Developing Anticancer Drugs in Orphan Molecular Entities — A Paradigm under Construction
A partir des données d'un essai de phase I incluant des adultes, d'un essai de phase I/II incluant des enfants et d'un essai de phase II incluant des adolescents et des adultes tous atteints d'un cancer présentant des fusions du gène TRK (nombre total de patients : 55 ; âgés de 4 mois à 76 ans), cette étude prospective évalue l'efficacité, du point de vue du taux de réponse globale, et la toxicité du larotrectinib, un inhibiteur sélectif de TRK
Genomic characterization of cancers has shown that some oncogenic alterations occur at very low frequency and are shared across tumor types. For example, NTRK translocations mediate malignant transformation and are observed in less than 1% of cancers and in more than 20 cancer types.1 The genes NTRK1, NTRK2, and NTRK3 encode for the tropomyosin receptor kinase (TRK) proteins TRKA, TRKB, and TRKC, respectively. In this issue of the Journal, Drilon et al.2 report a pooled analysis of three prospective clinical trials testing larotrectinib, a TRK inhibitor, in 55 patients. The overall response rate was 80%, and 71% of the responses .
New England Journal of Medicine , éditorial, 2017