U.S. and Jordanian Stem Cell Companies Sign Agreement

Stemedica Cell Technologies announced today the signing of an agreement with the Jordanian Stem Cell Company. The five-year, multi-million-dollar deal includes the purchase of technology from Stemedica’s wholly-owned subsidiary, Stemedica International, S.A., for the development, testing and commercialization of adult stem cell products. The first clinical trial under the new agreement is expected to begin in early 2010.

Based in Lausanne, Switzerland, Stemedica International is a wholly owned subsidiary of Stemedica Cell Technologies, headquartered in San Diego. Both are specialty biopharmaceutical companies that manufacture adult stem cells and stem cell factors for preclinical and human clinical trials conducted at research institutions and hospitals.

Headquartered in Amman, the Jordanian Stem Cell Company is chaired by His Royal Highness, Prince Asem Bin Nayef.

The signing took place at the European Burns Association Congress in Lausanne, Switzerland and was attended by Sam Alkhass, CEO of the Jordanian Stem Cell Company, as well as several representatives of Stemedica who included Dr. Mark Tager of Stemedica’s dermatological operations; Dr. Alex Kharazi, vice president of Stemedica’s research and manufacturing; Dr. Riccardo Nisato, director of manufacturing and business development at Stemedica; and Dr. Frank Schuller, CEO of Stemedica International.

As Sam Alkhass stated, "We are delighted to be working with Stemedica International. Jordan has an excellent reputation as a provider of innovative and quality-driven medical treatment. It was fundamental to our company’s future, and Jordan’s reputation, that we find the best possible partner within the stem cell industry. We believe we have accomplished this objective by securing a long-term exclusive relationship with Stemedica International."

As Nikolai Tankovich, M.D., Ph.D., Stemedica’s president and chief medical officer and vice chairman of Stemedica International, added, "Stemedica International will be providing our state-of-the-art stem cell technology to the Jordanian Stem Cell Company and will actively support their development and implementation of a clinical trial for burns. We will work closely with the leadership of the Jordanian Stem Cell Company to secure all necessary governmental approvals for the clinical trial. The trial is anticipated to begin sometime in the first quarter of 2010."

According to Dr. Schuller, "This is an important day for Stemedica International. This is a real testament to the quality and integrity of our products and the experience, dedication and professionalism of our team."

The Media is Criticized for Embryonic Stem Cell Bias

Not surprisingly, an organization that is focused on culture and the media issues a statement in which members of the U.S. media are criticized for their irresponsible bias in favor of embryonic stem cell research, while deliberately and systematically ignoring adult stem cell accomplishments.

According to Colleen Raezler of the Culture and Media Institute, reporter after reporter mistakenly features news that promotes embryonic stem cells as the ultimate cure for everything, despite the fact that embryonic stem cells have never been used to treat anything. Meanwhile, adult stem cells, which actually have been used to treat an increasing number of diseases and injuries, are routinely ignored by the media as a matter of policy.

Although this is a rather unsurprising statement of the obvious, its importance cannot be overemphasized. The unjustifiable, unconscienable and scientifically inaccurate bias throughout the media has the effect of misleading the public and creating false hope through unrealistic expectations. Even embryonic stem cell scientists themselves have tried to caution the public that clinical therapies from these cells are at least another decade away, if not more, yet there are very few members of the media who do not choose to turn a blind eye and a deaf ear to such expert advice. As Ms. Raezler further explains, members of the media are also "ignoring the fact that adult stem cell research has provided 73 different breakthroughs to help people with spinal cord injuries, Alzheimer’s disease, Parkinson’s disease, and diabetes," among other ailments.

Of course, no one would ever presume members of the media to be deliberately manipulative of the public. If they were, however – hypothetically, that is – they could not do a better job of misrepresenting the scientific truth than they currently are.

Hopefully, however, organizations such as the Culture and Media Institute will continue to challenge those members of the media who have chosen to relinquish their very serious responsibility to uphold and report the facts, and who instead consistently portray erroneous and unsubstantiated hyperbole as science.

