OnCo
termsTerm

Targeted therapy

aka targeted therapies, targeted drug, targeted drugs, targeted treatment, targeted treatments, targeted agent, targeted agents, molecularly targeted, precision oncology, precision medicine, personalised medicine, personalized medicine, actionable mutation, actionable mutations, actionable, matched therapy, genotype-directed, biomarker-directed

Drugs designed to hit a specific molecule the cancer depends on, usually a protein made by a mutated or amplified gene, while leaving normal cells relatively alone. The tumour is tested first to see whether it carries the target.

Targeted therapies are mostly small-molecule inhibitors (kinase inhibitors such as imatinib, osimertinib, sotorasib; PARP and CDK4/6 inhibitors) and antibodies (trastuzumab, cetuximab), and are given to patients whose tumours have the matching alteration, identified by genomic profiling or immunohistochemistry. When a tumour is truly dependent on the target (oncogene addiction) responses can be dramatic and side effects milder than chemotherapy, though the effects are rarely permanent because resistance evolves, and each new generation of drug is built to overcome the last generation's escape mutations. 'Precision oncology' is the broader programme of matching every patient to the right drug by testing; only a minority of tumours have an actionable target today.

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Clinic basics

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