Researchers at the College of Veterinary Medicine at Cornell University in Ithaca, New York have reported the effective treatment with an autolgous adult stem cell therapy of tendonitis in horses.

Led by Dr. Alan J. Nixon, the study was not so much a "clinical trial" in the ordinary sense of the term, but was perhaps more correctly an "experimental study", since the researchers sacrificed the horses at the end of the evaluation period in order to conduct thorough histological and mechanical analysis of the tendons.

In the study, the researchers artificially induced tendonitis in the superficial digital flexor tendons (SDFT) of both forelimbs of an undisclosed number of horses. Six days later, autologous mesenchymal stem cells (MSCs) were then derived from the bone marrow of each horse, expanded and injected into one of the SDFT lesions, while the other untreated, "control" limb received an injection of 1 milliliter of saline. The MSCs were also gene-enhanced with IGF-1 (insulin-like growth factor 1), which has been demonstrated to stimulate cellular proliferation and tendon fiber deposition in the core lesion of tendonitis. Ultrasound examination of the tendons was performed at the start of the trial as well as at 2, 4, 6 and 8 weeks after administration of the therapy. According to an online abstract, "the horses were euthanized at 8 weeks and SDFTs were mechanically tested to failure and evaluated for biochemical composition and histologic characteristics."

As Dr. Nixon explains, "The biochemical composition of the treated and untreated tendons were similar 8 weeks after treatment. However, tendons injected with the stem cells had significantly improved histology scores, indicating a more normal microscopic appearance in treated tendons than untreated tendons. Nonetheless, more research is needed regarding the optimal dose of stem cells and the use of gene enhancement techniques to augment the observed benefit before making this technology clinically available."

The researchers conclude, "These findings indicate a benefit to the use of MSCs and AdIGF-MSCs for the treatment of tendonitis."

The results of the trial will be published in an upcoming edition of the Journal of Orthopedic Research in an article entitled, "Mesenchymal stem cells and insulin-like growth factor-1 gene-enhanced mesenchymal stem cells improve structural aspects of healing in equine flexor digitorum superficialis tendons."

Tendon injuries are often catastrophic and fatal in horses, while even minor tendon injuries can end the career of a race horse. According to Dr. Nixon, "At present, few successful treatment options exist for horses with tendon injuries. While stem cell therapy has become a hot topic in equine medicine, there are few controlled studies clearly documenting the safety or efficacy of this treatment modality for tendonitis in horses."

If killing the horse is a requirement for conducting a "controlled" study, then most people would probably conclude it’s a good thing that there aren’t many controlled studies that have been conducted.

In fact, a number of companies throughout the world are already treating horses, as well as other animals, with autolgous adult stem cell therapies, and not only do the animals improve as a result of the therapy but the animals are also allowed to continue living and enjoy the remainder of their lives. Perhaps the most renowned of these companies is the U.S.-based Vet-Stem, which has treated over 3,000 horses and 2,000 dogs with autologous adult stem cell therapy for a variety of joint injuries and degenerative conditions that include tendonitis as well as ligament injuries and age-related osteoarthritis. None of the animals treated by Vet-Stem’s autolgous adult stem cell therapy ever had to be euthanized, but instead were allowed to live out their natural lives.

Additrionally, in September of 2007 Vet-Stem licensed their proprietary adult stem cell technology to the Central Veterinary Research Laboratory (CVRL) of Dubai in the United Arab Emirates, thereby allowing the CVRL to offer the same adipose-derived adult stem cell animal therapies throughout the Middle East. Sheik Mohammed bin Rashid al-Maktoum, the ruler of Dubai and the Prime Minister of the UAE, is an avid thoroughbred owner and a sponsor of the Dubai World Cup, the world’s most highly-prized horse race. As Vet-Stem founding CEO Dr. Harman described in 2007, "The Central Veterinary Research Laboratory will be an excellent partner in bringing this technology from the U.S. to the Middle East as they are already the most respected reference lab in the region." CVRL now provides stem cell services for the treatment of injuries not only in thoroughbred race horses and Arabian endurance horses, but also in racing camels, among other species, throughout the Middle East.

