ON THIS PAGE: You will learn about the different types of treatments doctors use for children with a central nervous system (CNS) tumor. Use the menu to see other pages.
In general, tumors in children are uncommon. This means it can be hard for doctors to plan treatments unless they know what has been most effective in other children. That is why more than 60% of children are treated as part of a clinical trial. A clinical trial is a research study that tests a new approach to treatment. The “standard of care” is the best treatments known based on previous clinical trials. Clinical trials may test such approaches as a new drug, a new combination of existing treatments, or new doses of current therapies. The health and safety of all children participating in clinical trials are closely monitored.
To take advantage of these newer treatments, children with a CNS tumor should be treated at a specialized cancer center. Doctors at these centers have extensive experience in treating children and have access to the latest research. If a pediatric cancer center is not nearby, general cancer centers sometimes have pediatric specialists who are able to be part of your child’s care.
How a CNS tumor is treated
In many cases, a team of doctors works with a child and the family to provide care. This is called a multidisciplinary team. Members of the multidisciplinary team for a child with a CNS tumor often include a:
Neurosurgeon: A doctor who specializes in treating a tumor in the brain or spine using surgery.
Pediatric oncologist: A doctor who specializes in treating children with cancer with chemotherapy or immunotherapy.
Radiation oncologist: A doctor who specializes in giving radiation therapy to treat a tumor.
Neurologist: A doctor who specializes in neurological problems such as seizures or movement or sensory disorders.
Neuro-radiologist: A doctor who specializes in imaging of the brain and spine.
Neuro-pathologist: A pathologist who specializes in the diagnosis of diseases of the CNS.
Cancer care teams also include physician assistants, nurse practitioners, oncology nurses, and pharmacists. Pediatric cancer centers often have extra support services for children and their families, such as child life specialists, dietitians, physical and occupational therapists, social workers, and counselors. Special activities and programs to help your child and family cope may also be available.
Treatment options and recommendations depend on several factors, including the type of CNS tumor, whether it is cancerous, the stage and/or grade of the tumor, possible side effects, the family’s preferences, and the child’s age and overall health. For instance, radiation therapy is often limited in children younger than 3 years old because extensive radiation therapy can cause learning and memory problems in the future. Your child’s care plan may also include treatment for symptoms and side effects, an important part of medical care. Take time to learn about all of your child’s treatment options and be sure to ask questions about things that are unclear.
Talk with your child’s doctors about the goals of each treatment and what you can expect during the treatment. These types of talks are called “shared decision-making.” Shared decision-making is when you and the doctors work together to choose treatments that fit the goals of your child’s care. Shared decision-making is particularly important for a CNS tumor because there are different treatment options. Learn more about making treatment decisions.
The common types of treatments used for specific types of CNS tumors that are common in children are described below.
Astrocytoma. Treatment options often depend on the grade of the tumor:
For low-grade astrocytoma, options include surgery, chemotherapy, radiation therapy, or combinations of these treatments. The types of treatments used depend on the tumor's location, how easily it can be removed with surgery, and the child's age. For some children with low-grade astrocytomas, surgery may be enough to cure the astrocytoma. Sometimes, depending upon the age of the child and the tumor's location, chemotherapy and/or radiation therapy may be recommended.
For high-grade astrocytoma, surgery plus radiation therapy and chemotherapy are usually given.
Learn more about the treatment of astrocytoma.
Diffuse intrinsic pontine glioma (DIPG). Also called brain stem glioma, treatment for DIPG generally includes radiation therapy. Currently, chemotherapy does not appear to lengthen the lives of children with DIPG. However, new drugs are being researched for DIPG in clinical trials. Learn more about the treatment options for DIPG.
Ependymoma. Treatment options for ependymoma include surgery and radiation therapy. Chemotherapy may also be used for infants and very young children with ependymoma. It may also be used for a short time to help shrink a tumor so it can be completely removed with surgery. Learn more about treatments for ependymoma.
Germ cell tumor. Surgery, chemotherapy, and/or radiation therapy are usually used to treat a germ cell tumor. Learn more about how a germ cell tumor is treated.
Medulloblastoma. Treatment options for medulloblastoma include surgery, radiation therapy, and chemotherapy. Learn more about these treatment options for medulloblastoma.
More details about each of the main types of treatment for childhood CNS tumors are described below.
Surgery is the removal of the tumor and some surrounding healthy tissue during an operation. The purposes of surgery involving the CNS are to remove tissue to make a diagnosis, to completely remove the tumor to improve the prognosis, and to relieve symptoms that are caused by the tumor compressing normal functioning nerves. Sometimes, a tumor spreads and grows between healthy nerve cells, making surgery difficult. In this situation, surgery may be limited and/or other treatments will be recommended.
