mechanismtargeted

Mechanismtargeted, often written mechanism-targeted therapy, is a term used to describe therapeutic approaches designed to intervene in a specific biological mechanism implicated in a disease, with the aim of altering disease progression rather than merely alleviating symptoms. It is a facet of precision medicine and is closely related to targeted therapy, though the emphasis is on the underlying mechanism rather than the disease symptom.

Approaches include small-molecule inhibitors, monoclonal antibodies, biologics, and nucleic acid–based therapies that block or modulate a defined target such as a receptor, enzyme, signaling node, or immune pathway. Drug discovery typically follows identification of a disease mechanism, target validation, development of agents that modulate the target, and evaluation in preclinical and clinical studies. Biomarkers and companion diagnostics are frequently used to select patients most likely to respond.

Examples include tyrosine kinase inhibitors that block BCR-ABL in chronic myeloid leukemia (imatinib), EGFR inhibitors in certain cancers (gefitinib), and HER2-targeting antibodies (trastuzumab); and anti-inflammatory biologics that neutralize cytokines or block their receptors in autoimmune diseases. In infectious diseases, agents that inhibit essential viral or bacterial enzymes are mechanism-targeted.

Limitations include the emergence of resistance through target mutation or pathway redundancy, adverse effects from interference with normal physiology, tumor microenvironment factors, and cost. Ongoing research seeks combination strategies, sequential targeting, and discovery of novel mechanisms to extend durability of responses.