Overview
Glioblastoma (GBM), also known as glioblastoma multiforme or grade 4 astrocytoma, is the most aggressive and most common primary malignant brain tumor in adults. Classified by the World Health Organization as a grade 4 glioma, it arises from astrocytes, the supportive glial cells of the central nervous system. Glioblastoma primarily affects the brain, most commonly the cerebral hemispheres, and is characterized by rapid, infiltrative growth into surrounding brain tissue, areas of necrosis, and robust new blood vessel formation (neovascularization). Key molecular subtypes include IDH-wildtype glioblastoma (the vast majority of cases) and, less commonly, tumors with IDH mutations, which carry a somewhat better prognosis. Symptoms depend on tumor location and size and may include persistent or worsening headaches, seizures, progressive neurological deficits such as weakness or numbness on one side of the body, speech difficulties, cognitive and personality changes, vision problems, and nausea or vomiting due to increased intracranial pressure. Symptoms often develop rapidly over days to weeks, reflecting the tumor's aggressive growth pattern. The current standard of care for newly diagnosed glioblastoma involves maximal safe surgical resection followed by concurrent radiation therapy and temozolomide chemotherapy (the Stupp protocol), followed by adjuvant temozolomide. Tumor treating fields (TTFields), a device-based therapy delivering alternating electric fields to the brain, may also be used in combination with temozolomide. Methylation of the MGMT gene promoter is an important biomarker that predicts better response to temozolomide. Despite multimodal treatment, prognosis remains poor, with a median overall survival of approximately 14 to 16 months and a five-year survival rate of less than 10%. Most cases are sporadic, though rare familial clustering has been observed, and prior ionizing radiation exposure is the only well-established environmental risk factor. Ongoing research includes immunotherapy, targeted molecular therapies, and novel drug delivery approaches.
Also known as:
Clinical phenotype terms— hover any for plain English:
Sporadic
Usually appears on its own, not inherited from a parent
Adult
Begins in adulthood (age 18 or older)
FDA & Trial Timeline
10 eventsVA Office of Research and Development — PHASE1
Jennifer Leddon — PHASE1
Catalina Lee Chang — PHASE1
National Institute of Neurological Disorders and Stroke (NINDS) — PHASE1, PHASE2
Jecho Biopharmaceuticals Co., Ltd. — PHASE1, PHASE2
Black Diamond Therapeutics, Inc. — PHASE2
Sabine Mueller, MD, PhD — PHASE1, PHASE2
Trogenix ltd — PHASE1, PHASE2
Tetragon Biosciences Ltd — PHASE1
University of Zurich — PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Glioblastoma.
20 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →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 Glioblastoma.
Community
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Start the conversation →Latest news about Glioblastoma
2 articlesCaregiver 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 Glioblastoma
What is Glioblastoma?
Glioblastoma (GBM), also known as glioblastoma multiforme or grade 4 astrocytoma, is the most aggressive and most common primary malignant brain tumor in adults. Classified by the World Health Organization as a grade 4 glioma, it arises from astrocytes, the supportive glial cells of the central nervous system. Glioblastoma primarily affects the brain, most commonly the cerebral hemispheres, and is characterized by rapid, infiltrative growth into surrounding brain tissue, areas of necrosis, and robust new blood vessel formation (neovascularization). Key molecular subtypes include IDH-wildtype g
How is Glioblastoma inherited?
Glioblastoma follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Glioblastoma typically begin?
Typical onset of Glioblastoma is adult. Age of onset can vary across affected individuals.
Are there clinical trials for Glioblastoma?
Yes — 20 recruiting clinical trials are currently listed for Glioblastoma on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Glioblastoma?
25 specialists and care centers treating Glioblastoma are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.