Study Identifies Key Steps in Stem Cell Therapy for Rare Bowel Disease, ET HealthWorld

London: A recent study by scientists from the Universities of Sheffield and UCL suggests that stem cell therapy could benefit patients with Hirschsprung disease. This condition is characterized by the absence of nerve cells in parts of the large intestine, leading to difficulties in bowel movements and potential blockages. These blockages can cause constipation and, in severe cases, a dangerous infection called enterocolitis. Hirschsprung disease affects about 1 in 5000 newborns and is typically diagnosed and treated with surgery shortly after birth. However, many patients continue to experience severe, lifelong symptoms and often require multiple surgeries.

Given the limitations of current treatments, researchers have been exploring alternative options. One promising approach involves using stem cell therapy to create nerve cell precursors, which can then develop into the missing nerve cells in the intestines of Hirschsprung disease patients. This method aims to enhance the intestine’s ability to function properly. Until now, this procedure had not been tested on human tissue from Hirschsprung disease patients.

The study, published in the journal Gut and funded by the Medical Research Council, is a collaborative effort that began in 2017. Researchers at the University of Sheffield focused on producing and analyzing nerve precursors from stem cells. These precursors were then sent to the UCL team, who prepared the patient gut tissue, performed the transplantation, maintained the tissue, and tested its functionality.

The study involved using tissue samples donated by patients from Great Ormond Street Hospital (GOSH) as part of their routine treatment. These samples were cultured in the lab and then transplanted with stem cell-derived nerve cell precursors. The transplanted cells developed into the necessary nerve cells within the gut tissue. Importantly, the transplanted gut samples showed a better ability to contract compared to control tissue, indicating improved gut functionality.

Dr. Conor McCann, the Principal Investigator from UCL Great Ormond Street Institute of Child Health, stated, “This study is a significant breakthrough in our cell therapy research for Hirschsprung disease. It highlights the benefits of collaborative expertise, which we hope will improve the lives of children and adults with this condition in the future.”

Dr. Anestis Tsakiridis, Principal Investigator at the University of Sheffield, added, “This has been an excellent collaboration, led by two talented early-career scientists, Dr. Ben Jevans and Fay Cooper. Our findings have laid the groundwork for developing a cell therapy for Hirschsprung disease, and we will continue our efforts to bring this treatment to the clinic in the coming years.”

The results of this study demonstrate, for the first time, the potential of stem cell therapy to enhance the functionality of the intestine in Hirschsprung disease patients. This could lead to improved symptoms and better outcomes for those affected by the disease. (ANI)