It is actually incorrect, therefore, to claim that "there are few controlled studies clearly documenting the safety or efficacy of this treatment modality for tendonitis in horses", since more than 3,000 case studies have been documented by Vet-Stem alone, not counting the other case studies conducted around the world by other companies that are performing similar therapies in other countries. It is also incorrect to claim that "more research is needed regarding the optimal dose of stem cells and the use of gene enhancement techniques to augment the observed benefit before making this technology clinically available", since, actually, such technology is, in fact, already clinicallly available.

Given the consistent success of such therapies as those used by Vet-Stem, one might seriously question the wisdom and necessity of conducting "controlled studies" in which the horses must be killed at the end of the study in order to determine the efficacy of the medical therapy. Horses are not generally bred and maintained in the same manner as laboratory mice, although in this Cornell study, one might easily get the impression that the two animals are interchangeable.

Ordinarily, veterinarians, like all physicians, measure the efficacy of a therapy by the ability of the patient to recover – not by killing the patient in order to examine the mechanical, biochemical and histological properties of the tissue that was treated. Such an approach to medical therapy would be considered entirely unacceptable in humans, and even in horses the concept seems to lack basic horse sense.

Deep in the heart of Texas, the Heart Hospital of Austin is one of approximately 40 hospitals throughout the United States and Canada that have been chosen to participate in an FDA-approved clinical trial for the use of adult stem cells in the treatment of heart attack patients.

Specifically, the double-blind, placebo controlled Phase II clinical trial will evaluate the efficacy of an allogeneic adult stem cell product developed by the biotech company Osiris Therapeutics. Known as Prochymal, this proprietary adult stem cell therapy has already demonstrated exceptionally strong safety results in the Phase I clinical trial, during which time Prochymal was found to be even safer than a placebo. The current Phase II clinical trial will evaluate the ability of Prochymal to regenerate damaged heart tissue following a heart attack. The first patient to be enrolled in the clinical trial, a 58-year-old male, was already treated on Monday, within days of his heart attack. Approximately 20 patients will be enrolled in the study over the next 6 to 8 months, all of whom will receive the Prochymal treatment within 7 days of having suffered a heart attack. Prochymal is administered intravenously, and the entire therapy takes less than an hour to complete. Of the 40 hospitals that are participating in the clinical trial, only the Heart Hospital of Austin and one other hospital, in Kansas, have already begun treating patients.

According to 74-year-old Miles Simmons, who is also enrolled in the study in Austin and is scheduled to receive the Prochymal therapy, “I have a lot of grandchildren and I love them and I would like to spend more time with them. I think it’s wonderful. If you have technology, then use it.” As Dr. Roger Gammon, a cardiologist with the Heart Hospital of Austin and the director of the clinical trial, explains, “It would change everything for people with heart attacks. Research in this area has just exploded, and a lot of funding is going to have to come from the government to really push it forward. What’s kind of unique and new is it’s given intravenously. The cells are drawn to the heart and they implant there. If it works, it will be fantastic.”

Prochymal is composed strictly of adult stem cells, not embryonic stem cells. More specifically, Prochymal is composed of mesenchymal stem cells that are derived from the bone marrow of healthy adult donors. As such, not only is Prochymal entirely removed from the ethical controversies surrounding embryonic stem cells, but Prochymal is also free of the numerous medical risks posed by embryonic stem cells, not the least of which is the strong natural tendency of embryonic stem cells to form teratomas (tumors), which all pluripotent stem cells must, by definition, be able to form. By sharp contrast, since adult stem cells are not pluripotent, they are incapable of forming teratomas and do not pose such risks. Even though Prochymal is composed of non-autologous, allogeneic (in which the donor and the recipient are not the same person) adult stem cells, there is no risk of immune rejection since mesenchymal stem cells have been proven in multiple studies to be “immune privileged”, “universal donor” cells.

Nevertheless, embryonic stem cells remain a contentious issue, and on Friday of this week the Texas State Senate is scheduled to debate the topic of funding for embryonic stem cell research. According to Republican Senator Tommy Williams, “I think people agree that there’s the potential for moral hazard here, and so I think it’s important to give our state agencies and research institutions guidance.” The Texas state budget was passed on Wednesday, but no state funds will be used for embryonic stem cell research until the Legislature sets a policy.

Meanwhile, adult stem cell products such as Prochymal eliminate any need for debate. In a similar type of clinical trial, a hospital in Houston is also using adult stem cells for the repair of neurological tissue in stroke patients.

