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Monday 11 of October, 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 (science.org)

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HRH Princess Caroline of Hanover, who through the Princess Grace Foundation, already supports medical research and anything that helps to relieve the sick children in France and around the world, has agreed to commit to our side so that our Center of Molecular medicine continues to meet the current challenges and fight diseases, and in particular the ones affecting children.

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