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Preventing by repurposing: from sleep sickness to diabetness, DMFO gets into the “sweet field”

Type 1 diabetes is an autoimmune disease that causes the body’s immune system to attack and destroy insulin-producing beta cells in the pancreas. Traditional management of type 1 diabetes has primarily involved replacing the missing insulin with injections which, though effective, can be expensive and burdensome. But no known molecule is able to prevent beta cell death and autoimmune diabetes at a clinical level. A new study led by researchers at the University of Chicago Medicine and Indiana University suggests that an existing drug could be repurposed to treat type 1 diabetes, potentially reducing dependence on insulin as the sole treatment.

The research focused on a known drug called alpha-difluoromethylornithine (DFMO), which inhibits an enzyme that plays a key role in polyamine metabolism. The latest translational results are a culmination of years of research: in 2010, while corresponding author Raghu Mirmira, MD, PhD, was at Indiana University, he and his lab performed fundamental biochemistry experiments on beta cells in culture. They found that suppressing the metabolic pathway altered by DFMO helped protect the beta cells from environmental factors, hinting at the possibility of preserving and even restoring these vital cells in patients diagnosed with type 1 diabetes.

The researchers confirmed their observations preclinically in zebrafish and then in mice before senior author Linda DiMeglio, MD, MPH, Edwin Letzter Professor of Pediatrics at Indiana University School of Medicine and a pediatric endocrinologist at Riley Children’s Health, launched a clinical trial to evaluate the safety and tolerability of the drug in type 1 diabetes patients. The results of the trial, which was funded by the Juvenile Diabetes Research Foundation (JDRF) and used DMFO provided by Panbela Therapeutics, indicated that the drug is safe for type 1 diabetes patients and can help keep insulin levels stable by protecting beta cells.

The research, “Inhibition of Polyamine Biosynthesis Preserves β-Cell Function in Type 1 Diabetes,” was published in Cell Medicine Reports this November. Importantly, DFMO has already been FDA-approved as a high dose injection since 1990 for treating African Sleeping Sickness and received breakthrough therapy designation for neuroblastoma maintenance therapy after remission in 2020. Pre-existing regulatory approval could potentially facilitate its use in type 1 diabetes, saving effort and expense and getting the treatment to patients sooner.

  • Edited by Dr. Gianfrancesco Cormaci, PhD, specialist in Clinical Biochiemistry.

Scientific references

Sims EK et al. Cell Reports Med 2023 Nov; in press.

Felton JL et al. Commun Med (Lond). 2023; 3(1):130.

Forlenza GP et al. JAMA. 2023 Mar; 329(12):990-99.

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Dott. Gianfrancesco Cormaci
Dott. Gianfrancesco Cormaci
Laurea in Medicina e Chirurgia nel 1998, specialista in Biochimica Clinica dal 2002, ha conseguito dottorato in Neurobiologia nel 2006. Ex-ricercatore, ha trascorso 5 anni negli USA alle dipendenze dell' NIH/NIDA e poi della Johns Hopkins University. Guardia medica presso la casa di Cura Sant'Agata a Catania. In libera professione, si occupa di Medicina Preventiva personalizzata e intolleranze alimentari. Detentore di un brevetto per la fabbricazione di sfarinati gluten-free a partire da regolare farina di grano. Responsabile della sezione R&D della CoFood s.r.l. per la ricerca e sviluppo di nuovi prodotti alimentari, inclusi quelli a fini medici speciali.

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