Medulloblastoma is a fast-growing tumor that forms in the cerebellum (the lower, back part of the brain). Medulloblastoma is the most common type of CNS embryonal tumor. CNS embryonal tumors are uncontrolled growths of cells in the brain. These tumors form in cells that are left over from fetal development, called embryonal cells. Pineoblastoma is a fast-growing type of brain tumor that forms in or around a tiny organ near the center of the brain called the pineal gland.
These tumors may be benign (not cancer) or malignant (cancer). Benign brain tumors grow and press on nearby areas of the brain but rarely spread to other parts of the brain. Malignant brain tumors are likely to grow quickly and spread into other parts of the brain. They may also spread to other parts of the body, but this is rare. When a tumor grows into and presses on an area of the brain or spreads to other parts of the brain, it may stop that part of the brain from working the way it should. Both benign and malignant brain tumors can cause serious signs or symptoms and need treatment.
Most medulloblastomas, other CNS embryonal tumors, and pineoblastomas in children are malignant. These tumors tend to spread through the cerebrospinal fluid to other parts of the brain and spinal cord.
Although cancer is rare in children, brain tumors are the second most common type of childhood cancer, after leukemia. This summary is about the treatment of primary brain tumors (tumors that begin in the brain).
The different types of CNS embryonal tumors include:
Most CNS embryonal tumors are medulloblastomas. Medulloblastomas are fast-growing tumors that form in brain cells in the cerebellum. The cerebellum is at the lower back part of the brain between the cerebrum and the brain stem. The cerebellum controls movement, balance, and posture. It is rare for medulloblastomas to spread to the bone, bone marrow, lung, or other parts of the body.
Other types of CNS embryonal tumors are fast-growing tumors and may form in brain cells anywhere in the brain, including the cerebrum, brain stem, or spinal cord. The cerebrum is at the top of the head and is the largest part of the brain. The cerebrum controls thinking, learning, problem-solving, emotions, speech, reading, writing, and voluntary movement. It is rare for these tumors to spread to the bone, bone marrow, lung, or other parts of the body.
There are many types of CNS embryonal (nonmedulloblastoma) tumors:
Cribriform neuroepithelial tumor forms in the ventricles in the brain. This tumor most often occurs in infants and young children. Cribriform neuroepithelial tumor occurs more often in boys.
Embryonal tumors with multilayered rosettes (ETMR) are rare tumors that form in the brain and spinal cord. ETMR most commonly occur in young children and are fast-growing tumors.
CNS neuroblastomas are a very rare type of neuroblastoma that form in the nerve tissue of the cerebrum or the layers of tissue that cover the brain and spinal cord. CNS neuroblastomas may be large and spread to other parts of the brain or spinal cord.
CNS high-grade neuroepithelial tumor is a very rare tumor that forms in the brain. This tumor occurs most often in children younger than 10 years, but can occur in older children and adolescents.
CNS Ewing sarcoma is a very rare tumor found in the brain or spine. This tumor most often occurs in children younger than 10 years.
CNS high-grade neuroepithelial tumor is a very rare tumor that forms in the brain or spinal cord. This tumor most often occurs in adolescents and females.
Medulloepithelioma is a fast-growing tumor that usually forms in the brain, spinal cord, or nerves just outside the spinal column. It occurs most often in infants and young children.
CNS atypical teratoid/rhabdoid tumor is also a type of embryonal tumor, but it is treated differently than other childhood CNS embryonal tumors. For more information, see Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment.
The pineal gland is a tiny organ in the center of the brain. The gland makes melatonin, a substance that helps control our sleep cycle. Pineoblastoma are usually malignant fast-growing tumors with cells that look very different from normal pineal gland cells. Pineoblastomas are not a type of CNS embryonal tumor but treatment for them is similar to treatment for CNS embryonal tumors.
Pineoblastoma is linked with inherited changes in the retinoblastoma (RB1) gene. A child with the inherited form of retinoblastoma (cancer that forms in the tissues of the retina) has an increased risk of pineoblastoma. When retinoblastoma forms at the same time as a tumor in or near the pineal gland, it is called trilateral retinoblastoma. MRI (magnetic resonance imaging) testing in children with retinoblastoma may detect pineoblastoma at an early stage when it can be treated successfully. It is rare for pineoblastoma to spread to the bone, bone marrow, lung, or other parts of the body.
Childhood medulloblastoma is caused by certain changes to the way brain cells function, especially how they grow and divide into new cells. Often, the exact cause of the cell changes is unknown. To learn more about how cancer develops, see What is Cancer?.
A risk factor is anything that increases the chance of getting a disease. Not every child with one or more of these risk factors will develop medulloblastoma, and it will develop in some children who don't have a known risk factor. Talk with your child's doctor if you think your child may be at risk.
