12/16/2025
Pediatric endocrinologists and diabetes researchers at Riley Children’s Health and Indiana University School of Medicine are leading a clinical trial investigating difluoromethylornithine (DFMO) as a disease-modifying therapy for type 1 diabetes. The study, titled “TADPOL” (Targeting type 1 Diabetes via POLyamines), evaluates whether DFMO can improve beta cell health for people with Type 1 diabetes.
DFMO is currently FDA-approved as an injection treatment for African Sleeping Sickness and given by mouth to reduce risk of relapse in patients with neuroblastoma. The TADPOL trial is designed as a double-blind, placebo-controlled study enrolling participants aged 4 to 40 years within 100 days of a type 1 diabetes diagnosis, a critical window in which residual beta cell function may still be present. Participants will engage in a 12-month protocol that includes six in-person visits and one by phone, with comprehensive diabetes management and monitoring.
Emily K. Sims, MD, pediatric endocrinologist at Riley Children’s and physician-scientist at Herman B Wells Center for Pediatric Research and Center for Diabetes and Metabolic Diseases serves as the study’s lead investigator. Dr. Sims and her team are exploring DFMO as part of a long-term strategy to deploy combination regimens of therapies to preserve insulin secretion in people with Type 1 diabetes. “We believe that combination regimens of these disease-modifying therapies, like DFMO, hold the potential to usher in a new era of type 1 diabetes treatment and meaningful change for those affected by the condition,” Sims shared.
Recently, a North Carolina family traveled to Indianapolis to enroll in the study, highlighting the national reach of Riley Children’s pediatric diabetes research and clinical programs. The family, seeking innovative care options after their son’s diagnosis, chose Riley Hospital for Children as their study site for its diabetes expertise and clinical leadership.
If successful, DFMO could represent a major advance in the management of pediatric T1D and inform future prevention strategies. The TADPOL trial exemplifies Riley Children’s broader mission to integrate clinical care with discovery to ensure that children with chronic diseases benefit from the latest scientific advances.
Learn more about Riley Children’s diabetes and endocrinology achievements in the annual report.
