HAWAII v1
Research type
Research Study
Full title
Investigating the interaction of the enteric nervous system with the extrinsic nervous system and development of cell therapies for Hirschsprung’s Disease Hirschsprung’s Advances; Working towards Autologous Intestinal Interventions (HAWAII)
IRAS ID
322649
Contact name
Rachel Harwood
Contact email
Sponsor organisation
Alder Hey Children's NHS Foundation Trust
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
9 years, 11 months, 30 days
Research summary
The overall aim of this study is to develop new treatments for babies born with Hirschsprung’s disease by promoting new nerves to form from stem cells already present in their bowel. Each year, approximately 170 babies in the UK are born with Hirschsprung’s Disease. This is a condition caused by nerve cells failing to develop at the end of the intestine before a baby is born. Children with Hirschsprung’s Disease are at high risk of developing enterocolitis, an infective and inflammatory process within the bowel which can be life-threatening. Surgery to remove the abnormal intestine is the current gold-standard treatment but children remain at increased risk of enterocolitis, 30% of children have ongoing constipation or incontinence, 10% need a permanent stoma, and fertility is reduced in adulthood. Research at the University of Liverpool and Alder Hey Children’s hospital first demonstrated the existence of stem cells that have the potential to produce nerves in the segment of intestine that lacks nerves. These findings have subsequently been replicated by other research groups.
We aim to stimulate the stem cells from the affected bowel to generate nerve cells in-situ as treatment for patients with Hirschsprung’s disease. We plan to isolate the stem cells and treat them with drugs that reproducibly stimulate the production of functioning nerve cells. Our research will help to develop therapies that can restore normal bowel function and improve surgical outcomes, transforming the lives of people with Hirschsprung’s Disease.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
23/NW/0291
Date of REC Opinion
3 Oct 2023
REC opinion
Further Information Favourable Opinion