that affect cognition, attention, and wakefulness. Our strategic approach to drug development builds on internally and externally generated knowledge to identify and develop proprietary and highly differentiated products, as well as enable the company to limit development risks and costs."

As further described on the website of Athersys, "MultiStem is a biologic product that is manufactured from human stem cells obtained from adult bone marrow or other nonembryonic tissue sources. The product consists of a special class of human stem cells that have the ability to express a range of therapeutically relevant proteins and other factors, as well as form multiple cell types. Factors expressed by MultiStem have the potential to deliver a therapeutic benefit in several ways, such as the reduction of inflammation, protection of damaged or injured tissue, and the formation of new blood vessels in regions of ischemic injury. These cells exhibit a drug-like profile in that they act primarily through the production of factors that regulate the immune system, protect damaged or injured cells, promote tissue repair and healing and most or all of the cells are cleared from the body over time."

One of the co-founders of Athersys, Dr. Van Bokkelan has served as CEO of the company since its founding, as well as the president of Athersys prior to 2006. Dr. Van Bokkelan holds a double B.A. in Economics and Molecular Biology from the University of California at Berkeley, and a Ph.D. in Genetics from Stanford.

Filed Under: Heart Failure, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
August 6, 2009 by

Proteomics Applied to Stem Cells

Scientists at The Scripps Research Institute and the Burnham Institute for Medical Research have just completed a large-scale comparative phosphoproteomic analysis of human embryonic stem cells (hESCs). The accomplishment is the first of its kind and also includes a detailed analysis of differentiated derivatives of hESCs. Data from the study may ultimately allow for greater control over the molecular mechanisms that determine whether or not stem cells differentiate into more specialized cells, or merely divide without differentiating.

Protein phosphorylation, a biochemical process which is essential for cell signaling, involves the modification of protein activity via the addition of a phosphate molecule. It is one of the most widely studied aspects of protein modification and commonly employed as a fundamental tool of proteomics. A logical extension of "genomics", proteomics is defined as the large-scale study of proteins, and in particular of the mechanisms by which the 3-dimensional structure of any particular protein influences biochemical function and activity. Similar to the concept of a genome, the "proteome" consists of the entire complement of proteins which any particular biological system or organism produces.

Combining both areas of specialization, the scientists in the current study used methods of "phosphoproteomic" analysis to catalogue 2,546 phosphorylation sites on 1,602 phosphoproteins, the identification of which has now shed new light on signaling pathways. Follow-up experiments further confirmed specific phosphoproteins and cell pathways that the researchers had identified using the phosphoproteomic data as a predictive tool in target samples. Among other findings, the scientists observed the various roles that phosphorylation plays in determining cell fate, especially in the phosphorylation of various kinases as well as specific transcription regulators such as epigenetic and transcription factors. In particular, proteins that are involved in the JNK (Jun N-terminal kinase) signaling transduction pathway were observed to be phosphorylated in undifferentiated hESCs, a finding which would suggest that JNK signaling inhibition may be involved in cell differentiation, as independently confirmed by other hESC studies. Additionally, phosphoproteins involved in the RTK (receptor tyrosine kinase) signaling pathways were found to be especially numerous in undifferentiated hESCs, and follow-up studies demonstrated that multiple RTKs can maintain hESCs in an undifferentiated state.

According to Dr. Laurence Brill, a member of the team of researchers who conducted the study, "This research will be a big boost for stem cell scientists. The protein phosphorylation sites identified in this study are freely available to the broader research community, and researchers can use these data to study the cells in greater depth and determine how phosphorylation events determine a cell’s fate."

Prior to this particular study, protein phosphorylation was poorly understood in hESCs, and even though the process is still not yet fully elucidated, the study nevertheless demonstrates the utility of phosphoproteomic data as a predictive and analytic tool in the determination of cell fate, at least within hESCs. Extrapolation to mechanisms of differentiation and cell fate in adult stem cells should prove to be of even greater and more immediate clinical utility, however, since adult stem cells are already being used in the treatment of a wide variety of diseases and injuries, unlike hESCs, which have yet to advance beyond the experimental laboratory stage.

Along with Dr. Laurence Brill, the team of scientists who conducted the study also include Drs. Sheng Ding and Evan Y. Snyder.

Filed Under: News, Stem Cells, Uncategorized Tagged With:
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