A recent study conducted by researchers at UCL and the University of Sheffield has shed light on the potential of stem cell therapy in treating individuals with Hirschsprung disease.
Hirschsprung disease is a rare condition characterized by the absence of nerve cells in the large intestine. This results in an inability of the intestine to contract and move stool, leading to possible blockages, constipation, and the risk of a severe bowel infection known as enterocolitis.
About 1 in 5000 babies are born with Hirschsprung disease, typically diagnosed soon after birth and managed with surgery. However, patients often experience long-term symptoms and may require multiple surgical interventions.
Given the challenges associated with current treatments, researchers have explored alternative options such as using stem cell therapy to generate nerve cell precursors that can help regenerate the missing nerves in the intestine of individuals with Hirschsprung disease. This approach aims to enhance intestinal function.
Although the concept has been investigated in the past, this study marks the first time this procedure has been conducted on human tissue from individuals with Hirschsprung disease.
The collaborative study, published in Gut and supported by the Medical Research Council, commenced in 2017 and involved researchers from UCL and the University of Sheffield.
Researchers at the University of Sheffield focused on producing and studying nerve precursors from stem cells, which were then sent to the UCL team. The latter team processed patient gut tissue, performed the transplantation of nerve cells, and assessed the functionality of the tissue segments.
The study utilized tissue samples donated by patients with Hirschsprung disease, cultured in the laboratory and transplanted with stem cell-derived nerve cell precursors. These precursors developed into essential nerve cells within the gut tissue.
Notably, the transplanted gut samples exhibited improved contraction ability compared to control tissue, suggesting enhanced gut functionality in individuals with the disease.
Principal Investigator Dr. Conor McCann from UCL Great Ormond Street Institute of Child Health expressed, “This study represents a significant advancement in our cell therapy research for Hirschsprung disease. It underscores the value of interdisciplinary collaboration in potentially benefiting children and adults affected by this condition in the future.”
Dr. Anestis Tsakiridis, Principal Investigator at the University of Sheffield, remarked, “This collaboration, led by the talented early career scientists, Dr. Ben Jevans and Fay Cooper, has been truly remarkable. Our findings lay the groundwork for the future development of cell therapy for Hirschsprung disease, and we are committed to progressing this towards clinical application in the coming years.”
The study results highlight the potential of stem cell therapy in enhancing intestinal function in individuals with Hirschsprung disease, offering the possibility of alleviating symptoms and improving outcomes for affected individuals.
Researchers are now seeking additional funding for clinical trials to further advance this promising treatment approach.
