Overview
Medulloblastoma is a malignant embryonal tumor of the cerebellum (posterior fossa) and is the most common malignant brain tumor in children. It arises from the cerebellum, which is located at the base of the brain and is responsible for coordination, balance, and motor control. Medulloblastoma is classified under ICD-10 as C71.6 (malignant neoplasm of the cerebellum). The tumor is highly aggressive and has a tendency to spread through the cerebrospinal fluid (CSF) to other parts of the central nervous system, a process known as leptomeningeal dissemination. Key symptoms include headaches, nausea and vomiting (particularly in the morning), problems with balance and coordination (ataxia), unsteady gait, visual disturbances such as double vision (diplopia), and signs of increased intracranial pressure including papilledema and hydrocephalus. In infants, an enlarging head circumference may be an early sign. As the tumor grows, it can obstruct the flow of cerebrospinal fluid, leading to obstructive hydrocephalus, which can cause lethargy, irritability, and cognitive changes. Medulloblastoma is now classified into four molecular subgroups: WNT-activated, SHH (Sonic Hedgehog)-activated, Group 3, and Group 4, each with distinct clinical behavior and prognosis. Some cases are associated with hereditary cancer predisposition syndromes, including Gorlin syndrome (PTCH1 mutations), Turcot syndrome (APC mutations), and Li-Fraumeni syndrome (TP53 mutations). Treatment typically involves a multimodal approach including maximal safe surgical resection, craniospinal irradiation (in children over 3 years of age), and adjuvant chemotherapy. Prognosis varies significantly by molecular subgroup, with WNT-activated tumors having the best outcomes (greater than 90% survival) and Group 3 tumors carrying a poorer prognosis. Current research is focused on risk-stratified therapy to reduce treatment-related toxicity in favorable-risk patients while intensifying therapy for high-risk groups.
Clinical phenotype terms— hover any for plain English:
Variable
Can be inherited in different ways depending on the underlying gene
Childhood
Begins in childhood, roughly ages 1 to 12
FDA & Trial Timeline
10 eventsNationwide Children's Hospital — PHASE3
University of Florida — PHASE1
St. Jude Children's Research Hospital
Sabine Mueller, MD, PhD — PHASE1, PHASE2
H. Lee Moffitt Cancer Center and Research Institute — PHASE2
Mohammad H. Abu Arja
C17 Council — EARLY_PHASE1
University of Florida — PHASE1
Tata Memorial Centre
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Medulloblastoma.
20 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →Rare Disease Specialist
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Medulloblastoma.
Community
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Common questions about Medulloblastoma
What is Medulloblastoma?
Medulloblastoma is a malignant embryonal tumor of the cerebellum (posterior fossa) and is the most common malignant brain tumor in children. It arises from the cerebellum, which is located at the base of the brain and is responsible for coordination, balance, and motor control. Medulloblastoma is classified under ICD-10 as C71.6 (malignant neoplasm of the cerebellum). The tumor is highly aggressive and has a tendency to spread through the cerebrospinal fluid (CSF) to other parts of the central nervous system, a process known as leptomeningeal dissemination. Key symptoms include headaches, nau
At what age does Medulloblastoma typically begin?
Typical onset of Medulloblastoma is childhood. Age of onset can vary across affected individuals.
Are there clinical trials for Medulloblastoma?
Yes — 20 recruiting clinical trials are currently listed for Medulloblastoma on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Medulloblastoma?
25 specialists and care centers treating Medulloblastoma are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.