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A neuroglial tumor that arises from the brain stem.
Biomarker and diagnostic research for brain stem glioma has been reported in the published literature.
1 FDA-approved treatment is available for brain stem glioma, including CARMUSTINE (CARMUSTINE, approved 1977). An additional 1 compound holds orphan drug designation.
Brand Name | Generic Name | Mechanism | Approved | Market Status |
|---|---|---|---|---|
8 clinical trials registered, 3 recruiting. Interventions under study include drug therapy, other interventions, procedural interventions, and medical devices. Pipeline includes 2 PHASE3, 1 PHASE2, 3 PHASE1. Research is primarily sponsored by academic and government institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT00840047](https://clinicaltrials.gov/study/NCT00840047) |
Data assembled from 4 of 12 sources · Last updated Sep 17, 2026, 11:25 PM UTC
CARMUSTINE
CARMUSTINE |
— |
1977 |
Available |
The following drugs have received orphan drug designation from the FDA for brain stem glioma. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor | Designated | Exclusivity End | Designation Status |
|---|---|---|---|---|---|
A10 & AS2-1 Antineoplaston | A10 & AS2-1 Antineoplaston | Burzynski Research Institute, Inc. | 2004 | — | Designated |
Gene therapy approaches for brain stem glioma have been reported in the published literature.
8 trials found
Methionine PET/CT Studies In Patients With Cancer |
PHASE2 |
St. Jude Children's Research Hospital |
ACTIVE_NOT_RECRUITING |
[NCT02887040](https://clinicaltrials.gov/study/NCT02887040) | Study of Antineoplaston Therapy + Radiation vs. Radiation Only in Diffuse, Intrinsic, Brainstem Glioma | PHASE3 | Burzynski Research Institute | UNKNOWN |
[NCT02864888](https://clinicaltrials.gov/study/NCT02864888) | Effect of Antineoplaston Therapy on the QT/QTc Interval In Subjects With Diffuse, Intrinsic, Brainstem Glioma | PHASE3 | Burzynski Research Institute | UNKNOWN |
[NCT05857969](https://clinicaltrials.gov/study/NCT05857969) | Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling | PHASE1 | Florida International University | RECRUITING |
[NCT04196413](https://clinicaltrials.gov/study/NCT04196413) | GD2 CAR T Cells in Diffuse Intrinsic Pontine Gliomas (DIPG) & Spinal Diffuse Midline Glioma(DMG) | PHASE1 | Stanford University | RECRUITING |
190 publications have been identified in PubMed for brain stem glioma. Research spans Basic Science / Preclinical (53%), Gene Therapy / Novel Therapeutics (18%), and Review / Meta-Analysis (14%).
Research Type | Count | % of Total |
|---|---|---|
Laboratory research | 101 | 53% |
New treatment approaches | 35 | 18% |
Research summaries | 26 | 14% |
Clinical study results | 10 | 5% |
Testing and diagnosis research | 8 | 4% |
Disease patterns and progression | 7 | 4% |
Patient case studies | 2 | 1% |
Other research | 1 | 1% |
Levine AB (2026). [PMID: 41117845](https://pubmed.ncbi.nlm.nih.gov/41117845/). *Neuro Oncol*. [Basic Science / Preclinical]
Cheng H (2026). [PMID: 41308767](https://pubmed.ncbi.nlm.nih.gov/41308767/). *Int J Biol Macromol*. [Basic Science / Preclinical]
Mikolajewicz N (2026). [PMID: 41570802](https://pubmed.ncbi.nlm.nih.gov/41570802/). *Neuron*. [Basic Science / Preclinical]
Zhou R (2026). [PMID: 41239946](https://pubmed.ncbi.nlm.nih.gov/41239946/). *Phytother Res*. [Basic Science / Preclinical]
Vaquié AM (2026). [PMID: 40975876](https://pubmed.ncbi.nlm.nih.gov/40975876/). *Mol Oncol*. [Gene Therapy / Novel Therapeutics]
Li J (2026). [PMID: 41061166](https://pubmed.ncbi.nlm.nih.gov/41061166/). *Annual review of pathology*. [Basic Science / Preclinical]
Horschitz S (2026). [PMID: 40188875](https://pubmed.ncbi.nlm.nih.gov/40188875/). *Journal of advanced research*. [Basic Science / Preclinical]
Jiang Z (2026). [PMID: 40973093](https://pubmed.ncbi.nlm.nih.gov/40973093/). *Neuro-oncology*. [Gene Therapy / Novel Therapeutics]
Guo M (2026). [PMID: 41507172](https://pubmed.ncbi.nlm.nih.gov/41507172/). *Nature communications*. [Basic Science / Preclinical]
Ding C (2026). [PMID: 41747254](https://pubmed.ncbi.nlm.nih.gov/41747254/). *Cancer Discov*. [Basic Science / Preclinical]