Embryonal tumor of neuroepithelial tissue

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ORPHA:251852
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26Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Embryonal tumor of neuroepithelial tissue is a broad category of rare, aggressive central nervous system (CNS) tumors that arise from primitive or undifferentiated neuroepithelial cells. These tumors predominantly occur in children and include several subtypes such as embryonal tumor with multilayered rosettes (ETMR), medulloepithelioma, CNS neuroblastoma, CNS ganglioneuroblastoma, and atypical teratoid/rhabdoid tumor, among others. They primarily affect the brain and, less commonly, the spinal cord, and are classified as high-grade (WHO grade IV) malignancies due to their rapid growth and tendency to disseminate through the cerebrospinal fluid. Key symptoms depend on tumor location but commonly include signs of increased intracranial pressure such as headaches, nausea, vomiting, and lethargy. Children may present with seizures, focal neurological deficits, developmental regression, visual disturbances, gait abnormalities, or cranial nerve palsies. In infants, an enlarging head circumference may be an early sign. The clinical course is often aggressive, with rapid symptom progression. Treatment typically involves a multimodal approach including maximal safe surgical resection, followed by chemotherapy and, depending on the patient's age and tumor subtype, craniospinal radiation therapy. Prognosis varies significantly by subtype, patient age, extent of resection, and presence of metastatic disease at diagnosis. Despite advances in treatment, many subtypes carry a guarded prognosis, and ongoing clinical trials are investigating novel targeted therapies and immunotherapies. Long-term survivors require careful monitoring for treatment-related late effects including neurocognitive impairment, endocrine dysfunction, and secondary malignancies.

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗NORD ↗

FDA & Trial Timeline

7 events
Dec 2025A Phase I/IIa Study of C5252 in Patients With Intracranial Tumor

ImmVira Pharma Co. Ltd — PHASE1, PHASE2

TrialNOT YET RECRUITING
Oct 2025T2 Star Magnetic Resonance Imaging and Biomarker Blood Testing to Predict the Change and Progress of Malignant Gliomas

John M. Buatti — NA

TrialRECRUITING
Sep 2024Feasibility of Aspirate Tissue Monitoring in Neuro-oncological Surgery

Kuopio University Hospital — NA

TrialRECRUITING
Sep 2024Inside a Neuron - Ex Vivo Evaluation of Cerebral and Cerebellar Neuronal Activity.

Fondazione IRCCS Policlinico San Matteo di Pavia

TrialRECRUITING
Jun 2024Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of the Oncolytic HSV1 MVR-C5252

Duke University — PHASE1

TrialRECRUITING
Oct 2023Novel Targeted Radiotherapy in Pediatric Patients With Inoperable Relapsed or Refractory HGG

Cellectar Biosciences, Inc. — PHASE1

TrialACTIVE NOT RECRUITING
Jan 2020Study of Vorasidenib (AG-881) in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO)

Institut de Recherches Internationales Servier — PHASE3

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Embryonal tumor of neuroepithelial tissue.

View clinical trials →

No actively recruiting trials found for Embryonal tumor of neuroepithelial tissue at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Embryonal tumor of neuroepithelial tissue community →

Specialists

Showing 25 of 26View all specialists →
WL
Wenbin Li
Specialist
PI on 3 active trials628 Embryonal tumor of neuroepithelial tissue publications
XQ
Xiaoguang Qiu
Specialist
PI on 1 active trial109 Embryonal tumor of neuroepithelial tissue publications
GL
Global Clinical Leader
Specialist
PI on 44 active trials4 Embryonal tumor of neuroepithelial tissue publications
AM
Ashley Ghiaseddin, MD
GAINESVILLE, FL
Specialist
PI on 3 active trials1 Embryonal tumor of neuroepithelial tissue publication
AM
Andrew Sloan, MD
Specialist
PI on 3 active trials1 Embryonal tumor of neuroepithelial tissue publication
TM
Tracy Batchelor, MD, MPH
BOSTON, MA
Specialist
PI on 5 active trials
HM
Helen E Heslop, MD
HOUSTON, TX
Specialist
PI on 6 active trials
PN
Phioanh Nghiemphu
LOS ANGELES, CA
Specialist
PI on 2 active trials
PM
Prashant Chittiboina, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 7 active trials
SP
Stanislaw R. Burzynski, MD, PhD
HOUSTON, TX
Specialist
PI on 29 active trials
SM
Stephen Bagley, MD MSCE
Specialist
PI on 1 active trial
JR
Jane Robertson
Specialist
PI on 3 active trials38 Embryonal tumor of neuroepithelial tissue publications
JG
John Grecula
COLUMBUS, OH
Specialist
PI on 1 active trial
JM
Joohee Sul, M.D.
ROCHESTER, NY
Specialist
PI on 3 active trials
WP
Wen-Ming Hsu, M.D, Ph.D
Specialist
PI on 1 active trial
NM
Nabil Ahmed, MD
HOUSTON, TX
Specialist
PI on 1 active trial
CM
Chirag G Patil, MD, MS
SAGINAW, MI
Specialist
PI on 1 active trial
DM
Douglas Ney, M.D
Specialist
PI on 1 active trial
GM
Gene Barnett, MD
PICKERINGTON, OH
Specialist
PI on 1 active trial1 Embryonal tumor of neuroepithelial tissue publication
DM
Diego Tosi, MD
Specialist
PI on 1 active trial
AM
Alain Herrera, MD
Specialist
PI on 1 active trial1 Embryonal tumor of neuroepithelial tissue publication
MP
Marjo Hahka-Kemppinen, MD, PhD
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Embryonal tumor of neuroepithelial tissue.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Embryonal tumor of neuroepithelial tissue

