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

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. ISSN 1066-5099


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.

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Item Type: Article (Commonwealth Reporting Category C)
Refereed: Yes
Item Status: Live Archive
Additional Information: © AlphaMed Press. Permanent restricted access to published version due to publisher copyright policy.
Faculty/School / Institute/Centre: No Faculty
Faculty/School / Institute/Centre: No Faculty
Date Deposited: 05 Nov 2014 06:54
Last Modified: 06 Mar 2015 01:13
Uncontrolled Keywords: adult stem cells; cell transplantation; cellular therapy; embryonic stem cells; mesenchymal stem cells; stem cell transplantation
Fields of Research (2008): 09 Engineering > 0910 Manufacturing Engineering > 091008 Manufacturing Safety and Quality
10 Technology > 1004 Medical Biotechnology > 100404 Regenerative Medicine (incl. Stem Cells and Tissue Engineering)
10 Technology > 1003 Industrial Biotechnology > 100302 Bioprocessing, Bioproduction and Bioproducts
Fields of Research (2020): 40 ENGINEERING > 4014 Manufacturing engineering > 401409 Manufacturing safety and quality
32 BIOMEDICAL AND CLINICAL SCIENCES > 3206 Medical biotechnology > 320606 Regenerative medicine (incl. stem cells)
31 BIOLOGICAL SCIENCES > 3106 Industrial biotechnology > 310602 Bioprocessing, bioproduction and bioproducts
Socio-Economic Objectives (2008): E Expanding Knowledge > 97 Expanding Knowledge > 970110 Expanding Knowledge in Technology
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