December 12, 2025
Cell transplant therapy offers new hope for treating type 1 diabetes
Scientists have successfully transplanted gene-edited insulin-producing cells into a person with type 1 diabetes, allowing him to produce some of his own insulin without immunosuppressive drugs.
This video is part of “Innovations in: Type 1 Diabetes“, an editorially independent special report produced with financial support from Vertex.
Karin Leong: What if people with type 1 diabetes could start making their own insulin?
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Scientists have just taken a big step in this direction. They treated the patient with 80 million lab-made insulin-producing cells designed to evade the immune system. This is the first time such a cell transplant has failed to cause rejection in a person, and the researchers say it opens up exciting possibilities for treating diabetes and other autoimmune diseases in the future.
About two million people in the United States currently live with type 1 diabetes. This is an autoimmune disease in which the body mistakenly destroys the cells in the pancreas that produce insulin. Without this hormone, people must rely on injections and pumps every day to control their blood sugar and avoid serious complications.
Scientists have tried to replace these insulin-producing cells before, but the body continues to attack them. And patients will have to take strong immunosuppressive drugs for life, which have their own long list of side effects.
This time, the researchers took donor cells and used the gene-editing technique CRISPR to deactivate two genes that normally signal the immune system to attack foreign cells, as well as boost expression of a gene that prevents the body's immune cells from attacking them.
Thus, 12 weeks after these cells were injected into the patient, they were still alive and producing insulin in his body. Of course, it wasn't a ton – about 7 percent of what he needed to completely stop taking insulin injections. But experts say a major milestone for his body is the ability to produce at least some insulin on its own and, most importantly, without the need for immunosuppressive drugs. They will continue to monitor him over the next year and test higher doses of these edited cells. And if all goes well, it could potentially lead us to a cure for type 1 diabetes.
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