As Dr. Gammon adds, “We are excited to be the first to treat a patient in this groundbreaking study and to lead the way in this important research. Austin Heart and the Heart Hospital of Austin were selected because of their proven history of excellence in cardiovascular research and strong performance in the earlier Phase I trial. There’s a lot of enthusiasm in the cardiovascular community about the potential of stem cell therapies for treating heart disease. Earlier studies have established confidence in the safety of the therapy, but more research is needed to study its effectiveness.”

Osiris Therapeutics is the leading stem cell therapeutic company in the world, involved in the research and development of therapeutic products that are based exclusively upon adult stem cells, not embryonic stem cells. Osiris was founded in 1992 and went public in 2006. Its products focus on the treatment of inflammatory, orthopedic and cardiovascular conditions. The company’s proprietary adult stem cell product Prochymal is the only stem cell therapeutic product currently designated by the FDA as both an Orphan Drug and as a Fast Track product. In addition to the current Phase II clinical trial for the regeneration of cardiac tissue following myocardial infarction, Prochymal is also in Phase II clinical trials for the regeneration of pancreatic beta islet cells in patients with Type I diabetes and for the repair and regeneration of damaged lung tissue in patients with chronic obstructive pulmonary disease. Prochymal is also currently being evaluated in three separate Phase III clinical trials, two of which are for graft vs. host disease (GvHD) and the third of which is for Crohn’s disease, both of which are potentially fatal conditions. Additionallly, the company’s adult stem cell product Chondrogen has also been approved to begin Phase II and III clinical trials for osteoarthritis of the knee. Osiris currently has 47 U.S. patents, each with one or more foreign counterparts. In 2008 Osiris formed a strategic alliance with Genzyme worth approximately $1.3 billion, and that same year the U.S. Department of Defense awarded Osiris a contract to develop Prochymal as a treatment for acute radiation syndrome.

The Heart Hospital of Austin has been ranked the number one leading hospital in Texas for overall cardiac services for 6 years in a row, since 2004, during which time it has also ranked among the top 5% of hospitals in the nation for overall cardiac services, and in 2009 it was also chosen as number one among hospitals in Texas for cardiac surgery.

In his profession, job-related injuries are common. So when Buzz, a 3-year-old male Border Collie, was recently injured in his job, it looked as though his career was over. Responsible for the herding of sheep on a 40-acre working farm near Ramona, California, Buzz was faced with surgery and a recovery period of at least 6 months after rupturing a kneecap tendon. According to his owner, John Doyle, “His career was over. He was through.”

However, after undergoing the surgery to repair his tendon, Buzz then also received 3 rounds of autologous adult canine stem cell therapy. Instead of having to endure a 6-month recovery period, Buzz returned to work a mere 6 weeks after receiving the stem cell treatment. As Mr. Doyle explains, “In 2 weeks, you could see that he was healing very quickly. He was able to do a lot more.”

As previously reported a number of times on this website, the California-based company Vet-Stem is demonstrating consistent veterinary success in the treatment of animals with autologous adult stem cell therapy. Although Vet-Stem was the first company to commercialize the process, a number of other companies throughout the world are now also utilizing the same type of technology, in which adult stem cells are derived from each animal’s own adipose (fat) tissue and readministered to the animal as a clinical therapy for the particular medical condition from which the animal suffers. This type of 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.

Vet-Stem was founded in 2002 as the result of stem cell research conducted at the University of Pittsburgh and UCLA in the late 1990s, when Dr. Bob Harman saw the commercial potential for veterinary applications of such stem cell technology. A veterinarian himself, as well as a statistician and former biotech entrepreneur who had already held the top executive title at 3 biotechnology companies prior to Vet-Stem, Dr. Harman is now the CEO of Vet-Stem as well as one of its founders. Based in San Diego, Vet-Stem patterned its initial clinical model upon the example of other companies that were already involved in human adult stem cell therapies, such as Cytori Therapeutics which had developed a proprietary separation apparatus that harvests human adult stem cells from adipose tissue at the patient’s bedside during reconstructive or cosmetic surgery. In a similar procedure, veterinarians extract approximately 2 tablespoons of adipose tissue from each animal, which are then sent to Vet-Stem where the adult stem cells are isolated, purified, expanded and returned within 48 hours to the veterinarian who then administers the stem cells to the animal.

