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Atlas / Drug / Busulfan

Busulfan

drug
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Also known as BusilvexBusulfan fresenius kabiBusulfanoBusulfanumBusulfexLeucosulfanMyelosanMyelosanumMyleranNCI-C01592NSC-750SulphabutinbusulphanSID17388889SID17389813SID26747279SID26752840SID49648470SID67633

Summary

Busulfan (CHEMBL820) is an approved small-molecule alkylating agent (ATC L01AB01); indicated across 95 conditions including chronic myeloid leukemia and leukemia.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: L01AB01
  • Indications: 95 conditions
  • Clinical trials: 487
  • Chemistry: 246.3 Da · C6H14O6S2

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL820
NameBusulfan
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID2478
ChEBICHEBI:28901
ATCL01AB01
Molecular formulaC6H14O6S2
Molecular weight246.3
InChIKeyCOVZYZSDYWQREU-UHFFFAOYSA-N

SMILES: CS(=O)(=O)OCCCCOS(=O)(=O)C

IUPAC name: 4-methylsulfonyloxybutyl methanesulfonate

ChEBI definition: A methanesulfonate ester that is butane-1,4-diol in which the hydrogens of the hydroxy groups are replaced by methanesulfonyl groups. An alkylating antineoplastic agent, it is used for the treatment of chronic myeloid leukemia (although it has been largely replaced by newer drugs). It is also used as an insect sterilant.

Pharmacological roles (ChEBI): alkylating agent, insect sterilant, antineoplastic agent, teratogenic agent, carcinogenic agent.

Also known as: Busilvex, Busulfan, Busulfan fresenius kabi, Busulfano, Busulfanum, Busulfex, Leucosulfan, Myelosan, Myelosanum, Myleran, NCI-C01592, NSC-750

Patent coverage: 38,184 distinct patent families (158,774 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: 8 (assay-derived). Sample: Lysine-specific demethylase 4E, Ubiquitin carboxyl-terminal hydrolase 2, Prelamin-A/C, Thyrotropin receptor, Polyunsaturated fatty acid lipoxygenase ALOX15, Matrix metalloproteinase-9, Aldehyde dehydrogenase 1A1, Lethal factor.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 8 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
LMNA6.1Potency794.3nMCHEMBL_ACT_3654899
MMP95.28IC505215nMCHEMBL_ACT_7609840

Target pathways

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

Indications & clinical

Indications

95 indications (5 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).

