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Site-directed therapy based on molecular tissue of origin or molecular targeted therapy
Source: Cancer of Unknown Primary (CUP) Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: May 7, 2025 · Captured 2026-09-09.
Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary.
Clinical, pathological, and molecular genetic tests have two goals. The first is to identify tumors that are responsive to available therapies. The second is to identify novel targeted therapies that might be applicable to the particular genetic profile. The U.S. Food and Drug Administration (FDA) has approved some novel targeted therapies when a molecular target is identified in a cancer, regardless of primary site or when no primary site is evident. Examples include tumors with the following actionable targets:[1-8]
High tumor mutational burden (TMB-H).
Elevated PD-1 or PD-L1 expression.
HER2 expression.
Pathogenic variants in NTRK, RET, or EGFR.
BRAF V600E pathogenic variant.
ROS1 gene fusion.
MET amplification.
Homologous recombination repair deficiency/microsatellite instability (MSI).
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Preserved source evidence · Not medical advice