A team of scientists from University College London, the University of Sheffield, and Queen's University Belfast announced on Mar. 31 a new stem cell therapy aimed at providing a permanent cure for Hirschsprung disease, a rare condition affecting newborns.
Hirschsprung disease leaves children without the network of nerves required for normal digestion, often resulting in life-threatening complications. Current treatments involve surgical removal of non-functional sections of the gut but do not always resolve symptoms and can lead to ongoing infections and bowel problems.
The research project, funded by £2.7 million from UK Research and Innovation, has developed a method to generate essential nerve cells from human stem cells in just six days—almost twice as fast as previous techniques. These lab-grown cells are described as "off-the-shelf" ready, meaning they can be frozen and transported safely to hospitals.
Dr Conor McCann from UCL Great Ormond Street Institute of Child Health said: "The award of this funding will provide a step change in allowing us to bring our stem cell-based treatment closer to the clinic. We hope that by bringing together the expertise of different groups across the UK we can drive this project towards a clinical trial that will hopefully benefit children with Hirschsprung disease and their families."
The new approach has already shown promise in restoring gut function in patient tissue samples, with further testing planned by clinical teams at UCL Great Ormond Street Institute. The researchers are also developing an economic framework to integrate the therapy into NHS care, with early modelling suggesting it could be more cost-effective than current long-term treatments.
Looking ahead, the team plans safety testing before moving toward first-in-human clinical trials. They also see potential for adapting this technology to treat other severe gut disorders such as achalasia and gastroparesis.
