E. Seront MD, PhD, V. Dekeuleneer MD, A. van Damme MD, PhD, L. Boon MD, PhD, M. Vikkula MD, PhD
Vascular malformations are rare diseases that result from anomalies in the angiogenesis process. They are subdivided into capillary, lymphatic, venous, arteriovenous, and mixed malformations, according to the type of affected vessels. Until a few years ago, therapeutic options were limited to sclerotherapy and/or surgery, but these treatments are rarely curative or not feasible. Most vascular malformations are caused by inherited or somatic mutations in various genes. These mutations are similar to oncogenic mutations detected in cancer, leading to hyperactivity of important signalling pathways, including MAPK and PI3K/AKT/mTOR cascades. This article highlights the role of targeted molecular inhibitors as therapies for vascular anomalies via repurposing of anticancer drugs.