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Stem cell therapy in severe pediatric motility disorders

Conor J McCann1, Osvaldo Borrelli2, Nikhil Thapar3

  • 1Stem Cells and Regenerative Medicine, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK.

Current Opinion in Pharmacology
|October 20, 2018
PubMed

Insights

Stem cell therapy shows promise for pediatric gastrointestinal motility disorders. Human enteric neural stem cells (ENSCs) successfully engrafted in animal models, offering hope for functional recovery and new treatments.

Area of Science:

  • Gastroenterology and Regenerative Medicine
  • Developmental Biology
  • Neuroscience

Background:

  • Pediatric gastrointestinal motility disorders are severe conditions impairing enteric neuromuscular function.
  • Current therapeutic options for these disorders are limited.
  • Stem cell-based treatments offer a promising avenue for improved or curative therapies.

Purpose of the Study:

  • To evaluate the potential of human enteric neural stem cells (ENSCs) for treating pediatric gastrointestinal motility disorders.
  • To assess the engraftment and functional rescue capabilities of transplanted ENSCs in vivo.

Main Methods:

  • Harvesting, propagation, and transplantation of human-derived ENSCs.
  • In vivo transplantation studies in animal models of enteric neuropathy.
  • Assessment of ENSC engraftment and functional recovery in recipient intestines.

Main Results:

  • Successful demonstration of harvesting, propagation, and transplantation of human ENSCs.
  • Confirmation of ENSC engraftment within the recipient intestine of animal models.
  • Evidence of functional rescue in animal models following ENSC transplantation.

Conclusions:

  • Human ENSCs are capable of engraftment and functional rescue in animal models of enteric neuropathy.
  • Stem cell-based therapies, including pluripotent stem cell-derived cells and pharmacological modulation, hold significant potential for clinical application in treating pediatric gastrointestinal motility disorders.
  • These findings pave the way for future clinical translation of stem cell therapies for these debilitating conditions.

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