Each year, the Galien France award rewards healthcare innovations in the following five areas: drugs, research, e-health, medical devices and patient support.
Pr Guillaume Canaud, from the translational medicine and targeted therapies unit at the Necker – Enfants malades hospital (AP-HP), has been awarded the Prix Galien 2022 for his work on dysharmonic overgrowth syndrome, due to a mutation in the PIK3CA gene, which causes excessive cell and tissue proliferation in the body.
This gene is responsible for major deformities and vascular swellings.
Pr Guillaume Canaud and his teams have been conducting work since 2016 1, demonstrating the major benefit of using alpelisib (BYL719, a drug used in breast cancer) for patients with CLOVES (Congenital Lipomatous Overgrowth, Vascular Malformation, Epidermal Nævi) syndrome or related disorders, who have seen significant improvements in their health status and quality of life.
More than 150 patients were treated, including two infants 2.
Further work 3 by Pr Canaud led in April 2022 to a marketing authorization from the American drug agency (FDA).
This drug is now considered the first and only treatment for adult and pediatric patients aged 2 years and older with PIK3CA mutation-related proliferation spectrum (PROS).
[1] Venot, Q., et al. Targeted therapy in patients with PIK3CA-related overgrowth syndrome. Nature 558, 540–546 (2018). https://doi.org/10.1038/s41586-018-0217-9
[2] Morin, G, et al., Treatment of two infants with PIK3CA-related overgrowth spectrum by alpelisib. J Exp Med (2022). https://doi.org/10.1084/jem.20212148
[3] Canaud G, et al. – EPIK-P1: Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib as Part of a Compassionate Use Programme / Presented at the 2021 ESMO Congress; September 17-21, 2021.