Grant Award Details

A new precision medicine based iPSC-derived model to study personalized intestinal fibrosis treatments in pediatric patients with Crohn’s disease
Grant Number: 
DISC2-13441
Project Objective: 
  • To develop an intestinal organoid based screening platform that will enable investigators to identify and study antifibrotic drugs for treating pediatric Crohn’s Disease
Investigator: 
Type: 
PI
Disease Focus: 
Intestinal Disease
Metabolic Disorders
Human Stem Cell Use: 
iPS Cell
Cell Line Generation: 
iPS Cell
Award Value: 
$776,340
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • A new precision medicine based iPSC-derived model to study personalized intestinal fibrosis treatments in pediatric patients with Crohn’s disease
Public Abstract: 

Research Objective

We propose to discover a tool that will utilize patient specific iPSC-derived human mini-guts to identify personalized antifibrotic treatments in pediatric Crohn’s disease patients

Impact

The major bottleneck in intestinal fibrosis research is the difficulty in obtaining patient-specific biologically relevant cells for in vitro modeling. This iPSC-derived tool would overcome it.

Major Proposed Activities

  • Procurement of patient specific cells from pediatric Crohn's disease patients that have intestinal fibrosis (Months 0-3)
  • Reprogram each patients harvested cells to form induced pluripotent stem cells (month 3-9)
  • Determine compounds via high throughput screening that attenuate the fibrotic response in each patient’s iPSC-derived cells (Months 9-15)
  • Validation of candidate compounds in corresponding biopsy-derived cells (Months 15-18)
  • Transcriptomic analysis between paired iPSC-derived cells and biopsy derived cells (Months 18-24)
Statement of Benefit to California: 

Crohn's disease is a recurring inflammatory disorder that affects the intestine, and the number of patients that suffer from this in the US continues to rise each year. There are numerous patients who have Crohn's disease in California and 20-30% of these patients will require surgery due to intestinal fibrosis. There is no therapy to prevent or treat this but the tool proposed would establish a platform that would allow numerous therapies to be tested in a personalized manner