ArticleMolecular cancer therapeutics2015
Repurposing the antihelmintic mebendazole as a hedgehog inhibitor.
Article in Molecular cancer therapeutics, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02644291 (Phase I Study of Mebendazole Therapy for Recurrent/Progressive Pediatric Brain Tumors), which is not on this map. Cited by 50 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Phase I Study of Mebendazole Therapy for Recurrent/Progressive Pediatric Brain Tumors
Who cites it
50 citing papers in PubMed, 102 citations in OpenAlex.
- A phase 2a clinical study on the safety and efficacy of individualized dosed mebendazole in patients with advanced gastrointestinal cancer.Scientific reports · 2021Trial
- USP5 regulates purine metabolism and represents a therapeutic target in esophageal cancer.Cell death & disease · 2026Article
- Characterization of the anticancer effect of mebendazole and its interaction with standard cytotoxic drugs in patient tumor cellsOncology reports · 2026Article
- Phase 1 study of mebendazole therapy for refractory/progressive or recurrent pediatric brain tumors.Neuro-oncology practice · 2025Article
- Mebendazole effectively overcomes imatinib resistance by dual-targeting BCR/ABL oncoprotein and β-tubulin in chronic myeloid leukemia cells.The Korean journal of physiology & pharmacology : official journal of the Korean Physiological Society and the Korean Society of Pharmacology · 2025Article
- USP5 Binds and Stabilizes EphA2 to Increase Nasopharyngeal Carcinoma Radioresistance.International journal of biological sciences · 2025Article
- Critical dysregulated signaling pathways in drug resistance: highlighting the repositioning of mebendazole for cancer therapy.Frontiers in pharmacology · 2025Review
- Chiral Hydroxy Metabolite of Mebendazole: Analytical and Semi-Preparative High-Performance Liquid Chromatography Resolution and Chiroptical Properties.Pharmaceuticals (Basel, Switzerland) · 2024Article
- In vitro and in vivo anticancer activity of mebendazole in colon cancer: a promising drug repositioning.Naunyn-Schmiedeberg's archives of pharmacology · 2024Article
- Review
- Repurposing approved non-oncology drugs for cancer therapy: a comprehensive review of mechanisms, efficacy, and clinical prospects.European journal of medical research · 2023Review
- Drug Repositioning for Refractory Benign Tumors of the Central Nervous System.International journal of molecular sciences · 2023Review
- Review
- Drug Repurposing in Oncology: A Systematic Review of Randomized Controlled Clinical Trials.Cancers · 2023Review
- Emerging Perspectives on the Antiparasitic Mebendazole as a Repurposed Drug for the Treatment of Brain Cancers.International journal of molecular sciences · 2023Review
- Pharmacokinetics of mebendazole in plasma and cerebrospinal fluid following a single oral dose in healthy dogs.Frontiers in veterinary science · 2023Article
- Drug Repurposing Flubendazole to Suppress Tumorigenicity via PCSK9-dependent Inhibition and Potentiate Lenvatinib Therapy for Hepatocellular Carcinoma.International journal of biological sciences · 2023Article
- Mebendazole prevents distant organ metastases in part by decreasing ITGβ4 expression and cancer stemness.Breast cancer research : BCR · 2022Article
- Review
- Double Repositioning: Veterinary Antiparasitic to Human Anticancer.International journal of molecular sciences · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 2 institutions in 1 country.
Funding
Abstract
The hedgehog (Hh) signaling pathway is activated in many types of cancer and therefore presents an attractive target for new anticancer agents. Here, we show that mebendazole, a benzamidazole with a long history of safe use against nematode infestations and hydatid disease, potently inhibited Hh signaling and slowed the growth of Hh-driven human medulloblastoma cells at clinically attainable concentrations. As an antiparasitic, mebendazole avidly binds nematode tubulin and causes inhibition of intestinal microtubule synthesis. In human cells, mebendazole suppressed the formation of the primary cilium, a microtubule-based organelle that functions as a signaling hub for Hh pathway activation. The inhibition of Hh signaling by mebendazole was unaffected by mutants in the gene that encodes human Smoothened (SMO), which are selectively propagated in cell clones that survive treatment with the Hh inhibitor vismodegib. Combination of vismodegib and mebendazole resulted in additive Hh signaling inhibition. Because mebendazole can be safely administered to adults and children at high doses over extended time periods, we propose that mebendazole could be rapidly repurposed and clinically tested as a prospective therapeutic agent for many tumors that are dependent on Hh signaling.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.