Entrectinib is a potent inhibitor of Trk-driven neuroblastomas in a xenograft mouse model

Highlights • Autocrine overexpression of TrkB and its ligand is found in over half of high-risk neuroblastomas. • Entrectinib (RXDX-101) is a potent, nontoxic inhibitor of TrkB-expressing neuroblastoma growth in vivo in mouse xenografts. • Entrectinib significantly enhances the efficacy of chemotherapy when used in combination, without additional toxicity. • Entrectinib is a very promising agent to treat TrkB-expressing neuroblastomas and other tumors with Trk receptor activation • A phase 1 clinical trial for recurrent/refractory pediatric solid tumors is planned.