Dogs are not the only animals to benefit from Vet-Stem’s veterinary technology, as horses and other larger animals have also been found to respond very well to adult stem cell therapies. In fact, among other partnerships, in September of 2007 Vet-Stem licensed their proprietary adult stem cell technology to the Central Veterinary Research Laboratory (CVRL) of Dubai in the United Arab Emirates, thereby allowing the CVRL to offer the same adipose-derived adult stem cell animal therapies throughout the Middle East. Sheik Mohammed bin Rashid al-Maktoum, the ruler of Dubai and the Prime Minister of the UAE, is an avid thoroughbred owner and a sponsor of the Dubai World Cup, the world’s most highly-prized horse race. As Dr. Harman described in 2007, “The Central Veterinary Research Laboratory will be an excellent partner in bringing this technology from the U.S. to the Middle East as they are already the most respected reference lab in the region.” CVRL now provides stem cell services for the treatment of injuries not only in thoroughbred race horses and Arabian endurance horses, but also in racing camels, among other species, throughout the Middle East. In the United States Vet-Stem has already treated over 3,000 horses and over 2,000 dogs with joint injuries and degenerative conditions that include tendon and ligament injuries as well as age-related osteoarthritis. Vet-Stem’s overall success rate is around 80% in the number of animals who are able to return to normal performance, a rate that is significantly above that of conventional surgical and pharmaceutical therapies.

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.

In fact, as Dr. Harman explains, “Our success in animals is directly translatable to humans, and we wish to share our evidence that stem cells are safe and effective.”

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.

Vet-Stem was initially funded by Toucan Capital of Maryland, which invests in early-stage life sciences companies and which provided an initial one million dollars in seed funding to Vet-Stem as well as an additional five million dollars to date. As with the other biotech companies that Dr. Harman has directed, Vet-Stem promises to be a highly profitable and sound investment, not only for its financial investors but also for its four-legged patients.

Researchers in Edinburgh, Scotland are developing a new medical procedure that “knits together” torn knee tissue with autologous adult stem cells. The technique will be tested in clinical trials with patients within the next year.

The research is being led by Dr. Anthony Hollander of Bristol University, who was a member of the U.S. team that successfully reconstructed a new trachea last year with autologous adult stem cells in the treatment of then 30-year-old Claudia Castillo, who received a new trachea that was grown from her own adult stem cells. (Please see the related news article on this website, entitled, “New Trachea Grown from Autologous Adult Stem Cells”, dated November 26, 2008, and originally reported in The Lancet).

Now, a similar procedure will offer a new form of therapy for injuries and degenerative conditions that are common to the knee. Although the new technique is targetted specifically for the repair of tears in the meniscus, which is a part of the knee cartilage in which sports injuries are especially common, the treatment will also offer relief to the many individuals who suffer from osteoarthritis.

Speaking at a conference of the Scottish Stem Cell Network in Edinburgh yesterday, and in reference to tears in the miniscus, Dr. Hollander stated, “At the moment, there’s no way to treat this. It is just cut out, and that leaves the patient very susceptible to osteoarthritis within a short number of years.” One such example is Alan McLaren, a former defender first for the Heart of Midlothian team and later for the Rangers, who was abruptly forced to end his professional sports career in 1999 at the age of 27 after suffering a cruciate ligament injury. Clearly, Mr. McLaren could have benefited from such a therapy, and he might have been able to continue his highly successful football career, had this type of stem cell therapy existed a decade ago.

The upcoming clinical trials will initially enroll 10 patients, in whom adult stem cells that have been grown on a membrane will be implanted directly into the lesions of their knees, and sewn up. As Dr. Hollander explains, “It is designed in a way that the cells will migrate across the lesion and literally knit it together. So instead of growing new tissue, it’s healing the lesion itself.”

Only autologous adult stem cells will be used in the clinical trial, derived from each patient’s own bone marrow.

A number of other researchers in Scotland are planning similar clinical trials, including Dr. Brendon Noble of the Centre for Regenerative Medicine at Edinburgh University, whose focus will be more oriented toward age-related osteoarthritis rather than sports injuries.