Your child's surgery will be carefully planned to reduce the risks of major side effects. However, as discussed in the Introduction, treatment of the CNS is challenging because of the very important functions of the brain and spinal cord. In certain situations, surgery can damage parts of the brain near the tumor, affecting arm and leg movement, breathing, swallowing, eye movement, and/or other functions. Before surgery, talk with the health care team about the possible side effects from the specific surgery your child will have. Learn more about the basics of surgery.
Sometimes, a CNS tumor cannot be removed by surgery because of its specific location. These tumors are called inoperable or unresectable. In these instances, the doctor will recommend other treatment options.
Radiation therapy is the use of high-energy x-rays or other particles to destroy tumor cells. A radiation therapy regimen, or schedule, usually consists of a specific number of treatments given over a set period of time.
The most common type of radiation treatment is called external-beam radiation therapy, which is radiation given from a machine outside the body. When radiation treatment is given using implants, it is called internal radiation therapy or brachytherapy.
Stereotactic radiosurgery is a way to deliver high doses of radiation therapy directly to a tumor and not to healthy tissue. This technique makes detailed, 3-dimensional maps of the brain and tumor so doctors can pinpoint where to direct the radiation treatment.
Another type of radiation therapy that may be used for a CNS tumor is called proton therapy. Proton therapy is a type of external-beam radiation therapy that uses a particle called a proton rather than an x-ray. At high energy, protons can destroy cancer cells. Proton therapy does not go completely through the body (and does not have an "exit dose"), so it may cause less damage to healthy tissue. In the past 2 decades, proton therapy has become more widely available in the United States.
Side effects from radiation therapy may include fatigue, mild skin reactions, upset stomach, headaches, and loose bowel movements. Most side effects go away soon after treatment is finished. However, in the long term, radiation therapy can sometimes cause problems with the growth and development of the child’s brain. Therefore, the doctor may choose to treat the tumor in another way.
Learn more about the basics of radiation therapy.
Chemotherapy is the use of drugs to destroy tumor cells, usually by keeping the tumor cells from growing, dividing, and making more cells. Chemotherapy for a child with a CNS tumor is usually prescribed by a pediatric oncologist or neurologist. These doctors specialize in treating a tumor with cancer medications.
Systemic chemotherapy gets into the bloodstream to reach tumor cells throughout the body. Common ways to give chemotherapy include an intravenous (IV) tube placed into a vein using a needle or as a pill or capsule that is swallowed (orally). Chemotherapy may also be given directly into the spinal canal to treat tumor cells on the surface of the brain and spine. This procedure, called intrathecal chemotherapy, is still being researched in clinical trials and may not be available everywhere.
A chemotherapy regimen, or schedule, usually consists of a specific number of cycles given over a set period time. A patient may receive 1 drug at a time or a combination of different drugs given at the same time.
Chemotherapy may be effective for many types of brain tumors. Depending on the tumor type, chemotherapy may be given immediately after a biopsy or surgery or after radiation therapy. In some instances, chemotherapy is used at the same time as radiation therapy.
The side effects of chemotherapy depend on the individual and the drug and dose used, but they can include fatigue, risk of infection, nausea and vomiting, hair loss, loss of appetite, and diarrhea. These side effects usually go away after treatment is finished.
Learn more about the basics of chemotherapy.
The medications used to treat a CNS tumor are continually being evaluated. Talking with your child’s doctor is often the best way to learn about the medications prescribed for your child, their purpose, and their potential side effects or interactions with other medications. It is also important to let your doctor know if your child is taking any other prescription or over-the-counter medications or supplements. Herbs, supplements, and other drugs can interact with cancer medications, causing unwanted side effects or reduced effectiveness. Learn more about your child’s prescriptions by using searchable drug databases.
Targeted therapy, also called targeted drug therapy or targeted chemotherapy, is a treatment using medication that targets the tumor’s specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. This type of medication treatment blocks the growth and spread of tumor cells and limits damage to healthy cells.
Not all tumors have the same targets. To find the most effective treatment, your doctor may run tests to identify the genes, proteins, and other factors in your tumor. This helps doctors better match each patient with the most effective treatment whenever possible. In addition, research studies continue to find out more about specific molecular targets and new treatments directed at them. Learn more about the basics of targeted treatments.
In 2022, the FDA approved dabrafenib (Tafinlar) in combination with trametinib (Mekinist) for the treatment of children with both low-grade and high-grade glioma with a BRAF V600E mutation that is either metastatic or that cannot be removed with surgery. To receive the combination, the disease must have either gotten worse following previous treatment or there must be no other satisfactory treatment options available. A BRAF V600 mutation is a specific change in the tumor’s BRAF gene. Dabrafenib is a targeted therapy that works by targeting the BRAF mutation, and trametinib is a targeted therapy that targets the MEK protein, which is involved in tumor growth and survival. Dabrafenib is given as an oral medication twice daily and trametinib is given as an oral medication once daily.
