Carmustine

Summary

Carmustine (CHEMBL513) is an approved small-molecule alkylating agent (ATC L01AD01) targeting GSR; indicated across 50 conditions including astrocytoma (excluding glioblastoma) and glioblastoma; with CIViC clinical evidence for 1 variant-indication association (e.g. MGMT Promoter Methylation in glioblastoma).

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L01AD01
• Targets: 1 (GSR)
• Indications: 50 conditions
• Clinical trials: 195
• Precision-oncology evidence (CIViC): 1 variant–indication association
• Chemistry: 214.05 Da · C5H9Cl2N3O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL513
Name	Carmustine
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	2578
ChEBI	CHEBI:3423
ATC	L01AD01
Molecular formula	C5H9Cl2N3O2
Molecular weight	214.05
InChIKey	DLGOEMSEDOSKAD-UHFFFAOYSA-N

SMILES: C(CCl)NC(=O)N(CCCl)N=O

IUPAC name: 1,3-bis(2-chloroethyl)-1-nitrosourea

ChEBI definition: A member of the class of N-nitrosoureas that is 1,3-bis(2-chloroethyl)urea in which one of the nitrogens is substituted by a nitroso group.

Pharmacological roles (ChEBI): alkylating agent, antineoplastic agent.

Also known as: BCNU, Becenun, Bicnu, Bischloroethyl nitrosourea, Camustine, Carmubris, Carmustina, Carmustine, Carmustine obvius, DTI-015, FDA-0345, Gliadel

Patent coverage: 35,722 distinct patent families (148,550 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
GSR	glutathione-disulfide reductase	Inhibition	4.15	0.2%	P00390

Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: Nuclear receptor ROR-gamma, RecQ-like DNA helicase BLM, Endonuclease 4, Peripheral myelin protein 22, Thyrotropin receptor, Menin/Histone-lysine N-methyltransferase MLL, Glutathione reductase, mitochondrial, Muscarinic acetylcholine receptor M1, Aldehyde dehydrogenase 1A1, Huntingtin.

Bioactivity

ChEMBL activities: 5 potent at pChembl ≥ 5 of 13 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
BLM	7.85	Potency	14.1	nM	CHEMBL_ACT_4745917
BLM	7.85	Potency	14.1	nM	CHEMBL_ACT_4921912
ALDH1A1	5.5	Potency	3162	nM	CHEMBL_ACT_4179069
GSR	5.09	IC50	8100	nM	CHEMBL_ACT_13876423
P0A6C1	5	Potency	10000	nM	CHEMBL_ACT_4084724

Target pathways

Aggregated over 1 target gene(s): GSR.

Top Reactome pathways

5 total, by targets touching each:

Pathway	Targets	Genes
Metabolism of ingested H2SeO4 and H2SeO3 into H2Se	1	GSR
Detoxification of Reactive Oxygen Species	1	GSR
Interconversion of nucleotide di- and triphosphates	1	GSR
TP53 Regulates Metabolic Genes	1	GSR
NFE2L2 regulating anti-oxidant/detoxification enzymes	1	GSR

Dominant GO biological processes

GO term	Targets
glutathione metabolic process	1
cellular response to oxidative stress	1
cell redox homeostasis	1
cellular oxidant detoxification	1

