Abstract
Developing clinical grade PSC-based cells for use in patients requires not only careful attention to cGMPs (current good manufacturing practices) but also careful consideration of a variety of issues that include accessing tissue in an ethically appropriate fashion and adhering to the current rules and regulations for specific local, national, and international jurisdictions, which, if not harmonized, will likely hinder progress. Countries have developed different ways to accelerate translation, and in this article, we discuss the issues specific to iPSC-based manufacture and how these issues are being resolved.
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Abbreviations
- BLA:
-
Biologics Licensing Application
- CAT:
-
Committee for Advanced Therapeutics
- CBER:
-
Center for Biologics evaluation and Research
- CHMP:
-
Committee for Medicinal Products for Human Use
- FDA:
-
Food and Drug Administration
- GLP:
-
Good Laboratory Practice
- IND:
-
Initial New Drug Application
- iPSC:
-
Induced Pluripotent stem cells
- NACs:
-
National APex
- PSC:
-
Pluripotent stem cells
- PMDA:
-
Pharmceuticals and Medical Devices Agency
- SME:
-
Small Medical entities
- HE-:
-
Hospital exemption
References
Ancans J. Cell therapy medicinal product regulatory framework in Europe and its application for MSC-based therapy development. Front Immunol. 2012;3:253.
Andrews PW, Cavanagro J, Deans R, Feigel E, Horowitz E, Keating A, Rao M, Turner M, Wilmut I, Yamanaka S. Harmonizing standards for producing clinical-grade therapies from pluripotent stem cells. Nat Biotechnol. 2014;32:724–6.
Arcidiacono JA, Blair JW, Benton KA. US Food and Drug Administration international collaborations for cellular therapy product regulation. Stem Cell Res Ther. 2012;3:38.
Byrne JA, Pedersen DA, Clepper LL, Nelson M, Sanger WG, Gokhale S, Wolf DP, Mitalipov SM. Producing primate embryonic stem cells by somatic cell nuclear transfer. Nature. 2007;450:497–502.
Caplan AI, West MD. Progressive approval: a proposal for a new regulatory pathway for regenerative medicine. Stem Cells Transl Med. 2014;3:560–3.
Cyranoski D. Stem cells cruise to clinic. Nature. 2013;494:413.
Eichler HG, Oye K, Baird LG, Abadie E, Brown J, Drum CL, Ferguson J, Garner S, Honig P, Hukkelhoven M, et al. Adaptive licensing: taking the next step in the evolution of drug approval. Clin Pharmacol Ther. 2012;91:426–37.
Hara A, Sato D, Sahara Y. New Governmental Regulatory System for Stem Cell–Based Therapies in Japan. Ther Innov Regul Sci. 2014;4:2:100137:1–3.
Lanzoni G, Oikawa T, Wang Y, Cui CB, Carpino G, Cardinale V, Gerber D, Gabriel M, Dominguez-Bendala J, Furth ME, et al. Concise review: clinical programs of stem cell therapies for liver and pancreas. Stem Cells. 2013;31:2047–60.
Martell K, Trounson A, Baum E. Stem cell therapies in clinical trials: workshop on best practices and the need for harmonization. Cell Stem Cell. 2010;7:451–4.
Morizane A, Doi D, Kikuchi T, Okita K, Hotta A, Kawasaki T, Hayashi T, Onoe H, Shiina T, Yamanaka S, et al. Direct comparison of autologous and allogeneic transplantation of iPSC-derived neural cells in the brain of a nonhuman primate. Stem Cell Rep. 2013;1:283–92.
Yamada M, Johannesson B, Sagi I, Burnett LC, Kort DH, Prosser RW, Paull D, Nestor MW, Freeby M, Greenberg E, Goland RS, Leibel RL, Solomon SL, Benvenisty N, Sauer MV, Egli D. Human oocytes reprogram adult somatic nuclei of a type 1 diabetic to diploid pluripotent stem cells. Nature. 2014;510:533–6.
Rao M. iPSC crowdsourcing: a model for obtaining large panels of stem cell lines for screening. Cell Stem Cell. 2013;13:389–91.
Rao M, Condic ML. Alternative sources of pluripotent stem cells: scientific solutions to an ethical dilemma. Stem Cells Dev. 2008;17:1–10.
Sipp D, Turner L. Stem cells. U.S. regulation of stem cells as medical products. Science. 2012;338:1296–7.
Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell. 2006;126:663–76.
Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K, Yamanaka S. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell. 2007;131:861–72.
Thomson JA, Itskovitz-Eldor J, Shapiro SS, Waknitz MA, Swiergiel JJ, Marshall VS, Jones JM. Embryonic stem cell lines derived from human blastocysts. Science. 1998;282:1145–7.
Turner M, Leslie S, Martin NG, Peschanski M, Rao M, Taylor CJ, Trounson A, Turner D, Yamanaka S, Wilmut I. Toward the development of a global induced pluripotent stem cell library. Cell Stem Cell. 2013;13:382–4.
Wilmut I, Beaujean N, de Sousa PA, Dinnyes A, King TJ, Paterson LA, Wells DN, Young LE. Somatic cell nuclear transfer. Nature. 2002;419:583–6.
Yi F, Liu GH, Izpisua Belmonte JC. Human induced pluripotent stem cells derived hepatocytes: rising promise for disease modeling, drug development and cell therapy. Protein Cell. 2012;3:246–50.
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Solomon, S., Rao, M.S. (2016). iPSC-Derived Products: Current Regulations. In: Terai, S., Suda, T. (eds) Gene Therapy and Cell Therapy Through the Liver. Springer, Tokyo. https://doi.org/10.1007/978-4-431-55666-4_11
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DOI: https://doi.org/10.1007/978-4-431-55666-4_11
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