Advancing translational cell therapy for Hirschsprung disease

Babies born with Hirschsprung disease are missing nerve cells in their lower bowel, and surgeons are now testing whether lab-grown replacement nerve cells can restore normal gut function. The standard treatment—removing the affected section of bowel—often leaves children with lifelong constipation, incontinence, or bowel inflammation. This project transplants enteric nervous system progenitors, derived from human pluripotent stem cells, into Hirschsprung-affected gut tissue to see whether the cells integrate and fire correctly. The team is also testing how common immunosuppressive drugs affect the transplanted cells, and developing genetically modified “universal” donor cells that could evade immune rejection without long-term drug regimens. If the approach works, it could replace a crude surgical resection with a regenerative repair that restores the gut’s own nerve network. That would shift treatment from managing chronic digestive problems to preventing them in the first place. The work is still at the preclinical stage—the cells are being tested in human gut tissue in the lab—so no timeline for patient use exists yet. But the study directly addresses the main barrier to any cell therapy for Hirschsprung disease: getting donor cells to survive, connect, and function inside a hostile immune environment.