Study shows diabetes drug improves symptoms in subtype of ALS in mice

Doctors Ranum and Zu
Laura Ranum, Ph.D., and Tao Zu, Ph.D.

New preclinical research shows a commonly used Type 2 diabetes drug can improve symptoms in a specific genetic form of ALS and frontotemporal dementia in mice.

In a new Proceedings of the National Academy of Sciences study, UF 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.

“We’ve shown in mice that the drug reduces the levels of RAN proteins and that neurons in the brain and spinal cord do better,” said Laura Ranum, Ph.D., director of UF’s Center for NeuroGenetics and the Kitzman Family Professor of Molecular Genetics and Microbiology. “Mouse behavior is improved, the way the mice walk is improved, inflammation in the brain is reduced and the survival of key motor neurons, which normally die in the disease, is increased with drug treatment.”

Read the UF Health News release.

Read an article about the study in The Conversation.


research instruments
Researcher piping red liquid
Researcher piping red liquid into tray
Researcher piping liquid in the lab
researchers discussing results of a scan