Meanwhile, Dr. Hollander is also developing further improvements in trachea transplants that are re-engineered with adult stem cells and grown in the laboratory, the technology for which is also believed to be applicable to the regeneration of other organs, including large blood vessels and the intestine.

Once again, veterinarians are healing canine injuries with adult stem cell therapy. This time, the vets and their four-legged patients are in Australia.

Veterinarians in Sydney are applying an already proven technique to the treatment of injuries and degenerative diseases in dogs. The technique, which was first developed in the U.S. by the company Vet-Stem, uses autologous adult stem cells derived from the adipose (fat) tissue of each dog to treat the dog of joint and bone injuries as well as age-related osteoarthritis. Already successful in other countries, the technique is now also showing great success in Australia.

According to Dr. Ben Herbert, associate professor at the University of Technology in Sydney and director of the University’s Proteomics Technology Centre of Expertise, “What we see is a pretty rapid, within the first couple of days, reduction in the animal’s pain and inflammation. We see animals that are a lot happier, then you go into a zone where the science tells us we are actually getting new cartilage. Longer term, going out now to the dogs treated 9 and 10 months ago, those dogs are still improving.”

One such example is Cassie, a 12-year-old border collie mixture whose favorite activity now involves chasing small wild animals. However, prior to receiving the adult stem cell therapy, Cassie suffered from severe osteoarthritis of the hips, and even slow walking was painful for the dog. According to Elizabeth Beyer, Cassie’s owner, “Before, going for a walk would be a bit of an ordeal. Now we can do a walk any day of the week. Her hips have improved, she’s walking faster. She chases possums and whatever else comes into the garden. It’s about quality of life.”

The treatment, which has been available at the Ku-Ring-Gai Veterinary Hospital in Sydney for less than a year, has already been used to treat 60 dogs in Australia, whose owners travel with the dogs from across the country to receive the therapy. Although the treatment is initially more expensive than conventional veterinary medicine, the benefits are also greater. Dramatic, positive results are seen immediately after receiving adult stem cell therapy, and the dogs usually do not need any further treatment of any type. Like Cassie, many other dogs who received the autologous adult stem cell treatment have also been cured of their ailments and no longer require long-term anti-inflammatory drugs nor painkillers, most of which do not offer a cure but in fact carry dangerous side effects and, over time, are considerably more expensive than the adult stem cell therapy. From a long-term perspective, therefore, the adult stem cell therapy is actually less expensive than conventional veterinary treatments, such as surgery and medication, which are not as effective and may need to be repeated throughout the dog’s life.

As Dr. Herbert explains, “These are the patient’s own cells. It’s effectively a transplant and it’s this idea of switching on the body’s own regenerative system.” Since the adult stem cells are autologous (in which the donor and recipient are the same dog), there is no risk of immune rejection, nor is there any need for the use of dangerous immune-suppressing drugs.

Overall, the procedure is quick, simple, minimally invasive, safe, effective, and less expensive than surgery or taking prescription medication for years. Although this type of adult stem cell therapy has already become quite popular in other countries such as the U.S., a number of independent researchers have published corroborating evidence throughout the medical and scientific literature on the safety and efficacy of such a therapy.

Currently, Dr. Herbert and his colleages are also developing a similar autologous adult stem cell treatment for dogs with kidney disease. As he explains, “This has given us the opportunity to immediately translate early-stage research into the clinic and get real clinical data. It’s on dogs and cats, but it’s in the real world.”

As Dr. Herbert further explains, “There is nothing really different about doing that in a human being and doing it in a dog.” He is quick to add, however, “The regulatory regime is easier to deal with in dogs.”

Indeed, it seems to be much easier for old dogs to learn new tricks, at least when it comes to adult stem cell therapy, than for the respective government regulatory agencies of various countries to do so.

The Australian company Mesoblast has received institutional ethics approval to begin clinical trials with its adult stem cell product, RepliCart, in the treatment of osteoarthritis of the knee.

