Archives: Treatment Types

List of Targeted Therapy Drugs Approved for Specific Types of Cancer

List of Targeted Therapy Drugs Approved for Specific Types of Cancer

The FDA has approved targeted therapy drugs for the treatment of some people with the following types of cancer.  Some targeted therapy drugs are listed more than once because they have been approved to treat more than one type of cancer. The generic drug name is listed first, with a brand name in parentheses.

Targeted therapy approved for bladder cancer

Targeted therapy approved for brain cancer

Targeted therapy approved for breast cancer

Targeted therapy approved for cervical cancer

Targeted therapy approved for colorectal cancer

Targeted therapy approved for dermatofibrosarcoma protuberans

Targeted therapy approved for endocrine and neuroendocrine tumors

Targeted therapy approved for endometrial cancer

Targeted therapy approved for esophageal cancer

Targeted therapy approved for head and neck cancer

Targeted therapy approved for gastrointestinal stromal tumor

Targeted therapy approved for giant cell tumor

Targeted therapy approved for kidney cancer

Targeted therapy approved for leukemia

Targeted therapy approved for liver and bile duct cancer

Targeted therapy approved for lung cancer

Targeted therapy approved for lymphoma

Targeted therapy approved for malignant mesothelioma

Targeted therapy approved for multiple myeloma

Targeted therapy approved for myelodysplastic and myeloproliferative disorders

Targeted therapy approved for neuroblastoma

Targeted therapy approved for ovarian epithelial, fallopian tube, and primary peritoneal cancers

Targeted therapy approved for pancreatic cancer

Targeted therapy approved for plexiform neurofibroma

Targeted therapy approved for prostate cancer

Targeted therapy approved for skin cancer

Targeted therapy approved for soft tissue sarcoma

Targeted therapy approved for solid tumors anywhere in the body

Targeted therapy approved for stomach (gastric) cancer

Targeted therapy approved for systemic mastocytosis

Targeted therapy approved for thyroid cancer

Monoclonal Antibodies

Monoclonal Antibodies

cancer cell showing a monoclonal antibody locking onto an antigen on the cell's surface Enlarge

Some monoclonal antibodies mark cancer cells so that the immune system will better recognize and destroy them.

Credit: National Cancer Institute

How do monoclonal antibodies work against cancer?

Monoclonal antibodies are immune system proteins that are created in the lab. Antibodies are produced naturally by your body and help the immune system recognize germs that cause disease, such as bacteria and viruses, and mark them for destruction. Like your body’s own antibodies, monoclonal antibodies recognize specific targets.

Many monoclonal antibodies are used to treat cancer. They are a type of targeted cancer therapy, which means they are designed to interact with specific targets. Learn more about targeted therapy.

Some monoclonal antibodies are also immunotherapy because they help turn the immune system against cancer. For example, some monoclonal antibodies mark cancer cells so that the immune system will better recognize and destroy them. An example is rituximab, which binds to a protein called CD20 on B cells and some types of cancer cells, causing the immune system to kill them. B cells are a type of white blood cell.

Other monoclonal antibodies bring T cells close to cancer cells, helping the immune cells kill the cancer cells. An example is blinatumomab (Blincyto®), which binds to both CD19, a protein found on the surface of leukemia cells, and CD3, a protein on the surface of T cells. This process helps the T cells get close enough to the leukemia cells to respond to and kill them.

a monoclonal antibody brings a t cell close to the cancer cell

Some monoclonal antibodies bring t cells close to cancer cells, helping them kill cancer cells.

Credit: National Cancer Institute

Which cancers are treated with monoclonal antibodies?

Many monoclonal antibodies have been approved to treat a wide variety of cancers.

To learn about specific treatments for your cancer, see the PDQ® adult cancer treatment summaries and childhood cancer treatment summaries.

What are the side effects of monoclonal antibodies?

Monoclonal antibodies can cause side effects, which can differ from person to person. The ones you may have and how they make you feel will depend on many factors, such as how healthy you are before treatment, your type of cancer, how advanced it is, the type of monoclonal antibody you are receiving, and the dose.

