A groundbreaking case study has provided a glimpse into the potential future of transplantation medicine for people with type 1 diabetes. A 42-year-old man, who was given gene-edited pancreatic cells via islet cell transplant, can now produce his own insulin without relying on drugs.
Researchers in Sweden and the US collaborated to conduct the study, which used CRISPR technology to modify donated islet cells that were resistant to rejection. The cells continued to produce insulin after four months, with no immune response detected. This breakthrough suggests an alternative approach to circumventing allorejection, a major obstacle to transplant success.
The goal of this research is to develop a self-sustained replacement supply of insulin for type 1 diabetics. However, the traditional approach of lifelong immunosuppressive therapy has its drawbacks, including weakened immunity to infections. The new approach aims to sidestep these risks by reducing immune response.
In the study, the researchers created three specific edits in the donated cells to quiet the immune system’s responses. Two edits depleted HLA class I and II antigens, while a third edit increased production of CD47 protein, which inhibits immune cell activity. The edited cells produced insulin normally, but some were not fully effective.
The patient experienced several adverse events, including mild inflammation. However, his immune system left the fully edited cells alone, even without immunosuppressants. This study is a proof-of-concept for the procedure and aims to demonstrate its safety rather than prove long-term efficacy.
Despite the limitations of this initial study, it offers encouraging signs that scientists are on the verge of making type 1 diabetes curable. Other research teams have reported early success in using similar transplants to cure the condition without immunosuppressant therapy.
Source: https://gizmodo.com/diabetic-man-with-gene-edited-cells-produces-his-own-insulin-no-transplant-drugs-required-2000640127