Chemotherapy and Supportive Care Medicines for Brain Tumors in Children

Chemotherapy (chemo) uses anti-cancer drugs that are usually given into a vein (IV) or taken by mouth. These drugs enter the bloodstream and reach almost all areas of the body.

These drugs can also be given directly into the cerebrospinal fluid (CSF) to treat brain tumors, through a lumbar puncture or a ventricular access device, such as an Ommaya reservoir.

Other types of medicines can be used to help prevent or relieve symptoms from brain or spinal cord tumors. These supportive care medicines are described below as well.

When might chemotherapy be used?

In general, chemo is used for faster growing tumors. Some types of brain tumors, such as medulloblastomas, tend to respond well to chemo.

Chemo is most often used along with other types of treatment, such as surgery and radiation therapy. It may be used instead of radiation therapy in children 3 years and younger.

Chemo drugs used to treat brain and spinal cord tumors in children

Some of the chemo drugs used to treat children with brain or spinal cord tumors include:

  • Carboplatin
  • Carmustine (BCNU)
  • Cisplatin
  • Cyclophosphamide
  • Etoposide
  • Irinotecan
  • Lomustine (CCNU)
  • Methotrexate
  • Procarbazine
  • Temozolomide
  • Thioguanine
  • Topotecan
  • Vinblastine
  • Vincristine

These drugs may be used alone or in combination, depending on the type of brain tumor.

How is chemo given for brain tumors?

Chemo is given in cycles, with each period of treatment followed by a rest period to give the body time to recover. Each cycle typically lasts a few weeks.

Because the brain has a protective barrier (the blood–brain barrier), some drugs cannot cross from the blood stream into the cerebrospinal fluid (CSF), the fluid that bathes the brain and spinal cord. For some chemo drugs that treat brain tumors but cannot reach high levels in the brain on their own, the drugs can be given directly into the CSF through a lumbar puncture or a ventricular access catheter such as an Ommaya reservoir. (See Surgery for Brain and Spinal Cord Tumors in Children.)

Possible side effects of chemotherapy

Chemo drugs can cause side effects. These depend on the type and dose of drugs, and how long treatment lasts.

Short-term side effects

Possible short-term side effects can include:

  • Hair loss
  • Mouth sores
  • Loss of appetite
  • Nausea and vomiting
  • Diarrhea
  • Increased chance of infections (from having too few white blood cells)
  • Easy bruising or bleeding (from having too few blood platelets)
  • Fatigue (from having too few red blood cells or other factors)

Most of these side effects go away once treatment is finished. Your cancer care team will watch closely for any problems. There are often ways to lessen these side effects during treatment. For example, drugs can be given to help prevent or reduce nausea and vomiting.

Be sure to ask your team about medicines to help reduce side effects and let them know if your child or teen is having symptoms so they can be managed. In some cases, the doses of the chemo drugs may need to be reduced, or treatment may need to be delayed or stopped to prevent the effects from getting worse.

Long-term side effects after treatment

Some chemo drugs can also have other, less common long-term side effects. Long term side effects of chemotherapy depend on the treatment given and, in some cases, the dose. These include:

Hearing loss: People treated with platinum chemotherapy, such as cisplatin and carboplatin, are at increased risk of developing hearing problems. A test called an audiogram will likely be done during and after treatment to check for any damage from chemo. Hearing is very important for young children’s speech and social development. If hearing has been affected by chemotherapy, your cancer care team may recommend seeing a hearing specialist, called an audiologist, to discuss hearing aids.

Kidney problems: Chemo drugs like cisplatin can affect the kidneys. Regular kidney function testing may be recommended by your child’s cancer care team to find and manage any kidney problems after treatment.

Fertility and pubertal development: Alkylating chemo drugs (cyclophosphamide, CCNU, BCNU, dacarbazine) and platinum chemo drugs (cisplatin, carboplatin) for brain tumors can affect organs such as the ovaries and testicles. Because these may be impacted by treatment, close attention to how younger children grow, develop, and progress through puberty will be an important part of their care after treatment is done. For more information, see Preserving Fertility in Children and Teens with Cancer.

Second cancers: After getting certain chemotherapy drugs, such as alkylating agents (cyclophosphamide, thiotepa), people are at increased risk of developing leukemia, most commonly acute myeloid leukemia (AML). This is rare, but it can occur.

Learning problems: Treatment that includes some types of chemotherapy (such as methotrexate) may affect learning ability in some people. Children and teens treated for brain tumors may need extra support in school.

Nerve problems: Some children can develop neuropathy, or damage to the nerves that control how the body moves and feels, after treatment with drugs such as vincristine, vinblastine, or platinum chemotherapy drugs (cisplatin, carboplatin). Symptoms of neuropathy, such as numbness, tingling, or tripping while walking often go away or get better once treatment is done. For some children, these symptoms may be lifelong.

Talk to your cancer care team about which specific long-term side effects you may need to watch for based on the treatment received.

More information about chemotherapy

For more general information about how chemotherapy is used to treat cancer, see Chemotherapy.

To learn about some of the side effects listed here and how to manage them, see Managing Cancer-related Side Effects.


Supportive care drugs for children with brain tumors

Children and young adults with brain or spinal cord tumors can often be given drugs to help with symptoms from the tumor or side effects of treatment. These drugs do not treat the tumor directly, but they can be an important part of your child's treatment plan.

Corticosteroids

Corticosteroid drugs, such as dexamethasone (Decadron), are often given to reduce swelling around brain tumors. This may help relieve headaches and other symptoms.

Steroids can also cause side effects, such as weight gain, mood changes, and high blood sugar, so they are used only for short periods of time, when possible.

Anti-seizure drugs (anticonvulsants)

Drugs may be given to lower the chance of seizures in people with brain tumors or to treat them if they happen. Many different anti-seizure drugs can be used.

Hormones

The pituitary gland, which lies at the base of the brain, helps control the levels of many different hormones in the body. If the pituitary gland has been damaged by the spread of the tumor or by treatment (surgery or radiation therapy), the hormones that the pituitary usually makes may need to be replaced with medication.

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Developed by the American Cancer Society medical and editorial content team with medical review and contribution by the American Society of Clinical Oncology (ASCO).

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Last Revised: February 9, 2026

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