Doctors and nurses cannot know for sure when or if side effects will occur or how serious they will be. So, it is important to know which signs to look for and what to do if you start to have problems.

Like most types of immunotherapy, monoclonal antibodies can cause skin reactions at the needle site and flu-like symptoms.

Needle site reactions include:

  • pain
  • swelling
  • soreness
  • redness
  • itchiness
  • rash

Learn more about skin changes caused by cancer treatment.

Flu-like symptoms include:

  • chills
  • fatigue
  • fever
  • muscle aches and pains
  • nausea
  • vomiting
  • diarrhea

Learn more about flu-like symptoms caused by cancer treatment.

Monoclonal antibodies can also cause:

  • mouth and skin sores that can lead to serious infections
  • high blood pressure
  • congestive heart failure
  • heart attacks
  • inflammatory lung disease

Monoclonal antibodies can cause mild to severe allergic reactions while you are receiving the drug. In rare cases, the reaction is severe enough to cause death.

Some monoclonal antibodies can also cause capillary leak syndrome. This syndrome causes fluid and proteins to leak out of tiny blood vessels and flow into surrounding tissues, resulting in dangerously low blood pressure. Capillary leak syndrome may lead to multiple organ failure and shock.

Cytokine release syndrome can sometimes occur with monoclonal antibodies, but it is often mild. Cytokines are immune substances that have many different functions in the body, and a sudden increase in their levels can cause:

  • fever
  • nausea
  • headache
  • rash
  • rapid heartbeat
  • low blood pressure
  • trouble breathing

How Monoclonal Antibodies Treat Cancer

Learn how monoclonal antibodies such as trastuzumab, pembrolizumab, and rituximab are used to treat cancer.

Targeted Therapy to Treat Cancer

Targeted Therapy to Treat Cancer

drawing of people in silhouette, a DNA strand, and a bottle of medication

Targeted therapy treats cancer by targeting proteins that control how cancer cells grow, divide, and spread.

Credit: National Cancer Institute

What is targeted therapy?

Targeted therapy is a type of cancer treatment that targets proteins that control how cancer cells grow, divide, and spread. It is the foundation of precision medicine. As researchers learn more about the DNA changes and proteins that drive cancer, they are better able to design treatments that target these proteins. 

What are the types of targeted therapy?

Most targeted therapies are either small-molecule drugs or monoclonal antibodies.

Small-molecule drugs are small enough to enter cells easily, so they are used for targets that are inside cells.

Monoclonal antibodies, also known as therapeutic antibodies, are proteins produced in the lab. These proteins are designed to attach to specific targets found on cancer cells. Some monoclonal antibodies mark cancer cells so that they will be better seen and destroyed by the immune system. Other monoclonal antibodies directly stop cancer cells from growing or cause them to self-destruct. Still others carry toxins to cancer cells. Learn more about monoclonal antibodies.

How Monoclonal Antibodies Treat Cancer

Learn how monoclonal antibodies such as trastuzumab, pembrolizumab, and rituximab are used to treat cancer.

Who is treated with targeted therapy?

For some types of cancer, such as chronic myelogenous leukemia (also known as CML), most people with that cancer will have a target for a certain drug, so they can be treated with that drug. But most of the time, your tumor will need to be tested to see if it contains targets for which there is a drug.

Testing your cancer for targets that could help choose your treatment is called biomarker testing. See Biomarker Testing for Cancer Treatment for more information.

You may need to have a biopsy for biomarker testing. A biopsy is a procedure in which your doctor removes a piece of the tumor for testing. There are some risks to having a biopsy. These risks vary depending on the size of the tumor and where it is located. Your doctor will explain the risks of having a biopsy for your type of tumor.

Look up your type of cancer on the list of targeted therapy drugs approved to treat specific cancers to learn more about drugs that may be an option for you.

How does targeted therapy work against cancer?

Most types of targeted therapy help treat cancer by interfering with specific proteins that help tumors grow and spread throughout the body. This is different from chemotherapy, which often kills all cells that grow and divide quickly. The following explains the different ways that targeted therapy treats cancer.

