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Targeted Therapy

What is Targeted Therapy?

Targeted therapy is designed to treat only the cancer cells and minimize damage to normal, healthy cells. Cancer treatments that “target” cancer cells may reduce treatment-related side effects and improve outcomes.

Targeted drugs often work by blocking cancer cells from copying themselves. This means they can help stop a cancer cell from dividing and making new cancer cells.

How Targeted Therapy Works

Targeted therapies find and attack specific areas or substances in cancer cells, or can detect and block certain kinds of messages sent inside a cancer cell that tell it to grow. Some of the substances in cancer cells that become the “targets” of targeted therapies are:

  • Too much of a certain protein on a cancer cell
  • A protein on a cancer cell that is not on normal cells
  • A protein that is mutated (changed) in some way on a cancer cell
  • Gene (DNA) changes that aren’t in a normal cell.

How is Targeted Therapy Different from Chemotherapy?

  • Targeted therapies act on specific molecular targets that are associated with cancer, whereas most standard chemotherapies act on all rapidly dividing normal and cancerous cells.
  • Targeted therapies are deliberately chosen or designed to interact with their target, whereas many standard chemotherapies were identified because they kill cells. 
  • Targeted therapies are often cytostatic (that is, they block tumor cell proliferation), whereas standard chemotherapy agents are cytotoxic (that is, they kill tumor cells). 

What Types of Targeted Therapies are Available?

Many different targeted therapies have been approved for use in cancer treatment. These therapies include hormone therapies, signal transduction inhibitors, gene expression modulators, apoptosis inducers, angiogenesis inhibitors, immunotherapies, and toxin delivery molecules.

  • Hormone therapies slow or stop the growth of hormone-sensitive tumors, which require certain hormones to grow. Hormone therapies act by preventing the body from producing the hormones or by interfering with the action of the hormones. Hormone therapies have been approved for both breast cancer and prostate cancer.
  • Signal transduction inhibitors block the activities of molecules that participate in signal transduction, the process by which a cell responds to signals from its environment. During this process, once a cell has received a specific signal, the signal is relayed within the cell through a series of biochemical reactions that ultimately produce the appropriate response(s). In some cancers, the malignant cells are stimulated to divide continuously without being prompted to do so by external growth factors. Signal transduction inhibitors interfere with this inappropriate signaling.
  • Gene expression modulators modify the function of proteins that play a role in controlling gene expression.
  • Apoptosis inducers cause cancer cells to undergo a process of controlled cell death called apoptosis. Apoptosis is one method the body uses to get rid of unneeded or abnormal cells, but cancer cells have strategies to avoid apoptosis. Apoptosis inducers can get around these strategies to cause the death of cancer cells.
  • Angiogenesis inhibitors block the growth of new blood vessels to tumors (a process called tumor angiogenesis). A blood supply is necessary for tumors to grow beyond a certain size because blood provides the oxygen and nutrients that tumors need for continued growth. Treatments that interfere with angiogenesis may block tumor growth. Some targeted therapies that inhibit angiogenesis interfere with the action of vascular endothelial growth factor (VEGF), a substance that stimulates new blood vessel formation. Other angiogenesis inhibitors target other molecules that stimulate new blood vessel growth.
  • Immunotherapies trigger the immune system to destroy cancer cells. Some immunotherapies are monoclonal antibodies that recognize specific molecules on the surface of cancer cells. Binding of the monoclonal antibody to the target molecule results in the immune destruction of cells that express that target molecule. Other monoclonal antibodies bind to certain immune cells to help these cells better kill cancer cells.
  • Monoclonal antibodies that deliver toxic molecules can cause the death of cancer cells specifically. Once the antibody has bound to its target cell, the toxic molecule that is linked to the antibody—such as a radioactive substance or a poisonous chemical—is taken up by the cell, ultimately killing that cell. The toxin will not affect cells that lack the target for the antibody—i.e., the vast majority of cells in the body.

Cancer vaccines and gene therapy are sometimes considered targeted therapies because they interfere with the growth of specific cancer cells.

What is Treated with Targeted Therapy?

The FDA has approved targeted therapies for the treatment of some patients with the following types of cancer (some targeted therapies have been approved to treat more than one type of cancer):

  • Bladder Cancer
  • Brain Cancer
  • Breast Cancer
  • Cervical Cancer
  • Colorectal Cancer
  • Head & Neck Cancer
  • Leukemia
  • Liver & Bile Duct Cancer
  • Lung Cancer
  • Kidney Cancer
  • GI Cancer
  • Multiple Myeloma
  • Malignant mesothelioma
  • Myelodysplastic/myeloproliferative
  • Neuroblastoma
  • Ovarian Cancer
  • Pancreatic Cancer
  • Plexiform neurofibroma
  • Prostate Cancer
  • Skin Cancer
  • Soft Tissue Sarcoma
  • Stomach Cancer
  • Systemic mastocytosis
  • Thyroid Cancer