The strategy behind the study is based on the knowledge that Type 1 diabetes occurs when T-cells from the immune system attack beta cells in the pancreas — the cells that produce insulin. Previously, one method researchers have used in an attempt to neutralize this attack is to reboot a patient’s own immune system by infusing that patient with his or her own blood stem cells through what’s known as an autologous bone marrow transplant. The problem has been that the blood stem cells of people with diabetes tend to be defective, which can promote inflammation.
Researchers at Boston Children’s Hospital, however, said they found a way to fix the defect in the patient’s blood stem cells. They do this by treating the blood stem cells with small molecules or with gene therapy. The treatment stimulates the cells to produce more of a protein called PD-L1, which has a strong anti-inflammatory effect. Then, when they’re introduced into the pancreas, the treated cells bind to receptors on the T-cells. The T-cells either die or become inactive. According to Paolo Fiorina, MD, senior investigator on the study, “There’s really a reshaping of the immune system when you inject these cells.”
Researchers tested the procedure on mice and reported they reversed Type 1 diabetes in the animals. All mice were cured of diabetes temporarily, and one-third of them were cured for the rest of their lives.
The next step, of course, is to try the treatment on people with diabetes. Accordingly, Boston Children’s researchers are partnering with a California therapeutics firm to refine the procedure for modifying the blood stem cells and then to begin a trial in humans. According to Fiorina, “The beauty of this approach is the virtual lack of any adverse effects, since it would use the patient’s own cells.”
Want to learn more about recent Type 1 diabetes research? Read “Protecting Beta Cells and Preventing Type 1 Diabetes,” “New Staging Classification for Type 1 Diabetes” and “IBM and JDRF: Organizations Partner to Combat Type 1 Diabetes.”