  • Help the immune system destroy cancer cells. One reason that cancer cells thrive is because they can hide from your immune system. Certain targeted therapies can mark cancer cells so it is easier for the immune system to find and destroy them. Other targeted therapies help boost your immune system to work better against cancer. Learn more about immunotherapy to treat cancer.
  • Stop cancer cells from growing by interrupting signals that cause them to grow and divide without order. Healthy cells in your body usually divide to make new cells only when they receive strong signals to do so. These signals bind to proteins on the cell surface, telling the cells to divide. This process helps new cells form only as your body needs them. But, some cancer cells have changes in the proteins on their surface that tell them to divide whether or not signals are present. Some targeted therapies interfere with these proteins, preventing them from telling the cells to divide. This process helps slow cancer’s uncontrolled growth.
  • Stop signals that help form blood vessels. To grow beyond a certain size, tumors need to form new blood vessels in a process called angiogenesis. The tumor sends signals that start angiogenesis. Some targeted therapies called angiogenesis inhibitors interfere with these signals to prevent a blood supply from forming. Without a blood supply, tumors stay small. Or, if a tumor already has a blood supply, these treatments can cause blood vessels to die, which causes the tumor to shrink. Learn more about angiogenesis inhibitors.

    Diagram illustrating the mechanism of Trastuzumab-deruxtecan targeting HER-2. The drug binds to the HER-2 receptor on the cell membrane. Inside the cell, it is transported to the lysosome, where the drug is released. This release leads to DNA damage and apoptotic cell death. Elements are labeled, including the drug, receptor, lysosome, and effects on DNA.

    Targeted Therapy Improves Survival for Metastatic “HER2-Low” Breast Cancer

    Enhertu expected to become new standard for treating metastatic breast cancer with low HER2 protein levels.

  • Deliver cell-killing substances to cancer cells. Some monoclonal antibodies are combined with cell-killing substances such as toxins, chemotherapy drugs, or radiation. Once these monoclonal antibodies attach to targets on the surface of cancer cells, the cells take up the cell-killing substances, causing them to die. Cells that don’t have the target will not be harmed.
  • Cause cancer cell death. Healthy cells die in an orderly manner when they become damaged or are no longer needed. But, cancer cells have ways of avoiding this dying process. Some targeted therapies can cause cancer cells to go through this process of cell death, which is called apoptosis.
  • Starve cancer of hormones it needs to grow. Some breast and prostate cancers require certain hormones to grow. Hormone therapies are a type of targeted therapy that can work in two ways. Some hormone therapies prevent your body from making specific hormones. Others prevent the hormones from acting on your cells, including cancer cells. Learn more about hormone therapy for prostate cancer and hormone therapy for breast cancer.

Are there drawbacks to targeted therapy?

Targeted therapy does have some drawbacks. 

  • Cancer cells can become resistant to targeted therapy. Resistance can happen when the target itself changes and the targeted therapy is not able to interact with it. Or it can happen when cancer cells find new ways to grow that do not depend on the target. Because of resistance, targeted therapy may work best when used with more than one type of targeted therapy or with other cancer treatments, such as chemotherapy and radiation.
  • Drugs for some targets are hard to develop. Reasons include the target’s structure, the target’s function in the cell, or both.

What are the side effects of targeted therapy?

When targeted therapy was first developed, scientists thought that it would be less toxic than chemotherapy. But they have learned that targeted therapy can also cause serious side effects. The side effects that you may have depends on the type of targeted therapy you receive and how your body reacts to it.

The most common side effects of targeted therapy include diarrhea and liver problems. Other side effects might include

  • problems with blood clotting and wound healing
  • high blood pressure
  • fatigue
  • mouth sores
  • nail changes
  • the loss of hair color
  • skin problems, which might include rash or dry skin

Very rarely, a hole might form through the wall of the esophagus, stomach, small intestine, large bowel, rectum, or gallbladder.

There are medicines for many of these side effects. These medicines may prevent the side effects from happening or treat them once they occur.

