Abstract

To address the cuticular barrier in anthelmintic discovery, this study establishes a bifurcated framework in Caenorhabditis elegans. First, HPLC-based quantification of 14 PIM inhibitors revealed that cuticular penetrance is primarily dictated by phylogenetic lineage (Clade V) rather than ecological niche. Specifically, C. elegans demonstrated a significant pharmacokinetic correlation with the hookworm Ancylostoma ceylanicum (Clade V) but not the whipworm Trichuris muris (Clade I), validating it as a superior surrogate model for hookworm bioaccumulation. Within this small molecule inhibitor library, SGI-12was identified as a lead candidate with superior potency against C. elegans and bioaccumulation. Second, target validation using a C. elegans prk-1; prk-2 double mutant demonstrated that while VB4182 induces spastic paralysis in wild-type nematodes, the kinase-deficient lines exhibit complete phenotypic resistance. Notably, these mutants displayed a constitutive "Long" (Lon) phenotype, suggesting that PRK-1/2 negatively regulate nematode growth. These findings identify PRK-1/2 as the specific in vivo targets of VB4182 and provide a validated methodology to decouple drug permeation from target inhibition in hookworm-targeted drug discovery.

Committee Chair

Makedonka Mitreva

Committee Members

Bruce A. Rosa, Michael Vahey

Degree

Master of Science (MS)

Author's Department

Biomedical Engineering

Author's School

McKelvey School of Engineering

Document Type

Thesis

Date of Award

Spring 5-2026

Language

English (en)

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