Already-Available Drug Could Help Treat Type 1 Diabetes by Miriam Tucker for Medscape.com, 20 November 2023.

The drug α-difluoromethylornithine (DFMO) may help preserve beta-cell function in people with new-onset type 1 diabetes, new preliminary data suggest.  “I think we have lots of potential to improve people’s quality of life who are living with type 1 diabetes if we can increase their endogenous insulin secretion. I think long-term combination therapy is going to be the answer,” said study author Emily K. Sims, MD, a pediatric endocrinologist at Indiana University School of Medicine, Indianapolis. The new safety study by Sims and colleagues was published November 1 in Cell Medicine Reports.

DFMO inhibits the polyamine biosynthesis pathway, which plays a role in the inflammatory responses in autoimmune diseases, including type 1 diabetes. It’s sold under the name eflornithine as an intravenous treatment for African sleeping sickness (trypanosomiasis) and as a cream for unwanted hair growth in women. It also has orphan designations for treating various cancers, including neuroblastoma.

In type 1 diabetes, the immune system destroys insulin-producing pancreatic beta cells. Insulin treatment is required. Recently, the monoclonal antibody teplizumab (Tzield, Sanofi) was approved as a treatment for delaying the onset of type 1 diabetes in people with autoantibodies that signify a preclinical stage of the condition. As yet, no agent has been approved for preserving beta-cell function after the onset of type 1 diabetes, but many are under investigation.

Read more:  Already-Available Drug Could Help Treat Type 1 Diabetes


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