Most side effects of targeted therapy go away after treatment ends.

Learn more about side effects caused by cancer treatment and ways to manage them.

What can I expect when having targeted therapy?

How is targeted therapy given?

Small-molecule drugs are pills or capsules that you can swallow.

Monoclonal antibodies are usually given through a needle in a blood vein.

Where do I go for targeted therapy?

Where you go for treatment depends on which drugs you are getting and how they are given. You may take targeted therapy at home. Or you may receive targeted therapy in a doctor’s office, clinic, or outpatient unit in a hospital. Outpatient means you do not spend the night in the hospital.

How often will I receive targeted therapy?

How often and how long you receive targeted therapy depends on

  • your type of cancer and how advanced it is
  • the type of targeted therapy
  • how your body reacts to treatment

You may have treatment every day, every week, or every month. Some targeted therapies are given in cycles. A cycle is a period of treatment followed by a period of rest. The rest period gives your body a chance to recover and build new healthy cells.

How will targeted therapy affect me?

Targeted therapy affects people in different ways. How you feel depends on how healthy you are before treatment, your type of cancer, how advanced it is, the kind of targeted therapy you are getting, and the dose. Doctors and nurses cannot know for certain how you will feel during treatment.

How will I know whether targeted therapy is working?

While you are receiving targeted therapy, you will see your doctor often. He or she will give you physical exams and ask you how you feel. You will have medical tests, such as blood tests, x-rays, and different types of scans. These regular visits and tests will help the doctor know whether the treatment is working.

Where can I find out about clinical trials of targeted therapy?

Clinical trials of targeted therapy and other cancer treatments take place in cities and towns across the United States and throughout the world. They take place in doctors’ offices, cancer centers, medical centers, community hospitals and clinics, and veteran and military hospitals.

To find clinical trials of targeted therapy use this advanced search form. Under “Keywords/Phrases,” type “targeted therapy.” Under “Trial Type,” select the box for “Treatment” trials.

If you need help finding trials, contact the Cancer Information Service, NCI’s contact center.

Biomarker Testing for Cancer Treatment

Biomarker Testing for Cancer Treatment

Doctor and patient looking at DNA genomic sequence on iPad

Biomarker testing (also called tumor testing, tumor profiling, or tumor genetic testing) finds changes in your cancer that could help you and your doctor choose your cancer treatment.

Credit: Darryl Leja, NHGRI

What is biomarker testing for cancer treatment?

Biomarker testing is a way to look for genes, proteins, and other substances (called biomarkers or tumor markers) that can provide information about cancer. Each person’s cancer has a unique pattern of biomarkers. Some biomarkers affect how certain cancer treatments work. Biomarker testing may help you and your doctor choose a cancer treatment for you.

There are also other kinds of biomarkers that can help doctors diagnose and monitor cancer during and after treatment. To learn more, visit the Tumor Markers fact sheet.

Biomarker testing is for people who have cancer. People with solid tumors and people with blood cancer can get biomarker testing.

Biomarker testing for cancer treatment may also be called:

  • tumor testing 
  • tumor genetic testing 
  • genomic testing or genomic profiling
  • molecular testing or molecular profiling 
  • somatic testing 
  • tumor subtyping 

A biomarker test may be called a companion diagnostic test if it is paired with a specific treatment.

Biomarker testing is different from genetic testing that is used to find out if someone has inherited mutations that make them more likely to get cancer. Inherited mutations are those you are born with. They are passed on to you by your parents.

How are biomarker tests used to select cancer treatment?

Biomarker tests can help you and your doctor select a cancer treatment for you. Some cancer treatments, including targeted therapies and immunotherapies, may only work for people whose cancers have certain biomarkers.

Conceptual illustration of personal genomic data.

Patients Discuss Use of Genomic Data in Cancer Care

Workshop participants discussed data sharing, privacy, and other issues.

For example, people with cancer that has certain genetic changes in the EGFR gene can get treatments that target those changes, called EGFR inhibitors. In this case, biomarker testing can find out whether someone’s cancer has an EGFR gene change that can be treated with an EGFR inhibitor.

