Understanding Targeted Therapy for Cancer

Targeted therapy uses drugs to identify and attack cancer cells (1). These drugs can find cancer cells so well because they look for genetic mutations and changes in protein synthesis, two hallmarks of cancer (2). Healthy tissues do not typically express these changes and are not readily harmed during targeted therapy. This article is for patients undergoing targeted cancer therapy, their carers, and other curious readers. It discusses the process of targeted therapy, compares it to other treatment types, and provides information about who it might be suitable for. 

AI generated image of female cancer patient baking.

What is Targeted Therapy?

Targeted cancer treatment is a cancer treatment that involves your whole body. The drug circulates throughout the bloodstream, into every body “system” in search of cancer cells (3).  It is also called a molecular targeted therapy since the drugs used block specific molecules. These molecules are proteins and can be receptors on the cancer cell surface or active enzymes inside cancer cells. Cancer cells depend on abnormal or overproduced molecules created by genetic mutations to spread. Drugs that target and block them stop cancer cells from dividing, surviving, spreading, and multiplying (4).

Targeted Therapy versus Biological Therapy

Biological therapy for cancer treatment relies on products or substances that occur in the body, such as antibodies and enzymes (5). Whereas targeted therapy relies on products that may or may not occur in the body. These products target and destroy cancer cells.  When a targeted therapy drug is also a biological one, it can be called biologic targeted therapy. 

How does Targeted Therapy Work?

Targeted therapy works by (6):

  • Blocking communication signals that help cancer cells divide and grow.
  • Preventing cancer cells from developing new blood vessels and getting their own blood supply.
  • Some targeted therapies may help the immune system recognise and respond to cancer cells more effectively, although this is not the primary mechanism of most targeted therapies.
  • Directly cause the death of cancer cells.

Cancer therapies can be given as a tablet which you can take at home, an infusion/ IV injection, or a subcutaneous injection (under your skin) which will have to be given at the hospital (7).

Types of Targeted Therapy

The different kinds of targeted therapy can be grouped into two main categories: small-molecule inhibitor drugs and monoclonal antibodies. Both of these groups target specific molecules that cause the growth of cancers.

Small Molecule Inhibitors

These drugs directly block the growth of cancer cells by disrupting the proteins inside the cell. 10 Examples are kinase inhibitors like CDK4/6 inhibitors and PARP inhibitors. Most small molecule inhibitors are taken orally.

Monoclonal Antibodies

These are manufactured or synthetic versions of antibodies that occur naturally in the body. They are very specific, in that they bind to a particular protein on a cancer cell, or the tissues around that cancer cell, and cause destruction. Monoclonal antibodies are usually administered by intravenous infusion or injections (11).

What Cancers Can be Treated with Targeted Therapy? 

Targeted therapy can be used to treat: 

  • Breast cancer.
  • Lung cancer.
  • Colorectal cancer.
  • Melanoma.
  • Head and neck cancers.
  • Kidney cancer.
  • Liver cancer.
  • Leukaemias and lymphomas.

Doctors recommend it when molecular tests reveal your cancer will respond to targeted treatments. If your cancer is unlikely to respond to targeted treatment, you can be considered ineligible. Targeted therapy is usually applied to later stages of cancer (stages III and IV) to slow down the progression of the disease. 

However, it is now increasingly being used to treat early stages of cancer. 

Targeted Therapy for Breast Cancer

Targeted therapy for breast cancer can be given as a tablet or an IV infusion/injection.  It is usually recommended because most breast cancers have identifiable molecular targets that the drug can work against. It can be used across all stages of breast cancer, even in the metastatic stages. 

The main types of targeted therapy for breast cancer include: HER2-targeted therapy, CDK4/6 inhibitors, PARP inhibitors, and others.

Targeted Therapy for Lung Cancer

Like with breast cancer, this can be given as an oral suspension, as an intravenous infusion, or as an injection into the skin or the muscle. It helps in lung cancer because lung cancer responds to molecular targeting and is usually given in the later stages. Main drugs for lung cancer targeted therapy target specific mutations like EGFR, ALK, ROS, and others.

DID YOU KNOW?

Biomarkers are specific proteins or molecules on the surface or inside a cell that serve as identification tags. Biomarker tests look for characteristics of a cancer, such as proteins, or other molecular markers, that may help guide treatment decisions. Genetic tests analyse DNA or other genetic material to identify changes that may influence cancer behaviour or treatment response (8, 9).

Targeted Therapy Compared to Other Treatments

When you're diagnosed with cancer, your doctor has many treatment methods in mind, such as surgery, anti-cancer drug therapy, and radiation. After comparing therapies, your medical team will advise you on the best option for you.  

Targeted Therapy versus Immunotherapy

Immunotherapy works by helping your body's immune system become strong enough to attack cancer cells, whereas targeted therapy attacks cancer cells on its own by blocking specific proteins, mutations, or signals to grow and spread.  

Targeted Therapy versus Chemotherapy

Chemotherapy works by damaging or interfering with rapidly dividing cells, including cancer cells. Because some healthy cells also divide rapidly, chemotherapy may affect normal tissues as well. Targeted therapy is newer and more innovative, targeting specific proteins and causing less damage to healthy cells. Although side effects with targeted therapy are not as severe as with chemotherapy, you may experience a few, and sometimes they can greatly impact your overall well-being. 

