treosulfan   Click here for help

GtoPdb Ligand ID: 8281

Synonyms: Grafapex® | Trecondi®
Approved drug
treosulfan is an approved drug (UK (1992), EMA (2019))
Compound class: Synthetic organic
Comment: Treosulfan is a DNA alkylating agent.
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View more information in the IUPHAR Pharmacology Education Project: treosulfan

2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/). All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 8
Hydrogen bond donors 2
Rotatable bonds 7
Topological polar surface area 143.96
Molecular weight 278.01
XLogP -3.29
No. Lipinski's rules broken 0

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)

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Canonical SMILES OC(C(COS(=O)(=O)C)O)COS(=O)(=O)C
Isomeric SMILES OC(C(COS(=O)(=O)C)O)COS(=O)(=O)C
InChI InChI=1S/C6H14O8S2/c1-15(9,10)13-3-5(7)6(8)4-14-16(2,11)12/h5-8H,3-4H2,1-2H3
InChI Key YCPOZVAOBBQLRI-UHFFFAOYSA-N

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
No information available.
Summary of Clinical Use Click here for help
The EMA granted treosulfan orphan drug status to be used as a conditioning treatment prior to allogenic haematopoietic progenitor cell transplantation (alloHSCT) . The FDA has granted orphan drug status for the treatment of ovarian cancer. Advanced clinical studies were carried out to determine safety and efficacy of treosulfan as a conditioning treatment and as a treatment for ovarian cancer and Ewing's sarcoma. Full EMA approval for use as a pre-alloHSCT conditioning regimen was granted in 2019 . The FDA approved treosulfan with fludarabine as a preparative regimen for alloHSCT in January 2025, for patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).
Mechanism Of Action and Pharmacodynamic Effects Click here for help
As a DNA alkylating (cross-linking) agent, treosulfan treatment irreversibly interferes with DNA replication, RNA transcription and causes general disruption of nucleic acid function. This action causes cell death.