Breakthrough in Diabetes Research: Patient Produces His Own Insulin After Gene Therapy
A research team at Uppsala University in Sweden has achieved a significant breakthrough with a diabetes patient. They successfully transplanted gene-edited, insulin-producing cells into a 42-year-old man with type 1 diabetes. The patient's immune system did not reject the cells, and they began to produce insulin.
The case was published in the journal The New England Journal of Medicine and offers a glimmer of hope for millions of people affected by the disease. Type 1 diabetes is an autoimmune disease where the pancreas produces little to no insulin. This vital hormone ensures that sugar absorbed from food is converted into energy for the body. Without insulin, sugar accumulates in the blood, which is why people with type 1 diabetes must inject insulin daily for life. To date, there has been no cure.
The study is an important step on the path to a "hypoimmune" cell therapy that could mean a cure for people with type 1 diabetes in the future.
Modified Donor Cells Prevent Rejection
Previous attempts using donor cells failed because the immune system rejected the transplanted cells. This would require the lifelong use of immunosuppressive drugs, which have significant side effects. The Uppsala University research team modified the cells with CRISPR technology before transplantation. They removed HLA-I and HLA-II antigens, which can trigger rejection by the recipient's immune system. Additionally, the cells were modified to produce more CD47 protein, which helps prevent attacks from the innate immune system.
Following tests on rodents and monkeys, the 42-year-old patient received a total of 17 injections of donor cells into his forearm in December 2024. The unmodified donor cells were rejected and destroyed by his body. However, the completely modified cells survived without immunosuppressive drugs and began to produce insulin three months after the injection.
Research Team Confident in Potential for Full Cure
The man is not yet cured and still requires insulin injections. Due to regulatory requirements, the research team administered only 7% of a potential curative dose. His blood and condition will now be monitored regularly for at least 15 years. Following this initial success, the scientists now plan to cultivate insulin-producing cells from stem cells, which would allow them to produce a much larger quantity than with donor cells.
The medical community is cautiously optimistic about the research team's breakthrough. It was only a single patient, and long-term observation is still ongoing. However, the results are so promising that there is great hope for wider application. The research team itself is confident that a higher dose could lead to complete independence from insulin.
The study is, in any case, an important step on the path to a "hypoimmune" cell therapy that could mean a cure for people with type 1 diabetes in the future.
