First Stem Cell–Derived Islet Therapy Achieves Insulin Independence in Type 1 Diabetes Patients

Published 2025-06-30

A groundbreaking study published on June 20, 2025, in The New England Journal of Medicine has revealed that a stem cell-based therapy developed by Vertex Pharmaceuticals successfully restored insulin production in patients with Type 1 diabetes. The therapy, known as VX-880 (zimislecel), uses fully differentiated, allogeneic islet cells derived from pluripotent stem cells and was tested in the FORWARD Phase 1/2 clinical trial. Administered as a single infusion into the portal vein, the therapy led to insulin independence in 10 out of 12 participants, along with normalization of HbA1c and elimination of severe hypoglycemic episodes over a one-year follow-up.

The study marks a pivotal advance in regenerative medicine, demonstrating for the first time that lab-grown islet cells can functionally replace insulin injections in a clinical setting. While the treatment currently requires immunosuppression, ongoing research is exploring encapsulation and immune-evasive strategies to eliminate this barrier. Phase 3 trials are now underway, with broader regulatory approval anticipated pending long-term efficacy and safety validation. This innovation represents a major step toward a functional cure for Type 1 diabetes.