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Cellular-Based Therapies for Paediatric GI Motility Disorders

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Pediatric Neurogastroenterology

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Abstract

Therapeutic options for many gastrointestinal motility disorders, especially the most severe, remain inadequate and limited to palliative interventions. Advances in molecular biology and genetics have led to the identification of stem cells as potential tools for curative therapies. The field of neural stem cell therapies for enteric neuropathies has seen tremendous progress in recent years. A variety of sources for such neural stem cells have been identified ranging from embryonic stem cells to induced pluripotent stem (iPS) cells. The latter have been harvested from postnatal human gut using minimally invasive techniques such as conventional endoscopy raising exciting possibilities for therapy including autologous stem cell transplantation. Key recent developments include reports of the efficient derivation and isolation of ENS progenitors from human pluripotent stem cells and their further differentiation into functional enteric neurons, successful in vivo transplantation of neural stem cells into the gastrointestinal tract and improved access to donor tissue. A number of key challenges remain, however, before effective clinical application. These include more effective assessment of transplant success and the need to better understand individual target diseases and, in turn, to tailor cellular tools to optimise their treatment. Nonetheless, the impressive recent progress raises further hope for the imminent use of stem cells as effective therapies for enteric neuromuscular diseases.

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Authors and Affiliations

  1. Department of Surgery, Harvard Medical School, Boston, MA, USA

    Ryo Hotta M.D., Ph.D.

  2. Stem Cells and Regenerative Medicine, UCL Institute of Child Health, London, UK

    Dipa Natarajan M.D. & Alan J. Burns Ph.D.

  3. Division of Neurogastroenterology and Motility, Department of Paediatric Gastroenterology, UCL Institute of Child Health and Great Ormond Street Hospital, London, UK

    Nikhil Thapar M.D., Ph.D.

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  1. Ryo Hotta M.D., Ph.D.
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  1. Division of Pediatric Gastroenterology Hepatology and Nutrition, CHU Sainte-Justine, Montreal, Québec, Canada

    Christophe Faure

  2. Department of Paediatric Gastroenterology, UCL Institute of Child Health, London, United Kingdom

    Nikhil Thapar

  3. Division of Pediatric Gastroenterology Hepatology, and Nutrition, Nationwide Children’s Hospital, Columbus, Ohio, USA

    Carlo Di Lorenzo

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Hotta, R., Natarajan, D., Burns, A.J., Thapar, N. (2017). Cellular-Based Therapies for Paediatric GI Motility Disorders. In: Faure, C., Thapar, N., Di Lorenzo, C. (eds) Pediatric Neurogastroenterology. Springer, Cham. https://doi.org/10.1007/978-3-319-43268-7_49

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  • DOI: https://doi.org/10.1007/978-3-319-43268-7_49

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