August 24, 2009 by

New Adult Stem Cell Facility Opens in Maryland

RNL Biostar Inc., a subsidiary of the Seoul-based company RNL Bio Inc., has been based in Rockville, Maryland since 2006. Now, in addition to its facility at the Technology Development Center, it plans to add a new facility in Germantown, worth $6 million, to which the company also plans to add 50 newly hired employees over the next 3 to 5 years. The new positions will include manufacturing technicians and lab personnel in addition to administrative personnel. According to Donna Lee, director of business development at RNL, "There will be pretty big growth spurts because of the amount of work we have."

The new RNL facility will be converted from an already existing structure, which currently occupies nearly 10,000 square feet and comprises a combination of labs and office suites. RNL Biostar projects that its facilities will occupy 20,000 square feet by 2014.

Currently RNL is in Phase II clinical trials for the testing of its proprietary adult stem cell products for the treatment of osteoarthritis and Buerger’s Disease, which is an inflammatory and blood clotting condition. Additionally, the company has received FDA authorization to commence clinical trials with its adult stem cell product in the treatment of spinal cord injury, which are scheduled to start at the end of the year.

RNL’s proprietary products are formulated from adult stem cells that are harvested from adipose (fat) tissue. According to Ms. Lee, "In Maryland, the fat samples come to our lab here first. People send in their fat and we extract stem cells out of the fat and ship them in nitrogen tanks to Korea, where cells are cultured and multiply. We have helped a lot of people with rheumatoid arthritis with a one-time IV injection. It’s really amazing to watch."

However, the U.S. citizens who donated their own adipose-derived adult stem cells must travel to China or Japan for the injections, since such treatments are not allowed in the U.S., due to a severely antiquated FDA.

RNL has also developed a stem-cell-based hand cream.

Filed Under: News, Spinal Cord Injury, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
August 23, 2009 by

India Boasts Profitable Adult Stem Cell Market

In sharp contrast to the regulatory setbacks experience in other nations, such as the U.S., India offers a commercially and legally attractive environment for adult stem cell entrepreneurs.

At last count, at least 15 new firms are scheduled to enter the Indian market, and 4 already existing firms are planning major expansion. In the latter category are Reliance Life Sciences, LifeCell International, Regenerative Medical Services, and Cryo-Save India. Though each of these companies is already supplying cell cultures to select hospitals, they are all planning even further expansion.

According to the Indian Council for Medical Research (ICMR), the 15 new firms who are scheduled to enter India’s market include private commercial ventures as well as academic research institutions. Additionally, a number of organizations are conducting clinical trials with adult stem cells in India, including but not limited to Stempeutics, the LV Prasad Eye Institute, the Nichi-In Centre for Regenerative Medicine, the Centre for Cellular and Molecular Biology, the National Centre for Cell Science, and Manipal Hospital in Bangalore, among others. According to the ICMR, at least 60 adult stem cell clinical trials are currently underway in India, most of which involve autologous (in which the donor and recipient are the same person) adult stem cells, and all of which are approved by the Drug Controller General of India. The entire field of adult stem cell therapy has enjoyed a new boost in India as a result of the recent decision by the Indian government to allow the patenting of micro-organisms and related laboratory processes.

According to Mayur Abhaya, executive director of LifeCell International, based in Chennai, the greatest therapeutic value thus far has been found in the type of adult stem cell known as mesenchymal stem cells (MSCs), which are already being used by doctors in hospitals in India to treat a wide variety of ailments, which include diabetes, heart disease, stroke, arthritis, multiple sclerosis, spinal cord injury, and blood disorders, to name a few.

LifeCell, which is currently the leading company in India for the storage of umbilical cord blood stem cells, has already banked cord-blood-derived stem cells for more than 12,000 clients. The company, which is also collaborating with Cryo-Cell in the U.S., is now planning to expand into the collection and storage of adult stem cells derived from bone marrow aspirate. Additionally, LifeCell has also begun clinical trials with Harvest Therapeutics in the treatment of critical limb ischemia with adult stem cells, and is planning further expansion into the storage of menstrual blood, now known to be a rich source of highly potent adult stem cells. As Mayur Abhaya further explains, "A few more clinical trials are being planned in other disease settings, apart from the proposed collaboration with Cryo-Cell to launch the menstrual blood stem cell banking service in India."