IndicationTrial phaseMONDOEFO
chronic myeloid leukemia4MONDO:0011996EFO:0000339
leukemia4MONDO:0005059EFO:0000565
neoplasm4MONDO:0005070EFO:0000616
Ewing sarcoma3MONDO:0012817EFO:0000174
acute lymphoblastic leukemia3MONDO:0004967EFO:0000220
acute myeloid leukemia3MONDO:0018874EFO:0000222
plasma cell myeloma3MONDO:0009693EFO:0001378
myelodysplastic syndrome3MONDO:0018881EFO:0000198
acute monocytic leukemia3MONDO:0007896EFO:0000221
acute myelomonocytic leukemia M43MONDO:0018871EFO:0000223
breast carcinoma3MONDO:0004989EFO:0000305
lymphoma3MONDO:0005062EFO:0000574
neuroblastoma3MONDO:0005072EFO:0000621
sarcoma3MONDO:0005089EFO:0000691
acute megakaryoblastic leukemia3MONDO:0018872EFO:0003025
acute myeloblastic leukemia without maturation3MONDO:0005224EFO:0003027
acute basophilic leukemia3MONDO:0019458EFO:0003029
chronic myelomonocytic leukemia3MONDO:0020311EFO:1001779
acute erythroid leukemia3MONDO:0017858EFO:0000218
myelodysplastic syndrome with single lineage dysplasia3MONDO:0005272EFO:0003802
myelodysplastic syndrome with excess blasts3MONDO:0019454EFO:0003811
breast neoplasm3MONDO:0021100MONDO:0007254
graft versus host disease3MONDO:0013730MONDO:0013730
ganglioneuroblastoma3MONDO:0005035EFO:0000502
thrombotic disease3MONDO:0000831MONDO:0000831
essential thrombocythemia3MONDO:0005029EFO:0000479
immune system disorder2MONDO:0005046EFO:0000540
B-cell chronic lymphocytic leukemia2MONDO:0004948EFO:0000095
Hodgkins lymphoma2MONDO:0004952EFO:0000183
central nervous system cancer2MONDO:0002714EFO:0000326
diffuse large B-cell lymphoma2MONDO:0018905EFO:0000403
lymphoid neoplasm2MONDO:0005157EFO:0001642
primary myelofibrosis2MONDO:0009692EFO:0002430
hematologic disorder2MONDO:0005570EFO:0005803
non-Hodgkin lymphoma2MONDO:0018908EFO:0005952
plasma cell leukemia2MONDO:0018689EFO:0006475
male breast carcinoma2MONDO:0005628EFO:0006861
juvenile myelomonocytic leukemia2MONDO:0011908EFO:1000309
inflammatory breast carcinoma2MONDO:0006804EFO:1000984
mantle cell lymphoma2MONDO:0018876EFO:1001469
thalassemia2MONDO:0000984EFO:1001996
beta thalassemia2MONDO:0019402Orphanet:848
plasmacytoma2MONDO:0005615EFO:0006738
Langerhans cell histiocytosis2MONDO:0018310EFO:1000318
epidermolysis bullosa2MONDO:0006541EFO:1000690
myeloproliferative neoplasm2MONDO:0020076EFO:0002428
central nervous system neoplasm2MONDO:0006130EFO:1000158
Niemann-Pick disease2MONDO:0001982EFO:1001380
kidney cancer2MONDO:0002367MONDO:0002367
T-cell non-Hodgkin lymphoma2MONDO:0015760MONDO:0015760
follicular lymphoma2MONDO:0018906MONDO:0018906
hematopoietic and lymphoid system neoplasm2MONDO:0002334MONDO:0044881
inborn error of immunity2MONDO:0003778HP:0002721
myelodysplastic/myeloproliferative disease2MONDO:0020077MONDO:0020077
fucosidosis2MONDO:0009254MONDO:0009254
Krabbe disease2MONDO:0009499MONDO:0009499
Sandhoff disease2MONDO:0010006MONDO:0010006
Tay-Sachs disease2MONDO:0010100MONDO:0010100
sickle cell disease2MONDO:0011382MONDO:0011382
severe combined immunodeficiency2MONDO:0015974MONDO:0015974
osteopetrosis2MONDO:0017198MONDO:0017198
GM1 gangliosidosis2MONDO:0018149MONDO:0018149
Gaucher disease2MONDO:0018150MONDO:0018150
chronic granulomatous disease2MONDO:0018305MONDO:0018305
adrenoleukodystrophy2MONDO:0018544MONDO:0018544
metachromatic leukodystrophy2MONDO:0018868MONDO:0018868
Wolman disease2MONDO:0019148MONDO:0019148
Fanconi anemia2MONDO:0019391MONDO:0019391
inherited hemoglobinopathy2MONDO:0019050MONDO:0044348
plasma cell neoplasm2MONDO:0004959EFO:0000200
brain neoplasm1MONDO:0021211EFO:0003833
cutaneous melanoma1MONDO:0005012EFO:0000389
hereditary disease1MONDO:0003847EFO:0000508
HIV infectious disease1MONDO:0005109EFO:0000764
anemia1MONDO:0002280EFO:0004272
T-B+ severe combined immunodeficiency due to gamma chain deficiency1MONDO:0010315EFO:0005555
combined immunodeficiency, X-linked1MONDO:0010730EFO:1001451
AIDS1MONDO:0012268EFO:0000765
blast phase chronic myelogenous leukemia, BCR-ABL1 positive1MONDO:0006115EFO:1000131
ovarian cancer1MONDO:0008170MONDO:0008170
retinoblastoma1MONDO:0008380MONDO:0008380
chordoma1MONDO:0008978MONDO:0008978
Wiskott-Aldrich syndrome1MONDO:0010518MONDO:0010518
osteogenesis imperfecta1MONDO:0019019MONDO:0019019
myeloid leukemia1MONDO:0004643MONDO:0004643

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

Clinical trials

Total trials: 487.

Phase distribution

PhaseTrials
PHASE2242
PHASE1/PHASE265
PHASE160
PHASE344
Not specified40
PHASE2/PHASE326
PHASE45
EARLY_PHASE15

