Stem-Cell Therapy Frees Type 1 Diabetes Patients From Insulin

For people living with type 1 diabetes, the immune system destroys the insulin-producing islet cells of the pancreas, forcing a lifetime of blood-sugar monitoring and insulin injections. A clinical trial published in the New England Journal of Medicine points toward a fundamentally different future, using lab-grown cells to restore the body's own ability to make insulin.

The therapy, called zimislecel, is an investigational treatment made from pluripotent stem cells that are coaxed into becoming fully functional, insulin-producing islet cells. These cells are then infused into the portal vein of the liver, where they take up residence and begin sensing blood sugar and releasing insulin much as a healthy pancreas would. In the phase 1/2 trial, 12 participants received the full dose.

“A clinical trial published in the New England Journal of Medicine points toward a fundamentally different future, using lab-grown cells to restore the body's own ability to make insulin.”

The results were remarkable. Ten of the 12 participants — about 83% — achieved insulin independence at one year, meaning they no longer needed injected insulin. All participants avoided severe hypoglycemic episodes, achieved hemoglobin A1c levels below 7%, and spent significantly more time in the healthy blood-sugar range. "Ten out of 12 patients achieved insulin independence, meaning they are no longer managing their disease every hour of every day," said Dr. Trevor Reichman, who directs the islet transplant program at UHN's Ajmera Transplant Centre.

There are important caveats. Because the cells come from a donor source, recipients must take immunosuppressant drugs to prevent rejection, which carry their own risks — so the therapy is currently aimed at patients with the most severe, hard-to-control disease. The trial was small and is now expanding into a larger phase 3 study, with regulatory submissions anticipated. Still, watching a stem-cell therapy free most participants from daily insulin is an extraordinary milestone, and a powerful glimpse of how regenerative medicine could one day transform life with type 1 diabetes.