Headquartered in Melbourne, Mesoblast is now approved to begin randomized, placebo-controlled, double-blind Phase II clinical trials for its off-the-shelf allogeneic stem cell product, “RepliCart”, in the treatment of osteoarthritis of the knee in patients who have undergone reconstruction of a ruptured anterior cruciate ligament (ACL) within 6 months of a traumatic knee injury. The clinical trials will enroll 24 patients between the ages of 18 and 40 who will either be administered RepliCart in combination with hyaluronan, or just hyaluronan alone. The primary endpoint of the study will measure safety at 12 months, and a secondary endpoint will measure the prevention of cartilage loss and of knee osteoarthritis over time. Preclinical trials were successful in demonstrating the ability of a single injection of the allogeneic stem cells contained in RepliCart to regenerate joint cartilage and prevent osteoarthritis in animal models.

According to orthopedic surgeon Andrew Shimmin, from the Melbourne Orthopedic Research Foundation and the lead investigator of the clinical trials, “ACL injury is very common in our young active sporting population and unfortunately the injury is associated with the early development of arthritis despite modern reconstructive procedures. Little has changed in the prevention and treatment of arthritis over the past 50 years, so the application of Mesoblast’s stem cell technology for reducing the progression of this degenerative process in the knee offers a new and exciting direction for the management of arthritis.”

As Mesoblast’s executive director, Dr. Silviu Itescu, explains, “Commencing this clinical trial in post-traumatic knee osteoarthritis is an important step towards accessing the huge commercial opportunity that exists today for Mesoblast in the osteoarthritis market.”

It has been estimated that more than 15 million people in the U.S. alone suffer from osteoarthritis, which is a degenerative disease characterized by loss of cartilage. Osteoarthritis constitutes the most common form of musculoskeletal disorders among the elderly, in whom it is the primary cause of disability and joint pain. In the past, joint replacement has been the only option for such patients, but now stem cell therapy offers the first type of treatment that may actually reverse damage and regenerate joint tissue.

For people who suffer a ruptured anterior cruciate ligament (ACL) of the knee, which is more commonly and simply known as ruptured knee, 70% of the patients will develop osteoarthritis 15 to 20 years earlier than the general population, regardless of whether or not they have undergone reconstructive knee surgery. In the U.S. alone there are approximately 300,000 new cases per year of osteoarthritis having developed after an acute traumatic incident, not only in the elderly but in people of varying ages.

Mesoblast Limited is focused on the treatment of orthopedic conditions via the rapid commercialization of proprietary adult stem cell products that are designed for the regeneration and repair of bone and cartilage. As such, Mesoblast has the worldwide exclusive rights for a series of patented technologies that were developed over a period of more than 10 years and which are built upon the utilization of adult mesenchymal precursor cells (MPCs). As described on their website, Mesoblast has acquired 39% equity in Angioblast Systems Inc., which is a private New York-based biotechnology company that is developing the platform MPC technology for the treatment of cardiac, vascular and eye diseases including the repair and regeneration of blood vessels and heart muscle. Together, Mesoblast and Angioblast are jointly funding and advancing the core technologies behind such therapies.

The biotech companies Osiris Therapeutics and Genzyme announced their signing of a collaboration for the commercialization of two adult stem cell products. According to the agreement, Osiris will commercialize the proprietary products Prochymal and Chondrogen in the U.S. and Canada, while Genzyme will commercialize the products throughout the rest of the world. Genzyme will make an upfront payment of $130 million to Osiris in additon to milestone and royalty payments that are estimated to be worth approximately $1.3 billion. Prochymal and Chondrogen are therapies that use allogeneic (in which the donor and recipient are different people) adult mesenchymal stem cells derived from bone marrow. Both of the products are late-stage treatments for a wide variety of diseases.

Although Osiris developed the therapies, Genzyme will provide the financial support for further marketing and commercialization of the products. This strategic alliance will leverage the leadership position that Osiris enjoys in the development of novel adult stem cell therapies, as well as Genzyme’s regulatory and marketing infrastructure outside of the U.S. and its expertise in the commercialization of cell therapies.

Genzyme and Osiris are already strategic allies and this is not their first collaboration. In 2007, the two companies forged an alliance to develop Prochymal for acute radiation syndrome, which resulted in both companies being awarded a $224.7 million contract in January of 2008 by the U.S. Department of Defense to develop Prochymal for the treatment of radiation-induced medical conditions related to warfare and terrorism. Once Prochymal is approved by the FDA for such indications, the Pentagon will buy 20,000 doses at $10,000 each.

