Glioblastoma
Recent clinical, regulatory, research and industry developments relating to this disease.
CHMP positive opinion: Kizfizo (EMA)
A Phase I Trial of Anti-Tim-3 in Combination With Anti-PD-1 and SRS in Recurrent GBM
A Pilot Study of APG-157 With Bevacizumab for Patients With Recurrent High-Grade Glioma
Neoadjuvant triplet immune checkpoint blockade in newly diagnosed glioblastoma.
The Impact of Metabolic Rewiring in Glioblastoma: The Immune Landscape and Therapeutic Strategies.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 3 clinical trials expected to report results, the earliest in Q4 2026.
- 7 industry developments reported.
- Q4 2026An Open Label, Multi-center Roll-over Study to Assess Long-term Effect in Pediatric Patients Treated With Tafinlar (Dabrafenib) and/or Mekinist (Trametinib)
- Q4 2027A Phase 3, Open-label, Randomized 2-arm Study Comparing the Clinical Efficacy and Safety of Niraparib With Temozolomide in Adult Participants With Newly-diagnosed, MGMT Unmethylated Glioblastoma
- Q4 2030Randomized Phase 2b Study of Safety And Efficacy Of TVI-Brain-1 Combined With Conformal Radiotherapy And Temozolomide Vs Standard Therapy In Newly Diagnosed MGMT Negative Glioblastoma Multiforme (GBM)
Clinical MilestonesView all 12Hide
- 2026-06-29Tofacitinib: Suppressing Tumor Invasion in Recurrent GBM PatientsResults posted
- 2026-04-22A Randomized Phase II/III Open-Label Study of Ipilimumab and Nivolumab Versus Temozolomide in Patients With Newly Diagnosed MGMT (Tumor O-6-Methylguanine DNA Methyltransferase) Unmethylated GlioblastomaResults posted
- 2025-11-21Safety and Efficacy Study of Retifanlimab and Epacadostat in Combination With Radiation and Bevacizumab in Patients With Recurrent GliomasResults posted
- 2026-09-29Randomized Phase 2b Study of Safety And Efficacy Of TVI-Brain-1 Combined With Conformal Radiotherapy And Temozolomide Vs Standard Therapy In Newly Diagnosed MGMT Negative Glioblastoma Multiforme (GBM)Results expected Q4 2030
- 2026-08-27An Open Label, Multi-center Roll-over Study to Assess Long-term Effect in Pediatric Patients Treated With Tafinlar (Dabrafenib) and/or Mekinist (Trametinib)Results expected Q4 2026
- 2026-07-01A Phase 3, Open-label, Randomized 2-arm Study Comparing the Clinical Efficacy and Safety of Niraparib With Temozolomide in Adult Participants With Newly-diagnosed, MGMT Unmethylated GlioblastomaResults expected Q4 2027
- 2026-09-02Efficacy and Safety Study of Neoadjuvant Efineptakin Alfa (NT-I7) and Pembrolizumab in Recurrent GlioblastomaPrimary completion
- 2026-02-12A Phase III Trial of Lomustine-Temozolomide Combination Therapy Versus Standard Temozolomide in Patients With Methylated MGMT Promoter GlioblastomaPrimary completion
- 2030-11-30A Phase II/III Randomized, Open-Label Study of Toca 511, A Retroviral Replicating Vector, Combined With Toca FC With Temozolomide and Radiation Followed by Adjuvant Temozolomide and Toca FC Compared to Temozolomide and Radiation Followed by Adjuvant Temozolomide in Patients With Newly Diagnosed GlioblastomaWithdrawn
- 2026-04-08Evaluation of the Feasibility of PD L 506 for Stereotactic Interstitial Photodynamic Therapy (iPDT) in Adult Patients With Newly Diagnosed Supratentorial IDH Wild-type GlioblastomaTerminated
- 2025-12-18Phase II Clinical Trial of HSV G207 With a Single 5 Gy Radiation Dose in Children With Recurrent High-Grade GliomaWithdrawn
- 2025-12-01Phase 2 Study of Ropidoxuridine as a Radiation Sensitizing Agent During Radiotherapy in Patients With Newly Diagnosed IDH-Wildtype Glioblastoma With Unmethylated MGMT PromoterTerminated
Industry & MarketView all 7Hide
- 2026-11-02PTK7-targeted gene therapy shows promise in glioblastomaIndustry · BioWorld Gene Therapy
- 2026-10-24Glioblastoma breakthrough reveals how brain cancer cells become more aggressiveIndustry · Science Daily
- 2026-10-09Earlier Use of TTFields Misses Mark in GlioblastomaIndustry · Medscape Medical News
- 2026-10-07MimiVax's cancer vaccine for glioblastoma narrowly misses Phase 2b goalIndustry · Endpoints News
- 2026-10-02Glioblastoma treatment gelsIndustry · Nature Neuroscience
Regulatory UpdatesViewHide
- 2026-10-16CHMP positive opinion — TemozolomideTreatment of adults with glioblastoma multiforme and children with malignant glioma
- 2030-11-30ClinicalA Phase II/III Randomized, Open-Label Study of Toca 511, A Retroviral Replicating Vector, Combined With Toca FC With Temozolomide and Radiation Followed by Adjuvant Temozolomide and Toca FC Compared to Temozolomide and Radiation Followed by Adjuvant Temozolomide in Patients With Newly Diagnosed GlioblastomaWithdrawn
- 2026-11-02IndustryPTK7-targeted gene therapy shows promise in glioblastomaIndustry · BioWorld Gene Therapy
- 2026-10-24IndustryGlioblastoma breakthrough reveals how brain cancer cells become more aggressiveIndustry · Science Daily
- 2026-10-16RegulatoryCHMP positive opinion — TemozolomideTreatment of adults with glioblastoma multiforme and children with malignant glioma
