This study looked at metformin—a common and widely used diabetes medication—to see if it had benefits for treating CCM. In a brain-specific CCM mouse model, metformin given through the stomach appeared to be safe and reduced the number of large lesions and the severity of hemorrhage. It also helped improve the function of cells and blood vessels, probably by restoring the functions of mitochondria, the parts of cells that generate energy.
The study suggested that one of these positive effects might be linked to metformin’s ability to lower levels of a protein called KLF4, which is known to play a key role in how CCM develops. In CCM, too much KLF4 leads to abnormal blood vessel behavior, including excessive mutant cell overgrowth and inflammation. By reducing KLF4 activity, metformin appeared to help restore more normal vessel function. However, metformin’s effect on CCM might be due to other mechanisms.
While these results are promising, it’s important to note that this research was done in mice, not humans. More studies are needed before we know whether metformin could be a safe and effective treatment for people living with CCM.
The Alliance to Cure Cavernous Malformation is gathering information on many important aspects of patient care through our International Patient Registry, including the medications people take. If you’re already on a medication like metformin or others, researchers might be interested in learning more about your experience down the road. If you’re not in the registry yet, we encourage you to join: https://ccmregistry.org/. Your participation can help move science forward and bring us closer to a cure.
Read the article abstract here.