The risk for medulloblastoma is increased in people who have any of the following inherited diseases:
It may not be clear from the family medical history whether a child with a brain tumor has an inherited condition that increased their risk. Genetic counselors and other specially trained health professionals can discuss your child's diagnosis and the family's medical history to understand:
Genetic counselors can also help you cope with your child's genetic testing results, including how to discuss the results with family members.
Children may not have symptoms of medulloblastoma, other CNS embryonal tumors, or pineoblastoma until the tumor has grown bigger. It's important to check with your child's doctor if your child has any symptoms below:
Infants and young children with these tumors may be irritable or grow slowly. Also they may not eat well or meet developmental milestones such as sitting, walking, and talking in sentences. These tumors may also cause an increase in the size of an infant's head.
These symptoms may be caused by conditions other than medulloblastoma, other CNS embryonal tumors, or pineoblastoma. The only way to know is to see your child's doctor. The doctor will ask you when the symptoms started and how often your child has been having them as a first step in making the diagnosis.
If your child has symptoms that suggest medulloblastoma, another type of CNS embryonal tumor, or pineoblastoma, the doctor will need to find out if these are due to cancer or another condition. They will ask about your child's personal and family health history and do a physical exam. Depending on the results, the doctor may recommend tests to find out if your child has a brain tumor. The results of these tests will also help you and your child's doctor plan treatment.
The following tests and procedures are used to diagnose medulloblastoma, other CNS embryonal tumors, and pineoblastoma:
If doctors think your child may have medulloblastoma, another type of CNS embryonal tumor, or pineoblastoma, a biopsy may be done. The biopsy is done by removing part of the skull and using a needle to remove a sample of tissue. Sometimes, a computer-guided needle is used to remove the tissue sample. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The piece of skull is usually put back in place after the procedure.
The following test may be done on the sample of tissue that is removed:
If your child has been diagnosed with medulloblastoma, other CNS embryonal tumor, or pineoblastoma, you may have questions about how serious the cancer is and your child's chances of survival. The likely outcome or course of a disease is called prognosis.
The prognosis and treatment options depend on:
Your child's cancer care team is in the best position to talk with you about your child's prognosis.
You may want to get a second opinion to confirm your child's diagnosis and treatment plan. If you seek a second opinion, you will need to get important medical test results and reports from the first doctor to share with the second doctor. The second doctor will review the pathology report, slides, and scans before giving a recommendation. The doctor who gives the second opinion may agree with the first doctor, suggest changes or another approach, or provide more information about your child's tumor.
To learn more about choosing a doctor and getting a second opinion, see Finding Cancer Care. You can contact NCI's Cancer Information Service via chat, email, or phone (both in English and Spanish) for help finding a doctor or hospital that can provide a second opinion. For questions you might want to ask at your appointments, see Questions to Ask Your Doctor About Cancer.
Cancer stage describes the extent of cancer in the body, such as the size of the tumor, whether it has spread, and how far it has spread from where it first formed. There is no staging system used for childhood medulloblastoma, other central nervous system (CNS) embryonal tumors, or pineoblastoma, but the tests and procedures done to diagnose the cancer are also used to help plan treatment.
Treatment of other CNS embryonal tumors and pineoblastoma in children is based on the child's age. Children aged 3 years and younger may be given different treatment than children older than 3 years.
Medulloblastomas are called average risk when all of the following are true:
Medulloblastomas are called high risk if any of the following are true:
In general, cancer is more likely to recur (come back) after treatment in patients with a high-risk tumor.
If your child is diagnosed with medulloblastoma, another type of CNS embryonal tumor, or pineoblastoma, they will be referred to a pediatric oncologist/neuro-oncologist. This is a doctor who specializes in staging and treating childhood cancers. They will recommend tests to determine the extent (stage) of cancer. Some of the tests used to diagnose the cancer are repeated after surgery. This is to find out how much tumor remains after surgery and to see if the cancer has spread from the brain to the spine or other parts of the body. It is important to know if the cancer has spread in order to plan the best treatment. For the list of diagnostic tests, see the General Information section.
The following tests may be used to find out if the cancer has spread beyond the brain and spinal cord:
Childhood medulloblastoma and other types of CNS embryonal tumors most often recur (come back) within 3 years after treatment but may come back many years later. Recurrent childhood medulloblastoma and other CNS embryonal tumors may come back in the same place as the original tumor and/or in a different place in the brain or spinal cord.
There are different types of treatment for children and adolescents with medulloblastoma, other types of CNS embryonal tumors, or pineoblastoma. You and your child's cancer care team will work together to decide treatment. Many factors will be considered, such as your child's overall health and whether the tumor is newly diagnosed or has come back.
A pediatric oncologist, a doctor who specializes in treating children with cancer, will oversee treatment of medulloblastoma, other CNS embryonal tumors, and pineoblastoma. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists and others:
Your child's treatment plan will include information about the cancer, the goals of treatment, treatment options, and the possible side effects. It will be helpful to talk with your child's cancer care team before treatment begins about what to expect. For help every step of the way, see our downloadable booklet, Children with Cancer: A Guide for Parents.
