A study led by researchers at UCL and the University of Sheffield has shown the potential of stem cell therapy to treat Hirschsprung disease, a rare condition where missing nerve cells in the large intestine prevent it from contracting and moving stool, leading to constipation and serious infections. Currently affecting about 1 in 5000 babies, Hirschsprung disease is usually treated with surgery soon after birth, but patients often suffer lifelong symptoms and require multiple surgeries. The study explored using stem cell therapy to generate nerve cell precursors that can produce the missing nerves in the intestine, potentially improving its functionality.

Researchers produced and analyzed nerve precursors from stem cells, which were then transplanted into gut tissue samples from patients with Hirschsprung disease by the UCL team. The transplanted samples showed improved contraction ability compared with control tissue, indicating enhanced gut functionality. The study’s success marks a breakthrough in cell therapy for Hirschsprung disease. It also lays the foundation for future clinical trials and potential new treatments to improve symptoms and outcomes for impacted individuals.

Reference: Important step forward in stem cell therapy for rare bowel disease. UCL News. Published June 27, 2024. Accessed July 31, 2024. https://www.ucl.ac.uk/news/2024/jun/important-step-forward-stem-cell-therapy-rare-bowel-disease

Link: https://www.ucl.ac.uk/news/2024/jun/important-step-forward-stem-cell-therapy-rare-bowel-disease