With a population of approximately 1.2 billion people, India ranks as the second most populous nation on earth, and the world’s most populous democracy. Second only to China, whose population is estimated at 1.3 billion people, India actually offers a larger and freer commercial market than can be found in China, which is still formally Communist and is therefore still highly restricted in the nature and extent of its commerce. It is hardly surprising, therefore, that entrepreneurs of all types eventually cast a serious eye toward India. Especially with the outdated federal regulatory laws that still govern medical research in other countries, such as the U.S., it is only logical and to be expected that adult stem cell companies would discover in India a welcoming atmosphere in which they are able to thrive.

Filed Under: Adult Stem Cells, Multiple Sclerosis, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , , ,
August 20, 2009 by

Adult Stem Cells Perk Up Pooch

Micki, an 11-year-old German Shepherd in Vancouver, Canada, had developed age-related osteoarthritis in 3 legs. As a result, the dog underwent a ligament operation that was not entirely successful and was accompanied by daily, heavy doses of anti-inflammatory medication, prescribed for indefinite use. Occasionally, Micki was in so much discomfort that a leg brace was required. When a second operation was prescribed for the ligament of a different leg, the dog’s owner, Sandy Draibye, a lawyer, decided that enough was enough. Instead of consenting to further surgery for her dog, Sandy opted for autologous adult stem cell therapy instead.

As Micki’s vet, Amanda Booth, explained, "This is her kid." Although Dr. Booth was personally unfamiliar with the stem cell treatment, she agreed to learn how to administer it anyway. She then harvested adipose (fat) tissue from the dog’s thigh and sent the tissue to the U.S. company Vet-Stem in California, where the stem cells are isolated and returned to Dr. Booth within 48 hours for injection directly into the dog’s injured ligaments and joints.

According to Dr. Booth, "If the other three legs had been in good shape, I would have held off on the stem cell surgery. I researched it before I agreed to do it. My first criterion was finding someone other than the company that says it works." After receiving a number of enthusiastic responses from other vets, Dr. Booth decided to take the plunge and begin acquiring her own experience with the procedure.

As previously reported a number of times on this website, the U.S. 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.

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 dramatically 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 and veterinary 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.

According to Sandy Draibye, owner of Micki, the 11-year-old German Shepherd, "It’s a lot of money, but she’s a lot of dog. I am not wealthy, but I can afford it. Everybody adores their dog. I don’t think they would give it a second thought."

In fact, as Sandy points out, most humans are so impressed by the news of such dramatic recovery in animals that people are becoming increasingly curious about the use of autologous adult stem cell therapy for the treatment of human ailments. As Sandy adds, "They’re wondering about their own joints."

Filed Under: Adult Stem Cells, Multiple Sclerosis, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , , ,
August 18, 2009 by

Adult Stem Cells Treat Heart Failure

At 68 years of age, Brent Benson was suffering from heart failure. As a biochemist who previously conducted tests for the Environmental Protection Agency, Benson was no stranger to physiology and medical science. By his own assessment, he says, "I’m essentially dying of heart failure. I was so far down, I had to try."

Now, however, approximately 5 months after receiving autologous adult stem cell therapy, Benson describes himself very differently. "My life is full and busy," he says. "I had lost that prior to this operation."

Autologous (in which the donor and recipient are the same person) adult stem cell therapy is catching on. Increasingly used in the treatment of a wide variety of diseases and injuries throughout the world, the procedure has shown such consistent success in patient improvement that the news finally seems to be spreading.

In Brent Benson’s particular case, he was able to participate in a clinical trial conducted at the University of Utah in Salt Lake City. After his own adult stem cells were extracted from his bone marrow via his hip, the cells were processed in a laboratory and later injected directly into his heart. He received a total of 30 injections, after which time his heart has exhibited considerable improvement, with his ejection fraction – a measure of the heart’s ability to pump blood – increasing from 15% to over 30%. While an ejection fraction between 55 and 70% is considered normal for healthy people, doctors expect Benson’s ejection fraction to continue to improve.

Heart failure is one of the most prevalent of all diseases, especially in developed nations. The American Heart Association estimates that in the U.S. alone there are more than 5.7 million people who live with heart failure, with approximately 670,000 new cases being diagnosed each year.

