Targeted Therapy for Childhood Leukemia

In recent years, new drugs that target specific parts of cancer cells have been developed. These targeted drugs work differently from standard chemotherapy drugs. They can be used instead of or along with chemo in some situations, and they have side effects that are different (and often less severe) than chemo. Some targeted drugs can be useful in certain childhood leukemias.

BCR-ABL inhibitors for CML (and some cases of ALL)

Nearly all children with chronic myeloid leukemia (CML) have an abnormal chromosome in their leukemia cells known as the Philadelphia chromosome. These chromosomes have a specific gene mutation known as BCR-ABL, which helps the leukemia cells grow.

Targeted drugs such as imatinib (Gleevec), dasatinib (Sprycel), and nilotinib (Tasigna) specifically attack cells that have this gene mutation. These drugs are very effective at controlling the leukemia for long periods of time in most children, although it’s not yet clear if the drugs can help cure CML.

A small number of children with acute lymphocytic leukemia (ALL) also have the Philadelphia chromosome in their leukemia cells. Studies have shown that their outcome is improved when these drugs are given along with chemotherapy drugs.

These drugs are taken daily as pills. Possible side effects include diarrhea, nausea, muscle pain, fatigue, and skin rashes. These are generally mild. A common side effect is swelling around the eyes or in the hands or feet. Some studies suggest this fluid buildup may be caused by the drugs’ effects on the heart. Other possible side effects include lower red blood cell and platelet counts at the start of treatment. These drugs might also slow a child’s growth, especially if used before puberty.

Gemtuzumab ozogamicin (Mylotarg) for AML

This is a targeted therapy that consists of a monoclonal antibody (a manmade immune protein) linked to a chemotherapy drug. The antibody acts like a homing signal, bringing the chemo drug to the leukemia cells, where it enters the cells and kills them when they try to divide into new cells.

This drug can be used to treat some children with acute myeloid leukemia (AML) that has come back after treatment or is no longer responding to treatment. It is given as an infusion into a vein (IV), typically for 3 doses, with 2 days in between each dose.

The most common side effects are fever, nausea and vomiting, low levels of blood cells (with increased risks of infection, bleeding, and fatigue), swelling and sores in the mouth, constipation, rash, and headaches.Less common but more serious side effects can include:

  • Severe liver damage, including veno-occlusive disease (blockage of veins in the liver)
  • Reactions during the infusion (similar to an allergic reaction). Your child will likely be given medicines before each infusion to help prevent this.
  • Serious or life-threatening infections, especially in people who have already had a stem cell transplant
  • Changes in the rhythm of the heart

Other targeted drugs are now being tested in clinical trials as well.

For more general information on targeted drugs, see Targeted Therapy.

The American Cancer Society medical and editorial content team

Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Last Medical Review: April 17, 2015 Last Revised: March 23, 2018

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