Stem-cell upstart Viacell Inc. and Genzyme Corp. are joining forces to develop a therapy they said has a chance of curing juvenile diabetes within years.
The collaboration with Genzyme is a strong endorsement for Viacell, which went public in January. Terms of the deal between the Cambridge firms were not disclosed, but Genzyme said it will pay for development of the experimental treatment until it is ready for human clinical trials, estimated to take two years.
''This is the only thing I see that has a potential to cure Type 1 diabetes," said Marc Beer, Viacell's chief executive.
Type 1, or juvenile, diabetes is a condition where the body's immune system destroys the islet cells in the pancreas, which produce insulin. That enzyme is crucial to maintaining a proper blood sugar level. Both Type 1 and Type 2, or adult onset, diabetes produce a range of painful and potentially fatal complications. People with Type 1 diabetes inject themselves with insulin several times a day to regulate their blood sugar. Researchers believe juvenile diabetes shortens a patient's life span by an average of 15 years.
Viacell's approach is to take pancreatic tissue from cadavers and isolate the stem cells that mature into islet cells. Using unique methods, Viacell grows those cells into enormous quantities of islet stem cells. Doctors then inject the cells into the main vein entering the liver. The cells are believed to take up residence in the liver -- and perhaps other organs -- and begin generating insulin.
Viacell is working to develop cell-based therapies that sidestep the controversy over stem cells, which are tiny, undifferentiated cells with the ability to develop into any type of body tissue. Many scientists use stem cells harvested from days-old embryos, such as those left over from fertility treatments. Many abortion opponents object to this technique because they say scientists should not destroy a potentially viable fetus for medical purposes. Viacell seeks to develop treatments from newborns' umbilical cord blood, which is rich in stem cells.
Viacell's brash attitude toward an intractable disease that affects 1 million to 2 million US patients elicited plaudits and caution in the diabetes community.
''The concept is very exciting and we hope it works," said Dr. Robert Goldstein, chief scientific officer of the Juvenile Diabetes Research Foundation International, the largest charity that funds research into Type 1 diabetes. ''We look forward to seeing how this industry collaboration can expedite the time to clinical trials."
Others questioned whether Viacell and Genzyme can get their experimental therapy -- which has so far been tested only in mice -- into the clinic within two years.
''Two years is very optimistic," said Susan Bonner-Weir, a senior investigator in islet transplantation and cell biology at the Joslin Diabetes Center in Boston. ''To get into the clinic they have to know what they are isolating, they have to be able to grow the stem cells, and they want to be sure the cells" function properly in the body, secreting insulin as needed based on blood sugar levels.
Researchers have worked for years to develop ways of replenishing islet cells in diabetic patients. Hundreds of diabetics have received islet cells under a special drug regimen that prevents the patients' bodies from rejecting the foreign cells. But there aren't enough donated organs to treat more than a tiny sample of patients.
''If you could create limitless amounts of tissue and convert it to islet-like clusters, you could treat many more patients," said Dr. Joel Habener, chief of the Molecular Endocrinology Lab at Massachusetts General Hospital. Viacell licenses Habener's patented research as the basis for the experimental diabetes treatment.
Under the terms of the deal with Genzyme, the firms will collaborate until the treatment is ready to enter human clinical trials. Then, Genzyme has first rights to negotiate a second deal to bring the drug to market. If those talks fail, Viacell can proceed on its own or with another partner.
There are a variety of close ties between Genzyme and Viacell. Genzyme made three separate equity investments in Viacell prior to the firm's January IPO. Jan van Heek, a Genzyme executive vice president, sits on Viacell's board. Beer, Viacell's chief executive, served as vice president of global marketing for a Genzyme division before he joined Viacell in 2000.
Beer said he expects the collaboration to turn into a long-term partnership. ''I'm going into this relationship with the clear intention that Genzyme will be our commercial partner," he said.
Viacell shares, which started trading in January at $7, yesterday gained 19 cents to close at $7.30. Genzyme rose $2.28, or 4.1 percent, to close at $57.88.
Jeffrey Krasner can be reached at firstname.lastname@example.org.