What is medulloblastoma?
Medulloblastoma is a cancerous tumor—also called cerebellar primitive neuroectodermal tumor (PNET)—that starts in the region of the brain at the base of the skull, called the posterior fossa.
- These tumors tend to spread to other parts of the brain and to the spinal cord.
How common is medulloblastoma?
- Medulloblastoma is the most common malignant brain tumor of childhood, and it accounts for about 20 percent of all childhood brain tumors.
- Between 250 and 500 children are found to have medulloblastoma each year in the United States.
- Most medulloblastoma tumors are found in children younger than age 16, but they can rarely occur in adults.
- Medulloblastoma is slightly more common in boys than in girls.
What are the symptoms of medulloblastoma?
If your child has medulloblastoma, the following symptoms may occur:
- Morning nausea or vomiting that gradually gets worse
- Problems with handwriting
- Visual problems (rare) at the time of diagnosis
If the tumor has spread to the spinal cord, symptoms may include:
- Back pain
- Trouble walking
- Problems controlling bladder and bowel functions
How is medulloblastoma treated?
- Surgery—is usually the first step in treating medulloblastoma:
- The goal is to take out as much of the tumor as possible.
- If the tumor has been completely removed, the patient is treated with reduced-dose radiation to the brain and spinal cord before receiving chemotherapy.
- If some tumor remains after surgery, or if the cancer has spread, the patient is given higher-dose radiation therapy before chemotherapy.
- Radiation therapy—uses high-energy X-rays or other types of radiation to kill cancer cells or stop them from growing.
- Chemotherapy (“chemo”)—uses powerful medicines to kill cancer cells or stop them from growing (dividing) and making more cancer cells:
- Chemo is injected or given orally, so that it can travel throughout the body.
- Combination therapy uses more than one type of chemo at a time.
What are the survival rates for medulloblastoma?
Survival rates in children with medulloblastoma depend on the patient’s age and how much the tumor spreads.
- If the disease has not spread, survival rates are around 70 to 80 percent.
- If the disease has spread to the spinal cord, the survival rate is about 60 percent.
- Children younger than age 3 often have lower survival rates because their disease tends to be more aggressive.
Why choose St. Jude for your child’s medulloblastoma treatment?
- St. Jude is the only National Cancer Institute-designated Comprehensive Cancer Center devoted solely to children.
- The hospital has one of the largest pediatric brain tumor programs in the United States.
- St. Jude has created more clinical trials for cancer than any other children’s hospital in the United States.
- The nurse-to-patient ratio at St. Jude is unmatched—averaging 1:3 in hematology and oncology, and 1:1 in the Intensive Care Unit.
- The program offers a closely knit group of doctors, scientists, nurses and support staff providing the many types of treatment and support services crucial to the complete care of children with brain tumors.
- Because St. Jude doctors and scientists work closely, they are always studying ways to better understand and treat the tumor. Through this partnership, they have discovered that not all medulloblastoma tumors are the same. The team members are now working to show that medulloblastoma can be better treated by focusing therapy on these differences.
- The current St. Jude clinical trial for treatment of medulloblastoma is the first to treat differences in medulloblastoma. Tests determine which type of medulloblastoma each child has and how aggressive that cancer is. Therapy is then based on these results and targeted to each patient’s tumor.
Contact the Brain Tumor Team any time, 24 hours a day, 7 days a week.
Call our Brain Tumor Coordinator, Tabatha E. Doyle, RN:
(901) 595-2544 or
Or email us at email@example.com
Phase I study of Savolitinib in Recurrent, Progressive or Refractory Medulloblastoma, High-Grade Glioma, or Diffuse Intrinsic Pontine Glioma
- Recurrent, refractory or progressive medulloblastoma, high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG)
- Evidence of genetic activation of the MET pathway (expansion co-hort)
- At least 5 years old and 21 years old or younger
SJDAWN: Molecular-Based Therapy for Aggressive Brain and Spinal Cord Tumors in Children and Young Adults
Molecularly-Driven Doublet Therapy for All Children with Refractory or Recurrent CNS Malignant Neoplasms and Young Adults with Refractory or Recurrent SHH Medulloblastoma
Ages for Study Enrollment
- Stratum A (ribociclib and gemcitabine): Between 1 and 24 years old with recurrent, progressive or refractory non-WNT, non-sonic hedgehog (SHH) (NWNS) medulloblastoma or ependymoma
- Stratum B (ribociclib and trametinib): Between 1 and 24 years old with recurrent, progressive, or refractory central nervous system (CNS) tumors, including:
- High-grade glioma
- Atypical teratoid rhabdoid tumor (ATRT)
- SHH and WNT medulloblastoma
- CNS embryonal tumors [previously called PNET])
- Stratum C (ribociclib and sonidegib): Between 10 and 39 years old with recurrent, progressive, or refractory SHH medulloblastoma and copy number loss of 9q or PTCH1 mutation. (This stratum is only open to patients with SHH medulloblastoma who are fully grown. Patients younger than 18 years old will have bone age to determine if fully grown)
Screening Phase Eligibility
- Participants with recurrent, progressive, or refractory brain tumors
- At least 1 year old and younger than 25 years old at the time of screening. Exception: Participants with recurrent, progressive or refractory medulloblastoma who are at least 1 year old and younger than 40 years of age at the time of study screening are eligible for screening.
- Participants and/or guardian have the ability to understand and the willingness to sign a written informed consent document according to institutional guidelines.
- Recurrent, progressive or refractory low grade glioma (LGG)
- Prior exposure to a CDK4/6 inhibitor
- History of clinically significant, uncontrolled heart disease and/or repolarization abnormalities
- History of QTc prolongation
SJELIOT: Phase I Study of a CHK1/2 Inhibitor Therapy in Combination with Chemotherapy for Children and Adolescents with Refractory or Recurrent Medulloblastoma Brain Tumors
Phase I Trial of a CHK1/2 Inhibitor Therapy, in Combination with Chemotherapy, for Children and Adolescents with Refractory/Recurrent Group 3, Group 4 or SHH Medulloblastoma Brain Tumors
- Recurrent, refractory or progressive medulloblastoma
- At least 1 year old and younger than 25 years old
Clinical and Molecular Risk-Directed Craniospinal Irradiation and Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed Medulloblastoma
Medulloblastoma (includes all variants of medulloblastoma and posterior fossa PNET)
- Diagnosis of newly medulloblastoma
- At least 3 years old and younger than 22 years old (Strata W, S or N) OR
- At least 22 years old and younger than 40 years old AND has SHH medulloblastoma (Stratum S)
- Has not received previous treatment with radiation therapy or chemotherapy
- Must start treatment within 36 days of surgery to remove the tumor
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