According to Dr. Henri A. Termeer, Genzyme’s chairman and CEO, “This partnership further strengthens Genzyme’s late-stage pipeline of products with the potential to support our growth beyond 2011. Osiris is the clear leader in stem cell technology, which holds the promise to transform standards of care in a number of therapeutic areas in which Genzyme already has a strong presence.”

Similarly, Dr. C. Randal Mills, president and CEO of Osiris, adds, “Today Genzyme and Osiris have forged a powerful partnership in the emerging field of stem cell therapy. This relationship greatly enhances our ability to effectively introduce this groundbreaking technology on a global basis.”

As stated by Edward Tenthoff, an analyst at Piper Jaffray & Co., the deal is the largest ever in the field of stem cell therapy. The stem cells under consideration are exclusively adult stem cells, which are derived from mature tissues instead of embryos. According to Tenthoff, “This is a huge validation. This is a major win for everyone involved.”

Osiris is an adult stem cell company which was founded in 1992 and went public in 2006. Yesterday the company’s stock gained 43 cents, or 2.8%, settling at $15.93 a share after the company announced a third-quarter profit of $5.3 million. Its products focus on the treatment of inflammatory, orthopedic and cardiovascular condtions. The company’s adult stem cell product Prochymal is the only stem cell therapeutic product currently designated by the FDA as both an Orphan Drug and as a Fast Track product. Prochymal is currently being evaluated in three separate phase III clinical trials, two of which are for graft vs. host disease (GvHD) and the third of which is for Chron’s disease, both of which are potentially fatal conditions. Prochymal is also in phase II clinical trials for the regeneration of pancreatic beta islet cells in patients with type I diabetes and for the repair and regeneration of damaged lung tissue in patients with chronic obstructive pulmonary disease. Data from all clinical trials are expected in 2009. Prochymal has also been approved to begin phase II clinical trials as a treatment for the regeneration of cardiac tissue following myocardial infarction. Additionallly, Chondrogen has also been approved to begin phase II and III clinical trials for osteoarthritis of the knee. Osiris currently has 47 U.S. patents, each with one or more foreign counterparts.

Genzyme, the world’s largest maker of drugs for rare genetic disorders, is well known for commercializing first-in-class biotechnologies. Founded in 1981, today Genzyme has a staff of more than 10,000 employees around the globe, with revenues of $3.8 billion. In 2007 Genzyme was awarded the National Medal of Technology, which is the highest honor awarded by the President of the United States for technological innovation. In addition to its large orthopedic franchise, Genzyme’s products are focused on rare genetic disorders, kidney disease, cancer, transplant and immune disease, diagnostic testing, cardiovascular disease, neurodegenrative diseases, endocrinology and other medical specializations in which patient needs are not adequately met. Most recently, Genzyme has also begun developing Mozobil, a novel proprietary product which stimulates the mobilization of the body’s own endogenous stem cells. Genzyme’s latest agreement with Osiris marks a major expansion by Genzyme into the field of cell transplant therapies.

As novel proprietary late-stage adult stem cell treatments which have already been shown to control inflammation, to prevent scarring and to promote tissue regeneration, both Prochymal and Chondrogen have the potential to treat a vast range of diseases. The mutual collaborative development and commercialization of these two adult stem cell therapies, by two of the most prominent biotech industry leaders, signifies an important milestone not only in the histories of these two leading companies, but also in the maturation of the adult stem cell industry. As David Meeker, executive vice president for Genzyme, explains, “The technology has evolved to a point where we have a level of confidence where we’re willing to make the deal. We’ll be working on the clinical development going forward and preparing for commercial launch.”

The partnership involves stem cell therapies which consist exclusively of adult stem cells, not embryonic stem cells, since embryonic stem cells have proven to be highly problematic in the laboratory and have therefore never advanced to the clinical stage.

Dr. Cheryl Adams, a pioneer in veterinary stem cell therapy, is one of only two vets in the state of Illinois who are certified to treat animals with stem cells. So far, among the 30 dogs whom she’s treated, she has achieved significant improvement without exception. In all cases, the stem cells used for the treatment were taken from the adult dog’s own body.

