Brain Neoplasms
Recent clinical, regulatory, research and industry developments relating to this disease.
Cancer neuroscience of brain metastasis: When in Rome, do as the Romans do.
Gut microbiota and their influence in brain cancer milieu.
Neurotransmitter power plays: the synaptic communication nexus shaping brain cancer.
Hormonal and neuronal interactions shaping the brain metastatic microenvironment.
Cytogenetic signatures favoring metastatic organotropism in colorectal cancer.
Neoadjuvant triplet immune checkpoint blockade in newly diagnosed glioblastoma.
The Impact of Metabolic Rewiring in Glioblastoma: The Immune Landscape and Therapeutic Strategies.
Breaking boundaries: role of the brain barriers in metastatic process.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 5 clinical trials expected to report results, the earliest in Q3 2026.
- Active recent publication activity.
- Q3 2026A Phase I, Multicenter Study to Assess the Safety, Tolerability, and Pharmacokinetics of Ascending Doses of AZD1390 in Combination With Radiation Therapy in Patients With Glioblastoma Multiforme and Brain Metastases From Solid Tumors
- Q4 2026An Open-label Multicenter Phase 1b-2 Study of Elacestrant in Combination With Abemaciclib in Women and Men With Brain Metastasis From Estrogen Receptor Positive, HER-2 Negative Breast Cancer
- Q3 2027A PHASE 1A/B OPEN-LABEL MASTER STUDY OF PF-07799544 AS A SINGLE-AGENT AND IN COMBINATION WITH OTHER TARGETED AGENTS IN PARTICIPANTS WITH BRAF-MUTANT MELANOMA AND OTHER SOLID TUMORS
- 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 2029DETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial): An Umbrella-Basket Platform Trial to Evaluate the Efficacy of Targeted Therapies in Rare Adult, Paediatric and Teenage/Young Adult (TYA) Cancers With Actionable Genomic Alterations, Including Common Cancers With Rare Actionable Alterations. Treatment Arm 03: Entrectinib in Adult, Paediatric and Teenage/Young Adult Patients With ROS1 Gene Fusion-Positive Cancers.
Research HighlightsViewHide
- 2025-08-22Brain Tumors, AI and Psychiatry: Predicting Tumor-Associated Psychiatric Syndromes with Machine Learning and Biomarkers.Șerban M · 2025
- 2025-11-10Artificial intelligence-driven intelligent nanocarriers for cancer theranostics: A paradigm shift with focus on brain tumors.Pourmadadi M · 2025
- 2025-09-01Cancer neuroscience of brain metastasis: When in Rome, do as the Romans do.Karreman MA · 2025
- 2025-11-01FDA Approval Summary: Vorasidenib for IDH-Mutant Grade 2 Astrocytoma or Oligodendroglioma Following Surgery.Barbato MI · 2025
Clinical MilestonesViewHide
- 2026-07-09A PHASE 1A/B OPEN-LABEL MASTER STUDY OF PF-07799544 AS A SINGLE-AGENT AND IN COMBINATION WITH OTHER TARGETED AGENTS IN PARTICIPANTS WITH BRAF-MUTANT MELANOMA AND OTHER SOLID TUMORSResults expected Q3 2027
- 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
- 2026-06-04An Open-label Multicenter Phase 1b-2 Study of Elacestrant in Combination With Abemaciclib in Women and Men With Brain Metastasis From Estrogen Receptor Positive, HER-2 Negative Breast CancerResults expected Q4 2026
- 2025-12-22A Phase I, Multicenter Study to Assess the Safety, Tolerability, and Pharmacokinetics of Ascending Doses of AZD1390 in Combination With Radiation Therapy in Patients With Glioblastoma Multiforme and Brain Metastases From Solid TumorsResults expected Q3 2026
- 2025-11-24DETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial): An Umbrella-Basket Platform Trial to Evaluate the Efficacy of Targeted Therapies in Rare Adult, Paediatric and Teenage/Young Adult (TYA) Cancers With Actionable Genomic Alterations, Including Common Cancers With Rare Actionable Alterations. Treatment Arm 03: Entrectinib in Adult, Paediatric and Teenage/Young Adult Patients With ROS1 Gene Fusion-Positive Cancers.Results expected Q4 2029
- 2026-07-09ClinicalA PHASE 1A/B OPEN-LABEL MASTER STUDY OF PF-07799544 AS A SINGLE-AGENT AND IN COMBINATION WITH OTHER TARGETED AGENTS IN PARTICIPANTS WITH BRAF-MUTANT MELANOMA AND OTHER SOLID TUMORSResults expected Q3 2027
- 2026-07-01ClinicalA 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
- 2026-06-30ClinicalA Phase I Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of JYP0322 in Patients With Solid TumorsPrimary completion
- 2026-06-04ClinicalAn Open-label Multicenter Phase 1b-2 Study of Elacestrant in Combination With Abemaciclib in Women and Men With Brain Metastasis From Estrogen Receptor Positive, HER-2 Negative Breast CancerResults expected Q4 2026
- 2025-12-22ClinicalA Phase I, Multicenter Study to Assess the Safety, Tolerability, and Pharmacokinetics of Ascending Doses of AZD1390 in Combination With Radiation Therapy in Patients With Glioblastoma Multiforme and Brain Metastases From Solid TumorsResults expected Q3 2026
