Targeted therapy

Targeted therapy uses drugs designed to interfere with specific molecules or pathways that contribute to disease, rather than acting broadly on cells or tissues.

Molecular target: A molecule whose activity, abundance, or interaction is deliberately altered by a therapeutic intervention. Targeted drugs are selected to act on a disease-linked molecule.
Precision oncology: Cancer care that uses tumor-specific molecular information to guide treatment decisions. It is the most prominent clinical setting for targeted drugs.
Chemotherapy: Treatment with drugs that kill or inhibit rapidly dividing cells, especially in cancer. Traditional chemotherapy often affects healthy dividing cells more broadly than targeted drugs.
Companion diagnostic: A medical test that provides information essential for the safe and effective use of a particular therapy. These tests identify patients whose disease has the drug’s target or biomarker.
Small-molecule inhibitor: A low-molecular-weight compound that binds a protein and reduces or changes its activity. Many targeted drugs enter cells to inhibit intracellular enzymes.
Imatinib: A tyrosine kinase inhibitor used to treat cancers including chronic myeloid leukemia and gastrointestinal stromal tumors. Its success against BCR::ABL1 helped establish targeted cancer therapy.
Immunotherapy: Treatment that changes immune activity to prevent or fight disease. Some targeted drugs act directly on disease molecules, while immunotherapy mobilizes immune responses.
Predictive biomarker: A measurable feature that indicates the likelihood of response to a particular treatment. It helps distinguish target presence from a target that actually predicts benefit.
Monoclonal antibody: A laboratory-produced antibody designed to bind a particular antigen. Antibodies can block extracellular targets or mark cells for immune attack.
HER2/neu: A cell-surface receptor encoded by ERBB2 that can drive growth when amplified or overexpressed in some cancers. HER2 status identifies tumors that may respond to drugs such as trastuzumab.

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