Targeted therapy focuses on the genetic characteristics of cancer cells. It uses drugs to attack mutated genes to prevent the cancer from surviving or spreading.

Introduced in 2004, targeted therapy is now a common personalized treatment for certain types of cancer. Research shows that, for some types of cancer, target therapies are more effective than chemotherapy drugs and have fewer side effects.

The number of targeted therapy drugs approved by the Food and Drug Administration (FDA) for treating various types of cancer continues to grow.

In 2006, only 5.13% of Americans with cancer were eligible for targeted therapy. By 2020, that number had grown to 13.6%.

Cancer changes the genes in your cells, and those mutations cause your cells to multiply and grow.

For targeted therapy, a doctor collects a sample of cancer cell tissue by performing a biopsy or surgery. They then send the sample to a lab for testing to determine the particular mutation that is causing the change in the cell’s genes. This is called biomarker or molecular testing.

Once your doctor identifies the mutated gene, you take targeted drugs in pill or intravenous (IV) form that will kill or slow that specific mutation. The cancer may decrease in size or disappear.


  • Targeted therapy kills only cancer cells, not healthy cells.
  • It changes the proteins in cancer cells to stop cancer from growing.
  • It stops the formation of new blood vessels and the supply of blood to your cancer cells.


  • If the targeted protein changes or cancer cells can grow without that particular protein, the cancer cells may resist targeted therapy.
  • You may experience side effects such as diarrhea and skin problems.
  • Only people with certain types of cancer are eligible for targeted therapy.
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Doctors use two common types of targeted therapy.

Monoclonal antibodies are lab-produced versions of your body’s antibodies. They target antigens, the unwanted proteins on or near your cells that may develop from cancer.

Small-molecule inhibitors are small enough to easily enter your cancer cells and attach themselves to targets, killing them or preventing them from spreading.

A healthcare professional administers monoclonal antibodies through an IV, but you can often take small-molecule inhibitors in capsule or tablet form.

Doctors can treat the following types of cancer with FDA-approved targeted therapy drugs:

Cancer typeFDA-approved targeted therapies
bladder cancerenfortumab vedotin (Padcev), sacituzumab govitecan (Trodelvy), and others
brain cancerbelzutifan (Welireg), dabrafenib (Tafinlar), everolimus (Afinitor), and trametinib (Mekinist)
breast cancerlapatinib ditosylate (Tykerb), margetuximab (Margenza), pertuzumab (Perjeta), trastuzumab (Herceptin), and others
cervical cancerbevacizumab (Avastin), pembrolizumab (Keytruda), and tisotumab vedotin-tftv (Tivdak)
colorectal cancerbevacizumab (Avastin), cetuximab (Erbitux), encorafenib (Braftovi), trastuzumab (Herceptin), and others
endometrial cancerdostarlimab-gxly (Jemperli), lenvatinib mesylate (Lenvima), and pembrolizumab (Keytruda)
esophageal cancernivolumab (Opdivo), fam-trastuzumab deruxtecan (Enhertu), ramucirumab (Cyramza), trastuzumab (Herceptin), and others
head and neck cancerscetuximab (Erbitux), nivolumab (Opdivo), and pembrolizumab (Keytruda)
kidney canceraxitinib (Inlyta), bevacizumab (Avastin), sorafenib (Nexavar), sunitinib (Sutent), and others
acute myeloid leukemiabosutinib (Bosulif), imatinib (Gleevec), nilotinib (Tasigna), and others
chronic lymphocytic leukemiaibrutinib (Imbruvica), idelalisib (Zydelig), venetoclax (Venclexta), and others
liver and bile duct cancersbevacizumab (Avastin), infigratinib (Truseltiq), pemigatinib (Pemazyre), regorafenib (Stivarga), sorafenib (Nexavar), and others
lung cancerafatinib (Gilotrif), bevacizumab (Avastin), osimertinib (Tagrisso), ramucirumab (Cyramza), and others
lymphomabelinostat (Beleogaq), bortezomib (Velcade), copanlisib (Aliqopa), ibrutinib (Imbruvica), and others
multiple myelomabortezomib (Velcade), daratumumab (Darzalex), lenalidomide (Revlimid), panobinostat (Farydak), and others
ovarian cancerbevacizumab (Avastin), mirvetuximab soravtansine (Elahere), olaparib (Lynparza), and others
pancreatic cancerbelzutifan (Welireg), erlotinib hydrochloride (Tarceva), everolimus (Afinitor), olaparib (Lynparza), and sunitinib malate (Sutent)
prostate cancerolaparib (Lynparza), rucaparib (Rubraca), talazoparib (Talzenna), and others
skin cancercetuximab (Erbitux), sonidegib (Odomzo), vismodegib (Erivedge), and others
soft tissue sarcomapazopanib (Votrient), tazemetostat (Tazverik), and others
stomach cancerfam-trastuzumab deruxtecan (Enhertu), pembrolizumab (Keytruda), ramucirumab (Cyramza), trastuzumab (Herceptin), and others
thyroid cancerlenvatinib (Lenvima), sorafenib (Nexavar), vandetanib (Capresla), and others

