Targeted therapy for childhood leukemia

Targeted therapy is sometimes used to treat childhood leukemia. It uses drugs to target specific molecules (such as proteins) on cancer cells or inside them. These molecules help send signals that tell cells to grow or divide. By targeting these molecules, the drugs stop the growth and spread of cancer cells and limit harm to normal cells. Targeted therapy may also be called molecular targeted therapy.

The healthcare team will consider your child’s personal needs to plan the drugs, doses and schedules of targeted therapy. Your child may also receive other treatments.

Targeted therapy drugs used for childhood leukemia

There are a few types of targeted therapy drugs used to treat childhood leukemia.

Tyrosine kinase inhibitors may be used to treat childhood leukemia that has the gene mutation BCR-ABL. This is also known as Philadelphia chromosome–positive leukemia. These drugs are taken by mouth (orally) and include:

  • imatinib (Gleevec)
  • dasatinib (Sprycel)
  • nilotinib (Tasigna)
  • ponatinib (Iclusig)

Monoclonal antibodies may be used to treat some children with acute myelogenous leukemia (AML) that has come back after treatment or is no longer responding to treatment.

These drugs are given through a needle in a vein (intravenously) and include:

  • gemtuzumab ozogamicin
  • inotuzumab ozogamicin (Besponsa)

Another targeted therapy drug that may be used to treat acute lymphoblastic leukemia (ALL) that has come back after treatment is blinatumomab (Blincyto).

Side effects

Side effects can happen with any type of treatment for childhood leukemia, but every child’s experience is different. Some children have many side effects. Other children have few or none at all.

Targeted therapy attacks cancer cells but doesn’t usually damage healthy cells, so there are usually fewer and less severe side effects than with chemotherapy or radiation therapy. Chemotherapy and radiation therapy can damage healthy cells along with cancer cells.

Side effects can happen any time during, immediately after or a few days or weeks after targeted therapy. Sometimes late side effects develop months or years after targeted therapy. Most side effects go away on their own or can be treated, but some side effects may last a long time or become permanent.

Side effects of targeted therapy will depend mainly on the type of drug or combination of drugs, the dose, how it’s given and your child’s overall health. Some common side effects of targeted therapy for childhood leukemia are:

Other side effects can develop months or years after treatment for childhood leukemia. Find out more about late effects for childhood leukemia.

Tell your child’s healthcare team if your child has these side effects or others you think might be from targeted therapy. The sooner you tell them of any problems, the sooner they can suggest ways to help your child deal with them.

Information about specific cancer drugs

Details on specific drugs change regularly. Find out more about sources of drug information and where to get details on specific drugs.

Questions to ask about targeted therapy

Find out more about targeted therapy. To make the decisions that are right for your child, ask the healthcare team questions about targeted therapy.

Expert review and references

  • American Cancer Society. Treating Childhood Leukemia. 2016:
  • National Cancer Institute. Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®) Patient Version. 2018.

Medical disclaimer

The information that the Canadian Cancer Society provides does not replace your relationship with your doctor. The information is for your general use, so be sure to talk to a qualified healthcare professional before making medical decisions or if you have questions about your health.

We do our best to make sure that the information we provide is accurate and reliable but cannot guarantee that it is error-free or complete.

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