Targeted Therapy

Targeted therapies generally treat cancer by interfering with specific molecules involved in cancer cells’ growth. Targeted therapies are typically considered cytostatic, meaning they stop or slow the growth and spread of cancer.1,2

Chemotherapy drugs attack cells that divide and grow quickly. They are typically cytotoxic, meaning they kill cells, including both cancer cells and some healthy cells in the body. When chemotherapy attacks normal cells, it can cause side effects.1,2

Targeted therapy addresses more specific features of cancer cells. It is a form of cancer treatment that attempts to do less damage to normal cells and cause fewer side effects.1,2

How does targeted therapy work?

Most kinds of targeted therapy drugs treat cancer by interfering with specific proteins that help cancer cells grow and spread in the body, or that are unique to cancer cells. Different drugs work in different ways, including:1,2

  • Stopping cancer cells from growing
  • Stopping signals that help form blood vessels
  • Delivering substances that kill cancer cells
  • Causing cancer cells to die

Epidermal Growth Factor Receptor (EGFR) Inhibitors

Roughly 20 percent of people with non-small cell lung cancer (NSCLC) in the United States have a tumor mutation linked to the epidermal growth factor receptor (EGFR) gene. This gene helps cells respond to their environment and can trigger cell growth, division, and survival.2,3

Changes in the EGFR gene (mutations) are most often found in tumors in people who have never smoked. People may be considered “never smokers” if they have smoked fewer than 100 cigarettes in their lifetime. EGFR mutations are also found in smokers and people with other tumor types. The tumors are usually adenocarcinomas (cancer that starts in the glands lining certain organs).2,3

Formulations of EGFR inhibitors

EGFR inhibitors for lung cancer include:2,3

Anaplastic Lymphoma Kinase (ALK) Inhibitors

Around 3 to 5 percent of lung cancer tumors have mutations (changes) on the anaplastic lymphoma kinase (ALK) gene. This gene plays an important role in cell growth, division, and maturation. ALK mutations are more common in light smokers and/or never smokers. These mutations are also linked to people with younger ages and adenocarcinomas.2-4

Formulations of ALK inhibitors

ALK inhibitors for lung cancer include:2-4

BRAF Inhibitors

A BRAF V600E mutation occurs in approximately 1 to 2 percent of people with NSCLC. This mutation is most often found in people who never smoked and women. The BRAF mutation can trigger an increase in the growth and spread of lung cancer cells.5

Formuations of BRAF inhibitors

Tafinlar® (dabrafenib) and Mekinist® (trametinib) are used together for certain people with BRAF mutated metastatic (has spread to other parts of the body) NSCLC.5

ROS1 Inhibitors

ROS1 positive lung cancer happens when there are changes to the ROS1 gene. This causes the ROS1 gene to fuel abnormal cell growth. Only approximately 1 to 2 percent of NSCLCs have a change in the ROS1 gene. They are commonly found in younger people, in light smokers and non-smokers, and in adenocarcinoma tumors.6-10

Formulations of ROS1 inhibitors

ROS1 inhibitors for lung cancer include:11

NTRK Inhibitors

NTRK positive lung cancer happens when a piece of the NTRK gene fuses to a nearby gene. This causes the NTRK gene to fuel uncontrolled cell growth and cancer. This kind of NSCLC is very rare, making up less than 1 percent of all cases. There is no one type of person who is more likely to have an NTRK fusion.9,12

Formulations of NTRK inhibitors

NTRK inhibitors for lung cancer include:11,13,14

MET Inhibitors

In certain NSCLCs, a cell may have a change in the MET gene. This change causes the cells to make an abnormal form of the MET protein, which helps the cells grow and spread.11

MET inhibitors attack the MET protein. MET inhibitors can be used to treat metastatic NSCLC, if the cancer cells have certain types of MET gene changes.11

Formulations of MET inhibitors

MET inhibitors for lung cancer include:11

  • Tabrecta® (capmatinib)
  • Tepmetko® (tepotinib)

RET Inhibitors

In a small number of NSCLCs, the cells have changes in the RET gene. These changes cause the cells to make an abnormal form of the RET protein, which helps cancer cells grow.11

RET inhibitors work by attacking the RET protein. They can be used to treat metastatic NSCLC if the cancer cells have certain types of RET gene changes.11

Formulations of RET inhibitors

RET inhibitors for lung cancer include:11

Monoclonal antibodies and angiogenesis inhibitors

Antibodies are proteins in the blood. They attach to antigens (such as bacteria or viruses) to mark them for destruction by the immune system. Monoclonal antibodies are man-made. This means they are created in a laboratory. They are designed to attach to specific antigens found on the surface of cancer cells.

These drugs work by targeting molecules that support the development of new blood vessels (angiogenesis). By blocking this process, these therapies can help reduce the blood supply to the tumors, resulting in slower growth.1,2

Formulations of angiogenesis inhibitors

Angiogenesis inhibitors for lung cancer include:1,2

  • Avastin® (bevacizumab)
  • Cyramza® (ramucirumab)

What are the possible side effects of targeted therapy?

Side effects can vary depending on the specific drug you are taking. Some common side effects of certain therapies include:2,11

  • Skin problems, including rashes, dry skin, and changes in the nails
  • Problems with blood clotting and wound healing
  • High blood pressure
  • Gastrointestinal perforation (a rare side effect that causes a hole in the intestines)

These are not all the possible side effects of targeted therapy. Talk to your doctor about what to expect or if you experience any changes that concern you during treatment with your specific targeted therapy regimen.

Things to know about targeted therapy

Certain side effects of targeted therapy have been linked to better outcomes or a better response to treatment. For example, some people develop a rash that looks like acne while being treated with Tarceva or Iressa. These people often have tumors that respond better to these drugs compared to those who do not get a rash.2

Another example is seen in people who take Avastin. People who develop high blood pressure while taking Avastin generally have tumors that respond better to treatment compared to those who do not develop high blood pressure.2

Before beginning treatment for lung cancer, tell your doctor about all your health conditions and any other drugs, vitamins, or supplements you are taking. This includes over-the-counter drugs.

Learn more about specific targeted therapies

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Written by Emily Downward & Karen Berger | Last reviewed: January 2022