What is targeted therapy?

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

Who is treated with targeted therapy?

Different types of cancer are treated differently. Some cancers are mostly treated with targeted therapy, while others are treated with mostly conventional chemotherapy.   In many cases, your tumor will need to be tested to see if it contains targets for which we have drugs.  Your doctor would like to tailor your cancer treatment to your particular kind of cancer and your particular targets, to get the best possible outcome for you and your family.  This is called individualized treatment, and it’s one of our guiding principles of practice here at Regatta Health.

To have your tumor tested for targets, you may need to have a biopsy. 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.

Testing for targets for targeted therapy usually involves sending a biopsy sample to a lab.  Patients often ask if they have to go somewhere or have additional procedures to do targeted therapy testing, but often doctors are able to test the biopsy already taken to establish the cancer diagnosis.  This type of testing occurs “behind the scenes” often in labs that are in different parts of the country from the patient.  Examples of targeted therapy testing labs include Neogenomics, Quest Laboratories,

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. They treat cancer in many ways. They can:

  • 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.
  • Stop cancer cells from growing. 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. Tumors need to form new blood vessels to grow beyond a certain size. In a process called angiogenesis, these new blood vessels form in response to signals from the tumor. Some targeted therapies called angiogenesis inhibitors are designed to 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.
  • Deliver cell-killing substances to cancer cells. Some monoclonal antibodies are combined with toxins, chemotherapy drugs, and 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.
  • Starve cancer of the 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.
Are there drawbacks to targeted therapy?

Targeted therapy does have some drawbacks. These include:

  • Cancer cells can become resistant to targeted therapy. For this reason, they may work best when used with other types 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?

Targeted therapy can cause side effects. The side effects you may have depend on the type of targeted therapy you receive and how your body reacts to the therapy.

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, and skin problems. Skin problems 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 are other risks of targeted therapy?

Since your tumor may be tested to find targets for treatment, there may be risks to the privacy of your personal information. The privacy of all information found from these tests is protected by law. But, there is a slight risk that genetic or other information from your health records may be obtained by people outside of the medical team.

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 your doctor’s office.

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.

(Excerpted from https://cancer.gov 4/19/21)