Repurposed drug shows promise for type 1 diabetes

Polyamine biosynthesis inhibition protects β cell function in type 1 diabetes.


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A recent study led by Indiana University School of Medicine and the University of Chicago Medicine offers promising possibilities for improving how we manage type 1 diabetes and reducing the need for insulin. The research, published in Cell Reports Medicine, explores the potential of repurposing the drug α-difluoromethylornithine (DFMO) to develop new therapies.

Type 1 diabetes is a chronic condition where the body’s immune system mistakenly attacks and destroys insulin-producing cells in the pancreas. This leads to high blood sugar levels, and patients currently need lifelong insulin treatment to survive. The study aims to make diabetes management more accessible for people with type 1 diabetes, who often struggle with daily insulin injections and frequent blood sugar monitoring.

These recent findings are the result of over a decade of research. In 2010, Dr. Raghu Mirmira and his team at IU School of Medicine discovered that blocking a specific metabolic pathway targeted by DFMO could protect beta cells from external factors, potentially preserving them in type 1 diabetes. They confirmed these findings in mice.

From 2015 to 2019, Dr. Linda DiMeglio, a pediatric endocrinologist and professor at IU School of Medicine, conducted a clinical trial to test DFMO’s safety in people newly diagnosed with type 1 diabetes. The problem, supported by the Juvenile Diabetes Research Foundation (JDRF) and with a drug supply from Panbela Therapeutics, suggested that DFMO might stabilize insulin levels by protecting beta cells.

DiMeglio, senior author of the study said, “After several years of bench-to-bedside studies, beginning with Drs. Mirmira and [Sarah] Tersey’s mouse models, it’s exciting to finally share the promising results from our pilot trial in humans. Now that we’ve established the 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.”

DFMO, approved by the FDA in 1990 for African Sleeping Sickness and granted breakthrough therapy designation in 2020 for neuroblastoma maintenance therapy, holds prior regulatory clearance. This history could speed up the approval process for type 1 diabetes treatment from decades to a few years.

With a new pill form, patients can take DFMO orally instead of getting injections, and it has minimal side effects. Dr. Mirmira, a professor at UChicago Medicine, is excited about having a treatment that works differently from other options.

The research team is already progressing to the next stage. Dr. Emily K. Sims, who initiated the study, leads a larger clinical trial across six centers to better understand DFMO’s impact on preserving beta cell function in type 1 diabetes. JDRF and Panbela Therapeutics also support this new study.

Dr. Emily K. Sims, a physician-scientist at IU School of Medicine, is optimistic about the potential of using DFMO, possibly in combination with other therapies, not only for people recently diagnosed with type 1 diabetes but also for those at risk of developing the condition.

She’s leading a new clinical trial to investigate DFMO’s effectiveness further. Driven by promising early results, they aim to modify the underlying disease process in type 1 diabetes. They’re inviting more participants to join this pioneering research with the hope that the knowledge gained today will lead to a brighter future for those affected by type 1 diabetes.

The repurposing of DFMO for type 1 diabetes management represents a significant step forward in the field. It promises improved treatment options, reduced dependency on insulin, and enhanced quality of life for individuals with this chronic condition. As ongoing research unfolds, the potential for a transformation in diabetes management becomes increasingly apparent, offering new hope to patients and healthcare providers alike.

Journal reference:

  1. Emily K. Sims, Abhishek Kulkarni, et al., Inhibition of polyamine biosynthesis preserves β cell function in type 1 diabetes. Cell Reports Medicine. DOI: 10.1016/j.xcrm.2023.101261.


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