TARGETED THERAPY
IN CANCER TREATMENT
Targeted therapy is a precision cancer treatment that attacks specific genes, proteins, or pathways driving tumor growth, improving outcomes while reducing damage to healthy cells.
analyticsAt a Glance
- check_circleBlocks specific molecular targets driving cancer growth and survival
- check_circleRequires biomarker or mutation testing (NGS, IHC) to confirm eligibility
- check_circleApproved drugs across EGFR, HER2, ALK, BRAF, KRAS, and other pathways
- check_circleOral or intravenous โ typically taken on a continuous or cyclic schedule
What Is Targeted Therapy โ and Why Does It Matter?
Targeted therapy is a category of cancer drugs designed to interfere with specific molecular targets โ proteins, enzymes, or receptors โ essential for cancer cell growth, survival, and spread. Unlike chemotherapy, which kills all rapidly dividing cells, targeted therapy is matched to a specific molecular defect identified by biomarker testing.
โImatinib achieved complete cytogenetic responses in 90%+ of CML โ from near-incurable to manageable chronic disease with a pill.โ
The Imatinib Revolution
Imatinib (Gleevec), approved in 2001 for BCR-ABL+ CML, demonstrated that targeting a single oncogenic driver could transform cancer management. CML went from requiring bone marrow transplantation to being controlled long-term with daily oral therapy.
Oncogene Addiction
Cancer cells that depend on a single activated pathway (EGFR, BRAF, ALK) are "addicted" to it. Blocking that pathway is highly toxic to the cancer cell while sparing normal cells โ the biological rationale for targeted therapy's selectivity and efficacy.
Targeted vs Chemotherapy
Chemotherapy: kills all rapidly dividing cells โ nausea, hair loss, myelosuppression. Targeted therapy: attacks specific cancer cell abnormalities โ different, often more manageable side effect profile. Efficacy depends entirely on presence of the target.
Why Biomarker Testing Is Essential
Without comprehensive molecular profiling (NGS), the most effective targeted therapy for your specific tumour cannot be identified. NGS tests hundreds of mutations, fusions, and amplifications in a single assay โ now the standard of care before initiating systemic therapy.
Major Classes of Targeted Therapy Agents
Targeted agents span multiple drug classes, each with distinct mechanisms, side effect profiles, and cancer type applications.
| Drug Class | Mechanism | Key Examples & Cancers |
|---|---|---|
| Tyrosine Kinase Inhibitors (TKIs) | Block intracellular kinase domains driving cancer proliferation | Imatinib (CML/GIST); osimertinib (EGFR+ NSCLC); alectinib (ALK+ NSCLC); vemurafenib (BRAF V600E melanoma) |
| Monoclonal Antibodies (mAbs) | Bind extracellular targets, blocking receptor activation or marking for immune destruction | Trastuzumab (HER2+ breast/gastric); cetuximab (EGFR+ CRC/H&N); bevacizumab (VEGF, multiple) |
| Antibody-Drug Conjugates (ADCs) | mAb delivers cytotoxic payload directly to antigen-expressing cancer cells | T-DM1, T-DXd (HER2+ breast); enfortumab vedotin (urothelial); sacituzumab govitecan (TNBC) |
| PARP Inhibitors | Exploit DNA repair deficiency (HRD/BRCA) via synthetic lethality | Olaparib, niraparib, rucaparib โ BRCA-mutated breast/ovarian/prostate/pancreatic cancer |
| CDK4/6 Inhibitors | Block cell cycle progression at G1/S checkpoint | Palbociclib, ribociclib, abemaciclib โ HR+/HER2- advanced breast cancer |
| VEGF/Angiogenesis Inhibitors | Block tumour blood vessel formation | Bevacizumab (multiple); sunitinib/axitinib (RCC); lenvatinib (thyroid/HCC/endometrial) |
| Proteasome Inhibitors | Disrupt protein degradation, inducing myeloma cell apoptosis | Bortezomib, carfilzomib, ixazomib โ multiple myeloma |
| mTOR Inhibitors | Block PI3K/AKT/mTOR signalling driving cancer growth | Everolimus (NET, RCC, breast); temsirolimus (RCC) |
Targeted Therapy by Cancer Type: Key Biomarkers and Agents
Targeted therapy has transformed outcomes in cancers with high rates of actionable molecular alterations. The following cancer types have the most established targeted therapy landscapes.
