Stem Cell Therapy Offers New Hope for Hirschsprung's Disease Patients

A new study published in the journal Gut suggests that stem cell therapy may be beneficial in treating rare bowel disease, such as Hirschsprung’s disease.

The research was conducted by scientists at the Universities of Sheffield and UCL. They found that Hirschsprung’s disease is associated with a small number of nerve cells in the large intestine being absent. This condition causes the intestine to be unable to contract and transport stool, which can lead to constipation and, in rare cases, an intestinal infection called enterocolitis.

Around 1 in 5000 babies are born with Hirschsprung disease. The condition is usually diagnosed shortly after birth and treated with surgery as soon as possible. However, many patients with Hirschsprung disease experience debilitating and lifelong symptoms that often require multiple surgical procedures.

Alternative treatment options for Hirschsprung disease are therefore crucial. One such option is stem cell therapy, which involves taking bone marrow or skin cells from a patient and genetically engineering them to become nerve cells. These nerve cells are then transplanted into the patient’s gut to replace the missing nerves in the large intestine.

This procedure has not been carried out on human tissue from people with Hirschsprung disease yet. The current study is a collaborative effort between UCL and the University of Sheffield that began in 2017. The researchers successfully transplanted nerve cell precursors into the gut tissue of GOSH patients with Hirschsprung disease. The results of the study showed that the transplanted gut samples were able to contract significantly more effectively than control tissue, suggesting improved functionality of the gut in those with Hirschsprung disease.

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