White paper on guidelines concerning enteric nervous system stem cell therapy for enteric neuropathies

Alan J Burns; Allan M Goldstein; Donald F Newgreen; Lincon Stamp; Karl-Herbert Schäfer; Marco Metzger; Ryo Hotta; Heather M Young; Peter W Andrews; Nikhil Thapar; Jaime Belkind-Gerson; Nadege Bondurand; Joel C Bornstein; Wood Yee Chan; Kathryn Cheah; Michael D Gershon; Robert O Heuckeroth; Robert M W Hofstra; Lothar Just; Raj P Kapur; Sebastian K King; Conor J McCann; Nandor Nagy; Elly Ngan; Florian Obermayr; Vassilis Pachnis; Pankaj J Pasricha; Mai Har Sham; Paul Tam; Pieter Vanden Berghe

doi:10.1016/j.ydbio.2016.04.001

White paper on guidelines concerning enteric nervous system stem cell therapy for enteric neuropathies

Dev Biol. 2016 Sep 15;417(2):229-51. doi: 10.1016/j.ydbio.2016.04.001. Epub 2016 Apr 5.

Authors

Alan J Burns¹, Allan M Goldstein², Donald F Newgreen³, Lincon Stamp⁴, Karl-Herbert Schäfer⁵, Marco Metzger⁶, Ryo Hotta², Heather M Young⁴, Peter W Andrews⁷, Nikhil Thapar⁸, Jaime Belkind-Gerson⁹, Nadege Bondurand¹⁰, Joel C Bornstein¹¹, Wood Yee Chan¹², Kathryn Cheah¹³, Michael D Gershon¹⁴, Robert O Heuckeroth¹⁵, Robert M W Hofstra¹⁶, Lothar Just¹⁷, Raj P Kapur¹⁸, Sebastian K King¹⁹, Conor J McCann⁸, Nandor Nagy²⁰, Elly Ngan²¹, Florian Obermayr²², Vassilis Pachnis²³, Pankaj J Pasricha²⁴, Mai Har Sham²⁵, Paul Tam²¹, Pieter Vanden Berghe²⁶

Affiliations

¹ Stem Cells and Regenerative Medicine, UCL Great Ormond Street Institute of Child Health, London, UK; Department of Clinical Genetics, Erasmus Medical Center, Rotterdam, The Netherlands. Electronic address: alan.burns@ucl.ac.uk.
² Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
³ Murdoch Childrens Research Institute, Royal Children's Hospital, Parkville 3052, Victoria, Australia.
⁴ Department of Anatomy and Neuroscience, University of Melbourne, Parkville, Victoria 3010, Australia.
⁵ University of Applied Sciences, Kaiserlautern, Germany; Clinic of Pediatric Surgery, University Hospital Mannheim, University Heidelberg, Germany.
⁶ Fraunhofer-Institute Interfacial Engineering and Biotechnology IGB Translational Centre - Würzburg branch and University Hospital Würzburg - Tissue Engineering and Regenerative Medicine (TERM), Würzburg, Germany.
⁷ Centre for Stem Cell Biology, Department of Biomedical Science, University of Sheffield, Sheffield, UK.
⁸ Stem Cells and Regenerative Medicine, UCL Great Ormond Street Institute of Child Health, London, UK.
⁹ Division of Gastroenterology, Hepatology and Nutrition, Massachusetts General Hospital for Children, Harvard Medical School, Boston, USA.
¹⁰ INSERM U955, 51 Avenue du Maréchal de Lattre de Tassigny, F-94000 Créteil, France; Université Paris-Est, UPEC, F-94000 Créteil, France.
¹¹ Department of Physiology, University of Melbourne, Parkville, Victoria 3010, Australia.
¹² School of Biomedical Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong.
¹³ School of Biomedical Sciences, The University of Hong Kong, Hong Kong.
¹⁴ Department of Pathology and Cell Biology, Columbia University, New York 10032, USA.
¹⁵ Department of Pediatrics, The Children's Hospital of Philadelphia Research Institute, Philadelphia, PA 19104, USA; Perelman School of Medicine at the University of Pennsylvania, Abramson Research Center, Philadelphia, PA 19104, USA.
¹⁶ Stem Cells and Regenerative Medicine, UCL Great Ormond Street Institute of Child Health, London, UK; Department of Clinical Genetics, Erasmus Medical Center, Rotterdam, The Netherlands.
¹⁷ Institute of Clinical Anatomy and Cell Analysis, University of Tübingen, Germany.
¹⁸ Department of Pathology, University of Washington and Seattle Children's Hospital, Seattle, WA, USA.
¹⁹ Department of Paediatric and Neonatal Surgery, The Royal Children's Hospital, Melbourne, Australia.
²⁰ Department of Anatomy, Histology and Embryology, Faculty of Medicine, Semmelweis University, Budapest, Hungary.
²¹ Department of Surgery, The University of Hong Kong, Hong Kong.
²² Department of Pediatric Surgery and Pediatric Urology, University Children's Hospital Tübingen, D-72076 Tübingen, Germany.
²³ The Francis Crick Institute, Mill Hill Laboratory, London, UK.
²⁴ Johns Hopkins Center for Neurogastroenterology, Baltimore, MD, USA.
²⁵ Department of Biochemistry, The University of Hong Kong, Hong Kong.
²⁶ Laboratory for Enteric NeuroScience (LENS), TARGID, University of Leuven, Belgium.

Abstract

Over the last 20 years, there has been increasing focus on the development of novel stem cell based therapies for the treatment of disorders and diseases affecting the enteric nervous system (ENS) of the gastrointestinal tract (so-called enteric neuropathies). Here, the idea is that ENS progenitor/stem cells could be transplanted into the gut wall to replace the damaged or absent neurons and glia of the ENS. This White Paper sets out experts' views on the commonly used methods and approaches to identify, isolate, purify, expand and optimize ENS stem cells, transplant them into the bowel, and assess transplant success, including restoration of gut function. We also highlight obstacles that must be overcome in order to progress from successful preclinical studies in animal models to ENS stem cell therapies in the clinic.

Keywords: Cell replacement therapy; Enteric nervous system; Enteric neuropathies; Hirschsprung disease; Stem cells.

Publication types

Review
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Animals
Cell- and Tissue-Based Therapy / methods*
Disease Models, Animal
Enteric Nervous System / pathology*
Gastrointestinal Tract / innervation
Gastrointestinal Tract / pathology*
Guidelines as Topic
Hirschsprung Disease / pathology
Hirschsprung Disease / therapy*
Humans
Intestinal Pseudo-Obstruction / pathology
Intestinal Pseudo-Obstruction / therapy*
Neural Stem Cells / transplantation*
Stem Cell Transplantation*

Abstract

Publication types

MeSH terms

Grants and funding