Non-Small Cell Lung Cancer (NSCLC)
The most comprehensive targeted therapy landscape in oncology. EGFR mutations (osimertinib), ALK fusions (alectinib), ROS1 fusions (crizotinib), BRAF V600E (dabrafenib+trametinib), MET exon 14 (capmatinib), RET fusions (selpercatinib), KRAS G12C (sotorasib/adagrasib). NGS is mandatory before systemic therapy in advanced NSCLC.
Breast Cancer
HER2+ disease: trastuzumab, pertuzumab, T-DM1, T-DXd. HR+/HER2-: CDK4/6 inhibitors + endocrine therapy as standard. Triple-negative: PARP inhibitors (BRCA-mutated), sacituzumab govitecan, pembrolizumab + chemo.
Gastrointestinal Cancers
CRC: anti-EGFR (cetuximab/panitumumab, RAS-WT only); BRAF V600E (encorafenib+cetuximab); HER2 amplified (trastuzumab+pertuzumab). GIST: imatinib. Gastric/GEJ: HER2+ (trastuzumab); claudin 18.2 (zolbetuximab).
Haematological Malignancies
CML: BCR-ABL TKIs (imatinib โ dasatinib โ ponatinib). CLL: BTK inhibitors (ibrutinib, acalabrutinib), venetoclax. AML: FLT3 (midostaurin/gilteritinib), IDH1/2 (enasidenib/ivosidenib). Multiple myeloma: proteasome inhibitors, IMiDs, anti-CD38 antibodies.
Acquired Resistance: Why It Happens and How It Is Overcome
Most patients who respond to targeted therapy eventually develop resistance. Understanding resistance mechanisms is essential โ and drives the sequencing of next-generation agents.
On-Target Resistance Mutations
Cancer cells develop secondary mutations in the targeted gene that prevent drug binding. EGFR T790M (osimertinib overcomes first-generation resistance); BCR-ABL T315I (ponatinib overcomes most other TKI resistance) โ the paradigm for next-generation drug development.
Bypass Pathway Activation
Cancer cells activate alternative signalling pathways that bypass the blocked target. MET amplification bypasses EGFR inhibition; RAS mutation bypasses HER2 inhibition. Liquid biopsy at progression identifies these mechanisms and guides next therapy selection.
Histological Transformation
EGFR-mutated NSCLC can transform to small cell lung cancer under TKI pressure. This transformation requires biopsy at progression โ not just molecular re-testing โ to identify the mechanism and switch to appropriate treatment.
Liquid Biopsy at Progression
ctDNA liquid biopsy detects resistance mutations emerging in circulating tumour DNA โ often earlier and less invasively than tissue re-biopsy. Critical for selecting the next-line targeted therapy and for identifying patients eligible for resistance-overcoming agents.
Targeted Therapy: Key Numbers
- 90%+Complete cytogenetic response with imatinib in CMLThe benchmark result that launched the targeted therapy revolution in oncology.
- 100+Targeted agents approved globally across cancer typesFrom TKIs to ADCs to PARP inhibitors โ the largest and fastest-growing drug category in oncology.
- 18โ24Months: typical duration of response before resistance in many solid tumour TKI settingsUnderstanding and planning for resistance is part of every targeted therapy treatment strategy.
- 30โ50%of advanced NSCLC patients have an actionable molecular alterationThe highest rate of any solid tumour โ why NGS is universally mandated in advanced NSCLC.