Surgery is used to diagnose and treat childhood medulloblastoma, other CNS embryonal tumors, and pineoblastoma as described in the General Information section of this summary.
After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy, radiation therapy, or both to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy.
Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. Medulloblastoma, other CNS embryonal tumors, or pineoblastoma in children may be treated with external beam radiation therapy. External beam radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer.
Certain ways of giving external radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of radiation therapy include the following:
Because radiation therapy can affect growth and brain development in young children, especially children who are 3 years or younger, chemotherapy may be given to delay or reduce the need for radiation therapy.
Radiation therapy to the brain can also affect growth and development in children older than 3 years. For this reason, clinical trials are studying new ways of giving radiation that may have fewer side effects than standard methods.
Chemotherapy (also called chemo) uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy may be given alone or with other types of treatment, such as radiation therapy.
To treat medulloblastoma, other CNS embryonal tumors, and pineoblastoma, chemotherapy is taken by mouth or injected into a vein. When given this way, the drugs enter the bloodstream and can reach cancer cells throughout the body. Chemotherapy drugs that can cross the blood-brain barrier and reach tumor cells in the brain are used. These include:
Combinations of these drugs may be used. Other chemotherapy drugs not listed here may also be used.
Learn more about Chemotherapy to Treat Cancer.
High doses of chemotherapy are given to kill cancer cells. This cancer treatment destroys healthy cells, including blood-forming cells. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.
Targeted therapy uses drugs or other substances to block the action of specific enzymes, proteins, or other molecules involved in the growth and spread of cancer cells.
Vismodegib may be used to treat recurrent medulloblastoma in children who have finished growing.
Targeted therapy is also being studied for the treatment of childhood medulloblastoma and other CNS embryonal tumors that have recurred (come back) after treatment.
Learn more about Targeted Therapy to Treat Cancer.
A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be the best treatment choice.
Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.
To learn more, see Clinical Trials Information for Patients and Caregivers. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment.
For information about the treatments listed below, see the Treatment Option Overview section.
Younger children with medulloblastoma
Treatment of newly diagnosed medulloblastoma in children aged 3 years and younger includes:
Other treatments that may be given after surgery include:
Children older than 3 years with average-risk medulloblastoma
Treatment of newly diagnosed average-risk medulloblastoma in children older than 3 years includes:
Children older than 3 years with high-risk medulloblastoma
Treatment of newly diagnosed high-risk medulloblastoma in children older than 3 years includes:
For information about the treatments listed below, see the Treatment Option Overview section.
Children aged 3 years and younger with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors
Treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors in children 3 years or younger includes:
Children older than 3 years with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors
Treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors in children older than 3 years includes:
Children with embryonal tumors with multilayered rosettes or medulloepithelioma
Treatment of newly diagnosed embryonal tumor with multilayered rosettes (ETMR) or medulloepithelioma may include:
Children with CNS neuroblastoma
Treatment of newly diagnosed CNS neuroblastoma may include:
For information about the treatments listed below, see the Treatment Option Overview section.
Children aged 3 years and younger
Treatment of newly diagnosed pineoblastoma in children aged 3 years and younger includes:
Children older than 3 years
Treatment of newly diagnosed pineoblastoma in children older than 3 years includes:
For information about the treatments listed below, see the Treatment Option Overview section.
Treatment for recurrent childhood medulloblastoma and other CNS embryonal tumors may include:
Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available.
Signs or symptoms caused by the tumor may begin before the cancer is diagnosed and continue for months or years. It is important to talk with your child's doctors about signs or symptoms caused by the tumor that may continue after treatment.
To learn more about side effects that begin during treatment for cancer, visit Side Effects.
Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include:
Children diagnosed with medulloblastoma may have certain problems after surgery or radiation therapy, such as changes in the ability to think, learn, and pay attention. Also, cerebellar mutism syndrome may occur after surgery. Signs of this syndrome include the following:
Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child and the types of symptoms to expect after cancer treatment has ended. To learn more, see Late Effects of Treatment for Childhood Cancer.
Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging. For a list of tests, see the General Information section.
Some of the imaging tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the brain tumor has recurred (come back). If the imaging tests show abnormal tissue in the brain, a biopsy may also be done to find out if the tissue is made up of dead tumor cells or if new cancer cells are growing. These tests are sometimes called follow-up tests or check-ups.
When a child has cancer, every member of the family needs support. Honest and calm conversations build trust as you talk with your child and their siblings. Taking care of yourself during this difficult time is also important. Reach out to your child's treatment team and to people in your family and community for support. To learn more, see Support for Families When a Child Has Cancer and the booklet Children with Cancer: A Guide for Parents.
For more information about childhood medulloblastoma and other central nervous system embryonal tumor, see the following:
For more childhood cancer information and other general cancer resources, visit:
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PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-cns-embryonal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389401]
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