Adult Stem Cells Applied to Cancer Therapy

The adult stem cell company Vitro Biopharma has announced pending patent applications on proprietary cell-based cancer therapies that are developed from human adult stem cells. Specifically, the therapies incorporate the natural properties of a specific type of adult stem cell known as mesenchymal stem cells (MSCs), a highly potent stem cell that is commonly found in bone marrow, adipose (fat) tissue and umbilical cord blood.

This new announcement for the use of MSCs in cell-based cancer therapies is believed to have wide-spread applications in the therapeutic treatment of certain types of cancer that are known to develop from a specialized set of cancer stem cells which are typically highly resistant to standard therapies such as chemotherapy and radiation. Among other forms of cancer, the glioblastoma tumors that are found in brain cancer would be an excellent candidate for treatment with Vitro’s new therapeutic approach. The novel cell-based therapies capitalize upon the natural tendency of MSCs to migrate to cancer stem cells, thereby utilizing the MSCs as a delivery mechanism to transport toxic agents that may selectively target and kill the cancer stem cells. Precisely toward such a goal, Vitro Biopharma currently has patent applications pending for their new proprietary adult stem cell therapies, for which the company is also actively pursuing strategic partnerships in the commercialization of these new therapeutic products. Vitro Biopharma currently has other related products that are already commercially available.

According to Dr. Pamela Rice, a member of Vitro’s scientific advisory board and an oncology researcher at the University of Colorado at Denver, "Cellular-mediated destruction of cancer stem cells represents a promising new avenue for the development of effective treatments that specifically target cancer stem cells. Vitro has developed an exciting portfolio of products to advance research in this field, which is one of the few approaches to show promise against intractable cancers such as most forms of brain cancer. Additionally, Vitro’s patent pending technology for cancer therapy represents an exciting and novel approach to the selective eradication of cancer stem cells that could usher in new promise in the fight against cancer."

Vitro Biopharma recently established a new manufacturing site in Golden, Colorado, comprised of both corporate as well as R&D facilities. Vitro’s commercial focus is on a series of products developed from MSCs and iPS (induced pluripotent stem) cells, as well as on cancer research that utilizes exclusively adult stem cells in the development of novel cancer therapies.

Horse Benefits From Adult Stem Cells

Continuing with an ongoing trend, and consistent with the latest developments in recent years, veterinary medicine remains several years ahead of human medicine in stem cell therapy.

As the latest development in veterinary medicine, Phil the horse has been treated with his own adult stem cells, derived from his own bone marrow just 2 weeks ago. Having suffered from a lame knee prior to receiving the therapy, Phil is now back to his usual self, unrestricted in motion and painfree. Increasingly, autologous (in which the donor and recipient are the same animal) adult stem cell therapy is being used in animals to treat a wide variety of ailments which include arthritis, tendonitis and even broken bones. Whether the condition is caused by an acute injury or by age-related degenerative conditions such as osteoarthritis, adult stem cell therapy continues to yield remarkable and consistent success in veterinary patients.

According to Martin Vidal, D.V.M., a veterinarian at the UC Davis Center for Equine Health, "The joint is a very delicate environment in horses. The horse has more appropriate mechanical demands than would be comparable to a human. The hope is that the stem cells will incite a reparative process." As Jeff Peck, D.V.M., a veterinarian at the Affiliated Veterinarian Specialists of Orlando, Florida, adds, "We know we’re not going to make those tissues normal, but it will suppress inflammation, slow the progression of degenerative changes in many cases, and also provide pain relief."

As previously reported a number of times on this website, 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. Furthermore, both VetCell and VetStem report a 100% safety rate. 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.

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."

Both Vet-Stem and VetCell use 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 these procedures are mesenchymal stem cells, which are highly potent adult stem cells that are found not only in bone marrow and adipose tissue but also in 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 either Vet-Stem’s or VetCell’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.

