Immunotherapy for Childhood Leukemia

Immunotherapy is the use of medicines to help a patient’s own immune system recognize and destroy cancer cells more effectively. Several types of immunotherapy are being studied for use against childhood leukemia, and some are now coming into use.

Chimeric antigen receptor (CAR) T-cell therapy

For this treatment, immune cells called T cells are removed from the child’s blood and genetically altered in the lab to have specific receptors (called chimeric antigen receptors, or CARs) on their surface. These receptors can attach to proteins on leukemia cells. The T cells are then multiplied in the lab and given back into the child’s blood, where they can seek out the leukemia cells and attack them.

Tisangenlecleucel (Kymriah) is a type of CAR T-cell therapy that targets the CD19 protein on certain leukemia cells. It can be used to treat childhood acute lymphoblastic leukemia (ALL) that has come back after treatment or that is no longer responding to treatment.

To make this treatment, T cells are removed from the child’s blood during a process called leukapheresis. Blood is removed through an IV line and goes into a machine that removes the T cells. The remaining blood then goes back into the body. This typically takes a few hours, and it might need to be repeated. The cells are then frozen and sent to a lab, where they are turned into CAR T cells and are multiplied. This process can take a few weeks.

For the treatment itself, the child typically gets chemotherapy for a few days to help prepare the body. Then they get the CAR T cells as an infusion into a vein. Because this treatment can have serious side effects (see below), it is only given in medical centers that have special training with this treatment.

In most children who have had this treatment, the leukemia could no longer be detected within a few months of treatment, although it’s not yet clear if this means that they have been cured.

Possible side effects

This treatment can have serious or even life-threatening side effects, which is why it needs to be given in a medical center that has special training in its use.

Cytokine release syndrome (CRS): CRS happens when T cells release chemicals (cytokines) that ramp up the immune system. This can happen within a few days to weeks after treatment, and can be life-threatening. Symptoms can include:

  • High fever and chills
  • Trouble breathing
  • Severe nausea, vomiting, and/or diarrhea
  • Severe muscle or joint pain
  • Feeling dizzy or lightheaded

Nervous system problems: This drug can have serious effects the nervous system, which can result in symptoms such as:

  • Headaches
  • Changes in consciousness
  • Confusion or agitation
  • Seizures
  • Trouble speaking and understanding
  • Loss of balance

Other serious side effects: Other possible side effects can include:

  • Serious infections
  • Low blood cell counts, which can increase the risk of infections, fatigue, and bruising or bleeding

It’s very important to report any side effects to the health care team right away, as there are often medicines that can help treat them.

Other types of immunotherapy are also being studied for use against leukemia (some of which are described in What’s New in Childhood Leukemia Research and Treatment?).

The American Cancer Society medical and editorial content team
Our team is made up of doctors and master's-prepared nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Last Medical Review: September 8, 2017 Last Revised: September 8, 2017

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