London [UK], Patients with Hirschsprung's disease may benefit from stem cell therapy, according to a recent study by scientists at the Universities of Sheffield and UCL.
In Hirschsprung's disease, a small number of nerve cells are missing in the large intestine. Due to the inability of the intestine to contract and transport stool, blockages can occur. This can lead to constipation and, in rare cases, a dangerous intestinal infection known as enterocolitis.
About 1 in 5,000 babies are born with Hirschsprung disease. The condition is usually detected soon after birth and treated with surgery as soon as possible; However, patients frequently suffer debilitating symptoms that last a lifetime and often require multiple surgical procedures.
Therefore, alternative treatment options are crucial. One option researchers have explored involves using stem cell therapy to generate nerve cell precursors, which then produce the missing nerves in the intestines of people with Hirschsprung's disease after transplant. This, in turn, should improve the functionality of the intestine.
However, until now this procedure had not been carried out 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 the production and analysis of nerve precursors from stem cells. They were then sent to the UCL team, who prepared the patient's intestinal tissue, carried out tissue transplantation and maintenance, and then tested the function of the tissue segments.
The study involved taking tissue samples donated by GOSH patients with Hirschsprung's disease as part of their routine treatment, which were then grown in the laboratory. The samples were then transplanted with stem cell-derived nerve cell precursors that then developed into crucial nerve cells within the intestinal tissue.
Importantly, transplanted intestine samples showed greater shrinkage capacity compared to control tissue, suggesting better intestine functionality in those with the disease.
Principal investigator Dr Conor McCann (UCL Great Ormond Street Institute of Children's 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 expertise. from different groups that will hopefully benefit children and adults living with Hirschsprung disease in the future.
Dr Anestis Tsakiridis, Principal Investigator at the University of Sheffield, said: "This has been a fantastic collaboration, led by two talented early career scientists, Dr Ben Jevans and Fay Cooper. Our findings have laid the foundation for the future development of a cell therapy against Hirschsprung's disease and we will continue our efforts to bring it to the clinic in the coming years.
The results of this study demonstrate for the first time the potential of stem cell therapy to improve gut functionality in people with Hirschsprung's disease, which, in turn, could lead to better symptoms and better outcomes for people with the disease. disease.
In Hirschsprung's disease, a small number of nerve cells are missing in the large intestine. Due to the inability of the intestine to contract and transport stool, blockages can occur. This can lead to constipation and, in rare cases, a dangerous intestinal infection known as enterocolitis.
About 1 in 5,000 babies are born with Hirschsprung disease. The condition is usually detected soon after birth and treated with surgery as soon as possible; However, patients frequently suffer debilitating symptoms that last a lifetime and often require multiple surgical procedures.
Therefore, alternative treatment options are crucial. One option researchers have explored involves using stem cell therapy to generate nerve cell precursors, which then produce the missing nerves in the intestines of people with Hirschsprung's disease after transplant. This, in turn, should improve the functionality of the intestine.
However, until now this procedure had not been carried out 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 the production and analysis of nerve precursors from stem cells. They were then sent to the UCL team, who prepared the patient's intestinal tissue, carried out tissue transplantation and maintenance, and then tested the function of the tissue segments.
The study involved taking tissue samples donated by GOSH patients with Hirschsprung's disease as part of their routine treatment, which were then grown in the laboratory. The samples were then transplanted with stem cell-derived nerve cell precursors that then developed into crucial nerve cells within the intestinal tissue.
Importantly, transplanted intestine samples showed greater shrinkage capacity compared to control tissue, suggesting better intestine functionality in those with the disease.
Principal investigator Dr Conor McCann (UCL Great Ormond Street Institute of Children's 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 expertise. from different groups that will hopefully benefit children and adults living with Hirschsprung disease in the future.
Dr Anestis Tsakiridis, Principal Investigator at the University of Sheffield, said: "This has been a fantastic collaboration, led by two talented early career scientists, Dr Ben Jevans and Fay Cooper. Our findings have laid the foundation for the future development of a cell therapy against Hirschsprung's disease and we will continue our efforts to bring it to the clinic in the coming years.
The results of this study demonstrate for the first time the potential of stem cell therapy to improve gut functionality in people with Hirschsprung's disease, which, in turn, could lead to better symptoms and better outcomes for people with the disease. disease.
