A recent research study led by the Indiana University School of Medicine, in collaboration with the University of Chicago Medicine, unveils exciting possibilities for managing type 1 diabetes and potentially reducing dependence on insulin. The study, published in Cell Reports Medicine, suggests that repurposing the drug α-difluoromethylornithine (DFMO) could pave the way for innovative therapies in the future.
Type 1 diabetes is a chronic condition where the immune system mistakenly attacks and destroys insulin-producing beta cells in the pancreas. This leads to high blood sugar levels, requiring lifelong insulin treatment for patient survival. Many individuals with type 1 diabetes find current treatments, such as daily insulin injections and frequent blood sugar monitoring, inconvenient and challenging.
The study's co-corresponding author, Dr. Raghu Mirmira, MD, PhD, and his team discovered in 2010 that inhibiting the metabolic pathway affected by DFMO could protect beta cells, suggesting potential preservation in type 1 diabetes. Subsequent validation in mice led to a clinical trial from 2015 to 2019, directed by Dr. Linda DiMeglio, MD, MPH, confirming DFMO's safety in newly diagnosed type 1 diabetes patients and suggesting its potential to stabilize insulin levels.
The promising results from the pilot trial in humans are now paving the way for larger clinical trials. Dr. DiMeglio, the senior author of the study, expresses excitement about advancing collaborative research to explore more potential benefits of DFMO in a broader clinical setting.
DFMO, FDA-approved since 1990 for high-dose injections to treat African Sleeping Sickness, and designated as a breakthrough therapy in 2020 for neuroblastoma maintenance therapy, may streamline its adoption as a type 1 diabetes treatment. Its prior regulatory clearance could significantly reduce the approval process timeline.
Dr. Mirmira, now a professor of medicine and endocrinologist at UChicago Medicine, emphasizes the new formulation of DFMO as a pill, making it more convenient for patients compared to regular injections. The researchers are already taking steps to investigate DFMO's potential further, launching a larger, six-center clinical study to define its impact on preserving beta cell function in type 1 diabetes. This study, funded by JDRF and supported by Panbela Therapeutics, aims to explore DFMO's efficacy and potential use in those at risk of developing type 1 diabetes. The researchers invite participants to join this groundbreaking research, emphasizing that the knowledge gained today has the potential to shape a brighter future for individuals impacted by type 1 diabetes.
