Central nervous system neoplasm

Summary

Central nervous system neoplasm (MONDO:0006130) is a cancer (an umbrella term covering 10 Mondo subtypes) and 141 clinical trials. Top therapeutic interventions include 2-mercaptoethanesulfonic acid, lomustine, and cabozantinib. A subtype of central nervous system disorder — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

• Classification: Cancer
• Umbrella term: 10 Mondo subtypes
• Clinical trials: 141

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	central nervous system neoplasm
Mondo ID	MONDO:0006130
EFO	EFO:1000158
NCIT	C9293
UMLS	C0085136
MedGen	88335
Anatomy (UBERON)	UBERON:0001017
Is cancer (heuristic)	yes

Also known as: brain/spinal cord tumor · brain/spinal cord tumour · central nervous system neoplasm · central nervous system neoplasm (disease) · central nervous system tumor · central nervous system tumour · CNS neoplasm · CNS tumor · CNS tumour · neoplasm of central nervous system · neoplasm of CNS · neoplasm of the central nervous system · tumor of central nervous system · tumor of CNS · tumor of the central nervous system · tumor of the CNS · tumour of central nervous system · tumour of CNS · tumour of the central nervous system · tumour of the CNS

Disease family

This is a subtype of central nervous system disorder. Genetic, therapeutic, and trial evidence is largely curated at the broader-term level — see the parent page for the associated-gene cohort and molecular evidence.

Classification path: disease › human disease › disease by body system or component › nervous system disorder › central nervous system disorder › central nervous system neoplasm

Related subtypes (18): autoimmune disorder of central nervous system, autonomic nervous system disorder, optic nerve disorder, spinal cord disorder, high pressure neurological syndrome, central nervous system vasculitis, encephalomyelitis, neurodegenerative disease, brain disorder, palsy, trigeminal neuralgia, infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly, sporadic fetal brain disruption sequence, congenital narrowing of cervical spinal canal, central nervous system infectious disorder, cerebrospinal fluid leak, SPAST-related motor disorder, tinnitus

Subtypes (10): central nervous system organ benign neoplasm, autonomic nervous system neoplasm, optic nerve neoplasm, central nervous system cancer, central nervous system germ cell tumor, central nervous system hematopoietic neoplasm, epidural abscess, tumor of meninges, brain neoplasm, spinal cord neoplasm

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

No tiered GWAS variants or ClinVar records for this disease.

Genes & proteins

No associated-gene cohort resolved for this disease. Atlas builds the molecular and therapeutic sections — associated genes, protein families, druggability, pathways, interactions, and drug associations — by aggregating over a disease’s associated genes (resolved via GWAS / GenCC / ClinVar / CIViC), and none resolved here. This is expected for antibody-mediated, autoimmune, or otherwise non-gene-defined conditions; the curated evidence for this disease is its clinical features, GWAS susceptibility, and clinical trials (above).

Function

No pathway enrichment — requires an associated-gene cohort.

Therapeutics

Drugs indicated for this disease

0 approved, 19 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Development status
Carboplatin	Phase 3 (in late-stage trials)
Carmustine	Phase 3 (in late-stage trials)
Cisplatin	Phase 3 (in late-stage trials)
Etoposide	Phase 3 (in late-stage trials)
Etoposide Phosphate	Phase 3 (in late-stage trials)
Filgrastim	Phase 3 (in late-stage trials)
Ifosfamide	Phase 3 (in late-stage trials)
Isotretinoin	Phase 3 (in late-stage trials)
Leflunomide	Phase 3 (in late-stage trials)
Lomustine	Phase 3 (in late-stage trials)
Methotrexate	Phase 3 (in late-stage trials)
Mitolactol	Phase 3 (in late-stage trials)
Porfimer Sodium	Phase 3 (in late-stage trials)
Prednisone	Phase 3 (in late-stage trials)
Procarbazine	Phase 3 (in late-stage trials)
Temozolomide	Phase 3 (in late-stage trials)
Thioguanine	Phase 3 (in late-stage trials)
Thiotepa	Phase 3 (in late-stage trials)
Vincristine	Phase 3 (in late-stage trials)

Earlier-phase candidates (phase 2, investigational — efficacy not yet established): 6-O-BENZYLGUANINE, Alcohol, Aldesleukin, Amifostine, Bevacizumab, Bleomycin, Bortezomib, Busulfan, Celecoxib, Cytarabine, Dactinomycin, Dalteparin Sodium, Fenretinide, Gefitinib, Glufosfamide, Hydrocortisone, Hydroxyurea, Interferon Alfa, Irinotecan, MUROMONAB-CD3, Motexafin Gadolinium, Nedaplatin, Oxaliplatin, Paclitaxel, Pazopanib, Penicillamine, Pomalidomide, Prinomastat, Rubitecan, Sargramostim, Sodium Phenylacetate, Temsirolimus, Thalidomide, Topotecan, Vitespen.

