Grant: Project Grant

Hirschsprung disease (HSCR) is a deadly congenital malformation where the enteric nervous system (ENS) is missing from the colon. Because the ENS controls gastrointestinal motility, the ENS-devoid segment remains contracted, resulting in obstruction by fecal material (megacolon). Untreated, children with HSCR are at high risk of developing fatal systemic infections as a result of gut-resident bacteria crossing the colon wall. Current treatment of HSCR consists of painful surgical removal of the ENS-devoid segment of the colon followed by re-connection of the innervated colon to the anus. Unfortunately, many children experience post-surgical complications like stool leakage and life-threatening inflammation, often leading to hospital re-admission and additional surgery. Together with our partner, Neurenati Therapeutics, our group is developing a novel therapeutic compound for HSCR named NEU-001. Studies in mice show that NEU-001 targets neuron precursors in the diseased segment and induces their differentiation into neurons, thereby regenerating the ENS and restoring intestinal function. Our ultimate goal is to bring this therapy to the clinic for the benefit of children with HSCR. To attract potential investors and achieve this goal, we propose to: 1-Evaluate the therapeutic value of NEU-001 for other gastrointestinal diseases (inflammatory bowel disease (IBD) and gut-first Parkinson disease (PD)) and thereby increase NEU-001 market size. 2- Initiate toxicity studies in animals required to support clinical trials for NEU-001 in humans. Patents have been filed to protect NEU-001 administration for the treatment of HSCR. Additional patents will be filed once NEU-001 efficacy for other indications is demonstrated. We believe NEU-001 represents a potentially curative treatment for several gastrointestinal diseases and are committed to bringing this therapy to clinical trials for the benefit of patients with HSCR, IBD and gut-first PD in Canada and worldwide.