Targeted therapies for cancer


Targeted therapy uses drugs to stop cancer from growing and spreading. It does this with less harm to normal cells than other treatments.

Standard chemotherapy works by killings cancer cells and some normal cells, targeted treatment zeroes in on specific targets (molecules) in or on cancer cells. These targets play a role in how cancer cells grow and survive. Using these targets, the drug disables the cancer cells so they cannot spread.

Alternative Names

Molecularly targeted anticancer agents; MTAs; Chemotherapy-targeted; Vascular endothelial growth factor-targeted; VEGF-targeted; VEGFR-targeted; Tyrosine kinase inhibitor-targeted; TKI-targeted; Personalized medicine - cancer

How Does Targeted Therapy Work?

Targeted therapy drugs work in a few different ways. They may:

  • Turn off the process in cancer cells that causes them to grow and spread
  • Trigger cancer cells to die on their own
  • Kill cancer cells directly

People with the same type of cancer may have different targets in their cancer cells. So if your cancer does not have a specific target, the drug will not work to stop it. Not all therapies work for all people with cancer. At the same time, different cancers may have the same target.

To see if a targeted therapy might work for you, your health care provider may:

  • Take a tiny sample of your cancer
  • Test the sample for the specific targets (molecules)
  • If the right target is present in your cancer, then you will receive

Some targeted therapies are given as pills. Others are injected into a vein (intravenous, or IV).

Who may get Targeted Therapy?

There are targeted therapies that can treat certain types of these cancers:

  • Leukemia and lymphoma
  • Breast cancer
  • Colon cancer
  • Skin cancer
  • Lung cancer
  • Prostate

Other cancers that may be treated with targeted therapies include brain, bone, kidney, lymphoma, stomach, and many others.

Your provider will decide whether targeted therapies may be an option for your type of cancer. In most cases, you will receive targeted therapy along with surgery, chemotherapy, hormonal therapy, or radiation therapy. You may receive these drugs as part of your regular treatment, or as part of a clinical trial.

Side Effects

Doctors thought that targeted therapies might have fewer side effects that other cancer treatment. But that turned out to be untrue. Possible side effects from targeted therapies include:

  • Diarrhea
  • Liver problems
  • Skin problems such as rash, dry skin, and nail changes
  • Problems with blood clotting and wound healing
  • High blood pressure

As with any treatment, you may or may not have side effects. They may be mild or severe. Fortunately, they usually go away after treatment ends. It is a good idea to talk with your provider about what to expect. Your provider may be able to help prevent or lessen some side effects.


Targeted therapies are promising new treatments, but they have limitations.

  • Cancer cells can become resistant to these drugs.
  • The target sometimes changes, so the treatment no longer works.
  • The cancer may find a different way to grow and survive that does not depend on the target.
  • Drugs can be difficult to develop for some targets.
  • Targeted therapies are newer and cost more to make. So they are more expensive than other cancer treatments.


Kummar S, Murgo AJ, Tomaszewski JE, Doroshow JH. Therapeutic targeting of cancer cells: era of molecularly targeted agents. In: Niederhuber JE, Armitage JO, Doroshow JH, Kastan MB, Tepper JE, eds. Abeloff's Clinical Oncology. 5th ed. Philadelphia, PA: Elsevier Saunders; 2014:chap 28.

National Cancer Institute website. Targeted cancer therapies. Updated November 6, 2017. Accessed December 15, 2017.

Review Date: 
Reviewed By: 
Todd Gersten, MD, Hematology/Oncology, Florida Cancer Specialists & Research Institute, Wellington, FL. Review provided by VeriMed Healthcare Network. Also reviewed by David Zieve, MD, MHA, Medical Director, Brenda Conaway, Editorial Director, and the A.D.A.M. Editorial team.