Of course, Phil the horse did not have to worry about an outdated federal regulatory agency preventing him from receiving his own adult stem cells as a therapy, unlike his human counterparts, at least in the United States. Until the U.S. FDA reverses its stance in which it insists that each person’s own adult stem cells must be categorized as a "drug", and therefore regulated as such, there’s not much hope of any human patient in the U.S. being allowed to receive autologous adult stem cell therapy, unless he or she is fortunate enough to be accepted into one of the very few FDA-approved clinical trials that are currently underway – and even then, half of all patients in each clinical trial will end up in the "control group", receiving a placebo instead of the adult stem cell therapy. It is precisely this politically motivated and scientifically erroneous stance by the FDA that is driving many U.S. adult stem cell companies, doctors and patients overseas where adult stem cell therapy is legally available. Fortunately, however, since the U.S. FDA does not govern veterinary medicine, U.S. animals are not forced to travel overseas for medical attention but instead are actually legally allowed to be treated with their own adult stem cells within the borders of the United States. With veterinary applications of adult stem cell therapies, in other words, there is no horsing around.

As Phil the horse would no doubt agree, it would seem as though the U.S. FDA lacks basic horse sense.

Stemgent Sponsors Harvard Seminar Series

The biotech company Stemgent announced today that it has pledged a financial gift to the Harvard Stem Cell Institute (HSCI) for its 2009-2010 Seminar Series.

According to Ian Ratcliffe, president and CEO of Stemgent, "We’re pleased to be able to fund this prestigious seminar series and we’re very proud of our ongoing relationshipwith HSCI through our Scientific Advisory Board which includes Harvard’s Lee Rubin and Doug Melton. HSCI has a top-notch research team, displays a global scientific acumen and it’s always a great pleasure to work with them."

As Brock Reeve, executive director of HSCI, adds, "The HSCI Seminar Series is focused on enabling scientific interchange among experts in the stem cell field from around the world. HSCI is very pleased that Stemgent, a company focused on addressing the needs of stem cell scientists, has agreed to partner with us by supporting this series."

Founded in 2004, the HSCI consists of 72 principal faculty and 146 affiliate faculty, thereby constituting the world’s largest organization of stem cell researchers. The HSCI describes itself as "a scientific collaborative that brings together researchers from across Harvard’s many affiliated hospitals, institutes and schools."

The HSCI further describes its seminar series as "an educational program for the stem cell science community in which pioneers in the national and international realm of stem cell research, including many from the Harvard-affiliated senior faculty, present contemporary stem cell topics. Speakers are nominated by HSCI faculty and selected by the HSCI."

With dual headquarters in San Diego and Boston, Stemgent is focused on the commercialization of proprietary reagents and other tools that have been optimized for stem cell research, including transfection reagents, viral-delivered transcription factors, cytokines, antibodies and cell lines, among other small-molecule products.

The precise financial terms of the sponsorship were not disclosed.

Eastern European Stem Cell Symposium Hosts International Speakers

The International Summer School for Stem Cells and Regenerative Medicine in Piran, Slovenia has just finished hosting an international panel of speakers from August 21st through the 29th. In addition to the formal conclusions drawn by the scientists during the week-long symposium, the mere list of topics which they addressed constitutes a revealing evaluation and excellent summarization of the field of regenerative medicine.

Among the sponsoring participants was the Florida-based U.S. company known as "Hard To Treat Diseases" (HTDS), whose medical director, Dr. Ivana Gadjanski, spoke at the symposium. Other speakers from the U.S. included Drs. Tanja Dominko and Raymond Page from the Worcester Polytechnic Institute in Massachusetts, and Dr. Darja Marolt from Columbia University in New York. Among the other speakers and countries represented were Dr. Stephen Minger from King’s College in London, Dr. Thomas Ekstrom from the Karolinska Institute in Stockholm, Sweden, Dr. Zoran Ivanovic from the blood bank EFS in Bordeaux, France, Dr. Smadar Cohen from the Ben-Gurion University of the Negev in Israel, and Dr. Dasa Cziskova from the Institute of Neurobiology in Kosice, Slovakia.

