Concise review: mind the gap: challenges in characterizing and quantifying cell- and tissue-based therapies for clinical translation

Article


Rayment, Erin A. and Williams, David J.. 2010. "Concise review: mind the gap: challenges in characterizing and quantifying cell- and tissue-based therapies for clinical translation." Stem Cells. 28 (5), pp. 996-1004. https://doi.org/10.1002/stem.416
Article Title

Concise review: mind the gap: challenges in characterizing and quantifying cell- and tissue-based therapies for clinical translation

ERA Journal ID3381
Article CategoryArticle
AuthorsRayment, Erin A. (Author) and Williams, David J. (Author)
Journal TitleStem Cells
Journal Citation28 (5), pp. 996-1004
Number of Pages9
Year2010
Place of PublicationDurham, NC. United States
ISSN1066-5099
1549-4918
Digital Object Identifier (DOI)https://doi.org/10.1002/stem.416
Web Address (URL)http://onlinelibrary.wiley.com/doi/10.1002/stem.416/pdf
Abstract

There are many challenges associated with characterizing and quantifying cells for use in cell- and tissue-based therapies. From a regulatory perspective, these advanced treatments must not only be safe and effective but also be made by high-quality manufacturing processes that allow for on-time delivery of viable products. Although sterility assays can be adapted from conventional bioprocessing, cell- and tissue-based therapies require more stringent safety assessments, especially in relation to use of animal products, immune reaction, and potential instability due to extended culture times. Furthermore, cell manufacturers who plan to use human embryonic stem cells in their therapies need to be particularly stringent in their final purification steps, due to the unrestricted growth potential of these cells. This review summarizes the current issues in characterization and quantification for cell- and tissue-based therapies, dividing these challenges into the regulatory themes of safety, potency, and manufacturing quality. It outlines current assays in use, as well as highlights the limits of many of these product release tests. Mode of action is discussed, with particular reference to in vitro surrogate assays that can be used to provide information to correlate with proposed in vivo patient efficacy. Importantly, this review highlights the requirement for basic research to improve current knowledge on the in vivo fate of these treatments; as well as an improved stakeholder negotiation process to identify the measurement requirements that will ensure the manufacture of the best possible cell- and tissue-based therapies within the shortest timeframe for the most patient benefit.

Keywordsadult stem cells; cell transplantation; cellular therapy; embryonic stem cells; mesenchymal stem cells; stem cell transplantation
ANZSRC Field of Research 2020401409. Manufacturing safety and quality
320606. Regenerative medicine (incl. stem cells)
310602. Bioprocessing, bioproduction and bioproducts
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Byline AffiliationsLoughborough University, United Kingdom
Institution of OriginUniversity of Southern Queensland
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