Biomarker testing could also help you find a study of a new cancer treatment (a clinical trial) that you may be able to join. Some studies enroll people based on the biomarkers in their cancer, instead of where in the body the cancer started growing. These are sometimes called basket trials. 

For some other clinical trials, biomarker testing is part of the study.  For example, studies like NCI-MATCH and NCI-COG Pediatric MATCH are using biomarker tests to match people to treatments based on the genetic changes in their cancers.

To find out if there are open trials for which you may be eligible, use the search tool at Find Clinical Trial. Or, contact the Cancer Information Service for help.

Is biomarker testing part of precision medicine?

Yes, biomarker testing is an important part of precision medicine, also called personalized medicine. Precision medicine is an approach to medical care in which disease prevention, diagnosis, and treatment are tailored to the genes, proteins, and other substances in your body.

For cancer treatment, precision medicine means using biomarker and other tests to select treatments that are most likely to help you, while at the same time sparing you from getting treatments that are not likely to help.

The idea of precision medicine isn’t new, but recent advances in science and technology have helped speed up the pace of this area of research. Scientists now understand that cancer cells can have many different changes in genes, proteins, and other substances that make the cells grow and spread. They have also learned that even two people with the same type of cancer may not have the same changes in their cancer. Some of these changes affect how certain cancer treatments work. 

Even though researchers are making progress every day, the precision medicine approach to cancer treatment is not yet part of routine care for most patients. But it’s important to note that even the “standard” approach to cancer treatment (selecting treatments based on the type of cancer you have, its size, and whether it has spread) is effective and is personalized to each patient. 

Should I get biomarker testing to select cancer treatment?

Talk with your health care provider to discuss whether biomarker testing for cancer treatment should be part of your care. Doctors usually suggest genomic biomarker testing (also called genomic profiling) for people with cancer that has spread or come back after treatment (what’s called advanced cancer). 

Biomarker testing is also done routinely to select treatment for people who are diagnosed with certain types of cancer—including non-small cell lung cancer, breast cancer, and colorectal cancer. 

It’s also a good idea to check with your health insurance provider to see if they will cover biomarker testing for your cancer. Biomarker testing is not available at every hospital. Check with your health care provider to see if biomarker testing is offered at the hospital or place where you get your cancer care.

How is biomarker testing done?

If you and your health care providers decide to make biomarker testing part of your care, they will take a sample of your cancer cells. If you have a solid tumor, they may take a sample during surgery. If you aren’t having surgery, you may need to have a biopsy of your tumor. 

If you have blood cancer or are getting a biomarker test known as a liquid biopsy, you will need to have a blood draw. You might get a liquid biopsy test if you can’t safely get a tumor biopsy, for example, because your tumor is hard to reach with a needle. 

Your samples will be sent to a special lab where they will be tested for certain biomarkers. The lab will create a report that lists the biomarkers in your cancer cells and if there are any treatments that might work for you. Your health care team will discuss the results with you to decide on a treatment. 

For some biomarker tests that analyze genes, you will also need to give a sample of your healthy cells. This is usually done by collecting your blood, saliva, or a small piece of your skin. These tests compare your cancer cells with your healthy cells to find genetic changes (called somatic mutations) that arose during your lifetime. Somatic mutations cause most cancers and can’t be passed on to family members.

Are there different types of biomarker tests?

Yes, there are many types of biomarker tests that can help select cancer treatment. Most biomarker tests used to select cancer treatment look for genetic markers. But some look for proteins or other kinds of markers.

Some tests check for a single biomarker. Others check for many biomarkers at the same time and may be called multigene tests or panel tests. One example is the Oncotype DX test, which looks at the activity of 21 different genes to predict whether chemotherapy is likely to work for someone with breast cancer. 

Some tests are for people with a certain type of cancer, like melanoma. Other tests look for biomarkers that are found in many cancer types, and such tests can be used by people with different kinds of cancer. 

