JAMA report on transplant treatment for newly diagnosed type 1 diabetes patients

New York, NY, April 11, 2007 — The Journal of the American Medical Association reported on April 11, 2007, that a procedure similar to a bone-marrow transplant was able to reverse type 1 diabetes in a small clinical trial in South America.
An editorial in the JAMA called the report “provocative.” It is that, and might some day have an important impact on understanding the autoimmune attack that causes type 1 diabetes and reversing it in newly diagnosed patients.
The treatment involves taking blood from the patient and isolating the hematopoietic stem cells that give rise to bone marrow and blood cells, destroying the patient’s existing immune system and other cells, then returning the patient’s own hematopoietic stem cells to the body to develop a new immune system. In the trial, the immune system was apparently reset or retrained, and after the procedure, the symptoms of diabetes were reversed.
The procedure—called autologous nonmyeloablative hematopoeitic stem cell transplantation—carries some significant risks and has usually been reserved for patients with either life-threatening disease, like leukemia, or organ-threatening autoimmune diseases. Because of those risks, it is not clear whether this trial would be approved in the U.S.
The trial presents a number of positives. It indicates that the immune system of people with type 1 diabetes can be retrained or reset, at least for a period of time. And it suggests that when the autoimmune response is interrupted, patients with some insulin-producing cells remaining (common in many newly diagnosed patients) can begin to regenerate enough additional beta cells to reduce or even eliminate their insulin requirements.
As with any early clinical trials involving new treatments or procedures, there are a number of issues that will need to be addressed before this approach can become widely available to patients with type 1 diabetes. The risks associated with a highly invasive treatment need to be better quantified, mitigated, and weighed against the benefits of the procedure. What exactly happens in the treatment—whether the immune system is “re-trained,” or just reset, with the risk of diabetes returning at some point—needs to be better understood. And the potential patient universe that could benefit from such a procedure needs to be outlined (as the trial involved just the recently diagnosed, who may have significant insulin-producing cells remaining) and the long-term effects have not been defined at this time.

Source- JDRF

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