Lomustine

Summary

Lomustine (CHEMBL514) is an approved small-molecule alkylating agent (ATC L01AD02); indicated across 30 conditions including neoplasm and brain neoplasm; with CIViC clinical evidence for 2 variant-indication associations (e.g. MGMT Promoter Methylation in glioblastoma).

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L01AD02
• Indications: 30 conditions
• Clinical trials: 94
• Precision-oncology evidence (CIViC): 2 variant–indication associations
• Chemistry: 233.69 Da · C9H16ClN3O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL514
Name	Lomustine
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	3950
ChEBI	CHEBI:6520
ATC	L01AD02
Molecular formula	C9H16ClN3O2
Molecular weight	233.69
InChIKey	GQYIWUVLTXOXAJ-UHFFFAOYSA-N

SMILES: C1CCC(CC1)NC(=O)N(CCCl)N=O

IUPAC name: 1-(2-chloroethyl)-3-cyclohexyl-1-nitrosourea

ChEBI definition: An N-nitrosourea that is urea in which one of the nitrogens is substituted by a 2-chloroethyl group and by a nitroso group, while the other nitrogen is substituted by a cyclohexyl group. An alkylating antineoplastic agent, it is used in the treatment of brain tumours, lung cancer, malignant melanoma and other solid tumours.

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

Also known as: CCN-U, CCNU, Ccnu, Ceenu, Gleostine, Lomustina, Lomustine, NSC-79037, Nitrosourea analogue, lomustine, LOMUSTINE, ccnu

Parent form; salt/anhydrous children: CHEMBL3250423, CHEMBL3250424, CHEMBL3250425

Patent coverage: 30,782 distinct patent families (127,882 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

Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: Alpha-2A adrenergic receptor, Alpha-1A adrenergic receptor, 3’,5’-cyclic-AMP phosphodiesterase 4A.

Bioactivity

ChEMBL activities: 1 potent at pChembl ≥ 5 of 3 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
ADRA2A	5.46	AC50	3499	nM	CHEMBL_ACT_25156595

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

30 indications (4 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
neoplasm	4	MONDO:0005070	EFO:0000616
brain neoplasm	4	MONDO:0021211	EFO:0003833
brain cancer	4	MONDO:0001657	MONDO:0001657
Hodgkins lymphoma	4	MONDO:0004952	EFO:0000183
glioblastoma	3	MONDO:0018177	EFO:0000519
acute myeloid leukemia	3	MONDO:0018874	EFO:0000222
anaplastic astrocytoma	3	MONDO:0016684	EFO:0002499
plasma cell myeloma	3	MONDO:0009693	EFO:0001378
medulloblastoma	3	MONDO:0007959	EFO:0002939
central nervous system neoplasm	3	MONDO:0006130	EFO:1000158
gliosarcoma	3	MONDO:0016681	EFO:1001465
plasma cell neoplasm	3	MONDO:0004959	EFO:0000200
paraganglioma	3	MONDO:0000448	EFO:1000453
cutaneous melanoma	2	MONDO:0005012	EFO:0000389
lymphoma	2	MONDO:0005062	EFO:0000574
melanoma	2	MONDO:0005105	EFO:0000756
sarcoma	2	MONDO:0005089	EFO:0000691
kidney cancer	2	MONDO:0002367	MONDO:0002367
lung neoplasm	2	MONDO:0021117	MONDO:0008903
breast neoplasm	2	MONDO:0021100	EFO:0003869
glioma	2	MONDO:0021042	MONDO:0100342
bone cancer	2	MONDO:0002129	EFO:1000350
spinal cord neoplasm	2	MONDO:0021234	EFO:0003828
bone neoplasm	2	MONDO:0019060	EFO:0003820
pancreatic neoplasm	2	MONDO:0021040	EFO:0003860
non-Hodgkin lymphoma	2	MONDO:0018908	EFO:0005952
ocular melanoma	2	MONDO:0006325	EFO:1000403

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

Clinical trials

Total trials: 94.