Indications & clinical

Indications

50 indications (13 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
astrocytoma (excluding glioblastoma)	4	MONDO:0019781	EFO:0000272
glioblastoma	4	MONDO:0018177	EFO:0000519
medulloblastoma	4	MONDO:0007959	EFO:0002939
plasma cell myeloma	4	MONDO:0009693	EFO:0001378
Hodgkins lymphoma	4	MONDO:0004952	EFO:0000183
neoplasm	4	MONDO:0005070	EFO:0000616
anaplastic astrocytoma	4	MONDO:0016684	EFO:0002499
non-Hodgkin lymphoma	4	MONDO:0018908	EFO:0005952
glioma	4	MONDO:0021042	MONDO:0003268
ependymoma	4	MONDO:0016698	EFO:1000028
neoplasm of mature B-cells	3	MONDO:0004949	EFO:0000096
breast carcinoma	3	MONDO:0004989	EFO:0000305
diffuse large B-cell lymphoma	3	MONDO:0018905	EFO:0000403
leukemia	3	MONDO:0005059	EFO:0000565
lymphoma	3	MONDO:0005062	EFO:0000574
anaplastic oligodendroglioma	3	MONDO:0016696	EFO:0002501
mantle cell lymphoma	3	MONDO:0018876	EFO:1001469
central nervous system neoplasm	3	MONDO:0006130	EFO:1000158
breast neoplasm	3	MONDO:0021100	MONDO:0007254
follicular lymphoma	3	MONDO:0018906	MONDO:0018906
paraganglioma	3	MONDO:0000448	EFO:1000453
brain neoplasm	2	MONDO:0021211	EFO:0003833
B-cell chronic lymphocytic leukemia	2	MONDO:0004948	EFO:0000095
peripheral T-cell lymphoma, not otherwise specified	2	MONDO:0004964	EFO:0000211
cutaneous melanoma	2	MONDO:0005012	EFO:0000389
sarcoma	2	MONDO:0005089	EFO:0000691
melanoma	2	MONDO:0005105	EFO:0000756
relapsing-remitting multiple sclerosis	2	MONDO:0005314	EFO:0003929
gliosarcoma	2	MONDO:0016681	EFO:1001465
hematologic disorder	2	MONDO:0005570	HP:0001871
stiff-person syndrome	2	MONDO:0008491	EFO:0007498
neuromyelitis optica	2	MONDO:0019100	EFO:0004256
myasthenia gravis	2	MONDO:0009688	EFO:0004991
Guillain-Barre syndrome, familial	2	MONDO:0007691	EFO:0009538
opsoclonus-myoclonus syndrome	2	MONDO:0015247	EFO:1001383
brain cancer	2	MONDO:0001657	MONDO:0001657
ovarian cancer	2	MONDO:0008170	MONDO:0008170
graft versus host disease	2	MONDO:0013730	EFO:0004599
plasma cell neoplasm	2	MONDO:0004959	EFO:0000200
colorectal neoplasm	2	MONDO:0005335	MONDO:0005575
primary cutaneous T-cell non-Hodgkin lymphoma	1	MONDO:0000607	EFO:0002913
plasma cell leukemia	1	MONDO:0018689	EFO:0006475
neuroblastoma	1	MONDO:0005072	EFO:0000621
Sezary syndrome	1	MONDO:0017844	EFO:1000785
mycosis fungoides	1	MONDO:0009691	EFO:1001051
hereditary breast ovarian cancer syndrome	1	MONDO:0003582	Orphanet:145

4 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 195.