Disease timeline:

New recruiting trial: Investigating Mechanistic Predictors of Interpatient Variability and Temozolomide (TMZ) Induced Haematological Toxicity for Glioma Patients

A new clinical trial is recruiting patients for Embryonal tumor of neuroepithelial tissue

New recruiting trial: Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of the Oncolytic HSV1 MVR-C5252

A new clinical trial is recruiting patients for Embryonal tumor of neuroepithelial tissue

New recruiting trial: Trilateral Retinoblastoma: Incidence and Outcomes

A new clinical trial is recruiting patients for Embryonal tumor of neuroepithelial tissue

New recruiting trial: MRE Scan for the Assessment of Differences in Tissue Stiffness Between Radiation Necrosis and Recurrent Glioma in Patients With Previously Treated Gliomas

A new clinical trial is recruiting patients for Embryonal tumor of neuroepithelial tissue

New recruiting trial: 18F-Fluciclovine PET-MRI in High-grade Glioma

A new clinical trial is recruiting patients for Embryonal tumor of neuroepithelial tissue

New recruiting trial: Evaluation of Diagnostic Performances of 18F-FDOPA PET KInetics

A new clinical trial is recruiting patients for Embryonal tumor of neuroepithelial tissue

New recruiting trial: Feasibility of Aspirate Tissue Monitoring in Neuro-oncological Surgery

A new clinical trial is recruiting patients for Embryonal tumor of neuroepithelial tissue

New recruiting trial: Study of GC101 TIL in Brain Glioma (Soochow2)

A new clinical trial is recruiting patients for Embryonal tumor of neuroepithelial tissue

New recruiting trial: T2 Star Magnetic Resonance Imaging and Biomarker Blood Testing to Predict the Change and Progress of Malignant Gliomas

A new clinical trial is recruiting patients for Embryonal tumor of neuroepithelial tissue

New recruiting trial: Three miRNA Signatures in Glioma: From Molecular Mechanisms to Potential Clinical Application

A new clinical trial is recruiting patients for Embryonal tumor of neuroepithelial tissue

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 Embryonal tumor of neuroepithelial tissue

What is Embryonal tumor of neuroepithelial tissue?

Embryonal tumor of neuroepithelial tissue is a broad category of rare, aggressive central nervous system (CNS) tumors that arise from primitive or undifferentiated neuroepithelial cells. These tumors predominantly occur in children and include several subtypes such as embryonal tumor with multilayered rosettes (ETMR), medulloepithelioma, CNS neuroblastoma, CNS ganglioneuroblastoma, and atypical teratoid/rhabdoid tumor, among others. They primarily affect the brain and, less commonly, the spinal cord, and are classified as high-grade (WHO grade IV) malignancies due to their rapid growth and ten

How is Embryonal tumor of neuroepithelial tissue inherited?

Embryonal tumor of neuroepithelial tissue follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Embryonal tumor of neuroepithelial tissue typically begin?

Typical onset of Embryonal tumor of neuroepithelial tissue is childhood. Age of onset can vary across affected individuals.

Which specialists treat Embryonal tumor of neuroepithelial tissue?

25 specialists and care centers treating Embryonal tumor of neuroepithelial tissue are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.