mebendazole   Click here for help

GtoPdb Ligand ID: 13427

Synonyms: Emverm® | Vermox®
Approved drug PDB Ligand
mebendazole is an approved drug (FDA (1974))
Compound class: Synthetic organic
Comment: Mebendazole is a long-established broad spectrum anthelmintic drug that is used in human and veterinary medicine. Benzimidazole class compounds like mebendazole interact with the colchicine-binding site of β tubulin and thereby disrupt polymerisation of microtubules. Repurposing of mebendazole for oncological indications has been proposed [1,5-6,8,11], with mechanism of action focussing on tubulin depolymerisation [3,10].

In 2024 mebendazole was identified as a repurposing candidate with potential to treat the rare genetic disorder autosomal dominant polycystic kidney disease (ADPKD) [2]. Transcriptomic and computational drug discovery tools were used to predict mebendazole's ADPKD potential, and this was validated in vitro (using patient-derived 3D kidney cyst cultures) and in mouse models of the disease.

mebendazole is commercially available from our sponsors

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 6
Hydrogen bond donors 2
Rotatable bonds 5
Topological polar surface area 79.79
Molecular weight 295.29
XLogP 1.92
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 COC(=O)NC1=NC2=C(C=C(C=C2)C(=O)C3=CC=CC=C3)N1
Isomeric SMILES COC(=O)NC1=NC2=C(N1)C=C(C=C2)C(=O)C3=CC=CC=C3
InChI InChI=1S/C16H13N3O3/c1-22-16(21)19-15-17-12-8-7-11(9-13(12)18-15)14(20)10-5-3-2-4-6-10/h2-9H,1H3,(H2,17,18,19,21)
InChI Key OPXLLQIJSORQAM-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/)
References
1. Bai RY, Staedtke V, Rudin CM, Bunz F, Riggins GJ. (2015)
Effective treatment of diverse medulloblastoma models with mebendazole and its impact on tumor angiogenesis.
Neuro Oncol, 17 (4): 545-54. [PMID:25253417]
2. Brownjohn PW, Zoufir A, O'Donovan DJ, Sudhahar S, Syme A, Huckvale R, Porter JR, Bange H, Brennan J, Thompson NT. (2024)
Computational drug discovery approaches identify mebendazole as a candidate treatment for autosomal dominant polycystic kidney disease.
Front Pharmacol, 15: 1397864. [PMID:38846086]
3. Choi HS, Ko YS, Jin H, Kang KM, Ha IB, Jeong H, Song HN, Kim HJ, Jeong BK. (2021)
Anticancer Effect of Benzimidazole Derivatives, Especially Mebendazole, on Triple-Negative Breast Cancer (TNBC) and Radiotherapy-Resistant TNBC In Vivo and In Vitro.
Molecules, 26 (17). [PMID:34500557]
4. Dakshanamurthy S, Issa NT, Assefnia S, Seshasayee A, Peters OJ, Madhavan S, Uren A, Brown ML, Byers SW. (2012)
Predicting new indications for approved drugs using a proteochemometric method.
J Med Chem, 55 (15): 6832-48. [PMID:22780961]
5. Gallia GL, Holdhoff M, Brem H, Joshi AD, Hann CL, Bai RY, Staedtke V, Blakeley JO, Sengupta S, Jarrell TC et al.. (2021)
Mebendazole and temozolomide in patients with newly diagnosed high-grade gliomas: results of a phase 1 clinical trial.
Neurooncol Adv, 3 (1): vdaa154. [PMID:33506200]
6. Hegazy SK, El-Azab GA, Zakaria F, Mostafa MF, El-Ghoneimy RA. (2022)
Mebendazole; from an anti-parasitic drug to a promising candidate for drug repurposing in colorectal cancer.
Life Sci, 299: 120536. [PMID:35385794]
7. Issa NT, Peters OJ, Byers SW, Dakshanamurthy S. (2015)
RepurposeVS: A Drug Repurposing-Focused Computational Method for Accurate Drug-Target Signature Predictions.
Comb Chem High Throughput Screen, 18 (8): 784-94. [PMID:26234515]
8. Mansoori S, Fryknäs M, Alvfors C, Loskog A, Larsson R, Nygren P. (2021)
A phase 2a clinical study on the safety and efficacy of individualized dosed mebendazole in patients with advanced gastrointestinal cancer.
Sci Rep, 11 (1): 8981. [PMID:33903692]
9. Nygren P, Fryknäs M, Agerup B, Larsson R. (2013)
Repositioning of the anthelmintic drug mebendazole for the treatment for colon cancer.
J Cancer Res Clin Oncol, 139 (12): 2133-40. [PMID:24135855]
10. Sasaki J, Ramesh R, Chada S, Gomyo Y, Roth JA, Mukhopadhyay T. (2002)
The anthelmintic drug mebendazole induces mitotic arrest and apoptosis by depolymerizing tubulin in non-small cell lung cancer cells.
Mol Cancer Ther, 1 (13): 1201-9. [PMID:12479701]
11. Williamson T, de Abreu MC, Trembath DG, Brayton C, Kang B, Mendes TB, de Assumpção PP, Cerutti JM, Riggins GJ. (2021)
Mebendazole disrupts stromal desmoplasia and tumorigenesis in two models of pancreatic cancer.
Oncotarget, 12 (14): 1326-1338. [PMID:34262644]