Phase distribution

Phase	Trials
PHASE2	104
PHASE3	35
PHASE1	28
PHASE1/PHASE2	20
Not specified	7
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02443077	PHASE3	ACTIVE_NOT_RECRUITING	Ibrutinib Before and After Stem Cell Transplant in Treating Patients With Relapsed or Refractory Diffuse Large B-cell Lymphoma
NCT00002545	PHASE3	COMPLETED	Radiation Therapy Plus Chemotherapy in Treating Patients With Supratentorial Glioblastoma Multiforme
NCT00002548	PHASE3	COMPLETED	SWOG-9321 Melphalan, TBI, and Transplant vs Combo Chemo in Untreated Myeloma
NCT00002556	PHASE3	COMPLETED	Combination Chemotherapy With or Without High Dose Cyclophosphamide and Recombinant Interferon Alfa-2b in Treating Patients With Previously Untreated Stage I-III Multiple Myeloma
NCT00002599	PHASE3	UNKNOWN	Combination Chemotherapy and Interferon Alfa With or Without Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Myeloma
NCT00002620	PHASE3	COMPLETED	Radiation Therapy in Treating Patients With Brain Cancer
NCT00002653	PHASE3	UNKNOWN	Combination Chemotherapy With or Without Cyclophosphamide and Prednisone in Treating Older Patients With Multiple Myeloma
NCT00002719	PHASE3	COMPLETED	Combination Chemotherapy With or Without G-CSF in Treating Older Patients With Acute Myeloid Leukemia
NCT00002766	PHASE3	COMPLETED	Comparison of Two Combination Chemotherapy Regimens in Treating Adults With Previously Untreated Leukemia or Lymphoma
NCT00002772	PHASE3	TERMINATED	S9623, Combination Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Women With Breast Cancer
NCT00002835	PHASE3	COMPLETED	Combination Chemotherapy in Treating Patients With Lymphoma
NCT00002836	PHASE3	COMPLETED	Filgrastim Plus Chemotherapy Compared With Filgrastim Alone In Treating Women Undergoing Peripheral Stem Cell Transplantation For Breast Cancer
NCT00003578	PHASE3	UNKNOWN	High Dose Chemotherapy With or Without Bone Marrow Transplantation in Treating Patients With Intermediate- or High-Grade Non-Hodgkin’s Lymphoma
NCT00003788	PHASE3	UNKNOWN	Surgery, Radiation Therapy, and Chemotherapy With or Without Photodynamic Therapy in Treating Patients With Newly Diagnosed or Recurrent Malignant Supratentorial Gliomas
NCT00003815	PHASE3	UNKNOWN	Chemotherapy and Radiation Therapy Plus Bone Marrow Transplantation in Treating Patients With Aggressive Non-Hodgkin’s Lymphoma
NCT00004031	PHASE3	COMPLETED	SWOG-9704 Chemoradiotherapy and Peripheral Stem Cell Transplantation Compared With Combination Chemotherapy in Treating Patients With Non-Hodgkin’s Lymphoma
NCT00004259	PHASE3	COMPLETED	Radiation Therapy Combined With Chemotherapy in Treating Patients With Anaplastic Astrocytoma or Mixed Gliomas
NCT00005090	PHASE3	TERMINATED	S9901 Combination Chemotherapy With or Without Peripheral Stem Cell Transplantation in Treating Men With Stage III or Stage IV Hodgkin’s Disease
NCT00005589	PHASE3	COMPLETED	Combination Chemotherapy Plus Peripheral Stem Cell Transplantation With or Without Rituximab in Treating Patients With Relapsed Non-Hodgkin’s Lymphoma
NCT00012051	PHASE3	COMPLETED	Chemotherapy and Peripheral Stem Cell Transplant With or Without Monoclonal Antibody Therapy in Treating Patients With Non-Hodgkin’s Lymphoma
NCT00016887	PHASE3	UNKNOWN	Chemotherapy Followed by Radiation Therapy and Peripheral Stem Cell Transplant Compared With Chemotherapy Plus Interferon Alfa in Treating Patients With Stage III or Stage IV Mantle Cell Lymphoma
NCT00017147	PHASE3	COMPLETED	S0001 RT and Carmustine With or Without O6BG in Patients With New Glioblastoma Multiforme or Gliosarcoma
NCT00025636	PHASE3	UNKNOWN	Combination Chemotherapy and Peripheral Stem Cell Transplant in Treating Patients With Relapsed Hodgkin’s Lymphoma
NCT00052923	PHASE3	COMPLETED	Stem Cell Transplantation With or Without Rituximab in Treating Patients With Relapsed or Progressive B-Cell Diffuse Large Cell Lymphoma
NCT00068952	PHASE3	COMPLETED	Study of IV Edotecarin Vs Temozolomide or Carmustine (BCNU) or Lomustine (CCNU) in Patients With Glioblastoma Multiforme
NCT00070187	PHASE2/PHASE3	COMPLETED	Immunotherapy Using Cyclosporine, Interferon Gamma, and Interleukin-2 After High-Dose Myeloablative Chemotherapy With Autologous Stem Cell Transplantation in Treating Patients With Refractory or Relapsed Hodgkin’s Lymphoma
NCT00083551	PHASE3	COMPLETED	UARK 98-026 TT II: Multiple Myeloma Evaluating Anti-Angiogenesis With Thalidomide and Post-Transplant Consolidation Chemotherapy
NCT00137995	PHASE3	COMPLETED	R-ICE Versus R-DHAP in Patients Aged 18-65 With Relapse Diffuse Large B-cell Lymphoma
NCT00209222	PHASE3	UNKNOWN	Efficacy of R-CHOP vs R-CHOP/R-DHAP in Untreated MCL
NCT00499018	PHASE3	UNKNOWN	Dose Dense Chemotherapy + Rituximab +/-Intensified High Dose Chemoimmunotherapy With Support of Peripheral Autologous Stem Cell in Diffuse Large B-Cell Lymphoma
NCT00761280	PHASE3	TERMINATED	Efficacy and Safety of AP 12009 in Patients With Recurrent or Refractory Anaplastic Astrocytoma or Secondary Glioblastoma
NCT00920153	PHASE3	TERMINATED	Three Different Therapy Regimens in Treating Patients With Previously Untreated Hodgkin Lymphoma
NCT01656980	PHASE3	UNKNOWN	Safety and Efficacy Study of Intracranially Implanted Carmustine to Treat Newly Diagnosed Malignant Glioma
NCT02366663	PHASE3	TERMINATED	BEAM vs. 90-Yttrium Ibritumomab Tiuxetan (Zevalin®)/BEAM With ASCT for Relapsed DLBCL
NCT03722355	PHASE3	COMPLETED	Hyperfractionated RT With BCNU Versus Conventional RT With BCNU for Supratentorial Malignant Glioma
NCT05466318	PHASE3	UNKNOWN	ChiCGB vs BEAM in High-risk or R/R Lymphomas
NCT00716066	PHASE2	ACTIVE_NOT_RECRUITING	Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases
NCT01511562	PHASE2	ACTIVE_NOT_RECRUITING	Combination Chemotherapy With or Without Autologous Stem Cell Transplant in Treating Patients With Central Nervous System B-Cell Lymphoma
NCT02570542	PHASE2	ACTIVE_NOT_RECRUITING	Study of the Impact of CD34+ Cell Dose on Absolute Lymphocyte Count Following High-Dose Therapy and Autologous Stem Cell Transplantation for Relapsed and Refractory Diffuse Large B-cell Lymphoma (DLBCL)
NCT02797470	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Gene Therapy in Treating Patients With Human Immunodeficiency Virus-Related Lymphoma Receiving Stem Cell Transplant

