Vascular malformations: a new hope due to molecular targeted anti-tumor strategies

Emmanuel Seront, Valérie Dekeuleneer, Julien Coulie, Ann Van Damme, Laurence Boon, Miikka Vikkula Published in the journal : May 2022 Category : Oncologie

Summary :

Vascular malformations are rare diseases that result from disturbances in the angiogenesis process. These malformations 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 or surgery, but these latter treatments were only rarely curative in nature or often not feasible. The majority of vascular malformations are caused by inherited or somatic mutations in various genes. Of note is that these mutations are similar to oncogenic mutations detected in cancer conditions, causing hyperactivity of essential signaling pathways, including the MAPK and PI3K/AKT/mTOR cascades. In this article, we have highlighted the role of targeted molecular inhibitors as possible therapies for vascular anomalies via repurposing of anticancer drugs.


- Current anti-cancer agents are promising therapeutic options in the management of vascular malformations

- Detecting mutations in these anomalies could help identify molecular targeted agents

- The improved understanding of the molecular pathways pertaining to vascular malformations could help identify new targets and targeting strategies, which could similarly represent future therapeutic options for cancer treatment.

Key Words

Vascular malformations, rapamycin, sirolimus, alpelisib, trametinib