- 18.9Median progression-free survival with first-line osimertinib in EGFR-mutant NSCLCA landmark benchmark for modern targeted therapy, showing how biomarker-matched treatment can deliver prolonged disease control in advanced lung cancer.
- 2.6XLonger survival with matched precision therapy versus unmatched treatmentRecent real-world precision oncology data showed patients treated with a genomically matched drug lived about 2.6 times longer, reinforcing the value of broad molecular profiling before treatment selection.
How CancerFax Helps Patients Access the Right Targeted Therapy
From molecular testing to accessing agents approved in China but not yet in your home country.
- 1
NGS Testing Guidance
Identification of the most appropriate molecular profiling panel if not yet done, and interpretation of existing NGS results to identify actionable targets and relevant approved or trial-stage targeted agents.
- 2
Treatment Option Identification
Review of all approved targeted agents relevant to the patient's molecular profile โ including agents approved in China or Japan but not yet in the patient's home country, which may be accessible through CancerFax.
- 3
Specialist Consultation
Facilitation of second-opinion consultations with molecular oncologists specialising in the patient's cancer type and targeted therapy landscape.
- 4
Access to China-Approved Agents
Several targeted agents have NMPA approval in China before or instead of FDA/EMA approval โ including some ADCs, TKIs, and HER2-targeted agents. CancerFax facilitates access for eligible patients.
- 5
Resistance Management
Support for patients who have progressed on first-line targeted therapy โ including coordination of liquid biopsy, re-biopsy, and identification of next-generation agents for resistance-overcoming therapy.
Explore Targeted Therapy in Detail
Each page covers one targeted therapy topic in depth.
- What is Targeted Therapy?
- How Targeted Therapy Works
- Targeted Therapy vs Chemotherapy
- Targeted Therapy vs Immunotherapy
- Types of Targeted Therapy Drugs
- Targeted Therapy for Lung Cancer (EGFR, ALK)
- Targeted Therapy for Breast Cancer (HER2)
- Targeted Therapy for Colorectal Cancer (RAS, BRAF)
- Targeted Therapy for Leukemia (BCR-ABL)
- Targeted Therapy for Melanoma (BRAF/MEK)
- Biomarker Testing Before Targeted Therapy
- How Long Does Targeted Therapy Work?
- Drug Resistance in Targeted Therapy
- Side Effects of Targeted Therapy
- Managing Side Effects of Targeted Drugs
- Success Rates of Targeted Therapy
- Cost of Targeted Therapy Worldwide
- Access to Targeted Therapy Abroad
- Clinical Trials in Targeted Therapy
- Future of Targeted Therapy
Frequently Asked Questions
What is targeted therapy for cancer?
Targeted therapy is a type of cancer treatment that uses drugs designed to act on specific molecules or genetic changes that help cancer cells grow and spread. Unlike chemotherapy, which affects rapidly dividing cells throughout the body, targeted therapy focuses on particular markers found in or on cancer cells. This often means fewer effects on healthy tissue, though side effects still occur. The right targeted therapy depends on the cancer type and its molecular profile, which is why proper testing and expert oncology review are essential before treatment begins.
How is targeted therapy different from chemotherapy?
Chemotherapy works broadly by attacking cells that divide quickly, including some healthy ones, which is why it often causes hair loss and other well-known side effects. Targeted therapy works more selectively by blocking the specific pathways a tumor relies on. Both have a role in cancer care, and in many cases they are used together or in sequence. The choice depends on the cancer type, stage, molecular findings, and the patient's overall condition.
Who is a candidate for targeted therapy?
Targeted therapy is suitable for patients whose tumors carry specific molecular changes that a drug can act on. This is determined through tests such as NGS, immunohistochemistry, and biomarker testing for markers like EGFR, ALK, ROS1, HER2, BRAF, KRAS, and others depending on the cancer type. Not every patient or every cancer has a targetable change, so eligibility is decided after complete investigations and review by a qualified oncologist.
