Celebrating Riley's Role in Diabetes Drug Approval

For an individual with a diagnosis of type 1 diabetes, living with the disease for almost three years means having to check their blood glucose more than 4,000 times. During that time, they will also experience about 200 hypoglycemic events, when a low blood sugar level triggers symptoms like sweating, fatigue, dizziness, tremors, and confusion.

Here is the good news: last November, the Federal Drug Administration approved teplizumab, an immunotherapy drug that delays the onset of type 1 diabetes in at-risk individuals by an average of 2.7 years. Researchers at the Herman B Wells Center for Pediatric Research at Indiana University School of Medicine played a key role in conducting clinical trials, and led data collection and analysis of the drug.

The team’s excitement is clear. “We’re so lucky to have the opportunity to participate in this because we’re watching how we care for our patients unfold and evolve in front of our eyes,” said Emily Sims, M.D., Riley Children’s Health endocrinologist and physician-scientist at the Wells Center.

Type 1 diabetes occurs when the body’s immune system attacks insulin-producing beta cells in the pancreas, causing abnormal blood glucose levels. A fortunate combination of developments brings the FDA approval of teplizumab alongside the launch of a new early-stage diabetes clinic at Riley Children’s Health. Early stage means that patients have antibodies but their beta cells are still functioning to a certain level. The clinic’s role is designed to identify early stage patients, monitor them in a way that is most beneficial, and ensure they are aware of research opportunities – and treatment opportunities like teplizumab – when they become available. It also provides a medical “home” where families can sit down face-to-face with caregivers to ask questions.

Riley Children’s endocrinologist Jamie Felton, M.D., leads the clinic, and she expects her patient numbers to continue growing. “My vision is helping families be more aware of treatments that are available that are already FDA approved as well as new research opportunities, because inevitably this is going to spark more interest,” said Dr. Felton. “The FDA approval has really energized people’s thoughts about therapies that could potentially prevent type 1 diabetes.”

Other Riley team members contributing to the teplimuzuab study include Carmella Evans-Molina, Ph.D., M.S., M.D., Linda DiMeglio, M.D., and Heba Ismail, MBBCH, Ph.D. “Support from donors allows our group to pursue high-risk and potentially high reward ideas, and build evidence for continued grant support and future proposals. We are incredibly grateful,” shared Dr. Evans-Molina.

Dr. Sims can’t imagine a more rewarding time to donate in support of Riley research. “This research is changing how we’re caring for patients right now,” she shared.