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Disease

Brain Neoplasms

Late-stage therapeutic developmentExtensively researchedRising momentum
114
Publications
17
Clinical trials
12
Related conditions
5
Related treatments
10
Related proteins
2025
Latest publication
Current focus
Isocitrate dehydrogenase biologyTherapeutic developmentTrastuzumab biologyGenetics & risk factorsInflammation & immunity
Latest activity
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Recent clinical, regulatory, research and industry developments relating to this disease.

Gut microbiota and their influence in brain cancer milieu.

Research2025-05-01Journal of neuroinflammation

Breaking boundaries: role of the brain barriers in metastatic process.

Research2025-01-08Fluids and barriers of the CNS

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 5 clinical trials expected to report results, the earliest in Q3 2026.
  • Active recent publication activity.
Research Highlights4View
Clinical Milestones6View
Results expected 5
Activity timeline10

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Carmustineapproved

Approval — Carmustine is indicated n adults in the following malignant neoplasms as a single ag… (2018)

Clinical trials

16 sponsors · 1 new · 0 completed in the last 12 months (net +1)

The current development programme across all trial phases.

Clinical programme
17
All trials
6
Active
7
Late-stage
5
Completed
Late-stage studies
Recruiting
Recently completed

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2018emaApprovalCarmustine· Carmustine is indicated n adults in the following malignant neoplasms as a single agent or in combination with other antineoplastic agents and/or other therapeutic measures (radiotherapy, surgery): Brain tumours (glioblastoma, brain-stem gliomas, medulloblastoma, astrocytoma and ependymoma), brain metastases Secondary therapy in non-Hodgkin’s lymphoma and Hodgkin’s disease as conditioning treatment prior to autologous haematopoietic progenitor cell transplantation (HPCT) in malignant haematological diseases (Hodgkin’s disease / Non-hodgkin’s lymphoma). source ↗

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

114 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
20132025
Recent publications
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

10 matches

Key proteins & gene products studied in this disease. Number shows shared papers.

Related conditions

12 matches

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.

Overview

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.

Causes

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.

Pathophysiology

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.

Risk factors

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.

Current standard of care

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.

Defined in MeSH

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.

References & data sources
  • 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.