Revolution in Diabetes Treatment: Repurposed Drug Shows Promise

In a recent study conducted by Indiana University School of Medicine in collaboration with the University of Chicago Medicine, a groundbreaking development in the treatment of type 1 diabetes has emerged. This research indicates that repurposing the drug α-difluoromethylornithine (DFMO) holds the potential to revolutionize the management of type 1 diabetes, with the aim of reducing the reliance on insulin. The findings of this research, which span over a decade of dedicated investigation, have been published in Cell Reports Medicine, shedding light on promising prospects for innovative therapies in the future.

Type 1 diabetes is a chronic condition characterized by the autoimmune destruction of insulin-producing beta cells in the pancreas. This process leads to persistently high blood sugar levels, necessitating lifelong insulin treatment for patients’ survival. The current methods of treatment, involving daily insulin injections and frequent blood sugar monitoring, present significant challenges for individuals living with type 1 diabetes.

The journey to these groundbreaking translational results began over a decade ago. In 2010, Dr. Raghu Mirmira, co-corresponding author of the study, led a research team at IU School of Medicine and made an initial discovery that inhibiting the metabolic pathway targeted by DFMO could potentially protect beta cells from environmental factors, hinting at the possibility of preserving them in type 1 diabetes. Subsequent validation of these findings in mice further supported this notion.

From 2015 to 2019, Dr. Linda DiMeglio, Edwin Letzter Professor of Pediatrics at IU School of Medicine, conducted a clinical trial that confirmed the safety of DFMO in individuals newly diagnosed with type 1 diabetes. This trial also suggested that DFMO might stabilize insulin levels by safeguarding beta cells. The study received funding from the Juvenile Diabetes Research Foundation (JDRF), with the drug provided by Panbela Therapeutics.

Dr. DiMeglio, the senior author of the study, expressed excitement about the promising results and the opportunity to advance research through a larger clinical trial. The preliminary safety of DFMO for individuals with type 1 diabetes has been established, and the research team looks forward to exploring its potential benefits on a broader scale.

DFMO has certain regulatory advantages due to its prior FDA approval for the treatment of African Sleeping Sickness since 1990. Furthermore, it received a breakthrough therapy designation for neuroblastoma maintenance therapy after remission in 2020. This regulatory history could expedite its adoption as a treatment for type 1 diabetes, potentially shortening the approval process considerably.

Notably, a new formulation of DFMO in the form of a pill offers a more convenient alternative to regular injections, and it is associated with a favorable side effect profile. Dr. Mirmira, who is currently a professor of medicine and an endocrinologist at UChicago Medicine, emphasized the unique mechanism of action that sets DFMO apart from existing treatments for type 1 diabetes.

The research team has already embarked on the next phase of their investigation into DFMO’s potential. Dr. Emily K. Sims, the first author and co-corresponding author of the study, initiated a larger, six-center clinical trial to comprehensively assess the impact of DFMO treatment on preserving beta cell function in type 1 diabetes. This new study is also supported by JDRF and Panbela Therapeutics.

Dr. Sims, who holds positions at the Herman B Wells Center for Pediatric Research and the Center for Diabetes and Metabolic Diseases at IU School of Medicine, envisions the potential of DFMO, possibly in combination therapy, not only for those recently diagnosed with type 1 diabetes but also for individuals at risk of developing the condition. She encourages more participants to join this pioneering research, emphasizing that the knowledge gained today has the potential to shape a brighter future for those affected by type 1 diabetes.

For further information on the new clinical trial, interested individuals can visit the study’s website.

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More about DFMO Diabetes Treatment

• Cell Reports Medicine Article
• [Clinical Trial Information](Provide the specific link to the clinical trial information if available)