What tests are needed before starting targeted therapy?
Before targeted therapy, doctors usually need a confirmed diagnosis through biopsy and pathology, along with molecular or genomic testing to identify treatable targets. Common tests include NGS panels, PD-L1, HER2, EGFR, ALK, ROS1, BRAF, KRAS, NRAS, and NTRK, with the exact panel depending on the cancer type. These results guide whether a targeted drug is appropriate and which one is most likely to help. Treatment decisions should not be made without complete testing and oncology review.
Which cancers can be treated with targeted therapy?
Many cancers now have targeted treatment options, including lung cancer, breast cancer, colorectal cancer, liver cancer, gastric cancer, certain leukemias and lymphomas, melanoma, kidney cancer, and others. Availability of a specific targeted drug depends on whether the tumor carries a matching molecular change. Research continues to expand the list of treatable targets each year, so options that were not available a few years ago may exist today.
What are the common side effects of targeted therapy?
Side effects vary by drug but can include skin changes, diarrhea, high blood pressure, fatigue, liver enzyme changes, and effects on wound healing or the heart in some cases. Because targeted therapy is more selective than chemotherapy, the side effect pattern is often different rather than simply milder. Side effects are usually manageable with proper monitoring, and patients should always report new symptoms to their care team promptly.
How long does targeted therapy treatment last?
The duration depends on the cancer type, how well the treatment is working, and how the patient tolerates it. Some patients take targeted drugs for many months or years as long as the cancer stays under control and side effects remain manageable. Treatment may continue, change, or stop based on regular scans and assessments. The plan is reviewed continuously by the treating oncologist.
Can cancer become resistant to targeted therapy?
Yes, cancers can develop resistance over time, meaning a drug that worked initially may stop being effective. When this happens, doctors may repeat molecular testing to look for new changes and consider a different targeted drug, a combination approach, or another treatment strategy. This is one reason ongoing monitoring and access to newer therapies and clinical trials can matter for patients on long-term treatment.
Is targeted therapy available in China and India, and how does CancerFax help?
Targeted therapy is available in major cancer centers in both India and China, and China in particular offers access to a wide range of advanced therapies and clinical trials. CancerFax helps patients and families review their medical records, identify whether their tumor has treatable molecular targets, coordinate second opinions, and explore treatment options across international oncology centers. Support includes medical record review, treatment planning guidance, hospital coordination, trial matching, and travel and translation help where needed.
How do I find out if targeted therapy is right for my case?
The first step is a careful review of your diagnosis, pathology, and any molecular testing already done, followed by identifying which tests may still be needed. CancerFax can help organize your records, highlight missing investigations, and arrange expert review so you understand your options clearly. Every recommendation should come from qualified oncologists based on complete information, and CancerFax supports you through that process from diagnosis confusion to confident next steps. You can reach the team at [email protected] for a personalized review.
How CancerFax Helps
CancerFax is a specialist cancer access and patient-navigation platform. We help patients and families understand their options, organise medical records, coordinate hospital communication, and support cross-border treatment planning where appropriate.
We help collect and organise reports, scans, pathology, biomarker results, and treatment history for structured case review.
We communicate with hospitals or trial teams to assess whether a case may be suitable for further screening.
We support appointment coordination, document submission, translation, and direct communication with international departments.
For international patients, we help with practical coordination โ travel planning, hospital admission guidance, and local support.
If this option is not suitable, we help explore other relevant treatments, clinical trials, or advanced care pathways.
From inquiry through to follow-up, our coordinators provide a single point of contact for the family.
CancerFax does not guarantee treatment access, eligibility, or clinical outcome. Our role is to help patients access accurate information, structured review, and appropriate specialist pathways.
Do You Have a Molecular Target That Needs the Right Drug?
Upload your NGS report and medical records โ our team will identify approved and trial-stage targeted agents relevant to your specific molecular profile.
This content is for informational purposes only. Always consult a qualified oncologist before making treatment decisions.