An excellent overview of the current state of affairs in the field of regenerative medicine can be gleaned by a quick glance at the topics that were addressed, which can be broadly divided into the following categories: 1) types of stem cells that are or are not suitable for clinical application, 2) ethical issues, 3) obstacles to therapy, 4) clinical trials, 5) technical issues pertaining to laboratory methods, 6) new ways of inducing pluripotency, 7) adult stem cells derived from umbilical cord blood, 8) adult stem cells derived from adipose tissue, and 9) means of enhancing endogenous stem cell activity. In the category of "obstacles to therapy", teratoma formation from any type of pluripotent stem cell was the dominant, most problematic concern. In the category of "clinical trials", the recent FDA-imposed halt on Geron’s embryonic stem cell trial was the focus of discussion. In regard to "technical issues pertaining to laboratory methods", topics of interest included the various types of biomaterials that are used for cellular scaffolds, the types of bioreactors that are used for growing cells, and the protocols for isolating cells from various tissues, among other themes. It is particularly noteworthy that both umbilical cord blood and adipose tissue received special attention as the most promising sources of the most versatile and the most clinically viable stem cells – all of which is indicative of the fact that gone are the days when stem cells were simplistically divided into the 2 fundamental categories of embryonic versus adult. Indeed, the only attention that embryonic stem cells received during this entire symposium was when the participants addressed the persistently recurring themes of teratoma formation and the types of stem cells that are unsuitable for clinical application. When it came to a discussion and analysis of clinically viable stem cells, only adult stem cells have proven to be qualified for this category, and people are now accustomed to drawing fine distinctions among the many different types of adult stem cells and their already very well elucidated characteristics.

The formal conclusion of the entire symposium, however, was that the most efficacious type of clinical therapy will be one that stimulates the powerful healing mechanisms of the body’s own naturally occurring supply of endogenous adult stem cells. Not only does this approach eliminate all ethical concerns and potential risks of immune rejection, but it has also proven to be among the most therapeutically efficacious of medical approaches.

The fact that this symposium was hosted by an Eastern European city, the name of which is unfamiliar to most Americans, is also not without its significance. Although there are some countries in the world, such as the U.S., who tend to think of themselves as the scientific center of the universe when it comes to stem cell research, in actuality there are many other countries which are advancing more rapidly in stem cell technology. Such a fact has nothing to do with Bush-era restrictions that were imposed on embryonic stem cell research, since embryonic stem cells are still ineligible for clinical use, even with unlimited funding, due to a number of dangers that they pose, not the least of which is teratoma (tumor) formation. Instead, the greatest hindrance to stem cell advancement in the U.S. is the entirely politically driven and egregiously unscientific insistence by the U.S. FDA that each person’s own endogenous adult stem cells must be classified as "drugs" and regulated as such. It is this unconscienable government policy – which persists throughout the Obama administration – that is driving the best, brightest and most capable U.S. clinics and doctors overseas, where they are setting up their offices in any and every other country on earth except the United States.

U.S. citizens should therefore be prepared to hear a lot more news throughout the future about impressive advances in adult stem cell therapies that are happening in places such as Piran, Slovenia.

Adult Stem Cell Clinical Findings Presented at European Conference

This week in Barcelona, at the 10th annual congress of the European Society of Cardiology, the U.S. company TCA Celluar Therapy is presenting its latest clinical findings on its own proprietary adult stem cell therapies in the treatment of two particular diseases, namely, critical limb ischemia (CLI) and refractory coronary ischemia (CI). TCA has already evaluated its therapies for both conditions in U.S. FDA-approved clinical trials.

According to Gabriel Lasala, M.D., president, co-founder and medical director of TCA, TCA is the only company in the world currently utilizing two different types of adult stem cells to treat both cardiac and vascular conditions. Most recently, Dr. Lasala and the company’s scientific director, Dr. Jose Minguell, have together treated 33 patients for CLI in both Phase I and Phase II clinical trials in which patients were treated with a combination of autologous endothelial progenitor cells and mesenchymal stem cells harvested via bone marrow aspiration. The combined cells were then infused directly into areas of ischemic tissue and blood vessel damage, from which new, mature and stable vessels were observed to form via the natural angiogenic properties of these particular adult stem cells. No adverse side effects were observed in any of the patients, all of whom experienced "a progressive improvement in all clinical parameters which are still persisting a year after treatment," according to the researchers.

Similarly, in CI clinical trials, Drs. Lasala and Minguell completed a Phase I protocol last year and are currently conducting Phase II trials in which 60 patients have been enrolled. Thus far all patients have exhibited "a significant improvement in the quality of life", according to the doctors, with the results suggesting that "recruitment of new capillaries could be a leading event involved in the improvement of CI," as the researchers explain. According to Drs. Lasala and Minguell, this is the first safety and feasibility study that tests the infusion of this particular combination of adult stem cells.

