2024 Health Care Heroes: Riley Children’s Health/ Indiana University School of Medicine

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From left, Dr. Emily Sims; Dr. Carmella Evans-Molina; Maria Spall;
Dr. Jamie Felton; Anna King and Dr. Linda DiMeglio. (IBJ photo/Eric Learned)

Honoree, Innovations in Health Care

Last August, Riley Hospital for Children achieved an important milestone, treating the first Indiana child with a new, 14-day autoimmune therapy called teplizumab, which delays the onset of stage 3 type 1 diabetes by an average of two to three years. For almost a century, it’s been possible to ameliorate the ravages of diabetes using insulin, but this is the first time it’s been stopped in its tracks—at least for a while.

The treatment doesn’t permanently prevent the onset of stage 3 type 1 diabetes (clinical diagnosis of type 1 diabetes), but delaying it even a couple of years can vastly improve patients’ quality of life—and might open the door for even more advanced and effective remedies.

Credit for this achievement belongs in part to Dr. Emily K. Sims, associate professor of pediatrics at the Indiana University School of Medicine and pediatric endocrinologist at Riley Children’s Health—along with Dr. Linda A. DiMeglio, a pediatric endocrinologist at Riley Children’s Health and the Edwin Letzter professor of pediatrics and division chief of pediatric endocrinology at the IU School of Medicine; and Dr. Jamie L. Felton, assistant professor of pediatrics at the IU School of Medicine, pediatric endocrinologist and Early Stage T1D Clinic director at Riley Children’s Health.

That team, using patient participants from Riley Children’s Health, served as one of only 28 sites across the country conducting clinical trials of teplizumab, helping to facilitate its approval by the FDA last November as the first disease-modifying drug cleared for subjects 8 years and older with stage 2 type 1 diabetes.

“When we did the analysis of the children and the adults who participated in the study, we could see that they were losing their ability to make their own insulin over time,” Sims said. “But after they got the drug, there was a reversal of the process, and they started to make more of their own insulin again.”

Riley Children’s Hospital recently opened a new clinic for patients at high risk for developing type 1 diabetes.

“The early-stage clinic was developed essentially as a result of FDA approval of teplizumab,” Felton said. “From a research standpoint, we’ve followed kids that have been at risk for developing type 1 diabetes for a long time, but there hasn’t been anything that we could offer them until now.”

Though the drug typically delays only the onset of type 1 diabetes, even buying just a couple of more good years can make a huge difference to a pediatric patient, both in reducing health care costs for their families and in allowing them to enjoy a childhood not centered around insulin injections and constantly monitoring their blood-sugar levels.

And with a drug that stops the progression of diabetes for up to three years in hand, even more effective compounds might be just around the corner.

“The ultimate goal would be that we have a number of therapies that we could offer people in different stages of risk,” Felton said. “A lot of the research that’s being done at IU and that my colleagues and I are working on now is about looking into other options.”•

Editor’s note: Previous references to Type 2 diabetes were removed from this story.

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