New DFMO drug is a game changer for patients with type 1 diabetes
In a recent study led by Indiana University School of Medicine Department of Pediatrics, physician-scientists unveil promising prospects for the management of type 1 diabetes in pediatric patients.
Pioneered by Linda DiMeglio, MD, MPH, division chief of pediatric endocrinology and diabetology at Riley Children’s Health, the study’s findings propose a novel approach to repurposing a-difluoromethylornithine (DFMO), an FDA-approved drug primarily used to treat African Sleeping Sickness and neuroblastoma maintenance therapy, to potentially reduce insulin dependency.
Type 1 diabetes, a chronic condition characterized by the immune system’s destruction of insulin-producing beta cells in the pancreas, has long required lifelong insulin treatment to regulate blood sugar levels. However, with the current standard treatments, including daily insulin injections and frequent blood sugar monitoring, these often pose significant challenges such as inconvenience as well as the risk of hypoglycemia or hyperglycemia.
The study, published in Cells Report Medicine, builds upon over a decade of research to find an alternative solution to the standard approach. With roots tracing back to 2010, Raghu G. Mirmira, MD, PhD, professor of medicine at UChicago Medicine, discovered that inhibiting the metabolic pathway targeted by DFMO could protect beta cells, hinting at potential preservation in type 1 diabetes. Subsequent validation in mice further bolstered these findings. Following that work, a clinical trial spearheaded by Dr. DiMeglio, funded by the Juvenile Diabetes Research Foundation (JDRF) with drugs provided by Panbela Therapeutics, demonstrated DFMO’s safety in newly diagnosed type 1 diabetes patients, suggesting its potential to stabilize insulin levels by protecting beta cells.
“After several years of bench-to-bedside studies, beginning with Dr. Mirmira and his team’s mice models, it's exciting to finally share the promising results from our pilot trial in humans,” Dr. DiMeglio said. “Now that we've established preliminary safety of DFMO for individuals with type 1 diabetes, we're thrilled about advancing our collaborative research to explore more of its potential benefits in a larger clinical trial."
Looking ahead, Emily K. Sims, MD, pediatric endocrinologist at Riley Children’s and contributor of the research, has launched a larger, new clinical trial across six centers to further explore DFMO’s efficacy in preserving beta cell function. Her work is also funded by the JDRF and supported by Panbela Therapeutics.
“As we dive into this new multicenter clinical trial to further investigate the efficacy of DFMO, we're driven by the promising results we've seen so far to modify the underlying disease process in type 1 diabetes," Dr. Sims said. "We invite more participants to join us in this pioneering research. With their help, the knowledge we gain today has the potential to shape a brighter future for those impacted by type 1 diabetes."
About pediatric endocrinology and diabetes
In addition to this research, the pediatric endocrinology and diabetes program at Riley Children’s delivers care for patients with diabetes and other endocrine conditions. Additionally, the program includes a special clinic with early-stage type 1 diabetes, offering access to disease-modifying therapy. Notably, it is the only comprehensive care center in the Midwest for congenital adrenal hyperplasia, and the team is active in more than 30 diabetes and endocrine-related studies.