Multi-colored model of DNA helix

FDA Approves Two Genomic Profiling Tests for Cancer

Tests can identify different cancer-associated genetic alterations.

Some tests, called whole-exome sequencing, look at all the genes in your cancer. Others, called whole-genome sequencing, look at all the DNA (both genes and outside of genes) in your cancer. 

Still other biomarker tests look at the number of genetic changes in your cancer (what’s known as tumor mutational burden). This information can help figure out if a type of immunotherapy known as immune checkpoint inhibitors may work for you.

Biomarker tests known as liquid biopsies look in blood or other fluids for biomarkers from cancer cells. There are two liquid biopsy tests approved by the Food and Drug Administration (FDA), called Guardant360 CDx and FoundationOne Liquid CDx. 

What do the results of a biomarker test mean?

The results of a biomarker test could show that your cancer has a certain biomarker that is targeted by a known therapy. That means that the therapy may work to treat your cancer. The matching therapy may be available as an FDA-approved treatment, an off-label treatment, or through participation in a clinical trial. 

The results could also show that your cancer has a biomarker that may prevent a certain therapy from working. This information could spare you from getting a treatment that won’t help you.

In many cases, biomarker testing may find changes in your cancer that won’t help your doctor make treatment decisions. For example, genetic changes that are thought to be harmless (benign) or whose effects are not known (variant of unknown significance) are not used to make treatment decisions. 

Based on your test results, your health care provider may recommend a treatment that is not FDA approved for your cancer type, but is approved for the treatment of a different type of cancer that has the same biomarker as your cancer. This means the treatment would be used off label, but it may work for you because your cancer has the biomarker that the treatment targets.

Some biomarker tests can find genetic changes that you may have been born with (inherited) that increase your risk of cancer or other diseases. These genetic changes are also called germline mutations. If such a change is found, you may need to get another genetic test to confirm whether you truly have an inherited mutation that increases cancer risk. 

Finding out that you have an inherited mutation that increases cancer risk may affect you and your family. For that reason, your health care provider may recommend that you speak with a genetic healthcare provider (such as a genetic counselor, clinical geneticist, or a certified genetic nurse) to help you understand what the test results mean for you and your family.

Will biomarker testing for cancer treatment help me?

Biomarker tests don’t help everyone who gets them. There are several different reasons why they may not help you. Biomarker testing may not help you if:

  • you are unable to safely get a biopsy needed for testing.
  • there is not enough tumor tissue in your biopsy sample to have biomarker testing done.
  • the test doesn’t find any biomarkers in your cancer that match with available therapies.
  • the test identifies a matching therapy that would be used off label, and your insurance doesn’t cover the cost.
  • the test identifies a matching therapy that is being tested in a clinical trial, and you are not able to participate in the trial.

Even if your test finds a biomarker that matches an available treatment, the therapy may not work for you. Sometimes other features of your cancer or your body affect how well a treatment works, such as how the medicine is broken down in your body. 

Another reason the treatment might not work is that not all of your cancer cells have the same biomarkers. That means that a biomarker test may find a treatment that will kill some, but not all, of your cancer cells. Cancer cells that are not killed by the treatment could keep growing, preventing the treatment from working or causing the cancer to quickly come back.

One other reason biomarker tests might not help is because the biomarkers in your cancer can change over time. But a test only captures a “snapshot” of the changes at one point in time. So, the results of a biomarker test done in the past may not reflect the biomarkers in your cancer now. Your health care provider may want to test your cancer again, for example, if it comes back after treatment. 

How much does biomarker testing for cancer treatment cost?

The cost of biomarker testing varies widely depending on the type of test you get, the type of cancer you have, and your insurance plan. 

For people with advanced cancer, some biomarker tests are covered by Medicare and Medicaid. Private insurance providers often cover the cost of a biomarker test if there is enough proof that the test is required to guide treatment decisions. Tests without enough proof to support their value may be considered experimental and are likely not covered by insurance.

Many clinical trials involve biomarker testing. If you join one of these clinical trials, the cost of biomarker testing might be covered. The study coordinator can give you more information about related costs.