Animals often share many of the same physical maladies as humans do, especially bone and joint problems that come with advanced age. Animals who suffer from such ailments are now offering strong clinical evidence for the efficacy of adult stem cell therapy. One case in point is an 8-year-old German Shepherd who had developed osteoarthritis and hip dysplasia. After removing some fat from the dog’s abdomen, Dr. Adams shipped the cells to the San Diego company, Vet Stem, where the stem cells were isolated from the fat and returned to Dr. Adams who injected the stem cells into the dog’s joints. According to the owner of the dog, Chicago Police Lieutenant Jim Gantz, “This science actually gives a chance to rejuvenate tissue, tendon, ligament and bone. So the dog can actually get better.” The procedure costs approximately $3,000, which is less than conventional therapies which may have to be repeated numerous times and are rarely completely effective. Adult stem cell therapy, by contrast, may only need to be administered once, after which results are often “spectacular and almost immediate”, according to Dr. Adams. In reference to the 8-year-old German Shepherd, Dr. Adams describes that, “His energy level went through the roof, and we increased his range of motion by 20 degrees on his right hip.” Similarly, a 12-year-old golden retriever also exhibited remarkable improvement after receiving a similar type of stem cell treatment. Previously unable to walk up or down stairs, the dog was treated with her own adult stem cells two years ago, after which time she regained her ability to climb and descend stairs. Now, two years later, she is still able to do so.

Vet Stem is currently also testing adult animal stem cells in the treatment of damaged kidney, liver and heart tissue in cats and dogs.

At the annual meeting of the American Academy of Orthopedic Surgeons held in San Francisco this week, researchers and physicians alike have agreed that regenerative medicine will be their most powerful tool throughout the future. Rather than surgically replacing joints, as has been the standard medical approach in the past, therapies of the future will focus on the use of stem cells and gene therapy in the new scientific field of tissue re-engineering.

Dr. Thomas Einhorn, chairman of the Department of Orthopedic Surgery and professor of orthopedic surgery, biochemistry and biomedical engineering at Boston University, performed his first hip “replacement” last year with a new technique that utilizes the patient’s own stem cells. As he stated, “It’s the future of our specialty.”

A handfull of companies, such as Stryker and Medtronic, already sell bone morphogenic proteins (BMPs) which are comprised of certain cytokines and growth factors that induce the formation of bone and cartilage. According to Dr. Scott Rodeo, who is co-chief of Sports Medicine and Shoulder Service at the Hospital for Special Surgery in New York and an associate team physician for the Super Bowl champion team, the New York Giants, BMPs in combination with stem cells have already been used in the successful repair of torn rotator cuffs in animal models, thereby offering a promising new type of therapy, which does not involve surgery, for one of the most common shoulder injuries to occur in sports and which normally has only been treatable in the past with surgery.

Previously, the tools of an orthopedic surgeon have typically resembled those of a car mechanic. The heavy metal and even the newer synthetic and composite materials from which artificial hip and knee joints are still fashioned may soon be replaced by BMPs in combination with adult stem cells that are easily and safely derivable from such sources as bone marrow, fat, and umbilical cord blood.

Athletes with sports injuries, and non-athletes with more ordinary conditions such as osteoarthritis and other degenerative diseases of the bones and joints, are expected to benefit greatly by these new techniques in which adult stem cell therapy is already making surgical joint replacement obsolete.

A team of scientists at the University of Edinburgh in Scotland have developed a “bioactive scaffolding” comprised of materials designed to interact with adult stem cells in such a way that even the most serious types of skeletal injuries may now be able to heal completely. Some types of injuries to bone and cartilage are too traumatic to heal without medical intervention, although the conventional types of orthopedic surgery have not always been successful in repairing such injuries. Now Dr. Brendon Noble and his colleagues at the MRC (Medical Research Council) Centre for Regenerative Medicine in Edinburgh have developed a composite “scaffold” which consists of a rigid mesh structure that has been impregnated with chemicals that facilitate the natural action of stem cells derived from bone marrow. Such a device would be useful not only for treating people who have suffered acute injury, but also for treating chronic conditions such as osteoarthritis and cancer of the bone. People of all ages who may suffer skeletomuscular trauma, especially the elderly, would benefit greatly from such a therapy.

Dr. Noble and his colleagues are working in collaboration with the Scottish National Blood Transfusion Service in the harvesting and culturing of blood-derived stem cells that are known to differentiate into bone, and which are incorporated into this new therapy.