Phase distribution

Phase	Trials
PHASE2	36
PHASE3	27
PHASE1	11
Not specified	8
PHASE2/PHASE3	5
PHASE1/PHASE2	5
EARLY_PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00887146	PHASE3	ACTIVE_NOT_RECRUITING	Radiation Therapy With Concomitant and Adjuvant Temozolomide Versus Radiation Therapy With Adjuvant PCV Chemotherapy in Patients With Anaplastic Glioma or Low Grade Glioma
NCT03970447	PHASE2/PHASE3	RECRUITING	A Trial to Evaluate Multiple Regimens in Newly Diagnosed and Recurrent Glioblastoma
NCT05095376	PHASE3	ACTIVE_NOT_RECRUITING	Testing the Addition of the Chemotherapy Drug Lomustine (Gleostine) to the Usual Treatment (Temozolomide and Radiation Therapy) for Newly Diagnosed MGMT Methylated Glioblastoma
NCT05382338	PHASE3	RECRUITING	A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing Loss
NCT05902169	PHASE3	RECRUITING	Sonocloud-9 in Association With Carboplatin Versus Standard-of-Care Chemotherapies (CCNU or TMZ) in Recurrent GBM
NCT05904119	PHASE3	RECRUITING	Lomustine With and Without Reirradiation for First Progression of Glioblastoma: a Randomized Phase III Study
NCT06419946	PHASE3	RECRUITING	Lomustine in Addition to Standard of Care in Patients With MGMT Methylated Glioblastoma
NCT07100730	PHASE3	RECRUITING	Study of TLX101-Tx Plus Standard of Care (SoC) Versus SoC Alone for the Treatment of Patients With Recurrent Glioblastoma
NCT00002569	PHASE3	COMPLETED	Radiation Therapy With or Without Chemotherapy in Treating Patients With Anaplastic Oligodendroglioma
NCT00002840	PHASE3	COMPLETED	Radiation Therapy With and Without Combination Chemotherapy in Patients With Resected Anaplastic Oligodendroglioma
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
NCT00003375	PHASE2/PHASE3	COMPLETED	Observation or Radiation Therapy With or Without Combination Chemotherapy in Treating Patients With Low-Grade Glioma
NCT00003603	PHASE3	UNKNOWN	Chemotherapy Plus Steroid Therapy in Treating Patients With Multiple Myeloma
NCT00003788	PHASE3	UNKNOWN	Surgery, Radiation Therapy, and Chemotherapy With or Without Photodynamic Therapy in Treating Patients With Newly Diagnosed or Recurrent Malignant Supratentorial Gliomas
NCT00004259	PHASE3	COMPLETED	Radiation Therapy Combined With Chemotherapy in Treating Patients With Anaplastic Astrocytoma or Mixed Gliomas
NCT00052455	PHASE3	COMPLETED	Temozolomide Compared to Procarbazine, Lomustine, and Vincristine in Treating Patients With Recurrent Malignant Glioma
NCT00053872	PHASE3	UNKNOWN	Radiation Therapy Plus Combination Chemotherapy in Treating Children With Medulloblastoma
NCT00068952	PHASE3	COMPLETED	Study of IV Edotecarin Vs Temozolomide or Carmustine (BCNU) or Lomustine (CCNU) in Patients With Glioblastoma Multiforme
NCT00085735	PHASE3	COMPLETED	Comparison of Radiation Therapy Regimens in Combination With Chemotherapy in Treating Young Patients With Newly Diagnosed Standard-Risk Medulloblastoma
NCT00278278	PHASE3	UNKNOWN	Combination Chemotherapy and Radiation Therapy With or Without Methotrexate in Treating Young Patients With Newly Diagnosed Gliomas
NCT00295815	PHASE3	COMPLETED	Enzastaurin Versus Lomustine in Glioblastoma
NCT00480064	PHASE3	COMPLETED	Lomustine and Intermediate Dose Cytarabine in Older Patients With AML
NCT00590837	PHASE3	COMPLETED	Adding Lomustine to Chemotherapy in Older Patients With Acute Myelogenous Leukemia (AML), and Allogeneic Transplantation for Patients From 60 to 65 Years Old
NCT00761280	PHASE3	TERMINATED	Efficacy and Safety of AP 12009 in Patients With Recurrent or Refractory Anaplastic Astrocytoma or Secondary Glioblastoma
NCT00777153	PHASE3	COMPLETED	Cediranib in Combination With Lomustine Chemotherapy in Recurrent Glioblastoma
NCT01149109	PHASE3	COMPLETED	Efficacy and Safety Study of Lomustine/Temozolomide Combination Therapy vs. Standard Therapy for Glioblastoma Patients
NCT01290939	PHASE3	COMPLETED	Bevacizumab and Lomustine for Recurrent GBM
NCT02414165	PHASE2/PHASE3	TERMINATED	The Toca 5 Trial: Toca 511 & Toca FC Versus Standard of Care in Patients With Recurrent High Grade Glioma
NCT02796261	PHASE3	UNKNOWN	Study to Evaluate Eflornithine + Lomustine vs Lomustine in Recurrent Anaplastic Astrocytoma (AA) Patients
NCT03025893	PHASE2/PHASE3	UNKNOWN	A Phase II/III Study of High-dose, Intermittent Sunitinib in Patients With Recurrent Glioblastoma Multiforme
NCT03758014	PHASE2/PHASE3	UNKNOWN	Studies of Chlorogenic Acid for Injection for Safety and Efficacy of Grade IV GBM Patients
NCT02724579	PHASE2	ACTIVE_NOT_RECRUITING	Reduced Craniospinal Radiation Therapy and Chemotherapy in Treating Younger Patients With Newly Diagnosed WNT-Driven Medulloblastoma
NCT03678883	PHASE2	ACTIVE_NOT_RECRUITING	9-ING-41 in Patients With Advanced Cancers
NCT04049669	PHASE2	ACTIVE_NOT_RECRUITING	Pediatric Trial of Indoximod With Chemotherapy and Radiation for Relapsed Brain Tumors or Newly Diagnosed DIPG
NCT04762069	PHASE2	ACTIVE_NOT_RECRUITING	A Study of Berubicin in Adult Subjects With Recurrent Glioblastoma Multiforme
NCT04863950	PHASE2	RECRUITING	Investigator-Initiated Study of Imipramine Hydrochloride and Lomustine in Recurrent Glioblastoma
NCT06177964	PHASE2	RECRUITING	Lerapolturev (PVSRIPO) in GBM
NCT06336291	PHASE2	ACTIVE_NOT_RECRUITING	A Study With L19TNF in Combination With Lomustine in Patients With Glioblastoma at Progression or Recurrence
NCT07120984	PHASE2	NOT_YET_RECRUITING	A Study to Evaluate the Safety and Efficacy of L19TNF With Alkylating Chemotherapy for Patients With Recurrent IDH-mutant Astrocytoma or Oligodendroglioma

Clinical evidence (CIViC)

Variant × indication × effect (2 predictive associations from 2 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
MGMT Promoter Methylation	Glioblastoma	Sensitivity/Response	Lomustine + Temozolomide	CIViC A	EID7303
EGFR Overexpression	Glioblastoma	Sensitivity/Response	Lomustine + Bevacizumab	CIViC B	EID10894

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 1 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).