The lab of Division of Transplantation Assistant Professor Matthew E. Brown, PhD, is hyper-focused on creating hypoimmune stem cells – cells that can evade recognition and destruction by the human immune system. Why? Because stem cells have the capability of developing into almost any type of cell in the body, and once differentiated into highly functional cells, if they are hypoimmune they can be transplanted into patients without the risk of rejection. With a new two-year, $700,000 grant from Breakthrough T1D, the leading global type 1 diabetes research and advocacy organization, he and his team will be creating and studying hypoimmune, insulin-producing, stem cell-derived islet-like clusters as a potential new treatment for type 1 diabetes.
Type 1 diabetes is a condition where the body’s immune system attacks and destroys beta cells, which are the insulin-producing cells in the pancreas. Without insulin, the body cannot regulate blood sugar levels. Transplantation of insulin-producing cells from a donor can be an effective treatment for patients with type 1 diabetes, but there are two major issues: a shortage of donor tissue and rejection of the transplanted cells by the patient’s immune system.
“By turning stem cells into islet cells, we’re able to produce islets in large numbers in the lab, making it possible to help more people living with type 1 diabetes,” explained Brown. “And by making these islets hypoimmune, we’re aiming to protect them from the patient’s immune system, which normally attacks and rejects transplanted cells as foreign invaders.”
Brown’s team will be testing their gene-edited cells in lab models that closely mimic human biology, using cutting-edge technology to study how these cells behave in a real immune system. Their goal is to develop a universal donor stem cell line that can be transplanted into any patient, regardless of their genetic background. Importantly, if they are successful, this approach could provide a new treatment for type 1 diabetes that eliminates the need for insulin and avoids lifelong use of immune-suppressing drugs, as the latter can have serious side effects.
“By developing gene-edited hypoimmune cells, our research could help advance curative therapies not only of type 1 diabetes, but also other conditions where the immune system plays a destructive role,” said Brown. “With continued research and clinical trials, this work has the potential to significantly improve the lives of the more than 1.5 million people in the U.S. who are living with type 1 diabetes.”