Targeted Therapy

Updated 
August 31, 2015
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Targeted therapies are treatments that target specific proteins or markers on or within cancer cells that help the cells to grow. Targeted therapies may be used alone or with other targeted therapies, hormonal therapies or chemotherapy medicines. Your doctor may also call these treatments tailored or biologicinfo-icon therapies.

Targeted therapies look for a specific feature of a cancer cellinfo-icon, attach to it and destroy it. Only cancers with that feature will respond to the therapyinfo-icon. Some examples are:

  • If the breast cancer tests positive for the HER2 receptorinfo-icon, you may be able to receive a targeted therapyinfo-icon that only targets HER2-positive cells
  • Medicines that block the growth of blood vessels that most tumors need to grow
  • Therapies that work like antibodies made by the immune systeminfo-icon

Unlike chemotherapyinfo-icon, targeted therapies only kill cancer cells, leaving healthy cells alone. For this reason, targeted therapies by themselves have far fewer side effects than chemotherapy medicines. But each targeted therapy has unique side effects that you should discuss with your doctor.  

Targeted therapies do not target the hormoneinfo-icon receptors estrogeninfo-icon and progesteroneinfo-icon. Instead, they target other parts of the cancer cell. Hormonal therapies, though, are a form of targeted therapy given for hormone receptor-positive breast cancer. Some targeted therapies can be used along with hormonal therapyinfo-icon to make hormonal therapy more effective at killing the cancer.

There are many kinds of targeted therapy, and researchers will likely find others as they learn more about what makes certain cancers grow. Targeted treatments are helping scientists and doctors personalize treatment plans so they can better treat different types of cancer.

Many forms of targeted therapy are being tested in clinical trials. They are given intravenously (by veininfo-icon) or by mouth as a pill. Some treatments have already been approved by the FDAinfo-icon, but others are only available in clinicalinfo-icon trials.

You will need tests to know whether the cancer will respond to targeted therapy. Sometimes, you may be offered a targeted therapy only in specific situations. You may be offered targeted therapy if you have

If you are interested in other targeted therapies, ask your doctor about a clinical trialinfo-icon.

Neoadjuvant vs. Adjuvant Targeted Therapy

Targeted therapyinfo-icon for breast cancer can be given either:

Neoadjuvant TherapyNeoadjuvant therapy is full-body, or systemicinfo-icon, treatment given as a first step to shrink tumors or slow cancer spread before surgery.

Neoadjuvant therapy is more commonly used for breast cancer that is locally advanced, or has traveled outside the breast to other nearby organs or tissues such as lymphinfo-icon nodes, skin or chest wallinfo-icon.

If you can have neoadjuvant therapy, you will have a needle biopsy to learn more about the type of breast cancer you have and decide which therapyinfo-icon will benefit you most.

You may be able to get neoadjuvant therapy if you have a HER2-positive tumorinfo-icon that is too large to remove with lumpectomy. The goal of neoadjuvant therapy would be to shrink the tumor to a size that could allow you to have lumpectomyinfo-icon instead of mastectomyinfo-icon.

Neoadjuvant therapy may change the timing of your treatment or allow you to avoid mastectomy, but studies show it does not lengthen life, or survival, over receiving therapy after surgery.

Targeted medicines that may be used as neoadjuvant therapy for HER2-positive breast cancer are

After neoadjuvant targeted therapy, you may have tests, such as a mammogram or MRI, to see how the cancer responded to therapy. Then, surgery will be scheduled.

After your surgery, a pathologistinfo-icon will check the tissueinfo-icon that was removed for any signs of cancer. In some cases, there may be a complete responseinfo-icon to therapy, meaning the doctor can find no evidence of remaining disease, or NED. When cancer completely goes away, it is linked to better overall outcomes. But you will still need to finish your treatment plan to lessen your risk for cancer return.

Adjuvant TherapyAdjuvant therapy is treatment given after surgery to lower the risk of the breast cancer coming back and increase the chance of long-term survival. The goal is to kill any cancer cells that may have traveled away from the breast or lymph nodes. If you are at higher risk of recurrenceinfo-icon, you are more likely to need adjuvant therapy.

The targeted therapy trastuzumab is given with chemotherapy as a standard adjuvant treatment for early-stageinfo-icon, HER2-positive breast cancer. Trastuzumab lowers the risk for distant recurrenceinfo-icon.

A number of other targeted therapy medicines are approved only for metastatic breast cancer. These are 

Bevacizumab (Avastin) and PARPinfo-icon inhibitors such as BSI-201, olaparibinfo-icon and ABT-888 are not FDAinfo-icon-approved but may be available to you through clinicalinfo-icon trials if you have stage IV breast cancerinfo-icon.

Many forms of neoadjuvant and adjuvant targeted therapy are being tested in clinical trials. Talk with your doctors about open clinical trials that may be available to you. You can also visit our section on Clinical Trials and Research Studies for more information.

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