Treating Leukemia in Children

Leukemia is the most common type of cancer in children, and treatment is based on the type of leukemia the child has. Examples of types of leukemia that occur in children include:

Types of treatment for childhood leukemia

There are several different types of treatment that may be used to treat leukemia in children, including:

  • Chemotherapy
  • Stem cell transplant
  • Targeted therapy
  • Radiation therapy
  • Immunotherapy2,3


Chemotherapy is the use of drugs to stop cancer cells and is often the main type of treatment for leukemia in children.1,2 Chemotherapy medications may be used in combination, and it works by targeting fast-growing cells, such as cancer cells. However, there are other fast-growing cells in the body that can also be affected, such as those in the gastrointestinal tract and hair.2,3

The way chemotherapy medications are delivered depends on the type of leukemia and how it is affecting the body. Some medications are given by mouth or injected into a vein or muscle. When leukemia is affecting the brain or spinal cord, chemotherapy may be injected into the space around the spinal cord and brain (called intrathecal chemotherapy, as shown below).2,3

Figure 1. Intrathecal chemotherapy

Human body showing central nervous system with needle injecting chemotherapy into space around the spinal column and brain

Stem cell transplants

Stem cell transplants are used in combination with high doses of chemotherapy. The high dose of chemotherapy destroys the cancer cells and also damages healthy blood cells. The transplant of stem cells, immature cells that can become new blood cells, is given to restore the bone marrow. Allogeneic stem cell transplants are typically the type of stem cell transplant utilized in childhood leukemia treatment. In allogeneic transplants, the stem cells are collected from a donor. Because the donor's tissue type should match the tissue type of the patient to the closest extent possible, siblings or close relatives are often donors. However, the donor does not necessarily have to be related to the patient.3,4

Targeted therapy

Targeted therapies are cancer treatments that block or slow the spread of cancer by interfering with specific areas of cancerous cells that are involved in the cancer cell's growth, or by focusing on particular characteristics that are unique to cancer cells. While chemotherapy drugs are often cytotoxic, meaning they kill cancer cells, targeted therapy is typically cytostatic, meaning it blocks the growth of cancer cells. The types of targeted therapy that may be used to treat childhood leukemias include:

  • Tyrosine kinase inhibitors (TKIs), which target a specific protein that is found on certain cancer cells
  • Monoclonal antibodies, which identify and block cancer growth or kill cancer cells, or may be used to deliver chemotherapy medications to cancer cells
  • Proteasome inhibitors, which break down proteins inside cancer cells2,3

Radiation therapy

Radiation therapy uses high-energy radiation, and it may be used in cases of childhood leukemia that have spread to the brain, cerebrospinal fluid (CSF), or testicles. Radiation may also be used in preparation for a stem cell transplant.3


Immunotherapy, also called biologic therapy, helps boost the patient's own immune system to fight the cancer. One type of immunotherapy that is being used in some cases of childhood leukemia is Chimeric antigen receptor (CAR) T-cell therapy. In CAR T-cell therapy, the child's own T-cells are removed from the body and altered in a laboratory to specifically target the cancer cells, then administered back into the patient's bloodstream to fight cancer cells.5

Clinical trials

Clinical trials are a type of research where new treatments are studied. Clinical trials are an important part of the scientific process to find and prove the safety and effectiveness of new treatments, and they offer patients a chance to receive the latest treatments and be closely monitored by healthcare professionals. Clinical trials can be found by talking to a doctor or through the website Patients can discuss treatment options with their doctor to determine if they might be eligible to participate in a clinical trial.2,6

By providing your email address, you are agreeing to our privacy policy. We never sell or share your email address.

Written by: Emily Downward | Last reviewed: October 2020