Targeted Therapy

Targeted Therapy encompasses therapeutic approaches designed to selectively interfere with specific molecular targets, pathways, or cellular mechanisms underlying disease while sparing normal tissues, contrasting with conventional treatments affecting all rapidly dividing cells. These precision medicines include small molecule inhibitors, monoclonal antibodies, antibody-drug conjugates, and cellular therapies engineered to recognise disease-specific markers. Targeted therapy development requires understanding disease molecular drivers, identifying suitable targets, and often employing companion diagnostics selecting appropriate patients.

The pharmaceutical industry has revolutionised oncology through targeted therapies addressing driver mutations, oncogenic pathways, or tumour microenvironment components. Small molecule kinase inhibitors targeting BCR-ABL in chronic myeloid leukaemia, EGFR mutations in lung cancer, or BRAF mutations in melanoma have achieved remarkable responses in selected patients. Resistance mechanisms including secondary mutations, pathway bypass, or phenotypic changes limit response duration, driving combination strategies or next-generation inhibitors. Companion diagnostics identify patients whose tumours harbour targetable alterations ensuring therapy reaches responsive populations. Biomarker development beyond initial targets identifies additional responsive populations or resistance predictors. Regulatory pathways increasingly require demonstration of target presence or pathway activation for approval. As genomic profiling becomes routine and understanding grows regarding actionable targets, targeted therapy continues transforming medicine through precision approaches matched to disease molecular characteristics.