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 efficacy in treating MS is linked to its ability to inhibit the Kdm6a gene, which is located on the X chromosome. This gene escapes X-chromosome inactivation, leading to higher expression in individuals with XX chromosomes. Dr. Voskuhl's team discovered that women with MS have higher Kdm6a activity than men, supporting the gene's role in the disease's sex bias.
"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," Dr. Voskuhl stated. "Embrace the differences, discover things for them, and then do clinical trials that target that particular sex."
Targeting Brain Inflammation in MS
Multiple sclerosis is an autoimmune disease where the body attacks its own nerves in the brain and spinal cord, causing a range of symptoms including mobility issues, cognitive problems, and speech difficulties. Women are approximately three times more likely than men to develop MS, a pattern observed in other autoimmune diseases as well.
Dr. Voskuhl emphasized the importance of targeting brain inflammation in MS treatment: "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 combination approach.
Future Directions and Clinical Trials
While the current study focuses on mouse models, Dr. Voskuhl is already leading a pilot trial at UCLA testing metformin in MS patients. However, based on the new findings regarding sex-specific effects, she suggests that future trials should focus primarily on women.
Dr. Voskuhl is also considering launching a startup in partnership with UCLA to further develop metformin for MS treatment. This approach would be modeled after CleopatraRx, a company she founded to develop estrogen-based treatments for cognitive symptoms associated with menopause.
As research continues, the pharmaceutical industry eagerly awaits further developments in sex-specific treatments for autoimmune diseases, with metformin potentially leading the way in multiple sclerosis therapy for women.
References
- Common diabetes drug alleviates brain inflammation in female mice with multiple sclerosis
A well-established diabetes drug alleviated brain inflammation in a female mouse model of multiple sclerosis, suggesting the long-used medicine could be repurposed to combat MS in people with female sex chromosomes, who are disproportionately affected by the autoimmune disease.
- Common diabetes drug alleviates brain inflammation in female mice with multiple sclerosis
A well-established diabetes drug alleviated brain inflammation in a female mouse model of multiple sclerosis, suggesting that the long-used medicine could be repurposed to combat MS in people with female sex chromosomes, who are disproportionally affected by the autoimmune disease.
Explore Further
What is the timeline and design of the current pilot clinical trials testing metformin in MS patients at UCLA?
How does metformin's mechanism of action in inhibiting the Kdm6a gene compare to other existing MS treatments targeting brain inflammation?
What is the projected market size for sex-specific MS treatments, particularly for women affected by the disease?
What are the safety and potential side effects of using metformin to treat brain inflammation in MS patients, based on preclinical and early clinical data?
Are there other pharmaceutical companies currently exploring sex-specific treatments for autoimmune diseases, and how do these compare to metformin’s development pathway?