Glioblastoma

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ORPHA:360OMIM:137800C71.9
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119Active trials41Specialists8Treatment centers

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

Abnormal nervous system physiologyHP:0012638Glioblastoma multiformeHP:0012174Abnormal cell morphologyHP:0025461Cerebral edemaHP:0002181ParalysisHP:0003470
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
Jul 2026Phase I Trial of pH Resections of GBM at VA

VA Office of Research and Development — PHASE1

TrialNOT YET RECRUITING
Jun 2026Pilot Study of Navigated Focused Ultrasound and Pembrolizumab in the Treatment of Recurrent WHO Grade 4 IDH-Wildtype Glioblastoma With Mismatch Repair Deficiency

Jennifer Leddon — PHASE1

TrialNOT YET RECRUITING
Jun 2026A Phase I First-in-human Trial of Bvax (B-cell Vaccination) in Addition to Standard of Care Chemoradiotherapy for Newly Diagnosed Glioblastoma

Catalina Lee Chang — PHASE1

TrialNOT YET RECRUITING
Apr 2026Novel Indenoisoquinolone CMYC/TOPOISOMERASE 1 Inhibitor (LMP744) in Recurrent Glioblastoma

National Institute of Neurological Disorders and Stroke (NINDS) — PHASE1, PHASE2

TrialNOT YET RECRUITING
Apr 2026A Phase Ib/II Clinical Trial to Evaluate the Safety and Efficacy of JL15003 Injection in Patients With Recurrent Glioblastoma (rGBM)

Jecho Biopharmaceuticals Co., Ltd. — PHASE1, PHASE2

TrialRECRUITING
Apr 2026Study of Silevertinib With Temozolomide for the Treatment of Newly Diagnosed GBM With Unmethylated MGMT and EGFRvIII

Black Diamond Therapeutics, Inc. — PHASE2

TrialRECRUITING
Mar 2026Combination ADI-PEG 20, TMZ, and RT for Treatment of Newly Diagnosed High-grade Glioma (HGG)

Sabine Mueller, MD, PhD — PHASE1, PHASE2

TrialNOT YET RECRUITING
Mar 2026Study of an AAV Mediated Dual-Payload Gene Therapy in Patients With High Grade Glioma

Trogenix ltd — PHASE1, PHASE2

TrialNOT YET RECRUITING
Mar 2026A Phase 1 Safety and Dose Finding Study of GLIX1 in Adults With Recurrent or Progressive High-grade Glioma

Tetragon Biosciences Ltd — PHASE1

TrialNOT YET RECRUITING
Feb 2026Pharmacoscopy for Patients With Refractory Primary Brain Tumors

University of Zurich — PHASE2

TrialNOT YET RECRUITING

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 →

Clinical Trials

20 recruitingView all trials with filters →
Phase 32 trials
Conventional Versus Hypofractionated Radiotherapy With Temozolomide in Elderly Glioblastoma
Phase 3
Actively Recruiting
PI: Chan Woo Wee, MD, PhD (Severance Hospital) · Sites: Seoul · Age: 7099 yrs
Repeated Superselective Intraarterial Cerebral Infusion (SIACI) of Bevacizumab With Temozolomide and Radiation Compared to Temozolomide and Radiation Alone in Newly Diagnosed GBM
Phase 3
Actively Recruiting
PI: John Boockvar, MD (Feinstein Institute for Medical Research/Lenox Hil) · Sites: New York, New York · Age: 1899 yrs
Phase 22 trials
A Study of BPM31510 With Vitamin K1 in Subjects With Newly Diagnosed Glioblastoma (GB)
Phase 2
Active
· Sites: Los Angeles, California; Palo Alto, California +9 more · Age: 1899 yrs
Enhanced Adjuvant Therapy for Newly Diagnosed GBM With Partial Surgical Resection or Short-term Progression
Phase 2
Actively Recruiting
· Sites: Hangzhou · Age: 1899 yrs
Other13 trials
The RESBIOP-study: Resection Versus Biopsy in High-grade Glioma Patients (ENCRAM 2202)
Actively Recruiting
PI: Jasper Gerritsen, MD PhD (Erasmus Medical Center) · Sites: San Francisco, California; Boston, Massachusetts +6 more · Age: 1890 yrs
Development and Implementation of an Intervention Enhancing Involvement of Relatives to Patients With Acquired Brain Injury or Malignant Brain Tumour
Actively Recruiting
· Sites: Copenhagen · Age: 1999 yrs
MRI Guided Radiotherapy and Radiobiological Data: the ISRAR Database (Irm Sequences for Radiobiological Adaptative Radiotherapy)
Actively Recruiting
· Sites: Lyon · Age: 1899 yrs
The SUPRAMAX Study: Supramaximal Resection Versus Maximal Resection for High-Grade Glioma Patients (ENCRAM 2201)
Actively Recruiting
PI: Jasper Gerritsen, MD PhD (Erasmus Medical Center) · Sites: San Francisco, California; Boston, Massachusetts +6 more · Age: 1890 yrs
The RECSUR-study: Resection Versus Best Oncological Treatment for Recurrent Glioblastoma (ENCRAM 2302)
Actively Recruiting
PI: Jasper Gerritsen, MD PhD (Erasmus Medical Center) · Sites: San Francisco, California; Boston, Massachusetts +6 more · Age: 1890 yrs
TTFields General Routine Clinical Care in ndGBM Patients: a French Prospective Non-Interventional Study (TIGER France)
Actively Recruiting
· Sites: Amiens; Avignon +22 more · Age: 1899 yrs
Clinical Significance of Liquid Biopsy in Brain Tumor Patients: a 5-ALA Guided Approach
Actively Recruiting
· Sites: Milan · Age: 1899 yrs
Collection of Blood and Urine Samples in Patients Receiving Radiation Therapy for Glioblastoma Multiforme
Actively Recruiting
PI: Kevin A Camphausen, M.D. (National Cancer Institute (NCI)) · Sites: Bethesda, Maryland · Age: 1899 yrs
The RECMAP-study: Resection With or Without Intraoperative Mapping for Recurrent Glioblastoma
Actively Recruiting
PI: Jasper Gerritsen, MD PhD (Erasmus Medical Center) · Sites: San Francisco, California; Boston, Massachusetts +6 more · Age: 090 yrs
The PALSUR-study: Palliative Care Versus Surgery in High-grade Glioma Patients (ENCRAM 2203)
Actively Recruiting
PI: Jasper Gerritsen, MD PhD (Erasmus Medical Center) · Sites: San Francisco, California; Boston, Massachusetts +6 more · Age: 1890 yrs
Analysis of Cerebrospinal Fluid Leakage After Surgery for Intracranial Tumors
Actively Recruiting
PI: Michal Senger, M.D. (Department of Neurosurgery, Medical University of ) · Sites: Warsaw · Age: 1899 yrs
Liquid Biopsy in Glioblastoma Treated With Chemoradiation and an Oxygen Therapeutic
Enrolling by Invitation
· Sites: Tucson, Arizona; New Haven, Connecticut +2 more · Age: 1899 yrs
Key Longitudinal Associations With Risk and Glioblastoma Outcomes
Actively Recruiting
PI: Quinn Ostrom, PhD (Duke University) · Sites: Durham, North Carolina · Age: 1899 yrs

