Towards stem cell therapies for gut disorders

Researchers at Great Ormond Street Hospital (GOSH) have taken the first steps towards using stem cells to repair damaged gut tissue in a pioneering new study that could hold promise for children and adults with serious gastrointestinal disorders.

In the study, which was led by National Institute for Health Research (NIHR) GOSH Biomedical Research Centre-supported Dr Conor McCann, researchers found that stem cells could be harvested from guts and expanded using a cocktail of molecules. After being transplanted into the colon these cells allowed damaged nerves in the gut to regrow. Stem cells have the potential to develop into many cells in the human body and therefore offer exciting possibilities for repairing many different types of damaged tissue. This is the first time that this type of gut stem cell (enteric neural stem cells) have been shown to restore the function of the digestive tract.

Gut disorders such as achalasia, gastroparesis, and Hirschsprung disease severely impact on quality of life and can be life threatening without intervention. Unfortunately, the root cause of many intestinal disorders is unknown and surgery is often required but in a significant proportion of patients, surgery is associated with poor outcomes. A stem cell based therapy could provide a much more attractive therapy for these life-limiting conditions and using stem cells harvested from the patient’s own colon (as in this study) means the chance of the transplanted tissue being rejected is low. So far Dr McCann’s team have shown that gut stem cells are effective at repairing damaged gut tissue in the lab and are now further refining this approach so that eventually it may be used in the clinic to treat adults and children with gastrointestinal conditions.

Other current research by Dr McCann’s team seeks to understand the underlying cause of human gut disease and to develop new diagnostic tests that may lead to the development of personalised treatments for these debilitating conditions.

Dr McCann was awarded a Top Abstract Prize at the largest conference in Europe dedicated to gastroenterology (UEGW 2016) and received a UCL Great Ormond Street Institute of Child Health Research Excellence Award for his paper in Nature Communications. Dr McCann has also been awarded the prestigious Core-Derek Butler Fellowship to continue his work, at the UCL Great Ormond Street Institute of Child Health, on the therapeutic potential of enteric neural stem cells in upper gastrointestinal tract disorders.

This research was supported by the National Institute for Health Research (NIHR) and by Great Ormond Street Hospital Children's Charity via an award to Dr Nikhil Thapar.