- 2026-10-09IndustryEarlier Use of TTFields Misses Mark in GlioblastomaIndustry · Medscape Medical News
- 2026-10-07IndustryMimiVax's cancer vaccine for glioblastoma narrowly misses Phase 2b goalIndustry · Endpoints News
- 2026-10-02IndustryGlioblastoma treatment gelsIndustry · Nature Neuroscience
- 2026-09-29ClinicalRandomized Phase 2b Study of Safety And Efficacy Of TVI-Brain-1 Combined With Conformal Radiotherapy And Temozolomide Vs Standard Therapy In Newly Diagnosed MGMT Negative Glioblastoma Multiforme (GBM)Results expected Q4 2030
- 2026-09-02ClinicalEfficacy and Safety Study of Neoadjuvant Efineptakin Alfa (NT-I7) and Pembrolizumab in Recurrent GlioblastomaPrimary completion
- 2026-08-27ClinicalAn Open Label, Multi-center Roll-over Study to Assess Long-term Effect in Pediatric Patients Treated With Tafinlar (Dabrafenib) and/or Mekinist (Trametinib)Results expected Q4 2026
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Generic approval — Carmustine is indicated n adults in the following malignant neoplasms as a single ag… (2018)
Generic approval — Temozolomide Sun is indicated for the treatment of: adult patients with newly diagnosed… (2011)
Clinical trials
The current development programme across all trial phases.
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes8
- Glioblastoma30
- Brain Neoplasms24
- Immune Checkpoint Inhibitors7
- Tumor Microenvironment5
- Glioma4
- Neoplasm Recurrence, Local4
- Bevacizumab2
- Cancer Vaccines2
Leading journals6
- Nature communications7
- Nature medicine4
- Neuro-oncology4
- The New England journal of medicine4
- Frontiers in immunology3
- The Journal of clinical investigation3
Leading researchers8
- Reardon DA5
- Zhang J5
- Heiland DH4
- Petrecca K4
- Weller M4
- Cloughesy TF3
- Delev D3
- Heimberger AB3
Affiliations (unnormalised)6
- Center for Neuro-Oncology4
- Department of Neurology and Brain Tumor Center4
- Sorbonne Université4
- Brigham and Women's Hospital3
- Dana-Farber Cancer Institute3
- Institute of Neuropathology3
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Disease biology
Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Disease profile
A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.
Glioblastoma is a malignant astrocytoma and the most common form of malignant primary brain tumor in adults. It is histologically marked by pleomorphism, nuclear atypia, microhemorrhage, and necrosis, and it can arise in the central nervous system with a predilection for the cerebral hemispheres, basal ganglia, and commissural pathways. Clinical presentation most often occurs in the fifth or sixth decade of life with focal neurologic signs or seizures.
The supplied grounding supports a molecularly complex disease rather than a single cause. Review abstracts emphasize genetic and epigenetic alterations, including IDH-wildtype biology, DNA methylation changes, and genomic reprogramming, but do not identify one definitive aetiology. No specific environmental or inherited cause is supported in the grounding.
Glioblastoma is characterized by aggressive tumor growth, marked cellular atypia, necrosis, and prominent angiogenesis. The literature grounding also describes a highly immunosuppressive tumor microenvironment, tumor heterogeneity, resistance-associated stem-like cells, and involvement of developmental pathways, DNA damage response, metabolism, and signal transduction. These features contribute to recurrence and therapeutic resistance.
The grounding supports adult age at presentation, most often in the fifth or sixth decade of life. It also indicates that glioblastoma is the most common malignant primary brain tumor in adults and is discussed as IDH-wildtype in current management reviews, but it does not provide additional validated risk factors. No other risk factors are supported by the supplied material.
Current standard management is feasible surgical resection followed by radiotherapy plus temozolomide chemotherapy. The supplied reviews also discuss targeted molecular therapies, agents targeting DNA damage response and metabolism, immunotherapies, viral therapies, and anti-angiogenesis approaches as emerging or investigational modalities. Bevacizumab and immune checkpoint blockade are among the co-studied treatment-related entities, but the grounding does not support them as universal standard therapy.
AI-generated summary grounded in MeSH and 6 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
A malignant form of astrocytoma histologically characterized by pleomorphism of cells, nuclear atypia, microhemorrhage, and necrosis. They may arise in any region of the central nervous system, with a predilection for the cerebral hemispheres, basal ganglia, and commissural pathways. Clinical presentation most frequently occurs in the fifth or sixth decade of life with focal neurologic signs or seizures.
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.