As Dr. Lasala further describes, "All patients experienced improvement in their walking tests, ankle brachial pressure index, oxygen pressure, angiography and quality of life. The similarity in the recovery of our patients is promising. We find that the stem cells, once re-injected, go about forming new blood vessels, thus increasing circulation dramatically. These findings, coupled with increase of blood flow in collateral vessels, suggest that the therapy is both safe and effective."

Headquartered in Covington, Louisiana, TCA Cellular Therapy is currently participating in a number of FDA-approved clinical trials, all of which are testing novel adult stem cell therapies.

Stem Cell Company Hosts Training Courses

The biotech company Stemcell Technologies announced today the schedule for their fall training program. The new curriculum includes a number of 2-day courses, namely, on mesenchymal stem cell biology (October 2nd and 3rd), mammary stem cell biology (October 17th and 18th), neural stem cell biology (October 30th and 31st), hematopoietic stem cell biology (October 30th and 31st), and lastly, human embryonic stem cell and iPS (induced pluripotent stem) cell biology (November 14th and 15th). All courses will be held at the company’s Vancouver headquarters and will be taught by scientists who have been personally involved in the actual development of many of the research tools and techniques that are commonly employed in stem cell laboratories around the world today. Hands-on instruction will be emphasized, in conjunction with some lectures.

According to Dr. Chantal Proulx, technical support manager at Stemcell Technologies, "We are dedicated to providing standardized tools and reagents in order to advance research, but this is only half the story. Providing detailed training courses to standardize the techniques that people use to grow their cells is also a key part of the equation."

As described on their website, the biotech company Stemcell Technologies is involved in "the development and marketing of specialty cell culture media, cell separation products and ancillary reagents for life science research." Stemcell Technologies currently delivers more than 900 products to research scientists in over 70 countries worldwide.

Headquartered in Vancouver, British Columbia, Canada, Stemcell Technologies also has corporate offices in France, Germany, the U.K., Australia and Singapore.

Researchers who wish to register for the courses may apply directly with the company.

Bioinformatics Company Launches Stem Cell Collaboration

A leading manufacturer of bioinformatics software for systems biology and drug discovery, GeneGo announced today that it has launched a collaborative endeavor with global pharmaceutical companies and academic centers. Known as the MetaMiner Stem Cell Project, the 24-month-long collaboration has been designed to create a comprehensive knowledge base for the properties and characteristics of different types of stem cells. The knowledge base will then in turn be applied to experimental R&D for the therapeutic use of stem cells of various types.

According to Yuri Nikolsky, CEO of GeneGo, "We are really excited about this project. Although one of the hottest areas of life science, no systematic effort was done on methodical annotation of current experimental knowledge on stem cells, and we intend to fill this gap. Understanding the biology of embryonic, adult and neoplastic stem cells is key in both drug discovery and fundamental research in many fields from embryology and ontogenesis to cancer and diabetes."

Similarly, Julie Bryant, GeneGo’s vice president of business development, adds, "We are very glad to be able to attract an excellent team of members for the cause. An industry-academia consortium model fits well with our development objectives in such a complex and controversial field. We believe that the members will see a strong positive return on their investment within several months from the project’s launch."

As described on their website, GeneGo provides "data mining and analysis solutions in systems biology". The company is focused on the development of "systems biology technology, such as compound-based pathway analysis, cheminformatics and bioinformatics software for life science research. The original computational MetaDiscovery platform allows an integration and expert analysis of different kinds of experimental data (mRNA expression, proteomics, metabolomics, microRNA assays and other phenotypic data) and relevant bioactive chemistry (metabolites, drugs, other xenobiotics) within the framework of curated biological pathways and networks. GeneGo’s flagship product, MetaCore 5.4, assists pharmaceutical scientists in the areas of target selection and validation, data mining in biology, identification of biomarkers for disease states and toxicology. The second product, MetaDrug 5.4, is designed for prediction of human metabolism, toxicity and biological effects for novel small molecule compounds. MetaBase represents the knowledge base for MetaCore."

