Oral elafin formulation for intestinal fibrosis

NIH RePORTER · NIH · R01 · $375,130 · view on reporter.nih.gov ↗

Abstract

Project Summary Prolonged Crohn’s disease leads to intestinal fibrosis, which is difficult to prevent or treat. The surgical resection may negatively impact a patient’s quality of life. Thus, new therapeutic approaches are actively sought after. We have recently shown that intestinal expression of antimicrobial peptide and protease inhibitor elafin is reduced in stricturing CD patients. Its potential protective effect in intestinal fibrosis has never been reported in the literature. Our preliminary data showed that elafin- overexpression inhibited preexisting colonic fibrosis in TNBS-mediated mice, Salmonella-infected mice, and SAMP1/YitFc mice. We generated an orally active elafin-Eudragit formulation for potential therapeutic applications. The elafin-Eudragit formulation reversed preexisting colonic fibrosis in TNBS-treated mice. We also discovered that elafin inhibits collagen expression in the intestinal fibroblasts by modulating several stricture-specific genes. We hypothesize that oral administration of modified elafin may be useful for treating colitis-associated intestinal fibrosis by affecting a stricture- specific pathway. We propose to (1) establish oral therapeutic regimens of modified elafin for treating intestinal fibrosis in a mouse model of preexisting intestinal fibrosis and (2) determine the cathepsin S- PAR2-miR205-ZEB1-mediated anti-fibrogenic mechanism of elafin in intestinal fibroblasts. The overall impact of this study will produce a comprehensive assessment of optimized oral elafin delivery approaches for intestinal fibrosis. This mechanistic study will reveal how and why elafin inhibits intestinal fibrosis.

Key facts

NIH application ID
10444865
Project number
1R01DK128142-01A1
Recipient
UNIVERSITY OF CALIFORNIA LOS ANGELES
Principal Investigator
Hon Wai Koon
Activity code
R01
Funding institute
NIH
Fiscal year
2022
Award amount
$375,130
Award type
1
Project period
2022-04-01 → 2026-02-28