Tissue agnostic oncology represents a new way of thinking about how cancers are treated, which is in contrast with how treatments have been developed in the past. A tissue agnostic treatment is a drug treatment that is used to treat any cancer, regardless of the site or origin but based on a specific molecular alteration that is targeted by the drug.

The checkpoint inhibitor pembrolizumab was the first drug to be FDA approved with a tumor-agnostic indication in tumors with microsatellite instability (MSI-high) or mismatch repair deficiency. More recently, the FDA also approved larotectrinib to treat all cancers with a gene alteration known as the neurotrophic receptor tyrosine kinase (NTRK) gene fusion.

Tissue-agnostic treatments are often studied in basket trials which can be very challenging due to the infrequency of some alterations. Moreover, there are many examples that targeting genetic alterations is not always as efficient throughout all tumor types. We therefore need a better understanding on the driving molecular alterations across tumor types in order to make a step forward in precision medicine for cancer treatment.