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Targeted Therapy

What is targeted therapy?

Targeted therapies can be used to treat different kinds of diseases. Here we will focus on their use to treat cancer. Some of the cancers that may be treated with targeted therapy include certain types of lung, pancreatic, head and neck, liver, colorectal, breast, and kidney cancers.

Targeted therapy is a new type of cancer treatment that uses drugs or other substances to identify and attack cancer cells while doing little damage to normal cells. These therapies attack the cancer cells' inner workings -- the programming that makes them different from normal, healthy cells. Each type of targeted therapy works differently, but all alter the way a cancer cell grows, divides, repairs itself, or interacts with other cells.

Targeted therapies are a major focus of cancer research today. Many future advances against cancer will likely come from this field.

This document explains:

  • the different ways that targeted therapy can work
  • some of the types of targeted therapy
  • how targeted therapies may change cancer treatment in the future

How does targeted therapy work?

Targeted therapies are used to keep cancer from growing and spreading. Normal cells must go through a process to become cancer cells and to be able to grow into tumors. (This process is called carcinogenesis.) Scientists have learned a lot about the molecules that are part of this process and the signals a cell gets to keep this process going.

Targeted therapies disrupt this process. These drugs target certain parts of the cellular changes and signals that are needed for a cancer to develop and keep growing. These drugs are often grouped by how they work, or what part of the cell they target.

Enzyme inhibitors

When most people think of enzymes, the first ones that come to mind are the digestive enzymes that break down the food we eat. But there are many other kinds of enzymes in our bodies, and they play a key role in controlling nearly everything our cells do. Some targeted therapies block certain proteins, or enzymes, that are signals for cancer cells to grow. Blocking these signals can keep the cancer from getting bigger and spreading. So even if the tumor is not getting smaller, its out-of-control growth has been changed. This may give regular chemotherapy (chemo) a better chance to work. The change in out of control growth may also help people live longer, even without adding other drugs. These drugs may be called small-molecule drugs, signal-transduction inhibitors, or enzyme-inhibitors.

Apoptosis-inducing drugs

Some targeted therapies change proteins within the cancer cells and cause the cells to die. These are called apoptosis-inducing drugs. (Apoptosis is the medical word for cell death. These drugs cause, or induce cell death.) Many cancer treatments (including radiation and chemotherapy) cause cell changes that lead to apoptosis. But targeted drugs in this group are different, because they are aimed right at the parts of the cell that control cell survival and death.

Angiogenesis inhibitors

Cancer cells need to form and be fed by new, healthy blood vessels that come from nearby healthy, tissue. The process of forming these blood vessels is called angiogenesis. Angiogenesis inhibitors are targeted therapies that stop (or inhibit) this process. Angiogenesis inhibitors stop the blood supply to tumors so they can't grow.

Many of these drugs work by blocking vascular endothelial growth factor (VEGF). VEGF is a family of protein growth factors made by some tumors. These VEGF proteins can attach to the VEGF receptors of blood vessel cells. This causes new blood vessels to form around the tumors. Blocking this process prevents angiogenesis, which would form new blood vessels that would feed tumor growth.

How is targeted therapy used?

Targeted cancer therapies do not damage bone marrow or blood cells like most standard chemo drugs do. They can be used alone or along with other drugs. Many are used with other cancer treatments, such as radiation or chemo. (This is called combination therapy.) They often help to boost the effects of the main treatment. At this time, targeted therapies are most often used along with other chemo drugs.

Targeted therapy is still relatively new compared to other forms of cancer treatment, like surgery, radiation, or regular chemo. It is only within the last 10 years or so that it has proven useful as a form of cancer treatment. And even though they mainly target the cancer cells, these drugs are not perfect -- they can cause side effects and sometimes serious reactions.

Types of targeted therapy in use today

Today there are many different types targeted therapies used to treat cancer. Looking at examples helps to understand how these drugs work. A few of the more commonly used targeted therapies are listed here, but this is not a complete list. There are many different targeted therapies in use and new ones are coming out all the time.

Some examples of small molecule targeted therapies

Gleevec® (imatinib mesylate)

This is one of the first targeted therapies used to treat cancer. It is used to treat gastrointestinal stromal tumor (or GIST, a rare cancer of the gastrointestinal tract) and certain kinds of leukemia. Gleevec targets abnormal proteins, or enzymes, that form on and inside cancer cells and promote uncontrolled growth.

