Why mebendazole?
Among the three substances on this site, MBZ has the most formal clinical oncology track: USA (Johns Hopkins preclinical + phase I, Michigan case report) → phase II India (recurrent GBM) → Egypt (colorectal) etc. See the collected USA page: USA — positive signals.
USA — positive human signals
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C Dobrosotskaya et al., 2011 — University of MichiganMebendazole monotherapy and long-term disease control in metastatic adrenocortical carcinoma.
Metastatic adrenal cortical cancer after multiple failed treatments. MBZ 100 mg × 2/day monotherapy: metastases regressed and disease stable for 19 months, good tolerance.
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B Gallia / Johns Hopkins, 2021 — phase IMedian OS 21 mo in newly diagnosed HGG + TMZ + MBZ (n=24); doses to 200 mg/kg; not a primary efficacy trial but a positive survival signal.
Preclinical — glioblastoma
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D Bai et al., 2011 — survival benefit in two GBM modelsAntiparasitic mebendazole shows survival benefit in 2 preclinical models of glioblastoma multiforme.
IC50 in GBM lines ~0.1–0.3 µM; microtubule disruption; mean survival extended up to ~63% in orthotopic mouse models.
Phase I — newly diagnosed high-grade glioma + TMZ
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B Gallia et al., 2021Mebendazole and temozolomide in patients with newly diagnosed high-grade gliomas… NCT01729260.
24 patients; dose escalation 25–200 mg/kg/day with adjuvant temozolomide. Grade 3 ALT/AST in some at 200 mg/kg, reversible. Authors: long-term safety acceptable; efficacy needs further study.
Phase II — recurrent glioblastoma (Patil 2022)
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A Patil et al., 2022 — IndiaMebendazole plus lomustine or temozolomide in patients with recurrent glioblastoma. EClinicalMedicine.
88 patients; both arms had MBZ (CCNU+MBZ vs TMZ+MBZ). MBZ well tolerated. Study missed its internal OS target (9-mo 55%) in this hard recurrent population — one of few formal “missed endpoints”, not proof that MBZ is harmful. See short limitations list.
Colorectal & GI
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A Hegazy et al. — small mCRC RCT (Egypt)MBZ 500 mg ×2 + FOLFOX4/bevacizumab vs standard (NCT03925662, n=40): higher ORR and longer PFS in MBZ arm (single-center, not independently replicated).
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B Mansoori et al., 2021 — GI monotherapy phase 2aRefractory GI cancer (NCT03628079): monotherapy without meaningful response — highlights mono vs combo difference.
Other registries
| ID | Description | Link |
|---|---|---|
| R NCT01837862 | Phase I/II MBZ in pediatric gliomas + standard | ClinicalTrials.gov |
| R NCT02644291 | Phase I MBZ recurrent pediatric brain cancers | ClinicalTrials.gov |
| R NCT03925662 | MBZ + chemo metastatic colorectal cancer | ClinicalTrials.gov |