Specialists

Showing 25 of 41View all specialists →
WL
Wenbin Li
Specialist
PI on 3 active trials628 Glioblastoma publications
XQ
Xiaoguang Qiu
Specialist
PI on 1 active trial109 Glioblastoma publications
EM
Elizabeth R Gerstner, MD
LIBERTY, MO
Specialist
PI on 2 active trials
WM
Wolfgang Wick, MD
Specialist
PI on 2 active trials1 Glioblastoma publication
AM
Ashley Ghiaseddin, MD
GAINESVILLE, FL
Specialist
PI on 3 active trials1 Glioblastoma publication
AM
Andrew Sloan, MD
Specialist
PI on 3 active trials1 Glioblastoma publication
TM
Tracy Batchelor, MD, MPH
BOSTON, MA
Specialist
PI on 5 active trials
DM
David Peereboom, MD
CLEVELAND, OH
Specialist
PI on 8 active trials
PN
Phioanh Nghiemphu
LOS ANGELES, CA
Specialist
PI on 2 active trials
JP
Jana Portnow
DUARTE, CA
Specialist
PI on 4 active trials
SP
Stanislaw R. Burzynski, MD, PhD
HOUSTON, TX
Specialist
PI on 29 active trials
JR
Jane Robertson
Specialist
PI on 3 active trials38 Glioblastoma publications
JG
John Grecula
COLUMBUS, OH
Specialist
PI on 1 active trial
PP
Paul Mulholland, MBBS, PhD
PEORIA, IL
Specialist
PI on 1 active trial
EM
Edward Pan, M.D.
ORLANDO, FL
Specialist
PI on 2 active trials
WD
Walter Stummer, Prof. Dr.
Specialist
PI on 2 active trials
JM
John A Boockvar, M.D.
BROOKLYN, NY
Specialist
PI on 1 active trial
FP
Francesco DiMeco, PI
Specialist
PI on 1 active trial
PM
Patrick Roth, MD
Specialist
PI on 1 active trial
CM
Chirag G Patil, MD, MS
SAGINAW, MI
Specialist
PI on 1 active trial
GM
Gene Barnett, MD
PICKERINGTON, OH
Specialist
PI on 1 active trial1 Glioblastoma publication

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 Glioblastoma.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Glioblastoma

2 articles
Clinical trialUNITERAREApr 4, 2026
New Recruiting Trial: A Phase Ib/II Clinical Trial to Evaluate the Safety and Efficacy of JL15003 Injection in Patients With Recurrent Glioblastoma (rGBM)
Researchers are testing a new drug called JL15003 to see if it can safely treat glioblastoma, a serious brain cancer that has come back after previous treatment
Clinical trialUNITERAREApr 3, 2026
New Recruiting Trial: Study of Silevertinib With Temozolomide for the Treatment of Newly Diagnosed GBM With Unmethylated MGMT and EGFRvIII
Researchers are looking for patients with a specific type of brain cancer called glioblastoma (GBM) to test a new drug combination. The study will test a drug c
See all news about Glioblastoma

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 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.