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UM DIABETES PIONEER WRITES JAMA EDITORIAL ON A PROMISING NEW CELLULAR THERAPY STRATEGY FOR DIABETES
A national leader in the management and treatment of diabetes at the University of Miami Leonard M. Miller School of Medicine has penned an editorial on a promising new treatment for the April 11 issue of the Journal of the American Medical Association. Jay S. Skyler, M.D., professor of medicine, pediatrics and psychology at UM’s Diabetes Research Institute, wrote about an experimental study by a team in Brazil, which is published in the same issue.
In the study, “Autologous Hematopoietic Stem Cell Transplantation in Early Onset Type 1 Diabetes Mellitus,” Brazilian researchers harvested hematopoietic stem cells from people newly diagnosed with type 1 diabetes. This is the same process which has been used for many years in bone marrow transplants. Each patient’s stem cells were then conditioned with cyclophosphamide, an alkylating agent, and an immune suppressor, antithymocyte globulin. Then the stem cells were injected back into the patient. “They isolated their cells, tried to purify the CD34 stem cells, and then gave them back to the same individual,” said Skyler. This is known as autologous hematopoietic stem cell transplantation, because the enriched stem cells are injected back into the person they came from in the first place. This strategy eliminates the need for tissue or blood typing and eliminates the risk of rejection.
“One of the nice things about autologous procedures like this is that they do not require long-term immunosuppression,” said Skyler. “If it re-sets the immune system, which is the hypothesis they put forward, it could really complement our islet cell transplants.” The Diabetes Research Institute at the UM Miller School of Medicine is a world leader in islet cell transplantation, a procedure which has successfully helped a number of patients reduce or eliminate the need for insulin.
“Their concept is that the stem cells then provide an arrest of the immune process,” said Skyler. “But it isn’t clear that the stem cells may also repopulate the pancreas.” Diabetes stems from an immune system attack on islet cells in the pancreas. Islet cells produce insulin, which regulate the body’s consumption of sugar for energy. When the immune system destroys the islets, the person develops diabetes which often requires daily insulin to maintain health. Of the 15 patients with diabetes involved in the Brazilian study, 14 were able to stop taking insulin – some for as long as 35 months. In his editorial, Skyler writes that the advantage of trying this therapy early in the disease process is that, if it works, it may help salvage the remaining islet cells, delaying or preventing more advanced diabetes.
Skyler details three critiques of the Brazilian study: It was very small, enrolling only 15 patients; there is no control group of people with diabetes receiving different care, for comparison; and there is no long-term data. In fact, Dr. Skyler feels that they should have followed this group of patients for a longer period before they publish their results.
Still, Skyler sees promise in the results. “I use the editorial to state that there is growing interest in using these and similar cell therapies in type 1 diabetes,” he said. UM researchers are already crafting their own cell therapy protocols. “There are lots of notions of things we can be doing in cell therapy, stem cells, dendritic cells, regulatory cells, and others and the time is right to pursue these.”
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April 9, 2007
Media Contact: Kelly Kaufhold
Medical Communications,
UM Leonard M. Miller School of Medicine
305-243-5184 / mobile 305-458-9654
kkaufhold@med.miami.edu
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