Dr. James Willerson is president of the Texas Heart Institute in Houston and a principal investigator of a separate clinical trial sponsored by NIH (the National Institutes of Health), also for the study of adult stem cell therapies in the treatment of heart failure. As Dr. Willerson explains, "When you put (adult) stem cells into a heart, some can differentiate to become blood vessels and others to become heart muscle cells." Additionally, Dr. Willerson adds, these types of adult stem cells, which are known as mesenchymal stem cells (MSCs), also contain growth factors and other beneficial "substances that recruit other cells and promote life," which makes the MSCs particularly efficacious as a clinical therapy. According to Dr. Willerson, "I believe we will be able to regenerate the whole heart of a human being with stem cells."

Dr. Douglas Losordo, director of the Program in Cardiovascular Regenerative Medicine at Northwestern University’s Feinberg School of Medicine, is conducting a pilot study in which 10 patients with heart failure are being treated with their own autologous adult stem cells, derived not from their bone marrow but instead from each patient’s own peripheral blood. As enthusiastic as he is about adult stem cell therapy, however, he is quick to point out that the field is not without its challenges. As Dr. Losordo explains, "Even as a wildly optimistic guy, I don’t imagine that anything I do on a single day in the cath lab is going to reverse 30 years of disease."

Similarly, Dr. Amit Patel, director of Cardiovascular Regenerative Medicine at the University of Utah and the leader of the study in which Mr. Benson is participating, believes that patients should not be shy about seeking adult stem cell treatment, but he also encourages them to conduct their own research and find a medical facility with specialists in a wide variety of fields, who can offer other forms of treatment in addition to adult stem cell therapy. According to Dr. Patel, "This is just one component of the entire continuum of heart failure treatment. It’s the sexiest one, but it’s only one component."

In a comment which is regarded by many as being more than just slightly odd, Dr. Patel also adds that, "With any cell-based therapy, there’s such a high placebo effect. There’s so much hype about the potential of stem cells. It’s positive reinforcement." In specific reference to Brent Benson, however, who lives at an altitude of 9,000 feet, Dr. Patel further acknowleges that, "Elevation, that’s the ultimate stress test." The fact that Benson has regained his energy and vigor following his adult stem cell therapy, while residing at such a high elevation, is concrete evidence for the powerful regenerative capacity of adult stem cells, and probably cannot be attributed solely to "the placebo effect". On the other hand, if someone with clinical heart failure can recover to the extent that their ejection fraction improves from 15% to more than 30%, for no other reason than "the placebo effect", then perhaps more attention should be paid to this grossly misunderstood phenomenon, and perhaps pharmaceutical companies might want to begin bottling and marketing "the placebo effect".

Whether inspite of or because of sudden, remarkable improvement in critically ill patients which is often explained as "just the placebo effect", Dr. Losordo adds that patients should not be afraid to pursue adult stem cell treatment, though he acknowledges that some patients have a difficult time knowing where or how to start. As he explains, "In my opinion, most patients are far too shy about picking up a phone and starting a line of inquiry. All the people involved in this stuff at medical centers, we want to help folks out."

In Brent Benson’s particular case, one of his cardiologists told him, "You don’t have a thing to lose. You don’t have much time left." For most people, a prognosis such as this would be enough to cure them of their shyness.

Not only is Benson now able to be physically active, without fatigue, while tending to the fruit trees on his farm and while also working on the deck at his home in Utah, at an eleveation of 9,000 feet, but also, as Benson now asserts, "I’ve gotten my sense of humor back."

Not bad, for "the placebo effect".

Filed Under: Heart Failure, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
August 18, 2009 by

Embryonic Stem Cell Trial Delayed

In the latest chapter of the ongoing saga over the first clinical trial ever to be conducted with human embryonic stem cells (hESCs), a new delay has presented itself.

Specifically, representatives of the Geron Corporation have announced today that U.S. federal regulators have placed a halt on the clinical trial that Geron was scheduled to conduct, before even one patient could be enrolled in the study.

Although a number of people among the non-scientific communities have expressed surprise over what they consider to be an unexpected announcement, there are many among the scientific community who find such a delay to be completely unsurprising and even predictable.

Frought with doubts and problems from the very start, the clinical trial was intended to use hESCs in the treatment of spinal cord injury. Because it would have been the first such study ever to be conducted with hESCs, the proposed clinical trial has repeatedly been the subject of widespread speculation and controversy. Now, this new ruling by the FDA, which brings all the momentum of the entire project to a grinding halt, has merely incited a new round of speculation and controversy.

