Gene and cell therapy approaches to treat diabetes

Transplantation of pancreatic islets has proven to be effective in controlling blood glucose levels in subjects with type 1 diabetes. The results of this procedure have demonstrated the potential to treat diabetes by transplanting as little as a teaspoon of insulin-producing cells into a diabetic patient. However, the promise of this procedure is currently restricted by two major challenges: transplantation is dependent upon the availability of tissue from recently deceased individuals; it also requires the use of chronic immunosuppression in the recipient, which carries the risk of side effects. Dr. Timothy Kieffer is investigating a variety of approaches to achieve the results of islet transplantation without relying on donor tissue. Ideally, he hopes to develop a therapy that uses the patient’s own cells and stimulates either the regrowth of insulin-producing beta cells, or the generation of new cells from adult stem cells. His team is also looking at ways to genetically modify the body’s gut cells to produce insulin or other anti-diabetic factors automatically in response to eating a meal.