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Targeted Therapy

Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific types of cancer cells with less harm to normal cells. Not all cancers have the same targets. Each type of targeted therapy works a little bit differently, but they all interfere with the growth and survival of cancer cells. To find the most effective treatment, your doctor may run tests to identify the genes, proteins and other factors in your cancer cells. This helps the doctor choose the most effective treatment for you based on the specific factors of your disease. Targeted therapy may be used alone or in combination with chemotherapy. Some types of targeted therapy include: 

Monoclonal Antibodies

Monoclonal antibodies are proteins that are made in the laboratory. They can bind to substances in the body, including cancer cells. Most are designed to attach to one specific substance. These drugs can be used alone to destroy cancer cells or to carry drugs, toxins or radioactive substances directly to the cancer cells. These drugs include

  • Blinatumomab (Blincyto®)
  • Inotuzumab ozogamicin (Besponsa®)

Chimeric Antigen Receptor (CAR) T-Cell Therapy

CAR T-cell therapy is a type of immunotherapy in which a patient's T cells (a type of white blood cell) are changed in the laboratory so that they will attack cancer cells.

  • Tisagenlecleucel (Kymriah®) is an FDA-approved for the treatment of patients up to age 25 years who have B-cell ALL that is refractory or in second or later relapse. This treatment is designed to help the body’s own immune system fight cancer. Each dose is made for a specific patient, using the patient’s own T cells (white blood cells that help the body fight infections and cancer). The T cells are collected from the patient and then genetically modified to add a new gene containing a CAR protein, so that the T cells can identify and kill leukemia cells with CD19 on their surface. These modified cells are infused back into the patient’s bloodstream to kill the cancer cells.

While CAR T-cell therapy can be an effective treatment, it is also associated with a relatively high rate of serious complications. As a result, it can only be given at specialized centers that have expertise in delivering this type of treatment.

Tyrosine Kinase Inhibitors (TKIs)

Tyrosine kinases are enzymes that are a part of many cell functions including cell signaling, growth and division. These enzymes may become too active in patients with an ALL subtype called Philadelphia chromosome-positive ALL (called Ph+ ALL)


For information about the drugs listed on this page, visit Drug Listings.


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