We are delighted to share a major research study funded in part by The Butterfly AVM Charity and published in the Journal of Investigative Dermatology (2025). This study investigates potential new treatments for arteriovenous malformations (AVMs), particularly those caused by mutations in the KRAS gene — a known driver of many sporadic AVM cases.
The researchers used a cutting-edge approach, combining computer-based (in silico) screening with laboratory testing in specially engineered endothelial cells that mimic AVM biology. Through this dual screening method, they identified several existing, FDA-approved drugs — including some statins and targeted cancer therapies — that showed selective effectiveness against AVM cells carrying KRAS mutations.
Importantly, the findings highlight that treatments based on cancer data alone may not be sufficient for AVMs, and that dedicated AVM-specific drug screening is essential. This pioneering work lays the foundation for future clinical testing and brings us closer to safer, targeted therapies for AVM patients.
Click here to read the full paper online
Click here to download the PDF