Regulatory considerations for the development of autologous induced pluripotent stem cell therapies

Regenerative Medicine
Melissa K Carpenter, Larry A Couture

Abstract

Induced pluripotent stem (iPS) cells offer tremendous opportunity for the creation of autologous cellular therapies, in which gene correction or the avoidance of immune response issues are desirable. In addition, iPS cells avoid the ethical concerns raised by the sourcing of human embryonic stem cells (hESCs) from embryos. iPS cells share many characteristics with hESCs and it is anticipated that existing experience with hESCs will translate to rapid progress in moving iPS cell-derived products toward clinical trials. While the potential clinical value for these products is considerable, the nature of current manufacturing paradigms for autologous iPS cell products raises considerable regulatory concerns. Here, the regulatory challenges posed by autologous iPS cell-derived products are examined. We conclude that there will be considerable regulatory concerns primarily relating to reproducibility of the manufacturing process and safety testing within clinically limited time constraints. Demonstrating safety of the final cell product in an autologous setting will be the single greatest obstacle to progressing autologous iPS cell-based therapies into the clinic.

References

Nov 6, 1998·Science·J A ThomsonJ M Jones
Dec 9, 2003·Nature Biotechnology·Jonathan S DraperPeter W Andrews
Jan 28, 2004·Developmental Dynamics : an Official Publication of the American Association of Anatomists·Elen S RoslerMelissa K Carpenter
Jul 9, 2005·Nature Biotechnology·Robert A Preti
May 2, 2006·Stem Cells and Development·M J CookeS A Przyborski
Nov 7, 2006·Trends in Biotechnology·Hannes HentzeAlan Colman
Jun 8, 2007·Nature·Keisuke OkitaShinya Yamanaka
Nov 22, 2007·Science·Junying YuJames A Thomson
Dec 27, 2007·Stem Cells and Development·Mahendra Rao
Feb 15, 2008·Stem Cells and Development·Mahendra Rao, Maureen L Condic
Feb 19, 2008·Nature Biotechnology·Kenji OsafuneDouglas A Melton
Aug 2, 2008·Stem Cells·Maureen L Condic, Mahendra Rao
Aug 5, 2008·Genes & Development·Junying Yu, James A Thomson
Aug 9, 2008·The Journal of Clinical Investigation·Salima Hacein-Bey-AbinaMarina Cavazzana-Calvo
Aug 30, 2008·Nature·Franz-Josef MüllerJeanne F Loring
Oct 11, 2008·Science·Keisuke OkitaShinya Yamanaka
Oct 22, 2008·Nature Biotechnology·Trond AasenJuan Carlos Izpisúa Belmonte
Oct 22, 2008·Cell Stem Cell·Jeremy Micah CrookAlan Colman
Dec 2, 2008·Cell Stem Cell·Nimet Maherali, Konrad Hochedlinger
Mar 21, 2009·Blood·Yuin-Han LohGeorge Q Daley
Apr 29, 2009·Cell Stem Cell·Hongyan ZhouSheng Ding
Jul 10, 2009·Nature Biotechnology·Melissa K CarpenterMahendra S Rao
Jul 11, 2009·Nature Biotechnology·Kyoko MiuraShinya Yamanaka
Oct 3, 2009·Cell Stem Cell·Alessandra GiorgettiJuan Carlos Izpisua Belmonte
Oct 7, 2009·Proceedings of the National Academy of Sciences of the United States of America·Ning SunJoseph C Wu
Oct 20, 2009·Nature Methods·Tongxiang LinSheng Ding
Nov 6, 2009·Cell Cycle·Barak Blum, Nissim Benvenisty

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Citations

Nov 10, 2011·Stem Cells and Development·Alexandre Teixeira VessoniOswaldo Keith Okamoto
Aug 6, 2014·Nature Biotechnology·Peter W AndrewsShinya Yamanaka
Mar 14, 2012·Stem Cell Reviews and Reports·Barbara von TigerstromBartha Maria Knoppers
Dec 26, 2015·Lancet Neurology·Joshua G HunsbergerP Murali Doraiswamy
Apr 4, 2015·Regenerative Medicine·Mahendra RaoSusan Solomon
Jun 28, 2016·Regenerative Medicine·Nicholas F Blair, Roger A Barker
Dec 6, 2014·Toxicologic Pathology·Joydeep BasuMahendra Rao
Jul 17, 2010·Regenerative Medicine·M William Lensch, Mahendra Rao

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Methods Mentioned

BETA
biopsies
xenograft

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