Professors Miikka Vikkula and Laurence Boon, both prominent members of the VASCERN Vascular Anomalies Working Group (VASCA WG) with the former being the Chair of this group, have been recognised with the prestigious Gagna & Van Heck International Prize for Incurable Diseases. This distinguished award, presented by the Fonds de la Recherche Scientifique (FNRS), honours their transformative work in advancing the understanding and treatment of rare vascular malformations.
Advancing Treatment for Vascular Malformations
For more than 30 years, Prof. Vikkula, a geneticist at the Institut de Duve of UCLouvain, and Prof. Boon, a clinician at the Cliniques universitaires Saint-Luc, have been working together to tackle these rare conditions, which affect around three people in every 1,000. Their research has led to the discovery of key genetic mutations behind certain hereditary vascular malformations, significantly improving the ability of clinicians to diagnose these complex conditions.
One of their most significant breakthroughs involved identifying the drug sirolimus, previously used in cancer treatments, as a highly effective therapy for vascular anomalies. Sirolimus has shown remarkable success in halting and even reversing disease progression in patients who had previously exhausted all other treatment options.
A Global First in Foetal Treatment
In a pioneering first, Prof. Boon and Prof. Vikkula successfully treated a foetus diagnosed with vascular malformations by administering sirolimus to the mother during pregnancy. This innovative approach has provided hope for families affected by these debilitating conditions.
Strengthening VASCERN’s Mission
As members of VASCERN’s VASCA Working Group, Professors Boon and Vikkula are key figures in the European Reference Network’s mission to improve care for patients with rare vascular diseases. Their work continues to strengthen Europe’s role as a leader in rare disease research and offers tangible benefits to patients suffering from vascular malformations.
For further information on the Gagna & Van Heck Prize and their groundbreaking research, visit the FNRS website.