Organized by Peter Andrews, University of Sheffield, UK
Hirschsprung disease (HSCR), a potentially lethal condition affecting 1 in 5,000 newborn children, is caused by the absence of enteric neurons in the distal bowel. This leads to a loss of propulsive gut motility, ultimately resulting in potentially lethal intestinal obstruction. Current treatment is complex, involves surgical removal of the affected region of the bowel, and often leaves patients with lifelong problems. Several groups around the world, that will be present at this ISSCR meeting, are well advanced in developing approaches using pluripotent stem cells or endogenous stem cells to provide enteric neural progenitors for transplant into the affected gut to relieve the condition. This Meet-up will bring together different teams and initiate an ongoing forum for addressing common issues in taking this approach forward to the clinic, e.g., comparing different cell lines, confirming safety, immunogenicity, appropriate clinical parameters and relevant endpoints for first-in-child clinical trials.