Central Nervous System Neoplasms
Recent clinical, regulatory, research and industry developments relating to this disease.
Adolescents and Young Adults With Cancer: CNS Tumors.
Prognostic and Predictive Biomarkers in Gliomas.
Central Nervous System Cancers, Version 3.2020, NCCN Clinical Practice Guidelines in Oncology.
The 2007 WHO classification of tumours of the central nervous system.
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 Q3 2026.
- Q3 2026Phase 1 Trial of Engineered HSV G207 in Children With Recurrent or Refractory Cerebellar Brain 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
- Q1 2035LOC-R01: Randomized Phase IB/II Study of Escalating Doses of Lenalidomide and Ibrutinib in Association With R-MPV as a Targeted Induction Treatment for Patients Aged 18 to 60 (up to 65 for Phase II) With a Newly Diagnosed Primary Central Nervous System Lymphoma
Clinical MilestonesViewHide
- 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
- 2026-05-15Phase 1 Trial of Engineered HSV G207 in Children With Recurrent or Refractory Cerebellar Brain TumorsResults expected Q3 2026
- 2025-09-25LOC-R01: Randomized Phase IB/II Study of Escalating Doses of Lenalidomide and Ibrutinib in Association With R-MPV as a Targeted Induction Treatment for Patients Aged 18 to 60 (up to 65 for Phase II) With a Newly Diagnosed Primary Central Nervous System LymphomaResults expected Q1 2035
- 2026-07-01A First-in-human Phase I Single-agent Dose-escalation, Food Effect and Dose Expansion Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System NeoplasmsPrimary completion
- 2026-06-04Loc3CAR: Locoregional Delivery of B7-H3-specific Chimeric Antigen Receptor Autologous T Cells for Pediatric Patients With Primary CNS TumorsPrimary completion
- 2026-07-01ClinicalA First-in-human Phase I Single-agent Dose-escalation, Food Effect and Dose Expansion Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System NeoplasmsPrimary 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
- 2026-06-04ClinicalLoc3CAR: Locoregional Delivery of B7-H3-specific Chimeric Antigen Receptor Autologous T Cells for Pediatric Patients With Primary CNS TumorsPrimary completion
- 2026-05-15ClinicalPhase 1 Trial of Engineered HSV G207 in Children With Recurrent or Refractory Cerebellar Brain TumorsResults expected Q3 2026
- 2025-09-25ClinicalLOC-R01: Randomized Phase IB/II Study of Escalating Doses of Lenalidomide and Ibrutinib in Association With R-MPV as a Targeted Induction Treatment for Patients Aged 18 to 60 (up to 65 for Phase II) With a Newly Diagnosed Primary Central Nervous System LymphomaResults expected Q1 2035
- 2025-08-11ClinicalA Phase II Trial of Oral Pazopanib Plus Oral Topotecan Metronomic Antiangiogenic Therapy for Recurrent Glioblastoma Multiforme (A)Without Prior Bevacizumab Exposure and (B) After Failing Prior BevacizumabResults posted
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes8
- Central Nervous System Neoplasms4
- Glioma4
- Brain Neoplasms2
- Astrocytoma1
- Biomarkers, Tumor1
- Cerebellar Neoplasms1
- Ependymoma1
- Medulloblastoma1
Leading journals6
- Neuro-oncology4
- Acta neuropathologica1
- Brain pathology (Zurich, Switzerland)1
- Cancer1
- International journal of molecular sciences1
- Journal of clinical oncology : official journal of the American Society of Clinical Oncology1
Leading researchers8
- Kruchko C4
- Barnholtz-Sloan JS3
- Ostrom QT3
- Gittleman H2
- Louis DN2
- Stroup NE2
- Ahluwalia M1
- Aldape K1
Affiliations (unnormalised)6
- Case Comprehensive Cancer Center2
- Dan L. Duncan Comprehensive Cancer Center2
- 10Roswell Park Comprehensive Cancer Center.1
- 10th Military Research Hospital and Polyclinic1
- 11Moffitt Cancer Center.1
- 12UC San Diego Moores Cancer Center.1
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.
Central nervous system neoplasms are benign or malignant tumors that arise in, or secondarily involve, the brain, spinal cord, or meninges. The literature emphasizes that this is a broad disease category encompassing many distinct tumor types, with classification increasingly based on both histology and molecular features.
The supplied grounding does not support a single unified cause for central nervous system neoplasms. It does indicate that some entities are associated with specific genetic profiles and that familial tumor syndromes, including rhabdoid tumor predisposition syndrome, can involve the nervous system.
These neoplasms reflect abnormal neoplastic growth within central nervous system tissues, with biological behavior varying by tumor type and grade. The literature highlights that molecular alterations are now integrated into classification because they help define tumor identity, pathophysiology, and clinical behavior.
The supplied grounding does not support general risk factors for all central nervous system neoplasms. It does note familial tumor syndromes involving the nervous system and tumor-specific genetic alterations as relevant to classification and prognosis.
Management is described at the level of diagnosis, therapy, and drug therapy, with treatment depending on tumor type, grade, and molecular features. The literature supports multimodal neuro-oncology care, including surgery and other tumor-directed therapies, and for malignant tumors it emphasizes molecularly informed management and clinical trial participation where appropriate.
AI-generated summary grounded in MeSH and 5 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
Benign and malignant neoplastic processes that arise from or secondarily involve the brain, spinal cord, or meninges.
- 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.