Targeted Therapy
If your treatment plan includes targeted therapy, knowing how it works and what to expect can help you prepare for treatment and make informed decisions about your care.
What is targeted therapy?
Targeted therapy is a type of precision or personalized medicine that treats cancer by targeting specific features, changes, mutations, or substances in or on cancer cells.
There are many kinds of targeted therapies. They are designed to stop cancer cells from growing and spreading while limiting damage to normal, healthy cells. Each type works in a specific way. For example, they might:
- Target specific biomarkers (genes and proteins that help cancer cells survive and grow)
- Change the tissue or environment that cancer cells grow in
- Target other types of cells that help a cancer grow, like blood vessel cells
What is the goal of targeted therapy?
If your doctor has recommended targeted therapy, it’s important to understand the goal of treatment. This can depend on your type of cancer, its stage (size and location), what biomarkers (gene and protein changes) it has, and how far it has spread. Targeted therapy can have 3 possible treatment goals:
Whenever possible, the goal of targeted therapy is to cure the cancer—destroying it completely so it doesn’t come back. Doctors may call this “treatment with curative intent.” They may avoid saying “cure” because it can take many years to know for sure if the cancer is truly gone. Cure is the hope, but it’s not always possible.
If a cure is not possible, targeted therapy may be used to slow the growth of the cancer or shrink it. This can help people feel better and live longer. The cancer might not go away completely, but it can often be controlled and managed as a chronic disease, like heart disease or diabetes. Sometimes the cancer may go away for a while but come back later.
Targeted therapy can also be used to help people feel better. This is called palliation. It may be given even when the cancer has spread or can’t be controlled to improve comfort and quality of life. For example, it may help shrink a tumor that’s pressing on an area and causing pain.
Palliative targeted therapy is just one part of palliative care, which includes things like pain relief and anti-nausea medicine. Palliative treatments can be used at any stage of cancer.
How is targeted therapy different from chemotherapy?
Chemotherapy and targeted therapy are both treatments for cancer, but they are different types of drugs. They work in different ways and cause different side effects.
- Targeted therapy acts on specific types of cancer cells and mostly leaves normal, healthy cells alone. Traditional chemo, however, is cytotoxic to most cells, meaning it can damage normal, healthy cells too.
- Some targeted therapies work by blocking cancer cells from copying themselves. This means they can help stop a cancer cell from dividing and making new cancer cells. Traditional chemo, however, kills cancer cells that have already been made.
Both chemo and targeted therapy are systemic treatments. They travel through the bloodstream to reach all parts of your body. This means they can both treat cancer that has spread (metastasized) to parts of the body far from where the original (primary) tumor started, or blood cancers, such as leukemia, that have spread throughout the body. This makes them different from local treatments like surgery and radiation, which only affect one part of the body.
Which cancers can be treated with targeted therapy?
Targeted therapies are not yet available for all cancers. The biomarkers (gene changes, proteins, and other substances) these treatments target can be different for different cancers, and even for different people with the same type of cancer. Targeted therapy is most often used for:
- Breast cancer
- Colorectal cancer
- Gastrointestinal stromal tumors
- Leukemias (acute, chronic, or specific subtypes)
- Liver (hepatocellular) cancer
- Lung cancer
- Melanoma
- Ovarian cancer
- Pancreatic cancer
- Prostate cancer
- Stomach cancer
- Thyroid cancer
Biomarker testing is often needed to know whether your cancer might respond to targeted therapy.
Learn about targeted therapy for your cancer type
What is it like to get targeted therapy?
Most people get targeted therapy as infusions or injections at a clinic or hospital or by taking medicine by mouth at home. Each type has its own routine, side effects, and safety steps.
Targeted therapy schedules
Some targeted therapy is given at regular intervals called cycles. A cycle may be a dose of targeted therapy (sometimes with other treatments) on one or more days, followed by several days or weeks without treatment. You may also take targeted therapy every day with no rest breaks, or only 1 or more days a week or month.
Each targeted therapy is given on a schedule that makes the most of its anti-cancer actions and minimizes side effects. If you are getting more than one treatment, your treatment plan will say how often and exactly when each should be given.
Many types of targeted therapy are given as an infusion through an IV. The process is much like getting chemo infusions. If you are getting a combination of chemo and targeted therapy, they might be given at the same time or during separate appointments, depending on your treatment plan.
Subcutaneous (SC or SQ) targeted therapy is given as an injection or shot into the subcutaneous tissue (fatty tissue) right under your skin (similar to subcutaneous chemo). Not all types of targeted therapy can be given as a SQ injection, but the number is increasing. Giving targeted therapy this way is just as effective, but takes less time and doesn’t require an IV.
Oral targeted therapy is targeted therapy that can be taken by mouth as pills, capsules, or liquids. Most small-molecule targeted therapies are oral. Examples include imatinib, sunitinib, lenalidomide, and everolimus. It may be given in rounds or cycles and is often given along with traditional chemotherapy (chemo). Because oral targeted therapy is taken at home, it’s important to understand exactly how and when to take it, and what to do if you have any problems. Our information on oral chemotherapy can help you know what to expect and what to ask your care team.
Targeted therapy safety
Many targeted therapies, including small-molecule drugs and a few monoclonal antibodies, are considered hazardous agents that require special handling, similar to chemotherapy. But because many targeted therapies are newer, there isn’t as much information about the long-term effects of exposure. It’s important to follow the safety precautions recommended by your cancer care team. Usually, these are the same precautions used for chemotherapy safety.
What are the side effects of targeted therapy?
Targeted therapy has different side effects than standard or traditional chemotherapy (chemo). Some targeted therapies have very few side effects, and others can cause more or more serious problems. There are many different types of targeted therapies, and their side effects depend largely on the type that's given and what it targets.
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- References
Developed by the American Cancer Society medical and editorial content team with medical review and contribution by the American Society of Clinical Oncology (ASCO).
American Society of Clinical Oncology (ASCO). Understanding targeted therapy. Accessed at cancer.net. Content is no longer available.
American Society of Clinical Oncology. What is Targeted Therapy? Accessed at cancer.net. Content is no longer available.
Groves E. Targeted and Immunotherapy. In Maloney-Newton S, Hickey M, Brant JM, eds. Mosby’s Oncology Nursing Advisor: A Comprehensive Guide to Clinical Practice. 3rd ed. St. Louis: Elsevier; 2024:409-428.
National Cancer Institute (NCI). Targeted therapy to treat cancer. Updated May 31, 2022. Accessed at https://www.cancer.gov/about-cancer/treatment/types/targeted-therapies/targeted-therapies-fact-sheet on June 2, 2025.
Roman D. Targeted therapy. In Olsen MM, LeFebvre KB, Walker SL, Dunphy EP, eds. Chemotherapy and Immunotherapy Guidelines and Recommendations for Practice. 2nd ed. Pittsburgh, PA: Oncology Nursing Society; 2023:113-201.
Last Revised: June 2, 2025
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