Overview
Central nervous system embryonal tumors (CNS embryonal tumors) are a heterogeneous group of rare, highly malignant brain tumors that arise from embryonal (primitive) cells in the central nervous system. This category encompasses several subtypes, including medulloblastoma, atypical teratoid/rhabdoid tumor (AT/RT), embryonal tumor with multilayered rosettes (ETMR), and other previously classified primitive neuroectodermal tumors (CNS-PNETs). These tumors predominantly affect infants and young children, though they can occasionally occur in adolescents and adults. They are characterized by rapid growth and a tendency to spread through the cerebrospinal fluid pathways. Clinical presentation depends on tumor location but commonly includes signs of increased intracranial pressure such as headaches, nausea, vomiting, and lethargy. Depending on the specific site within the brain or spinal cord, patients may experience ataxia, cranial nerve palsies, seizures, developmental regression, visual disturbances, and focal neurological deficits. In infants, an enlarging head circumference may be an early sign. Behavioral changes and cognitive decline can also be prominent features. Treatment typically involves a multimodal approach including maximal safe surgical resection, craniospinal irradiation (in children over approximately three years of age), and intensive chemotherapy. Specific treatment protocols vary by tumor subtype, patient age, and extent of disease at diagnosis. Despite aggressive therapy, prognosis remains guarded for many subtypes, particularly AT/RT and ETMR, though outcomes for standard-risk medulloblastoma have improved significantly. Ongoing research into molecular classification has led to more refined risk stratification and the development of targeted therapeutic approaches. Long-term survivors often require monitoring for treatment-related late effects including neurocognitive impairment, endocrine dysfunction, and secondary malignancies.
Sporadic
Usually appears on its own, not inherited from a parent
Childhood
Begins in childhood, roughly ages 1 to 12
FDA & Trial Timeline
10 eventsSt. Jude Children's Research Hospital — PHASE2
The Netherlands Cancer Institute — PHASE3
Grupo Español Multidisciplinar de Melanoma — PHASE2
Washington University School of Medicine — EARLY_PHASE1
Emory University — PHASE1
University of Florida — PHASE1
University of Manchester — NA
Nationwide Children's Hospital — PHASE2
University Health Network, Toronto — EARLY_PHASE1
Beijing Tiantan Hospital — EARLY_PHASE1
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Central nervous system embryonal tumor.
4 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 Central nervous system embryonal tumor.
Community
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Start the conversation →Latest news about Central nervous system embryonal tumor
Disease timeline:
New recruiting trial: Engineering Immune Organoids to Study Pediatric Cancer
A new clinical trial is recruiting patients for Central nervous system embryonal tumor
New recruiting trial: Genetic and Molecular Risk Profiles of Pediatric Malignant Brain Tumors in China
A new clinical trial is recruiting patients for Central nervous system embryonal tumor
New recruiting trial: Atovaquone Combined With Radiation in Children With Malignant Brain Tumors
A new clinical trial is recruiting patients for Central nervous system embryonal tumor
New recruiting trial: PLX038 in Primary Central Nervous System Tumors Containing MYC or MYCN Amplifications
A new clinical trial is recruiting patients for Central nervous system embryonal tumor
New recruiting trial: Systemic Biomarkers to Predict Radiation-Induced Neurocognitive Decline
A new clinical trial is recruiting patients for Central nervous system embryonal tumor
New recruiting trial: Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed Medulloblastoma
A new clinical trial is recruiting patients for Central nervous system embryonal tumor
New recruiting trial: Chemo-immunotherapy Using Ibrutinib Plus Indoximod for Patients With Pediatric Brain Cancer
A new clinical trial is recruiting patients for Central nervous system embryonal tumor
New recruiting trial: Phase I Trial to Determine the Dose and Evaluate the PK and Safety of Lutetium Lu 177 Edotreotide Therapy in Pediatric Participants With SSTR-positive Tumors
A new clinical trial is recruiting patients for Central nervous system embryonal tumor
New recruiting trial: Melanoma Metastasized to the Brain and Steroids
A new clinical trial is recruiting patients for Central nervous system embryonal tumor
New recruiting trial: GD2-CAR T Cells for Pediatric Brain Tumours
A new clinical trial is recruiting patients for Central nervous system embryonal tumor
Caregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Central nervous system embryonal tumor
What is Central nervous system embryonal tumor?
Central nervous system embryonal tumors (CNS embryonal tumors) are a heterogeneous group of rare, highly malignant brain tumors that arise from embryonal (primitive) cells in the central nervous system. This category encompasses several subtypes, including medulloblastoma, atypical teratoid/rhabdoid tumor (AT/RT), embryonal tumor with multilayered rosettes (ETMR), and other previously classified primitive neuroectodermal tumors (CNS-PNETs). These tumors predominantly affect infants and young children, though they can occasionally occur in adolescents and adults. They are characterized by rapid
How is Central nervous system embryonal tumor inherited?
Central nervous system embryonal tumor follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Central nervous system embryonal tumor typically begin?
Typical onset of Central nervous system embryonal tumor is childhood. Age of onset can vary across affected individuals.
Are there clinical trials for Central nervous system embryonal tumor?
Yes — 4 recruiting clinical trials are currently listed for Central nervous system embryonal tumor on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Central nervous system embryonal tumor?
25 specialists and care centers treating Central nervous system embryonal tumor are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.