Clinical evidence (CIViC)

Variant × indication × effect (1 predictive associations from 1 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
MGMT Promoter Methylation	Glioblastoma	Sensitivity/Response	Carmustine	CIViC B	EID308

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

18 molecules share ≥1 primary target. Top 18 by shared-target count:

Molecule	Source	Status	Shared targets
MENADIONE	ChEMBL + PubChem	Phase 4 (approved)	GSR
METHYLENE BLUE	ChEMBL + PubChem	Phase 4 (approved)	GSR
NIFEDIPINE	ChEMBL + PubChem	Phase 4 (approved)	GSR
GLUTAMIC ACID	ChEMBL + PubChem	Phase 3 (approved)	GSR
LYSINE	ChEMBL + PubChem	Phase 2 (approved)	GSR
ELLAGIC ACID	ChEMBL	Phase 2	GSR
MOLIBRESIB	ChEMBL	Phase 2	GSR
Ceftriaxone	PubChem	Approved	GSR
Cefuroxime	PubChem	Approved	GSR
Chlorambucil	PubChem	Approved	GSR
Chlorhexidine	PubChem	Approved	GSR
Chlorpromazine	PubChem	Approved	GSR
Fluphenazine	PubChem	Approved	GSR
Ketotifen	PubChem	Approved	GSR
Meloxicam	PubChem	Approved	GSR
Promethazine	PubChem	Approved	GSR
Quercetin	PubChem	Approved	GSR
Trifluoperazine	PubChem	Approved	GSR