An active drug for TRK-positive paediatric solid tumours
Mené sur 24 patients pédiatriques (âge médian : 4,5 ans) atteints d'une tumeur solide de stade localement avancé ou métastatique et présentant des fusions des gènes NTRK, cet essai de phase I/II évalue la dose maximale tolérée et l'activité antitumorale du larotrectinib, un inhibiteur sélectif de TRK
Despite the promise of personalised medicine and molecular targeted agents, these approaches have translated into few successful new treatments for childhood cancers. Some drugs that have shown activity in this setting include BRAF inhibitors for V600-mutant gliomas or Langerhans cell histiocytosis, and ALK inhibitors for cancers harbouring ALK-translocations.1,2 In The Lancet Oncology, Theodore W Laetsch and colleagues report the phase 1 results of a first-in-child, phase 1/2 trial of the TRK inhibitor larotrectinib, showing encouraging activity in tumours with NTRK fusions
The Lancet Oncology , commentaire, 2017