Reversing Type 1 Diabetes. Researchers at Boston Children’s Hospital recently published research that could eventually lead to a cure for type 1 diabetes.
The strategy behind the study is based on the recognition that type 1 diabetes occurs when the T cells of the immune system attack the beta cells of the pancreas, the cells that produce insulin. Previously, researchers who tried to neutralize this attack have reinitiated the patient’s own immune system by infusing this patient with their own blood stem cells through a so-called autologous bone marrow transplant. The problem was that the blood stem cells of people with diabetes tend to have flaws, which can promote inflammation.
The researchers at Boston Children’s Hospital, however, said they had found a way to remedy the defect in the patient’s blood stem cells. They do it by treating blood stem cells with small molecules or with gene therapy. The treatment stimulates the cells to produce more than one protein called PD-L1, which has a strong anti-inflammatory effect. Then, when they are introduced into the pancreas, the treated cells bind to the receptors in the T cells. The T cells die or become inactive. According to Paolo Fiorina, MD, principal investigator of the study, “there is really a reorganization of the immune system when these cells are injected.”
The researchers tested the procedure in mice and reported that they reversed type 1 diabetes in animals. All the mice were temporarily cured of diabetes, and a third of them were cured for the rest of their lives.
The next step, of course, is to try to treat people with diabetes. As a result, Boston Children’s researchers are working with a Californian therapeutic company to refine the process of modifying blood stem cells and then begin a study in humans. According to Fiorina, “the beauty of this approach is the lack of side effects, since it would use the patient’s own cells.”