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What is targeted therapy?

2026-07-04 23:30:31

Targeted therapy overview

Targeted therapy is a precision medical treatment for diseases such as cancer, which inhibits tumor growth or spread by identifying and attacking specific molecular targets (such as mutated genes or abnormal proteins). Unlike traditional chemotherapy, its advantage lies in reducing damage to normal cells, improving efficacy and reducing side effects. The main contents include:Mechanism of action(such as targeting EGFR, HER2 and other targets),Applicable people(requires genetic testing to match),Common medicines(such as trastuzumab, imatinib) andlimitations(drug resistance, high cost). The following will be developed in layers.

Core principles of targeted therapy

What is targeted therapy?

The core of targeted therapy is "precision strike". Cancer cells often carry unique molecular markers (such as specific gene mutations or protein overexpression), and drugs cansmall molecule inhibitors(eg gefitinib blocks EGFR) ormonoclonal antibodies(eg, bevacizumab inhibits angiogenesis) plays a role. For example, trastuzumab significantly prolonged survival in patients with HER2-positive breast cancer. This treatment relies on genetic testing or immunohistochemical analysis to ensure that the drug matches the target.

Applicable diseases and patient screening

Targeted therapy is not suitable for all cancers and is currently mainly used fornon-small cell lung cancer(ALK/ROS1 mutation),breast cancer(HER2 positive),chronic myelogenous leukemia(BCR-ABL fusion gene) etc. Patients need to passbiopsy or liquid biopsyConfirm the target exists. For example, about 15%-20% of lung cancer patients have EGFR mutations, and osimertinib can be used. However, some tumors may not benefit due to target loss or heterogeneity, and must be combined with radiotherapy and chemotherapy.

Drug types and representative products

Targeted drugs are divided intoOral small molecule drugsandInjectable macromolecular antibodiesTwo categories. The following table lists some representative drugs and manufacturers:

Drug nametargetIndicationsManufacturer
Imatinib (Gleevec)BCR-ABLchronic myelogenous leukemiaNovartis
Trastuzumab (Herceptin)HER2breast cancerRoche
Osimertinib (Tagressa)EGFR T790Mnon-small cell lung cancerAstraZeneca

Strengths, limitations and future prospects

Although targeted therapy can improve the quality of life, it still facesdrug resistance(For example, patients with EGFR mutations develop T790M mutation after taking first-generation drugs),high cost(The annual treatment fee can reach hundreds of thousands of yuan) and other issues. Future research directions include combination immunotherapy and the development of new generation inhibitors (such as fourth-generation EGFR drugs targeting drug-resistant mutations). Patients need to weigh the pros and cons and make a personalized choice under the guidance of a doctor.

Citing sources

1. American Cancer Society’s popular science guide on targeted therapy.
2. Chinese Society of Clinical Oncology (CSCO) "Guidelines for the Diagnosis and Treatment of Primary Lung Cancer" (2023 Edition).
3. Nobel Prize winner Brian Druker’s discussion of the development of imatinib (Nature).
4. Manufacturer and product information: Data disclosed on the official websites of Novartis (Gleevec), Roche (Herceptin), and AstraZeneca (Taressa).

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