Other targeted drugs for the treatment of CNS tumors continue to be studied in clinical trials. Talk with your doctor about whether treatment with a targeted chemotherapy drug through a clinical trial may be recommended for your child and their specific diagnosis.
Side effects of targeted chemotherapy treatments can vary. Talk with your doctor about possible side effects for a specific medication and how they can be managed.
A CNS tumor and its treatment cause physical symptoms and side effects, as well as emotional, social, and financial effects. Managing all of these effects is called palliative care or supportive care. It is an important part of your child’s care that is included along with treatments intended to slow, stop, or eliminate the tumor.
Palliative care focuses on improving how your child feels during treatment by managing symptoms and supporting patients and their families with other, non-medical needs. Any person, regardless of age or type and grade of the tumor, may receive this type of care. And it often works best when it is started right after the diagnosis. People who receive palliative care along with treatment for the tumor often have less severe symptoms, better quality of life, and report that they are more satisfied with treatment.
Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional and spiritual support, and other therapies. Your child may also receive palliative treatments similar to those meant to get rid of the tumor, such as chemotherapy, surgery, or radiation therapy.
Before treatment begins, talk with your child’s doctor about the goals of each treatment in the recommended treatment plan. You should also talk about the possible side effects of the specific treatment plan and palliative care options. Many patients also benefit from talking with a social worker and participating in support groups. Ask your doctor about these resources, too.
During treatment, your child’s health care team may ask you to answer questions about your child’s symptoms and side effects and to describe each problem. Be sure to tell the health care team if your child is experiencing a problem. This helps the health care team treat any symptoms and side effects as quickly as possible. It can also help prevent more serious problems in the future.
A remission is when the tumor cannot be detected in the body and there are no symptoms. This may also be called having “no evidence of disease” or NED.
A remission may be temporary or permanent. This uncertainty causes many people to worry that the tumor will come back. While many remissions are permanent, it is important to talk with the doctor about the possibility of the tumor returning. Understanding your child’s risk of recurrence and the treatment options may help you feel more prepared if the tumor does return. Learn more about coping with the fear of recurrence.
If the tumor returns after the original treatment, it is called a recurrent tumor. It may come back in the same place (called a local recurrence), nearby (regional recurrence), or in another place (distant recurrence).
If a recurrence happens, a new cycle of testing will begin again to learn as much as possible about it. After this testing is done, you and your child’s doctor will talk about the treatment options. Often the treatment plan will include the treatments described above, such as surgery, chemotherapy, and radiation therapy, but they may be used in a different combination or given at a different pace. Your child’s doctor may suggest clinical trials that are studying new ways to treat a recurrent CNS tumor. Whichever treatment plan you choose, palliative care will be important for relieving symptoms and side effects.
For a recurrent CNS tumor, the treatment options depend on 3 factors:
The type of tumor
Whether the tumor recurred where it originally started or in another part of the brain or body
The type of treatment given for the original tumor
Depending on your child’s situation, the doctor may recommend surgery, radiation therapy, chemotherapy, and/or a stem cell/bone marrow transplantation.
A recurrent tumor may bring up emotions such as disbelief or fear. You and your family are encouraged to talk with your child's health care team about these feelings and ask about support services to help you cope. Learn more about dealing with a tumor recurrence.
Although treatment is successful for many children with a tumor in the brain or spinal cord, sometimes it is not. If a child’s tumor cannot be cured or controlled, this is called an advanced or terminal tumor. This diagnosis is stressful, and an advanced CNS tumor may be difficult to discuss. However, it is important to have open and honest conversations with your child’s health care team to express your family’s feelings, preferences, and concerns. The health care team has special skills, experience, and knowledge to support patients and their families and is there to help.
Hospice care is designed to provide the best possible quality of life for people who are expected to live less than 6 months. Parents and guardians are encouraged to talk with the health care team about hospice options, which include hospice care at home, a special hospice center, or other health care locations. Nursing care and special equipment can make staying at home a workable option for many families. Some children may be happier and more comfortable if they can attend school part-time or keep up other activities and social connections. The child’s health care team can help parents or guardians decide on an appropriate level of activity. Making sure a child is physically comfortable and free from pain is extremely important as part of end-of-life care. Learn more about caring for a terminally ill child and advanced care planning.
The death of a child is an enormous tragedy, and families may need support to help them cope with the loss. Pediatric cancer centers often have professional staff and support groups to help with the process of grieving. Learn more on grieving the loss of a child.
The next section in this guide is About Clinical Trials. It offers more information about research studies that are focused on finding better ways to care for children with a CNS tumor. Use the menu to choose a different section to read in this guide.