Tumor-agnostic therapy. Current status and future outlook.

Tumor-agnostic therapy, also known as site-agnostic, genetically defined and biomarker-defined therapy, treats cancer based on its genetic and molecular features without regard to where the cancer started in the body. It has the potential to revolutionalize how we think about treating cancer.

The first tumor-agnostic therapy was approved in May 2017 for cancers with a microsatellite-instability-high genotype. Since then two agents have been approved for cancers expressing NTRK fusion.

The tumor-agnostic approach has implications for clinical practice, trial design, testing and reimbursement. To get a sense of what might be next for tumor-agnostic therapy, we conducted a systematic search of ongoing clinical trials and present the results here.

It’s difficult to predict what the next approval may be, but the sheer volume of agents being looked at (including some quite old targeted therapies that could get a new usage) is exciting and can only lead to better options for patients.

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