Data from a recent study suggests that treatment using the drug fasudil could prevent lesions from cerebral cavernous malformation, a brain abnormality that can facilitate bleeding, epilepsy and stroke. 

The study reports that the drug fasudil prevented the formation of lesions when tested in a genetic mouse model. Issam Awad, MD, professor of surgery at the University of Chicago Medical Center, Ill, served as senior author of the study and calls the results of the mouse model “very exciting,” thanks to its evidence of a drug effect on cavernous angiomas in living animals. “The results were dramatic,” Awad says, “the prevalence of legions went down significantly, and the legions that developed in the mice were simpler and smaller and did not show any inflammation or bleeding.”

Cerebral cavernous malformation (CCM), also known as cavernous angioma, is reportedly estimated to occur in one out of 200 individuals. The CCM lesion is detected by MRI, but cannot be treated unless it is large enough to require brain surgery, Awad says. 

According to Awad, researchers in Chicago and at Duke University, Durham, NC, created CCM lesions in living mice by reducing the gene CCM1, also known as KRIT1. In a parallel collaborative effort by Mark H. Gingsberg, MD, University of California, San Diego, Calif, researchers reported that knocking down the CCM1 gene resulted in elevated activity of a signal called ROCK, which makes blood vessels “leaky.” Researchers hypothesized that the use of drug inhibitor of ROCK might prevent CCM lesions. Fasudil is reported to be the only specific ROCK inhibitor available for animal studies and human use.

After a 4-month duration, researchers reported that the genetically modified mice displayed fewer and less severe lesions and fewer signs of hemorrhage than similar mice given the placebo treatment. 

Awad also acknowledges that while the treatment does not cure the disease, if translated to human therapy it would be, “A bit like treating multiple sclerosis (MS), where many treatments do not eliminate the primary disease trigger, but can muffle it, slow it down and make it not as serious, and therefore allow a patient to effectively live with the disease, as opposed to having the disease dictate their health.”

The study was recently published in the journal Stroke.

Source: University of Chicago Medical Center