Iressa® (gefitinib)

Iressa is used to treat advanced non-small cell lung cancer. This drug targets the epidermal growth factor receptor (EGFR). This receptor is found on the surface of many normal cells, but on certain cancer cells there are many more copies. EGFR takes in the signal telling the cell to grow and divide. When Iressa blocks this signal, it can slow or stop cell growth.

Sutent® (sunitinib)

This enzyme-inhibitor is used to treat advanced kidney cancer and some gastrointestinal stromal tumors (or GIST, a rare cancer of the gastrointestinal tract). It is a type of VEGF receptor inhibitor that blocks an enzyme called tyrosine kinase. By doing this it keeps tumors from making their own blood vessels to help them grow and spread.

Velcade® (bortezomib)

This enzyme-inhibitor may be used to treat multiple myeloma that does not respond to other treatments. Velcade is a proteasome inhibitor. A proteasome is a complex of enzymes that helps destroy proteins that the cell no longer needs. Some of these proteins help to regulate cell function and growth. Velcade stops the proteasome from breaking down these proteins, which in turn causes the cancer cells to die

Other drug that may be called targeted therapies

There are other cancer treatments that are included in the group of drugs called targeted therapies. Some examples of these are:

  • Monoclonal antibodies, such as
  • Campath® (alemtuzumab)
  • Erbitux® (cetuximab)
  • Mylotarg® (gemtuzumab ozogamicin)
  • Rituxan® (rituximab)
  • Herceptin® (trastuzumab)
  • Avastin® (bevacizumab)
  • Cancer vaccines
  • Gene therapy
  • Cytokines, such as
  • Interleukins
  • Interferons
  • Granulocyte-macrophage colony-stimulating factor

The targeted therapies listed above are often grouped as immunotherapies, or treatments that work with your immune system to fight cancer. But some of them also act as angiogenesis inhibitors (bevacizumab) or enzyme inhibitors (cetuximab and trastuzumab). You can learn more about these treatments in our documents, Immunotherapy and Gene Therapy.

How targeted therapies may change cancer treatment in the future

Targeted cancer therapies are a huge part of cancer research today. They are hoped to be a better way to treat cancer. Some doctors think that in the future these treatments may be able to be custom-made for each patient based on the molecular targets made by the patient's tumor. Targeted cancer therapies are designed to kill cancer cells with little damage to normal, healthy cells. This means less side effects and better quality of life.

Most cells have had more than one step of the normal growth process altered as they've become cancer. Researchers are looking at ways to use different targeted therapies in combination to target more of these pathways. Some cancer cells become resistant to these therapies, too, and combinations of treatment may help prevent this. Researchers are also looking for better ways to use targeted therapy along with chemo and radiation to treat cancer.

Researchers are also looking at which cancers are best treated with targeted therapies and when treatment should start. Targeted therapies have helped many people with cancer, but this type of treatment only helps some of the people who receive it. Researchers are looking for ways to predict which patients will be helped the most, so that only the patients who will benefit will be treated. At this time, most targeted therapies are used to treat advanced cancers. Studies are also being done to figure out the best ways to manage the side effects of these treatments.

Researchers have made important progress in this field in recent years. Newer, more effective treatments are now being tested that will have a greater impact on the outlook for people with cancer in the future.

Additional resources

More information from your American Cancer Society

We have selected some related information that may also be helpful to you. You can find these on our Web site or order them from our toll-free number, 1-800-ACS-2345 (1-800-227-2345).

Along with the above, the American Cancer Society has information about many different types of cancer and how they are treated.

National organizations and Web sites*

Along with the American Cancer Society, other sources of information and support include:

National Cancer Institute
Toll-free number: 1-800-4-CANCER 1-800-422-6237
Web site: www.cancer.gov

*Inclusion on this list does not imply endorsement by the American Cancer Society.

No matter who you are, we can help. Contact us anytime, day or night, for cancer-related information and support. Call us at 1-800-ACS-2345 or visit www.cancer.org.

References

Kwak EL, Clark JW, Chabner B. Targeted Agents: The Rules of Combination. Clin Cancer Res. 2007;13:5232-5237.

Ma J, Waxman DJ. Combination of antiangiogenesis with chemotherapy for more effective cancer treatment. Molecular Cancer Therapeutics. 2008;7:3670-3684.

National Cancer Institute. Targeted Cancer Therapies: Questions and Answers. Accessed at www.cancer.gov/cancertopics/factsheet/Therapy/targeted on January 12, 2009.

Viele CS. Keys to Unlock Cancer: Targeted Therapy. Oncology Nursing Forum. 2005;32: 935-940.

Last Medical Review: 03/16/2009
Last Revised: 03/16/2009

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