Stem cell technology shows promise for rare intestinal disease
A recent study has shown that stem cell technology could offer a viable treatment for Hirschsprung disease, potentially reducing the need for multiple surgeries.
Hirschsprung disease is a rare genetic disorder occurring in around 1 in every 5,000 live births. Patients are born with some nerve cells missing in a portion of the large intestine, meaning it can’t contract normally to move stool. This can cause chronic constipation and colon swelling, leaving patients prone to painful enterocolitis and at risk of bowel perforation.
The condition is generally diagnosed early and patients undergo surgery to remove the defective part of their colon. Still, patients often remain afflicted with lifelong debilitating symptoms and many must undergo numerous surgeries as there are currently no other viable treatment options.
The team of researchers from the University of Sheffield and University College London (both UK) sought to develop an alternative strategy using stem cell technology to repopulate the intestines with functional nerve cells. The team based in Sheffield developed nerve cell precursors from embryonic stem cells, which were then shipped down to University College London. The London-based researchers cultured intestinal tissue samples donated by Hirschsprung disease patients and seeded them with the stem-cell-derived nerve precursors, which developed into nerve cells.
Critically, the intestinal samples that were seeded with stem-cell-derived nerve cell precursors displayed better contraction than control samples – a promising sign that stem cell seeding could be a viable avenue for Hirschsprung disease treatment.
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“This study is a real breakthrough in our cell therapy work for Hirschsprung disease,” explains Connor McCann, principal investigator of the study. “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.”
The team is currently seeking funds to continue their research and progress to clinical trials.