Existing cancer drug reduces blood vessel malformations in the brain
Researchers have identified a substance that reduces blood vessel lesions in the brain in the disease cerebral cavernous malformation (CCM). The substance is already used to treat brain tumors in children and could therefore be repurposed relatively rapidly to treat CCM, hopefully replacing surgery, currently the only treatment option.
Researchers have discovered a drug already used to treat brain tumors in children that can reduce blood vessel malformations in the brain caused by the disease cerebral cavernous malformation (CCM). CCM results in fragile, malformed blood vessels that can lead to hemorrhages and strokes. Currently, the only treatment is invasive surgery to remove the lesions.
In this study published in Science Translational Medicine, researchers identified dabrafenib, a BRAF inhibitor, which successfully minimized both the number and size of vascular malformations in CCM mouse models. Dabrafenib works by targeting two key pathways contributing to CCM development and progression. In addition to reducing lesion size and number, dabrafenib stabilized vessels and decreased leakage in the mouse model, suggesting potential benefits for patients with retinal vasculature abnormalities associated with CCM.
Dabrafenib's already approved status by the FDA and EMA, coupled with an established safety profile, positions it as a promising candidate for rapid clinical translation to treat CCM.
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