Repurposing niclosamide ethanolamine for alveolar echinococcosis reveals a disconnect between in vitro efficacy and in vivo outcome in the intraperitoneal infection model
PLOS Neglected Tropical Diseases·
- DOI
- 10.1371/journal.pntd.0014016
- PMID
- 42672097
- PMCID
- —
- OpenAlex
- W7204849537
- Study type
- Journal article
- Publisher
- Public Library of Science (PLoS)
- Article type
- journal-article
- Integrity
- current
Why this research matters now
Alveolar echinococcosis is a lethal neglected disease when untreated, and current therapies with albendazole or mebendazole are parasitostatic rather than curative. The findings underscore ongoing challenges in developing effective treatments and highlight the importance of bridging in vitro promise with in vivo performance.
Structured evidence summary
Research question
The study evaluated whether niclosamide ethanolamine, a formulation with improved systemic exposure, could effectively treat Echinococcus multilocularis infection both in laboratory cultures and in a mouse model of alveolar echinococcosis.
Study design
The investigators conducted in vitro assays using E. multilocularis metacestode vesicles to measure drug efficacy, followed by in vivo testing in a mouse model using intraperitoneal infection.
Population and setting
The study used E. multilocularis metacestode vesicles in laboratory culture and experimentally infected mice with intraperitoneal E. multilocularis infection.
Main findings
Niclosamide and niclosamide ethanolamine showed potent activity against E. multilocularis metacestode vesicles in vitro, with IC50 values below 1 micromolar. However, this laboratory efficacy did not translate to the mouse infection model, where no meaningful anti-parasitic activity was observed.
Public-health relevance
Alveolar echinococcosis is a lethal neglected disease when untreated, and current therapies with albendazole or mebendazole are parasitostatic rather than curative. The findings underscore ongoing challenges in developing effective treatments and highlight the importance of bridging in vitro promise with in vivo performance.
Important limitations
The authors attribute the lack of in vivo activity to potential factors including the infection model used, limited drug uptake by the parasite in the animal, or rapid metabolization of the compound.
GIDS interpretation
This article is discoverable through disease classifier links for echinococcosis and topic links for One Health and Treatment. It represents peer-reviewed research on experimental therapeutics for a neglected parasitic disease and does not report surveillance data or outbreak investigations.
Related GIDS surveillance
Literature context does not validate, explain, or change a surveillance signal. Exact and contextual relationships are shown separately.
Evidence relationships
This article has 7 auditable classifier relationships to diseases, places, topics, and study design.