Study finds key step in stem cell therapy for rare bowel disease, ET HealthWorld

Study finds key step in stem cell therapy for rare bowel disease, ET HealthWorld


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LONDON: Patients with Hirschsprung's disease may benefit from stem cell therapy, according to a recent study conducted by scientists from the Universities of Sheffield and UCL.

In the case of Hirschsprung's disease, a small number of nerve cells in the large intestine are absent. Due to the shrinkage of the intestine and its inability to transport stool, blockages can occur. This can result in constipation and, in rare cases, a dangerous bowel infection called enterocolitis.

About 1 in 5,000 babies are born with Hirschsprung's disease. The condition is usually detected soon after birth and treated with surgery as soon as possible, although patients often suffer from debilitating, lifelong symptoms, often requiring multiple surgical procedures.

Therefore alternative treatment options are important. One option explored by researchers involves generating nerve cell precursors using stem cell therapy, which after transplantation produces missing nerves in the intestine of people with Hirschsprung's disease. This in turn should improve the functionality of the intestine.

However, this procedure has not yet been performed on human tissue from people with Hirschsprung's disease.

The research, published in Gut and funded by the Medical Research Council, is a collaborative effort between researchers at UCL and the University of Sheffield that began in 2017.

Researchers at the University of Sheffield focused on producing and analysing neural precursors from stem cells. These were then sent to the UCL team, who prepared the patient's intestinal tissue, transplanted and maintained the tissue and then tested the function of the tissue segments.

The study involved tissue samples donated by GOSH patients suffering from Hirschsprung's disease as part of their routine treatment, which were then cultured in the laboratory. The samples were then transplanted with stem cell-derived nerve cell precursors, which then developed into vital nerve cells within the intestinal tissue.

Importantly, the transplanted intestine samples showed increased contractile ability compared to control tissue, indicating improved intestinal function in people with the disease.

Principal investigator, Dr Conor McCann (UCL Great Ormond Street Institute of Child Health), said: “This study is a real breakthrough in our cell therapy work for Hirschsprung's disease. It really shows the benefit of bringing together the expertise of different groups, which will hopefully benefit children and adults with Hirschsprung's disease in the future.”

Principal investigator Dr Anestis Tsakiridis from the University of Sheffield said: “This has been a fantastic collaboration, led by two talented early career scientists, Drs Ben Jevans and Fay Cooper. Our findings have laid the foundation for the future development of cell therapies against Hirschsprung's disease and we will continue our efforts to bring this into the clinic over the next few years”.

The results of this study show for the first time that stem cell therapy can improve intestinal function in people with Hirschsprung's disease, leading to improved symptoms and better outcomes for those with the disease. (ANI)

  • Published on July 2, 2024 at 04:42 PM IST

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