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Stem Cells Transl Med. 2015 Apr;4(4):389-400. doi: 10.5966/sctm.2014-0110. Epub 2015 Feb 26.

Concise review: workshop review: understanding and assessing the risks of stem cell-based therapies.

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Medical Research Council Centre for Drug Safety Science, Division of Molecular & Clinical Pharmacology, Institute of Translational Medicine, and Institute of Integrative Biology, University of Liverpool, Liverpool, U.K.; Division of Molecular and Systems Toxicology, Department of Pharmaceutical Sciences, Pharmazentrum, University of Basel, Basel, Switzerland; Laboratorios Almirall, S.A. Laurea Miro, Sant Feliu de Llobregat, Spain; SANOFI-Research & Development, Disposition, Safety and Animal Research, Alfortville, France; Uppsala Biomedicinska Centrum BMC, Husarg, Uppsala University, Uppsala, Sweden; Centre for Stem Cell Biology, Department of Biomedical Science, University of Sheffield, Sheffield, U.K.; Developmental & Regenerative Biomedicine, School of Biomedicine, Faculty of Human and Medical Sciences, University of Manchester, Manchester, U.K.; Medical Research Council Centre for Regenerative Medicine, University of Edinburgh, Edinburgh, U.K.; Department of Gastroenterology, Hepatology and Infectious Diseases, Heinrich-Heine University Düsseldorf, Düsseldorf, Germany; OakMore Solutions Ltd., Faversham, U.K.; ReNeuron Limited, Guildford, U.K.; Medicines and Healthcare Products Regulatory Agency, London, U.K.; Paul Ehrlich Institut, Langen, Germany; Faculty of Science, Leiden Academic Centre for Drug Research, Toxicology, Leiden, The Netherlands; Stem Cell Unit, Department of Genetics, Institute of Life Sciences, Hebrew University of Jerusalem, Givat-Ram, Jerusalem, Israel; U.K. Stem Cell Bank, National Institute for Biological Standards and Control, Medicines and Healthcare Products Regulatory Agency, Potters Bar, U.K.; Cellectis AB, Göteborg, Sweden; GSK, David Jack Centre for Research & Development, Ware, U.K.; Personalised Healthcare and Biomarkers, AstraZeneca, Macclesfield, U.K.; CN-bio Innovations Limited, Centre for Innovation & Enterprise, Begbroke, U.K.; Institute of Veterinary Pathology, University of Zurich, Zurich, Switzerland; School of Bio Scien


The field of stem cell therapeutics is moving ever closer to widespread application in the clinic. However, despite the undoubted potential held by these therapies, the balance between risk and benefit remains difficult to predict. As in any new field, a lack of previous application in man and gaps in the underlying science mean that regulators and investigators continue to look for a balance between minimizing potential risk and ensuring therapies are not needlessly kept from patients. Here, we attempt to identify the important safety issues, assessing the current advances in scientific knowledge and how they may translate to clinical therapeutic strategies in the identification and management of these risks. We also investigate the tools and techniques currently available to researchers during preclinical and clinical development of stem cell products, their utility and limitations, and how these tools may be strategically used in the development of these therapies. We conclude that ensuring safety through cutting-edge science and robust assays, coupled with regular and open discussions between regulators and academic/industrial investigators, is likely to prove the most fruitful route to ensuring the safest possible development of new products.


Adult stem cells; Autologous stem cell transplantation; Cellular therapy; In vivo tracking; Pluripotent stem cells; Stem cell; Stem cell expansion; Stem cell transplantation

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