Diabetes Drug Approval Re-energizes Research Community

When Linda DiMeglio, M.D., Riley Children’s Health endocrinologist and division chief of pediatric endocrinology and diabetology, began treating patients at Riley Children’s, she imagined a cure for diabetes would be in the foreseeable future.

That was 25 years ago. Her dream, shared by so many, may be within reach, thanks in part to Dr. DiMeglio and her colleagues at the Riley-affiliated Herman B Wells Center for Pediatric Research at Indiana University School of Medicine.

In November 2022, the Federal Drug Administration (FDA) approved the drug teplizumab (Tzield), a new immunotherapy that delays the onset of Type 1 diabetes in at-risk individuals by an average of almost three years. Wells Center researchers helped conduct the clinical trials that led to this landmark achievement, the culmination of nearly 20 years of investigation and clinical trials.

The Wells Center team says this game-changing development was only possible thanks to a massive collaboration among individuals with Type 1 diabetes, researchers, advocacy groups and funders who have supported this research, refusing to consider insulin as the only lifesaving treatment.

The Indiana team contributing to the teplizumab study comprises a rich network of clinical and research faculty, including Carmella Evans-Molina, M.D., Ph.D., M.S., director of the Indiana Diabetes, Indiana Diabetes Research Center; Jamie Felton, M.D., assistant professor of pediatrics; Heba Ismail, MBBCH, Ph.D., assistant professor of pediatrics; Emily Sims, M.D., associate professor of pediatrics, and Dr. DiMeglio.

“This is an impressive team of researchers,” says Dr. Evans-Molina. “We have people doing rigorous basic science, translational research and these clinical trials.”

“Since the early ’90s, people have been trying to delay the onset of Type 1 diabetes. This is the first successful drug to do that,” says Dr. Sims, Wells Center assistant director of faculty development and translational research. Dr. Sims is especially proud that her team not only participated in the trial that led to this approval but that analyses she performed using metabolic data from the clinical trial contributed to the FDA’s decision.

“This drug is designed for individuals who are in stage two of the disease which is marked by the presence of two antibodies and problems with blood glucose to delay them from transitioning to stage three which needs to be treated with insulin,” explains Dr. Evans-Molina.

“It’s really quite impactful when you consider the burden of injecting insulin; checking blood glucose multiple times a day; and thinking about everything you eat and counting carbohydrates,” she adds. “With Tzield, children will be able to delay stage three by a median of 32.5 months, or about two and a half years.”

New clinic helps children with diabetes

A fortunate convergence of developments brings the FDA approval of Tzield alongside the launch of the new Riley Early-Stage Diabetes Clinic with Dr. Felton serving as director. The clinic is a medical home for children, families and primary care physicians looking for resources, screenings, diagnosis, treatment guidelines, medical management and clinical trial information.

“Patients who engage in early education and monitoring are healthier when they do start to use insulin,” says Dr. Sims. “They can avoid conditions like diabetes ketoacidosis (DKA) which is a condition in which their acid level is very high; they’re unstable; and require time in the intensive care unit for an insulin drip,” she explains. “It can be life threatening, and very stressful for families.” The Riley Early-Stage Diabetes Clinic’s goal is to work with families to help them know what to expect and remain as healthy as possible.

Research team has an eye on the future

“We want to continue to partner with public and private funders – people who’ve put their trust in us,” said Dr. Evans-Molina. While they’ve prioritized getting the clinic set up, Dr. Evans-Molina says their vision is bold and will require collaboration with state and national funders, policymakers and health insurers to develop a population-based screening approach so children would be screened for diabetes risk during their well-child visits.

“That is our dream and the wave of the future we’d like to see happen. Rolling out a public health initiative, that’s a very costly endeavor. Our approach is to understand and partner with Riley Children’s Foundation donors, the Helmsley Charitable Trust and the Juvenile Diabetes Research Foundations of the world.”

Despite her team’s amazing success, Dr. Evans-Molina says the biggest accomplishment is not the FDA approval of Tzield alone, but the myriad of research and clinical care resources that are and will be available to get children and families the care they need.

“I’m thinking of these developments as a love letter to donors,” Dr. Evans-Molina says.