Stem Cell Treatment May Cure Severe Type 1 Diabetes, Study Finds

A groundbreaking stem cell treatment developed by Vertex Pharmaceuticals has allowed 10 out of 12 patients with severe type 1 diabetes to stop insulin therapy after one year. While the trial included some side effects and two unrelated deaths, the results mark a major step forward and have progressed to phase 3 clinical testing. ScienceAlert reports: The pancreas’s islet cells are responsible for maintaining most of our bodies’ insulin levels. Donor transplants of healthy versions of these cells have shown promise in treating type 1 diabetes in the past, but multiple donors are required, and donors are rare. So University of Toronto surgeon Trevor Reichman and colleagues infused 12 patients with islet cells derived from human stem cells in a treatment known as zimislecel. The patients also received immunosuppressive treatment before and after their zimislecel infusion. The islets not only produced insulin inside their bodies, but they did so at safe levels, reducing the patients’ dependence on costly doses of insulin. “These findings showed that zimislecel islet cells were functional and self-regulated appropriately,” the researchers write in their paper.

The mild to moderate side-effects, including decreased kidney function and the anticipated drop in immune cells, were all linked with the immunosuppressive therapy. Sadly, two additional participants died during the trial; one from an infection arising from surgery and the other from complications due to an unrelated condition. As there were no serious adverse events attributed to the new islet cell therapy, the clinical trials are have progressed into phase 3. “These findings provide evidence that pancreatic islets can be effectively produced from pluripotent stem cells and used to treat type 1 diabetes,” Reichman and team conclude. The research has been published in the journal NEJM.