Diabetes Drug Metformin Shows Promise in Treating Multiple Sclerosis in Women

A groundbreaking study published in Science Translational Medicine on October 15, 2025, reveals that metformin, a common diabetes medication, may have potential in treating multiple sclerosis (MS) in women. The research, led by Dr. Rhonda Voskuhl at the University of California, Los Angeles (UCLA), demonstrates that metformin alleviates brain inflammation in female mice with MS, opening new avenues for sex-specific treatments in autoimmune diseases.

Metformin's Sex-Specific Effects on MS

The study found that metformin's effectiveness in treating MS symptoms was limited to female mice, attributed to its ability to inhibit the Kdm6a gene located on the X chromosome. This gene, which escapes X-chromosome inactivation, is more expressed in individuals with XX chromosomes, explaining the drug's sex-specific effects.

Dr. Voskuhl explained, "There are 23 or 25 drugs for MS, they all target the immune system. The Holy Grail of MS is we don't have treatments to combine with an anti-inflammatory that go to the brain." Metformin's ability to target microglia, immune cells in the brain, makes it a promising candidate for this role.

Implications for MS Treatment and Research

The findings highlight the importance of considering sex differences in medical research and treatment development. Women are approximately three times more likely than men to develop MS, a pattern observed in other autoimmune diseases such as rheumatoid arthritis and lupus.

Dr. Voskuhl emphasized, "The bottom line is, sex differences should be used to discover new treatments, and they may need to be tailored for women or tailored for men. Embrace the differences, discover things for them, and then do clinical trials that target that particular sex."

Future Directions and Clinical Trials

Currently, Dr. Voskuhl is leading a pilot trial at UCLA testing metformin in MS patients of both sexes. However, based on the recent findings, she suggests that future trials should focus specifically on women.

The research team is also exploring the potential of other compounds targeting Kdm6a, which could lead to more targeted treatments. Dr. Voskuhl is considering launching a startup in partnership with UCLA to further pursue metformin as an MS treatment, following a model similar to her previous venture, CleopatraRx.

As the pharmaceutical industry continues to explore repurposing existing drugs for new indications, this research underscores the potential for well-established medications like metformin to address unmet medical needs in complex diseases such as multiple sclerosis.