For the first time, a patient with type 1 diabetes has received a transplant of pancreatic islet cells from a donor without requiring immunosuppressive drugs or experiencing rejection. The breakthrough, achieved by researchers at Sweden’s Uppsala University and documented in the New England Journal of Medicine, utilized genetically modified cells engineered to evade immune response.
The experimental therapy, named UP421, involved implanting CRISPR-edited donor pancreatic cells into the patient’s forearm. While the transplant did not aim for therapeutic benefit, the cells remained functional for 12 weeks, producing insulin without immunosuppression. Dr. Lorenzo Piemonti, head of regenerative medicine at Milan’s San Raffaele Hospital, emphasized this is a proof of concept: “The transplanted cell quantity was low, and insulin output minimal, but we now have evidence that edited cells can survive in humans without immunosuppressants.”
Future validation could expand access to islet transplantation, eliminating immunosuppression risks and daily insulin injections for countless patients, noted Raffaella Buzzetti, president of the Italian Diabetes Society. Further trials are required to confirm the approach.