Clinical trials & evidence

Clinical trials

Clinical trials: 141.

Phase distribution (across all retrieved trials)

Phase	Trials
Not specified	55
PHASE1	35
PHASE2	30
PHASE1/PHASE2	14
PHASE3	5
PHASE4	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04081701	PHASE4	RECRUITING	68-Ga DOTATATE PET/MRI in the Diagnosis and Management of Somatostatin Receptor Positive CNS Tumors.
NCT00002875	PHASE3	COMPLETED	Radiation Therapy Plus Combination Chemotherapy in Treating Children With Medulloblastoma
NCT00002944	PHASE3	COMPLETED	Combination Chemotherapy in Treating Children With Progressive Brain Tumors
NCT00078988	PHASE3	COMPLETED	High-Dose Chemotherapy Plus Autologous Stem Cell Transplantation Compared With Intermediate-Dose Chemotherapy Plus Autologous Stem Cell Transplantation With or Without Isotretinoin in Treating Young Patients With Recurrent High-Grade Gliomas
NCT00085098	PHASE3	COMPLETED	Radiation Therapy Compared With Chemotherapy and Radiation Therapy in Treating Patients With Newly Diagnosed Primary Central Nervous System (CNS) Germ Cell Tumor
NCT00716976	PHASE3	COMPLETED	Sodium Thiosulfate in Preventing Hearing Loss in Young Patients Receiving Cisplatin for Newly Diagnosed Germ Cell Tumor, Hepatoblastoma, Medulloblastoma, Neuroblastoma, Osteosarcoma, or Other Malignancy
NCT02650401	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options
NCT02797366	PHASE2	RECRUITING	Proton Radiotherapy for Primary Central Nervous System Tumours in Adults
NCT02867592	PHASE2	ACTIVE_NOT_RECRUITING	Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors
NCT02932280	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Safety and Dose Finding Study of Neratinib in Children and Young Adults With Cancer That Has Returned or Not Responded to Treatment
NCT03270059	PHASE2	RECRUITING	Gadolinium and Ferumoxytol MRI in Diagnosing Patients With Abnormalities in the Central Nervous System
NCT03834961	PHASE2	ACTIVE_NOT_RECRUITING	Larotrectinib in Treating Patients With Previously Untreated TRK Fusion Solid Tumors and TRK Fusion Relapsed Acute Leukemia
NCT03838042	PHASE1/PHASE2	RECRUITING	INFORM2 Study Uses Nivolumab and Entinostat in Children and Adolescents With High-risk Refractory Malignancies
NCT05235074	PHASE1/PHASE2	RECRUITING	OH2 Oncolytic Viral Therapy in Central Nervous System Tumors
NCT05386108	PHASE1/PHASE2	RECRUITING	Study of Abemaciclib and Elacestrant in Participants With Brain Metastasis Due to ER+/HER-2- Breast Cancer
NCT06528691	PHASE2	RECRUITING	Entrectinib as a Single Agent in Upfront Therapy for Children <3 Years of Age With NTRK1/2/3 or ROS1-FUSED CNS Tumors
NCT07539441	PHASE1/PHASE2	RECRUITING	A Study of Mirdametinib in People With Central Nervous System Tumors
NCT00002594	PHASE2	COMPLETED	Combination Chemotherapy Followed by Bone Marrow and/or Peripheral Stem Cell Transplantation in Treating Patients With Recurrent Medulloblastoma or CNS Germ Cell Tumors
NCT00003203	PHASE2	COMPLETED	Carboplatin and Vincristine Plus Radiation Therapy Followed By Adjuvant Chemotherapy in Treating Young Patients With Newly Diagnosed CNS Embryonal Tumors
NCT00003573	PHASE2	COMPLETED	Etoposide Plus Radiation Therapy Followed by Combination Chemotherapy in Treating Children With Newly Diagnosed Advanced Medulloblastoma
NCT00005812	PHASE2	TERMINATED	Temozolomide in Treating Patients With Leptomeningeal Metastases From a Solid Tumor or Lymphoma
