Les chélateurs spécifiques du cuivre : une voie pour la thérapie de la maladie de Wilson

(Text in French) Copper specific chelators: a drug candidate for the therapy of Wilson’s disease. Among orphan diseases, some seem more orphan than others... This is the case of Wilson’s disease. While other genetic diseases have attracted the attention of academic or industrial researchers, it must be recognized that this “copper disease”, which requires lifelong treatment of patients, is currently only treated by two old molecules, of moderate efficacy, and having significant adverse effects. It is in this context that a new specific copper chelator, TDMQ20, proved very effective during its evaluation on TX mice, a classic model of this disease which is due to the deficiency of the ATP7B protein, responsible for the elimination of excess copper in the liver. Its pharmacological properties are directly linked to the particular structure of the copper-TDMQ20 complex and make it an effective competitor of the molecules currently in clinical use.