Assessing the safety of stem cell therapeutics

Chris E.P. Goldring; Paul A. Duffy; Nissim Benvenisty; Peter W. Andrews; Uri Ben-David; Rowena Eakins; Neil French; Neil A. Hanley; Lorna Kelly; Neil R. Kitteringham; Jens Kurth; Deborah Ladenheim; Hugh Laverty; James McBlane; Gopalan Narayanan; Sara Patel; Jens Reinhardt; Annamaria Rossi; Michaela Sharpe; B. Kevin Park

doi:10.1016/j.stem.2011.05.012

Assessing the safety of stem cell therapeutics

Chris E.P. Goldring, Paul A. Duffy, Nissim Benvenisty, Peter W. Andrews, Uri Ben-David, Rowena Eakins, Neil French, Neil A. Hanley, Lorna Kelly, Neil R. Kitteringham, Jens Kurth, Deborah Ladenheim, Hugh Laverty, James McBlane, Gopalan Narayanan, Sara Patel, Jens Reinhardt, Annamaria Rossi, Michaela Sharpe, B. Kevin Park

Medical and Dental Sciences

Research output: Contribution to journal › Review article › peer-review

182 Citations (Scopus)

Abstract

Unprecedented developments in stem cell research herald a new era of hope and expectation for novel therapies. However, they also present a major challenge for regulators since safety assessment criteria, designed for conventional agents, are largely inappropriate for cell-based therapies. This article aims to set out the safety issues pertaining to novel stem cell-derived treatments, to identify knowledge gaps that require further research, and to suggest a roadmap for developing safety assessment criteria. It is essential that regulators, pharmaceutical providers, and safety scientists work together to frame new safety guidelines, based on "acceptable risk," so that patients are adequately protected but the safety "bar" is not set so high that exciting new treatments are lost.

Original language	English
Pages (from-to)	618-628
Number of pages	11
Journal	Cell stem cell
Volume	8
Issue number	6
DOIs	https://doi.org/10.1016/j.stem.2011.05.012
Publication status	Published - 3 Jun 2011

Bibliographical note

Funding Information:
C.E.P.G., R.E., N.F., N.A.H., L.K., N.R.K., H.L., and B.K.P. would like to thank the Medical Research Council (MRC); Frank Bonner, Ernie Harpur, and Rebecca Lumsden at Stem Cells for Safer Medicines; and The Association of British Pharmaceutical Companies (ABPI). C.E.P.G and R.E. thank Gillian Wallace and acknowledge the support of Life Technologies. N.H. acknowledges the support of The Wellcome Trust and The National Institute for Health Research. Peter Andrews wishes to thank the MRC and Yorkshire Cancer Research (YCR). An Industry-Academic-Regulator workshop that was held in Liverpool, UK, on Nov. 24, 2010, formed the initiative for the development of this manuscript.

ASJC Scopus subject areas

Molecular Medicine
Genetics
Cell Biology

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10.1016/j.stem.2011.05.012

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Goldring, C. E. P., Duffy, P. A., Benvenisty, N., Andrews, P. W., Ben-David, U., Eakins, R., French, N., Hanley, N. A., Kelly, L., Kitteringham, N. R., Kurth, J., Ladenheim, D., Laverty, H., McBlane, J., Narayanan, G., Patel, S., Reinhardt, J., Rossi, A., Sharpe, M., & Park, B. K. (2011). Assessing the safety of stem cell therapeutics. Cell stem cell, 8(6), 618-628. https://doi.org/10.1016/j.stem.2011.05.012

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abstract = "Unprecedented developments in stem cell research herald a new era of hope and expectation for novel therapies. However, they also present a major challenge for regulators since safety assessment criteria, designed for conventional agents, are largely inappropriate for cell-based therapies. This article aims to set out the safety issues pertaining to novel stem cell-derived treatments, to identify knowledge gaps that require further research, and to suggest a roadmap for developing safety assessment criteria. It is essential that regulators, pharmaceutical providers, and safety scientists work together to frame new safety guidelines, based on {"}acceptable risk,{"} so that patients are adequately protected but the safety {"}bar{"} is not set so high that exciting new treatments are lost.",

author = "Goldring, {Chris E.P.} and Duffy, {Paul A.} and Nissim Benvenisty and Andrews, {Peter W.} and Uri Ben-David and Rowena Eakins and Neil French and Hanley, {Neil A.} and Lorna Kelly and Kitteringham, {Neil R.} and Jens Kurth and Deborah Ladenheim and Hugh Laverty and James McBlane and Gopalan Narayanan and Sara Patel and Jens Reinhardt and Annamaria Rossi and Michaela Sharpe and Park, {B. Kevin}",

