Stem cell therapy shows promise in treating lifelong disease Image By HPN Staff Key Points In a trial of 12 patients with type 1 diabetes, 10 were cured and 2 saw major improvement using the stem cell therapy Zimislecel. The therapy rebuilds pancreatic islet cells, restoring natural insulin production; however, patients must take lifelong immunosuppressants to avoid rejection. Vertex Pharmaceuticals plans to seek FDA approval next year, potentially making this the first functional cure for type 1 diabetes available in the U.S. A stem cell therapy tested on a dozen people with type 1 diabetes seems to have cured 10 of them and significantly improved life for two others, according to a new study published in The New England Journal of Medicine. The treatment used stem cells to grow new islet cells, a crucial part of the pancreas that regulates blood sugar. The head of the diabetes center at the University of California in San Francisco, who was not involved in the study, told The New York Times that the study is “trailblazing work” with the potential to change the lives of people living with this disease. Why it matters Some 2 million Americans have type 1 diabetes, which means their body doesn’t produce enough insulin needed to regulate blood sugar. The cause is not known, and those who suffer from type 1 diabetes must regularly take insulin to control their blood sugar. The disease typically appears in children, as opposed to type 2 diabetes, which may emerge in adults and is often tied to obesity. Routine insulin shots can be expensive despite policy efforts to lower costs. This study focused on a stem cell therapy called Zimislecel to rebuild the islet cells along with immunosuppressive therapy so the body would accept the new cells. Patients “began to need less insulin within a few months of being infused with new islet cells, and most stopped needing the hormone altogether at about six months,” The Times reported. Or as the study itself put it: “All 12 participants in parts B and C were free of severe hypoglycemic events and had a glycated hemoglobin level of less than 7%; these participants spent more than 70% of the time in the target glucose range (70 to 180 mg per deciliter). Ten of the 12 participants (83%) had insulin independence and were not using exogenous insulin at day 365.” The bigger picture The treatment is owned by Vertex Pharmaceuticals, which partnered with a Harvard University researcher named Doug Melton, who has studied type 1 diabetes for decades. The company hopes to apply to the Food and Drug Administration to approve Zimislecel next year, The Times reported. It noted that patients who use the treatment may need to stay on immunosuppressant drugs the rest of their lives to keep their bodies from rejecting the new islet cells. That increases the risk of other infections. Additional details Melton is famous in research circles, in part because he dedicated himself to a cure for type 1 diabetes after his infant son developed the disease. About a decade later, his daughter was also diagnosed with the disease. “I’m not inventing anything,” Melton told The Harvard Gazette for a profile published earlier this year. “I’m trying to copy nature.”
