New Study Shows Common Diabetes Drug Improves Symptoms in Genetic Form of ALS in Mice

University of Florida neuroscientists showed in a mouse-model study that metformin, a widely prescribed drug approved by the U.S. Food and Drug Administration to treat Type 2 diabetes, reduces levels of specific mutant proteins central to the most common genetic form of ALS and frontotemporal dementia, or FTD, two intractable neurodegenerative diseases.

The study, published in the Proceedings of the National Academy of Sciences, or PNAS, paves the way for additional research into possible future treatments, not only for the specific type of ALS and FTD linked to a mutation in the C9orf72 gene but many other neurological diseases caused by similar so-called “repeat expansion” mutations, the researchers said.

Led by UF neurogeneticists Laura Ranum, Ph.D., and Tao Zu, Ph.D., with important contributions from McGill University biochemist Nahum Sonenberg, Ph.D., the current research builds upon previous work by the Ranum lab showing repeat expansion mutations, like the mutation in the “chromosome 9 open reading frame 72,” gene is known as C9orf72, can produce unexpected proteins called “repeat-associated non-AUG proteins,” or RAN proteins.

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