Like chemotherapy, targeted therapy may cause side effects. But they may be fewer or less severe than chemotherapy side effects.

The possible side effects may depend on the type of drugs you’re taking. Other medications may help prevent or reduce side effects.

The common side effects of targeted therapy are:

  • diarrhea
  • liver problems such as hepatitis
  • skin issues such as an acne-like rash on your face, chest, and back that may itch or burn

The more serious possible side effects include:

Side effects usually subside once your targeted therapy is complete, but this may vary depending on the particular drugs and your overall health.

The effectiveness of targeted therapy depends on factors such as the type of cancer and the specific genetic characteristics of the cancer cells.

Research indicates that targeted cancer therapy has improved response rates and delayed cancer progression for lung cancer, metastatic breast cancer, and other incurable cancers.

A 2022 study of 58 people with advanced non-small cell lung cancer found that the median survival rate for those who received targeted therapy was nearly double that of those who received other therapies.

The researchers noted that targeted therapy also more effectively reduced the risk of cancer progression or recurrence.

Targeted therapy has significantly improved the survival rate for people with HER2-positive breast cancer. In clinical trials, the drug trastuzumab (Herceptin) combined with chemotherapy improved the 3-year disease-free survival rate by up to 52% and reduced the risk of relapse by about 30%.

How long does targeted therapy last?

The duration of targeted therapy depends on factors such as:

  • the type of cancer
  • the drugs being used
  • your response to the treatment

A doctor may determine whether you’ll receive targeted therapy for a defined amount of time, continuously, or intermittently. You may need to take targeted therapy drugs every day for months or years.

To ensure that your targeted therapy is working and your cancer cells have shrunk or disappeared, you’ll see a doctor for regular checkups that include blood tests and scans.

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Targeted therapy uses drugs that kill only mutations in your cancer cells.

Chemotherapy uses drugs that kill all cells that multiply quickly and may be cancerous, including healthy cells.

Also, unlike chemotherapy, targeted therapy can prevent cancer cells from dividing and creating new cancer cells. Chemotherapy attacks duplicate cells after they’ve been created.

Immunotherapy boosts your immune system’s ability to destroy cancer cells.

Like targeted therapy, immunotherapy uses monoclonal antibodies that attach to cancer cells, which your immune system can then destroy.

Doctors may combine targeted therapy with immunotherapy to enhance its cancer-fighting abilities.

Targeted therapy has proven to be an effective treatment for certain types of cancer, especially incurable lung cancer and metastatic breast cancer. Doctors may use targeted therapies alone or in combination with chemotherapy or immunotherapy.

Although targeted therapy is currently available for only certain genetic mutations, researchers are identifying more cancer genes and developing targeted drugs to treat them.