Founded in 2000 by Dr. Tatiana Nikolskaya, a molecular biologist from the University of Chicago, GeneGo is headquartered in St. Joseph, Michigan, with offices in San Diego and Moscow.

Skin Cells are Reprogrammed to Produce Insulin

Using ordinary skin cells derived from patients with Type I diabetes, scientists were able to reprogram the cells to create new cells that produce insulin. The announcement heralds a potentially revolutionary type of therapy for the millions of people who suffer from Type I diabetes.

In a procedure which is now commonly reproduced by stem cell scientists around the world, the researchers de-differentiated ordinary somatic (non-stem-cell) skin cells into a more primitive state, known as iPS (induced pluripotent stem) cells. In a new variaton on the theme, however, the iPS cells were then reprogrammed and re-differentiated into a new type of cell, one which resembles the insulin-producing beta islet cells of the pancreas. Specifically, the skin samples were obtained from two white males, one of whom had been diagnosed with Type I diabetes at 3 years of age, and the other of whom was first diagnosed at 21 years of age. Led by Dr. Douglas Melton, codirector of the Harvard Stem Cell Institute and a leading investigator at the Howard Hughes Medical Institute, the team of researchers reprogrammed the fibroblasts into iPS cells using 3 of the 4 genes that are commonly used for the iPS reprogramming procedure. Although the new cells do not produce insulin as efficiently as naturally occurring pancreatic cells do, nevertheless the new cells are responsive to changes in blood sugar levels. The procedure signifies an especially important accomplishment since the skin cells were not randomly taken from any donor but instead were specifically taken from patients who are suffering from Type I diabetes, thereby yielding a new type of cell which is "patient-specific" and which therefore matches the individual’s unique genetic profile, in addition to being free of any risk of immune rejection.

The next step now is to create an animal model of Type I diabetes in which the new cells can be studied. Eventually, the ultimate goal is to develop a clinical therapy from the procedure which can be used in human patients to replace the pancreatic beta islet cells that are destroyed by Type I diabetes.

According to Susan Solomon, J.D., CEO of the New York Stem Cell Foundation, which cofunded the study, "This is a big deal. Tackling the basic biology of Type 1 diabetes, which is a very complex disease, is a critical step. With these cells, we can see in a dish what’s happening to the immune system, and if you don’t understand the immune response, you get nowhere with Type 1 diabetes." As Dr. Meri Firpo of the Stem Cell Institute at the University of Minnesota further adds, "This is very preliminary data, but now we could potentially look at the interaction between immune system cells and insulin-producing cells to find the root cause or trigger, which we think might vary from patient to patient."

Meanwhile, however, such a therapy is still in the developmental stage, and the new insulin-producing cells are currently disqualified from clinical use since the genetic manipulation that is used for reprogramming the cells poses too many medical risks. Among other problems, cells from mice that have been reprogrammed according to this method have been found to develop into teratomas (tumors) when the cells were readministered to the mice. According to Julia Greenstein of the Juvenile Diabetes Research Foundation (JDRF), the most immediate applications of Dr. Melton’s new achievement "are primarily research-related". As she further explains, "Our hope is that understanding all of these things will come together – that once we’ve figured out how to make the cell source, we will have also figured out how to block the immune response, but there’s a lot of basic science one has to do to get there." Nevertheless, "There’s an incredible amount of exciting research that has the capacity to impact the disease in the long-term," she adds.

Believed to be of autoimmune origin, Type I diabetes destroys the insulin-producing beta islet cells of the pancreas. Though not as common as Type II diabetes, Type I diabetes is currently untreatable by conventional medical therapies, which offer no known cure for the disease. Since there is a strong genetic susceptibility, researchers believe that "patient-specific" therapies which are derived from each patient’s own unique cells should offer the most efficacious type of treatment. Such therapies would also eliminate any need for dangerous immunosuppressive drugs – if or when such therapies are ever actually developed from iPS cells, some day, at some undetermined point in the future.

Of course, "patient-specific" therapies already exist, today, and have already been derived from autologous adult stem cells and are already being used in clinics around the world for a wide variety of diseases and injuries, without any risk of immune rejection and without any need for dangerous immunosuppression – should anyone be interested to notice.