A new stem cell therapy may soon revolutionize the treatment of type 1 diabetes. At the 85th American Diabetes Association conference, held in Chicago in June 2025, researchers presented the results of clinical trials conducted by Vertex Pharmaceuticals of Boston. The presented data could signify a breakthrough in the treatment of this chronic autoimmune disease.
An experimental drug called Zimislecel was administered to 12 patients with severe, insulin-dependent type 1 diabetes. The therapy consisted of a single intravenous infusion of pancreatic islet cells grown from pluripotent stem cells. Ten of the twelve patients showed significant improvement after just one dose. One year after receiving the drug, they no longer required insulin. The remaining two patients also showed significant improvement in their health, allowing for a significant reduction in their daily insulin doses. The reported side effects were mild and included transient renal dysfunction and gastrointestinal symptoms. No serious complications directly related to cell therapy were reported.
The mechanism of action of Zimislecel is based on restoring the pancreas physiological function. The pancreatic islet cells produced in the laboratory implanted themselves in the patients’ pancreas and began to secrete insulin in response to blood glucose levels. This restoration of the natural function of beta cells allowed for glycemic control without the need for daily external insulin administration. In addition, after only three months of therapy, all patients experienced a resolution of dangerous episodes of hypoglycemia, which had previously threatened their lives and significantly reduced their quality of life.
According to an article published in the New England Journal of Medicine, directed differentiation of stem cells opens up the possibility of creating inexhaustible sources of tissue for clinical use. The researchers emphasize that although these are early-stage results and require confirmation in subsequent stages, they already provide significant evidence of the potential of cell therapies in the treatment of autoimmune diseases. At the same time, work is underway to develop methods that will allow patients to become independent of immunosuppressive drugs, which would further increase the safety and accessibility of this method.
For the academic community, these results open up new avenues of research and collaboration between medical universities, biotechnology departments, and the pharmaceutical industry.
Source: DziennikNaukowy.pl, Science Alert, New England Journal of Medicine