NCT00027846	PHASE2	COMPLETED	Observation or Radiation Therapy and/or Chemotherapy and Second Surgery in Treating Children Who Have Undergone Surgery for Ependymoma
NCT00031590	PHASE2	TERMINATED	Low-Dose Radiation and Combination Chemotherapy Following Surgery in Children With Newly Diagnosed Medulloblastoma
NCT00047320	PHASE2	COMPLETED	Neoadjuvant Chemotherapy With or Without Second-Look Surgery Followed by Radiation Therapy With or Without Peripheral Stem Cell Transplantation in Treating Patients With Intracranial Germ Cell Tumors
NCT00066248	PHASE2	COMPLETED	Cyproheptadine and Megestrol in Preventing Weight Loss in Children With Cachexia Caused By Cancer or Cancer Treatment
NCT00100802	PHASE2	COMPLETED	Radiation Therapy, Temozolomide, and Lomustine in Treating Young Patients With Newly Diagnosed Gliomas
NCT00107471	PHASE1/PHASE2	TERMINATED	Topotecan, G-CSF, and Radiation Therapy in Treating Young Patients With Newly Diagnosed Brain Stem Glioma
NCT00187226	PHASE2	COMPLETED	A Study for Image-Guided Radiation Therapy in Pediatric Brain Tumors and Side Effects
NCT00238264	PHASE2	COMPLETED	Radiation Therapy in Treating Young Patients With Gliomas
NCT00400816	PHASE2	COMPLETED	Temozolomide in Treating Patients With Newly Diagnosed Anaplastic Oligodendroglioma or Mixed Oligoastrocytoma
NCT00407433	PHASE2	COMPLETED	Clinical Studies of Gemcitabine-Oxaliplatin
NCT00619112	PHASE2	COMPLETED	Temozolomide in Treating Patients With Recurrent High-Grade Glioma
NCT01164189	PHASE2	COMPLETED	Bevacizumab in Recurrent Grade II and III Glioma
NCT01217437	PHASE2	COMPLETED	Temozolomide and Irinotecan Hydrochloride With or Without Bevacizumab in Treating Young Patients With Recurrent or Refractory Medulloblastoma or CNS Primitive Neuroectodermal Tumors
NCT01582152	PHASE1/PHASE2	TERMINATED	Phase I/II Bevacizumab Versus Bevacizumab Plus TPI 287 for Recurrent Glioblastoma
NCT01735747	PHASE2	UNKNOWN	Temozolomide, Nedaplatin, Vincristine, and Radiotherapy as First-line Treatment in Newly Diagnosed Primary CNS Lymphoma
NCT01931098	PHASE2	COMPLETED	Oral Pazopanib Plus Oral Topotecan Metronomic Antiangiogenic Therapy for Recurrent Glioblastoma Multiforme (A) Without Prior Bevacizumab Exposure and (B) After Failing Prior Bevacizumab
NCT01985451	PHASE2	UNKNOWN	Pemetrexed and Temozolomide in Treating Patients With Relapsed Primary Central Nervous System Lymphoma （PCNSL）
NCT02095132	PHASE1/PHASE2	COMPLETED	Adavosertib and Irinotecan Hydrochloride in Treating Younger Patients With Relapsed or Refractory Solid Tumors
NCT02100891	PHASE2	TERMINATED	Phase 2 STIR Trial: Haploidentical Transplant and Donor Natural Killer Cells for Solid Tumors

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
2-MERCAPTOETHANESULFONIC ACID	4	5
LOMUSTINE	4	5
CABOZANTINIB	4	3
FILGRASTIM	4	3
PROCARBAZINE	4	3
ENTRECTINIB	4	2
GADOBENATE DIMEGLUMINE	4	2
LAROTRECTINIB	4	2
THIOTEPA	4	2
VINCRISTINE SULFATE	4	2
AMINOLEVULINIC ACID	4	1
AVAPRITINIB	4	1
BORTEZOMIB D-MANNITOL	4	1
CARBOPLATIN	4	1
CEMIPLIMAB	4	1
CISPLATIN	4	1
CYPROHEPTADINE HYDROCHLORIDE	4	1
ELACESTRANT	4	1
FERUMOXYTOL	4	1
GADOTERIDOL	4	1
GALLIUM CITRATE GA 67	4	1
INFIGRATINIB	4	1
ISOTRETINOIN	4	1
MEGESTROL ACETATE	4	1
MEMANTINE HYDROCHLORIDE	4	1
NERATINIB	4	1
PAZOPANIB	4	1
SARGRAMOSTIM	4	1
SODIUM THIOSULFATE	4	1
SONIDEGIB	4	1