Authors: Jade Parker, Editor
A first-of-its-kind drug, termed larotrectinib, which targets TRK fusions has been demonstrated to be effective in 93% of pediatric patients tested. The drug is the first to be granted FDA breakthrough therapy designation for patients with a specific fusion of two genes in the cancer cell, no matter what cancer type.
TRK fusions occur in many types of cancer and whilst only small percentages occur in common adult cancers, this fusion occurs frequently in some rare pediatric cancers – such as infantile fibrosarcoma, cellular congenital mesoblastic nephroma and papillary thyroid cancer.
Findings from the trial were recently published in The Lancet Oncology.