- 2025-11-24ClinicalDETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial): An Umbrella-Basket Platform Trial to Evaluate the Efficacy of Targeted Therapies in Rare Adult, Paediatric and Teenage/Young Adult (TYA) Cancers With Actionable Genomic Alterations, Including Common Cancers With Rare Actionable Alterations. Treatment Arm 03: Entrectinib in Adult, Paediatric and Teenage/Young Adult Patients With ROS1 Gene Fusion-Positive Cancers.Results expected Q4 2029
- 2025-11-10ResearchArtificial intelligence-driven intelligent nanocarriers for cancer theranostics: A paradigm shift with focus on brain tumors.Pourmadadi M · 2025
- 2025-11-01ResearchFDA Approval Summary: Vorasidenib for IDH-Mutant Grade 2 Astrocytoma or Oligodendroglioma Following Surgery.Barbato MI · 2025
- 2025-09-01ResearchCancer neuroscience of brain metastasis: When in Rome, do as the Romans do.Karreman MA · 2025
- 2025-08-22ResearchBrain Tumors, AI and Psychiatry: Predicting Tumor-Associated Psychiatric Syndromes with Machine Learning and Biomarkers.Șerban M · 2025
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approval — Carmustine is indicated n adults in the following malignant neoplasms as a single ag… (2018)
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
- Brain Neoplasms70
- Glioblastoma23
- Glioma11
- Tumor Microenvironment11
- Immune Checkpoint Inhibitors7
- Lung Neoplasms7
- Breast Neoplasms5
- Carcinoma, Non-Small-Cell Lung4
Leading journals6
- Neuro-oncology12
- Nature communications11
- International journal of molecular sciences6
- Frontiers in immunology5
- Nature medicine5
- The New England journal of medicine4
Leading researchers8
- Barnholtz-Sloan JS6
- Weller M6
- Curigliano G5
- Heiland DH5
- Reardon DA5
- Wen PY5
- Zhang J5
- Kruchko C4
Affiliations (unnormalised)6
- Brigham and Women's Hospital7
- Center for Neuro-Oncology7
- Dana-Farber Cancer Institute7
- Mayo Clinic6
- University of California6
- Department of Pathology and Laboratory Medicine5
Disease biology
Key proteins & gene products studied in this disease. Number shows shared papers.
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.
Brain neoplasms are tumors arising within the intracranial components of the central nervous system, including the cerebral hemispheres, basal ganglia, hypothalamus, thalamus, brain stem, and cerebellum. They include primary tumors that originate in brain tissue and secondary tumors that represent metastases to the brain. Primary brain neoplasms may be benign or malignant, and they are also classified by age of onset, histologic type, and location.
Brain neoplasms are divided into primary and secondary forms, with secondary disease arising from metastatic spread from solid tumors. For primary gliomas, the literature identifies a subset caused by Mendelian disorders, including neurofibromatosis, tuberous sclerosis, and Li-Fraumeni syndrome. Genomic analyses have also identified heritable risk alleles and molecular biomarkers associated with glioma risk and prognosis.
The literature emphasizes malignant progression, widespread invasion throughout the brain, and destruction of normal brain tissue, particularly in malignant astrocytic gliomas such as glioblastoma. Reported biological themes include altered signal transduction, gene expression regulation, transcriptome changes, DNA methylation, DNA mismatch repair, tumor microenvironment effects, tumor escape, and cell differentiation. Molecular features discussed in glioma include isocitrate dehydrogenase mutation and O6-methylguanine-DNA methyltransferase methylation, and immune-related mechanisms are also relevant through tumor-infiltrating lymphocytes and immunotherapy research.
Reported risk factors for glioma include Mendelian disorders such as neurofibromatosis, tuberous sclerosis, and Li-Fraumeni syndrome. Genome-wide association studies have identified heritable risk alleles associated with increased glioma risk. Molecular markers such as IDH mutation and MGMT methylation are discussed in the literature as biomarkers of prognosis rather than established general risk factors.
For brain metastases, surgery is a reasonable option, particularly for large tumors with mass effect, and symptomatic brain metastases should receive local therapy. The literature also discusses local therapy as the main approach for brain metastases, with deferral only in selected situations. For glioblastoma and other malignant primary brain tumors, current management includes targeted molecular therapies, agents targeting DNA damage response and metabolism, immunotherapies, and viral therapies, although outcomes remain poor.
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.
Neoplasms of the intracranial components of the central nervous system, including the cerebral hemispheres, basal ganglia, hypothalamus, thalamus, brain stem, and cerebellum. Brain neoplasms are subdivided into primary (originating from brain tissue) and secondary (i.e., metastatic) forms. Primary neoplasms are subdivided into benign and malignant forms. In general, brain tumors may also be classified by age of onset, histologic type, or presenting location in the brain.
- 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.