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A recent study by Vertex Pharmaceuticals is testing a new stem cell-derived therapy for people with type 1 diabetes, particularly those at high risk for severe hypoglycemia.
The investigational therapy, VX-880, uses fully differentiated pancreatic islet cells to replace damaged ones, with the goal of helping patients produce insulin naturally. The treatment was tested on 17 patients, and thus far, three have become insulin-independent within one year of receiving the full dose. These patients also achieved an A1c level below 7%, a significant improvement.
The VX-880 therapy is delivered through an infusion into the hepatic portal vein, and patients are required to take immunosuppressive drugs to prevent their bodies from rejecting the transplanted cells.
Experts from The University of Chicago presented these findings at the American Diabetes Association’s 84th Scientific Sessions. They noted that no severe adverse events were directly related to the VX-880 product itself, although there were some complications from the infusion procedure and the required immunosuppression.
While the results so far are promising, ongoing research aims to further improve the therapy by reducing or eliminating the need for immunosuppressive drugs. Several strategies are being explored, including encapsulating the islet cells or using gene-editing techniques to prevent immune rejection. The study has been expanded to include more patients to fast-track FDA approval.
This innovative approach holds great potential for treating type 1 diabetes, offering an alternative to traditional treatments like insulin therapy and pancreas transplants. With further development, it could significantly improve the quality of life for those living with this condition.
