Tumor-agnostic therapy in oncology

Tumor-agnostic therapy is considered as a promising therapeutic approach in oncology, however classification, validation of the targets and standardized methodology for their evaluation is mandatory. The development and approval of the tumor-agnostic drugs should be based on biomarker driven clinical trials. To date three validated biomarkers are known, the high microsatellite instability (MSI-H), the fusion of the neurotrophic-receptor-tyrosine-kinase (NTKR) genes, and the high mutation burden (TMB-H) of the tumors. Pembrolizumab (anti-PD-1 antibody) was the first approved tumor-agnostic drug, the MSI-H status of the tumor was the first indication, then later the TMB-H status was also approved. Larotrectinib and entrectinib are approved for the treatment of NTKR fusion-positive tumours.