Targeted Therapy versus Radiation

Radiation is not a systemic cancer treatment. It acts locally by destroying cancer cells in a specific area, whereas targeted therapy is a systemic drug that focuses on specific molecular abnormalities within cancer cells. Modern radiation therapy techniques can focus radiation more precisely on a tumour while reducing exposure to nearby healthy tissues.   

What Factors influence Treatment Outcomes

The goals of targeted therapy depend on the stage of your cancer and the treatment setting. Goals can be: 

  • To cure the cancer completely.
  • To reduce the risk of the cancer coming back.
  • To control cancer spread and extend life.
  • To improve the quality of your life.

Outcomes of targeted therapy are typically determined by (12):

  • The type of cancer (biology of the tumour): the presence of specific biomarkers, genes, and secondary mutation status.
  • Past treatment history.
  • Your health status: how you adhere to the drug, how well you tolerate it, and how well your body metabolises it.

What Happens after Targeted Therapy?

After you've received targeted therapy, you'll be monitored routinely with scans, blood tests and physical examinations. Targeted therapy is usually less toxic than chemotherapy and so the side effects may not be as many but may still be distressing. 

Pros and Cons

Pros of targeted cancer therapy: 

  • It’s highly precise at targeting cancer cells while sparing healthy ones.
  • It is not as toxic as chemotherapy.
  • Its administration is convenient.

Cons of targeted cancer therapy: 

  • Only effective against cancers with specific targets.
  • Some cancer cells can adapt to it and become resistant.
  • Can cause some side effects, like severe rash or other skin changes, high blood pressure.

Key Takeaways

Targeted therapy is a newer approach to cancer treatment, which uses drugs that track and attack cancer cells by following a trail of genetic changes or alterations in proteins. Usually, your doctor will recommend targeted therapy when your cancer produces traceable changes that a drug can track. It may be used alone or combined with another form of cancer treatment. 

References:

  1. [1] Smith CEP, Prasad V. Targeted Cancer Therapies. Am Fam Physician. 2021 Feb 1;103(3):155-163. PMID: 33507053.
  2. [2] Pessoa J, et al. Altered Expression of Proteins in Cancer: Function and Potential Therapeutic Targets. Front Oncol. 2022 Jun 22;12:949139. doi: 10.3389/fonc.2022.949139. PMID: 35814397; PMCID: PMC9257268.
  3. [3] Geyer T, et al. Systemic Anticancer Treatment Near the End of Life: a Narrative Literature Review. Curr Treat Options Oncol. 2023 Oct;24(10):1328-1350. doi: 10.1007/s11864-023-01115-x. Epub 2023 Jul 27. PMID: 37501037; PMCID: PMC10547806.
  4. [4] Tang Z, et al. New insights into molecules and pathways of cancer metabolism and therapeutic implications. Cancer Commun (Lond). 2021 Jan;41(1):16-36. doi: 10.1002/cac2.12112. Epub 2020 Nov 10. PMID: 33174400; PMCID: PMC7819563.
  5. [5] Papież MA, et al. Biological Therapies in the Treatment of Cancer-Update and New Directions. Int J Mol Sci. 2021 Oct 28;22(21):11694. doi: 10.3390/ijms222111694. PMID: 34769123; PMCID: PMC8583892.
  6. [6] Shuel SL. Targeted cancer therapies: Clinical pearls for primary care. Can Fam Physician. 2022 Jul;68(7):515-518. doi: 10.46747/cfp.6807515. PMID: 35831091; PMCID: PMC9842142.
  7. [7] Karati D, et al. A Comprehensive Review on Targeted Cancer Therapy: New Face of Treatment Approach. Curr Pharm Des. 2023;29(41):3282-3294. doi: 10.2174/0113816128272203231121034814. PMID: 38038008.
  8. [8] Califf RM. Biomarker definitions and their applications. Exp Biol Med (Maywood). 2018 Feb;243(3):213-221. doi: 10.1177/1535370217750088. PMID: 29405771; PMCID: PMC5813875.
  9. [9] Panchariya DC, et al Genetic marker: a genome mapping tool to decode genetic diversity of livestock animals. Front Genet. 2024 Oct 17;15:1463474. doi: 10.3389/fgene.2024.1463474. PMID: 39483851; PMCID: PMC11524813.
  10. [10] Wang Y, et al. Targeted cancer therapy: A comprehensive review of strategies in drug development. Crit Rev Oncol Hematol. 2026 Jun;222:105303. doi: 10.1016/j.critrevonc.2026.105303. Epub 2026 Mar 27. Erratum in: Crit Rev Oncol Hematol. 2026 Jul;223:105349. doi: 10.1016/j.critrevonc.2026.105349. PMID: 41905570.
  11. [11] Zahavi D, et al. Monoclonal Antibodies in Cancer Therapy. Antibodies (Basel). 2020 Jul 20;9(3):34. doi: 10.3390/antib9030034. PMID: 32698317; PMCID: PMC7551545.
  12. [12] Taghizadeh H, et al. Outcome of Targeted Therapy Recommendations for Metastatic and Recurrent Head and Neck Cancers. Cancers (Basel). 2020 Nov 15;12(11):3381. doi: 10.3390/cancers12113381. PMID: 33203166; PMCID: PMC7696688.

Disclaimer

This information is provided for educational purposes only. It is general in nature and is not intended to replace professional medical advice, diagnosis, or treatment. Always consult your healthcare team regarding any questions or concerns about your health or care.

  • Cancer
  • Treatment
  • Cancer Care

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