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Lundi 11 octobre 2021
New therapeutic perspectives for patients with lymphatic anomalies linked to a PIK3CA gene mutation

A study conducted by Prof. Guillaume Canaud, physician at the Necker Enfants Malades Hospital and Laboratory Director at the Necker Enfants Malades Institute has just been published in the journal Science Translational Medicine

This team created a mouse model that develops lymphatic anomalies linked to the PIK3CA gene mutation similar to those of patients. The team then demonstrated the efficacy of treatment with alpelisib (a molecule approved for use in breast cancer) in this animal model and then in 6 patients.

This is the culmination of 3 years' work which opens up important therapeutic prospects for patients. The result is a better quality of life and a significant aesthetic improvement.

Link to the article: Alpelisib administration reduced lymphatic malformations in a mouse model and in patients (

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S.A.R. la Princesse Caroline de Hanovre qui, à travers la Fondation Princesse Grace, soutient déjà la recherche medicale et tout ce qui contribue à soulager les enfants malades en France et dans le monde, a accepté de s'engager à nos cotés pour que ce centre de medecine moleculaire, tourné entre autres vers les pathologies des enfants, prenne de vitesse les maladies et continue à relever les defis actuels.

INEM - Organigramme