Top trials by phase / activity

NCTPhaseStatusTitle
NCT00361140PHASE4COMPLETEDBusulfan Safety/Efficacy as Conditioning Prior to Hematopoietic Cell Transplantation (HCT)
NCT01339988PHASE4UNKNOWNBusulfan and Cyclophosphamide Instead of Total Boby Irradiation (TBI) and Cyclophosphamide for Hematological Malignancies Hematocrit (HCT)
NCT03171831PHASE4UNKNOWNHaploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major
NCT04009525PHASE4COMPLETEDHematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
NCT06069180PHASE4UNKNOWNThe Optimization of Conditioning Regimen for HLA Matched HSCT in SAA
NCT01704716PHASE3RECRUITINGHigh Risk Neuroblastoma Study 1.8 of SIOP-Europe (SIOPEN)
NCT01949129PHASE2/PHASE3RECRUITINGAllogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia
NCT02724163PHASE3RECRUITINGInternational Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia
NCT03126916PHASE3RECRUITINGTesting the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL)
NCT04217278PHASE2/PHASE3ACTIVE_NOT_RECRUITINGA Trial of Treatments to Assess the Effects on Outcome of Adults With AML and MDS Undergoing Allogeneic SCT
NCT04221035PHASE3RECRUITINGHigh-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)
NCT04547049PHASE3ACTIVE_NOT_RECRUITINGA Study Comparing Haploidentical Hematopoietic Stem Cell Transplantations (HSCTs) From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies
NCT04708054PHASE2/PHASE3RECRUITINGVenetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS
NCT05457556PHASE3ACTIVE_NOT_RECRUITINGMismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome
NCT05674539PHASE3ENROLLING_BY_INVITATIONReduced Intensity Conditioning Regimens for Acute Myeloid Leukemia and Myelodysplastic Syndrome
NCT05991908PHASE3RECRUITINGRandomized Study of Conditioning of Fludarabine Combined With Single or Dual Alkylating Agents in Myeloid Malignancies
NCT06079879PHASE3RECRUITINGA Study of Bomedemstat (IMG-7289/MK-3543) Compared to Best Available Therapy (BAT) in Participants With Essential Thrombocythemia and an Inadequate Response or Intolerance of Hydroxyurea (MK-3543-006)
NCT06297772PHASE3NOT_YET_RECRUITINGCompare the Efficacy and Safety of Dec-FB4 and FB4 as Conditioning Regimen for AML-MR
NCT06613035PHASE3NOT_YET_RECRUITINGTwice-per-weekSelinexor, 2 Days Melphalan
NCT07052422PHASE2/PHASE3NOT_YET_RECRUITINGVEN+DAC+Bu2Flu4 vs Bu2Flu5 Conditioning Regimen for Elderly Myeloid Malignancies Undergoing Allo-HSCT
NCT00002549PHASE3UNKNOWNCombination Chemotherapy Followed by Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Acute Myelogenous Leukemia
NCT00002701PHASE3UNKNOWNCombination Chemotherapy With or Without Bone Marrow Transplantation in Treating Patients With Acute Promyelocytic Leukemia
NCT00002771PHASE3UNKNOWNChemotherapy, Interferon, and Bone Marrow Transplantation in Treating Patients With Chronic Myelogenous Leukemia
NCT00002789PHASE3COMPLETEDBone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Chronic Myeloid Leukemia
NCT00002798PHASE3COMPLETEDCombination Chemotherapy With or Without Bone Marrow Transplantation in Treating Children With Acute Myelogenous Leukemia or Myelodysplastic Syndrome
NCT00002868PHASE3COMPLETEDInterferon-alfa With or Without Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Newly Diagnosed Chronic Myelogenous Leukemia
NCT00002961PHASE3TERMINATEDChemotherapy With or Without Total-Body Irradiation Prior to Bone Marrow Transplantation in Treating Children With Acute Lymphoblastic Leukemia
NCT00002989PHASE3UNKNOWNCombination Chemotherapy With or Without Idarubicin and Peripheral Stem Cell Transplantation in Treating Patients With Leukemia or Myelodysplastic Syndrome
NCT00003414PHASE3COMPLETEDGraft-Versus-Host Disease in Treating Patients With Recurrent or Refractory Lymphoma or Hodgkin’s Disease
NCT00003816PHASE2/PHASE3COMPLETEDCombination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer
NCT00003972PHASE3COMPLETEDCombination Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Stage II or Stage IIIA Breast Cancer
NCT00004128PHASE3UNKNOWNCombination Chemotherapy, Interleukin-2, and Peripheral Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia
NCT00005866PHASE3COMPLETEDS9920 Busulfan Compared With Cyclophosphamide in Patients Undergoing Total-Body Irradiation Plus Peripheral Stem Cell Transplantation for Advanced Myelodysplastic Syndrome or Related Acute Myeloid Leukemia
NCT00006363PHASE3COMPLETEDCombination Chemotherapy With or Without PSC 833, Peripheral Stem Cell Transplantation, and/or Interleukin-2 in Treating Patients With Acute Myeloid Leukemia
NCT00020566PHASE3UNKNOWNCombination Chemotherapy With or Without Peripheral Stem Cell Transplantation, Radiation Therapy, and/or Surgery in Treating Patients With Ewing’s Sarcoma
NCT00025402PHASE3UNKNOWNChemotherapy and Biological Therapy With or Without Bone Marrow or Peripheral Stem Cell Transplant in Treating Patients With Chronic Myelogenous Leukemia
NCT00030719PHASE3UNKNOWNCombination Chemotherapy With or Without Filgrastim Before Surgery, High-Dose Chemotherapy, and Radiation Therapy Followed by Isotretinoin With or Without Monoclonal Antibody in Treating Patients With Neuroblastoma
NCT00049517PHASE3COMPLETEDCombination Chemotherapy With or Without Monoclonal Antibody Therapy in Treating Patients With AML Leukemia
NCT00176839PHASE2/PHASE3TERMINATEDStem Cell Transplantation for Hematological Malignancies
NCT00176852PHASE2/PHASE3COMPLETEDStem Cell Transplant for Hemoglobinopathy

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 5 clinical and 23 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

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

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.2
BioBTree
v2.0.2

Sources 17

This page pulls from 17 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclinical_trialsefogtopdb_interactiongtopdb_ligandhgncmeshmondopatent_compoundpharmgkbpharmgkb_guidelinepubchemuniprot