University of Wisconsin News and Events, August 10, 2009

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.