note = "Funding Information: C.E.P.G., R.E., N.F., N.A.H., L.K., N.R.K., H.L., and B.K.P. would like to thank the Medical Research Council (MRC); Frank Bonner, Ernie Harpur, and Rebecca Lumsden at Stem Cells for Safer Medicines; and The Association of British Pharmaceutical Companies (ABPI). C.E.P.G and R.E. thank Gillian Wallace and acknowledge the support of Life Technologies. N.H. acknowledges the support of The Wellcome Trust and The National Institute for Health Research. Peter Andrews wishes to thank the MRC and Yorkshire Cancer Research (YCR). An Industry-Academic-Regulator workshop that was held in Liverpool, UK, on Nov. 24, 2010, formed the initiative for the development of this manuscript.",

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Goldring, CEP, Duffy, PA, Benvenisty, N, Andrews, PW, Ben-David, U, Eakins, R, French, N, Hanley, NA, Kelly, L, Kitteringham, NR, Kurth, J, Ladenheim, D, Laverty, H, McBlane, J, Narayanan, G, Patel, S, Reinhardt, J, Rossi, A, Sharpe, M & Park, BK 2011, 'Assessing the safety of stem cell therapeutics', Cell stem cell, vol. 8, no. 6, pp. 618-628. https://doi.org/10.1016/j.stem.2011.05.012

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AU - Goldring, Chris E.P.

AU - Duffy, Paul A.

AU - Benvenisty, Nissim

AU - Andrews, Peter W.

AU - Ben-David, Uri

AU - Eakins, Rowena

AU - French, Neil

AU - Hanley, Neil A.

AU - Kelly, Lorna

AU - Kitteringham, Neil R.

AU - Kurth, Jens

AU - Ladenheim, Deborah

AU - Laverty, Hugh

AU - McBlane, James

AU - Narayanan, Gopalan

AU - Patel, Sara

AU - Reinhardt, Jens

AU - Rossi, Annamaria

AU - Sharpe, Michaela

AU - Park, B. Kevin

N1 - Funding Information: C.E.P.G., R.E., N.F., N.A.H., L.K., N.R.K., H.L., and B.K.P. would like to thank the Medical Research Council (MRC); Frank Bonner, Ernie Harpur, and Rebecca Lumsden at Stem Cells for Safer Medicines; and The Association of British Pharmaceutical Companies (ABPI). C.E.P.G and R.E. thank Gillian Wallace and acknowledge the support of Life Technologies. N.H. acknowledges the support of The Wellcome Trust and The National Institute for Health Research. Peter Andrews wishes to thank the MRC and Yorkshire Cancer Research (YCR). An Industry-Academic-Regulator workshop that was held in Liverpool, UK, on Nov. 24, 2010, formed the initiative for the development of this manuscript.

PY - 2011/6/3

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N2 - Unprecedented developments in stem cell research herald a new era of hope and expectation for novel therapies. However, they also present a major challenge for regulators since safety assessment criteria, designed for conventional agents, are largely inappropriate for cell-based therapies. This article aims to set out the safety issues pertaining to novel stem cell-derived treatments, to identify knowledge gaps that require further research, and to suggest a roadmap for developing safety assessment criteria. It is essential that regulators, pharmaceutical providers, and safety scientists work together to frame new safety guidelines, based on "acceptable risk," so that patients are adequately protected but the safety "bar" is not set so high that exciting new treatments are lost.

AB - Unprecedented developments in stem cell research herald a new era of hope and expectation for novel therapies. However, they also present a major challenge for regulators since safety assessment criteria, designed for conventional agents, are largely inappropriate for cell-based therapies. This article aims to set out the safety issues pertaining to novel stem cell-derived treatments, to identify knowledge gaps that require further research, and to suggest a roadmap for developing safety assessment criteria. It is essential that regulators, pharmaceutical providers, and safety scientists work together to frame new safety guidelines, based on "acceptable risk," so that patients are adequately protected but the safety "bar" is not set so high that exciting new treatments are lost.

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SN - 1934-5909

VL - 8

SP - 618

EP - 628

JO - Cell stem cell

JF - Cell stem cell

IS - 6

ER -

Assessing the safety of stem cell therapeutics

Abstract

Bibliographical note

ASJC Scopus subject areas

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