The U.S. federal regulators decided to impose a halt on the clinical trial as a direct result of new data that Geron disclosed to the FDA, regarding dose escalation in preclinical animal studies. In January of this year, the FDA lifted an additional halt that it had previously imposed on Geron in May of 2008. This time, the new halt is based on safety concerns triggered by the new higher-dose data.

According to Joseph Pantginis, an analyst with the Merriman Curhan Ford Group in New York, "They are at the forefront, which a lot of times is a bit of a curse. They have to hit all the hurdles and be the first one to learn about how these cells behave."

In an official statement issued by representatives of Geron, the company "will work closely with the FDA to facilitate their review of the new data and to release the clinical hold."

Anna Krassowska, a spokesperson for Geron, stated to the press that the company had no further comment.

The news release posted on Geron’s website was brief, at only two paragraphs in length, not counting the customary "forward-looking statements" and "safe harbor" disclaimers. As stated on the comapny’s website, "GRNOPC1, a cell therapy for neurologically complete, subacute spinal cord injury, has been placed on clinical hold by the FDA pending the agency’s review of new nonclinical animal study data submitted by the company. A clinical hold is an order that the FDA issues to a sponsor to delay a proposed trial or to suspend an ongoing trial. … No patients have yet been treated in this study."

As the New York Times article concludes, "While thousands of patients around the world have been treated with adult stem cells and have shown mixed results, no humans have been given cells derived from embryos in an approved trial."

Indeed, it would seem as though, once again, embryonic stem cells remain confined to the experimental, laboratory stage, unable to progress to clinical trials. Meanwhile, by comparison, numerous types of adult stem cells have already advanced to Phase III clinical trials and beyond, and in fact have already been in use as viable therapies in clinics around the world for years.

Filed Under: News, Spinal Cord Injury, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
August 14, 2009 by

A New Quiet Leader is Emerging in the Adult Stem Cell Industry

George Reed, a 73-year-old policeman, was told by his cardiologist that nothing more could be done for him. Having already been through several coronary bypass surgeries, stent procedures, defibrillators and pacemakers, to no avail, Mr. Reed was known by his doctors as a "no-option patient". Since his heart was so weakened by diffuse coronary artery disease, he was disqualified from undergoing even one more invasive procedure.

In fact, there was actually one other option which still remained: adult stem cell therapy. Fortunately for Mr. Reed, he happened to reside in what is quickly becoming a geographic center for pioneering adult stem cell research, namely, Cleveland, Ohio.

A case in point is The Center for Stem Cell and Regenerative Medicine (CSCRM), which was founded in 2003 with a $19.4 million award from the state of Ohio. Located on the campus of Case Western Reserve University, the CSCRM is now known as a "scientific matchmaker", bringing together a number of researchers from academia and private industry who collaborate in the R&D of adult stem cell therapies.

In the 6 years since its inception, the CSCRM has received an additional $13 million in state funding, and it has spun off four new start-up companies, namely, Arteriocyte in 2004, Cell Targeting in 2005, and both Invenio Therapeutics and Juventas Therapeutics in 2007. Together with another Cleveland-based biopharmaceutical company, Athersys, which was one of CSCRM’s original founding partners, the four new start-ups have raised over $235 million in venture capital. Thus far, researchers at CSCRM have conducted 51 clinical trials in which more than 250 patients were treated with adult stem cells, and more than 60 patients were treated with other novel therapies.

According to Debra Grega, Ph.D., executive director of CSCRM, "When we got started in 2003, stem cells were considered very esoteric and not very practical. Now that we’ve progressed into early-stage clinical evaluations and actually are treating patients, we’ve gotten the attention of large pharmaceutical companies, which was absent until now."

Thanks to the collaboration between such pharmaceutical companies, academia and CSCRM, much of which is focused on the development of clinical therapies derived from adult stem cells, patients such as George Reed are now given new hope.

As the local Cleveland article noted, "With little fanfare, Cleveland has become one of the leaders in the relatively young field of adult stem cell therapy."

It would seem as though fanfare and accolades are overdue.

Filed Under: Heart Failure, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
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 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

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: , , , , ,
« Previous Page
Next Page »