Un nouveau médicament révolutionne le traitement d’une neuropathie


Les personnes soufrant d’amylose héréditaire à transthyrétine (TTR), une maladie génétique rare mais mortelle, bénéficient désormais d’un médicament à base d’ARN interférant, qui réduit les atteintes neurologiques.

L’amylose héréditaire à transthyrétine (TTR) est une maladie génétique rare mais mortelle : lorsque le gène codant pour la TTR – une protéine fabriquée dans le foie – est muté, celle-ci se disloque et s’accumule dans les nerfs, entraînant perte de mobilité et de sensibilité, douleurs, troubles digestifs et cardiaques et enfin la mort, sans que les traitements existants ne puissent rien y faire.

Mais voilà qu’un nouveau médicament, baptisé Patisiran (ou Onpattro, son nom commercial), bouleverse la donne : « Chez environ la moitié des patients, il réduit les atteintes neurologiques et améliore même la qualité de vie. Pour l’autre moitié, il permet au moins de ralentir la progression de la maladie », s’enthousiasme David Adams, neurologue au CHU Bicêtre (APHP) et instigateur principal d’un essai clinique dont les résultats ont été publiés en juillet.

Autorisé aux Etats-Unis et en Europe

Son principe est inédit : il s’attaque à l’ARN messager, la molécule intermédiaire entre l’ADN et la protéine. Une portion d’ARN (les scientifiques parlent d’ARN interférant) est encapsulée dans des nanoparticules qui ciblent le foie, pour y être libérée et s’accrocher à l’ARN messager : la fabrication de la TTR est alors stoppée. Le Patisiran a déjà reçu les autorisations de mise sur le marché aux États-Unis et en Europe.


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