The role of animal models in evaluating reasonable safety and efficacy for human trials of cell-based interventions for neurologic conditions

Alan Regenberg; Debra J.H. Mathews; David M. Blass; Hilary Bok; Joseph T. Coyle; Patrick Duggan; Ruth Faden; Julia Finkel; John D. Gearhart; Argye Hillis; Ahmet Hoke; Richard Johnson; Michael Johnston; Jeffrey Kahn; Douglas Kerr; Patricia King; Joanne Kurtzberg; S. Matthew Liao; John W. McDonald; Guy McKhann; Karin B. Nelson; Mahendra Rao; Andrew W. Siegel; Kirby Smith; Davor Solter; Hongjun Song; Jeremy Sugarman; Angelo Vescovi; Wise Young; Henry T. Greely; Richard J. Traystman

doi:10.1038/jcbfm.2008.98

The role of animal models in evaluating reasonable safety and efficacy for human trials of cell-based interventions for neurologic conditions

Alan Regenberg, Debra J.H. Mathews, David M. Blass, Hilary Bok, Joseph T. Coyle, Patrick Duggan, Ruth Faden, Julia Finkel, John D. Gearhart, Argye Hillis, Ahmet Hoke, Richard Johnson, Michael Johnston, Jeffrey Kahn, Douglas Kerr, Patricia King, Joanne Kurtzberg, S. Matthew Liao, John W. McDonald, Guy McKhannKarin B. Nelson, Mahendra Rao, Andrew W. Siegel, Kirby Smith, Davor Solter, Hongjun Song, Jeremy Sugarman, Angelo Vescovi, Wise Young, Henry T. Greely, Richard J. Traystman

Global Public Health

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

Abstract

Progress in regenerative medicine seems likely to produce new treatments for neurologic conditions that use human cells as therapeutic agents; at least one trial for such an intervention is already under way. The development of cell-based interventions for neurologic conditions (CBI-NCs) will likely include preclinical studies using animals as models for humans with conditions of interest. This paper explores predictive validity challenges and the proper role for animal models in developing CBI-NCs. In spite of limitations, animal models are and will remain an essential tool for gathering data in advance of first-in-human clinical trials. The goal of this paper is to provide a realistic lens for viewing the role of animal models in the context of CBI-NCs and to provide recommendations for moving forward through this challenging terrain.

Original language	English (US)
Pages (from-to)	1-9
Number of pages	9
Journal	Journal of Cerebral Blood Flow and Metabolism
Volume	29
Issue number	1
DOIs	https://doi.org/10.1038/jcbfm.2008.98
State	Published - Jan 2009

Keywords

Clinical trials
Modeling
Neurologic conditions
Stem and progenitor cells

ASJC Scopus subject areas

Neurology
Clinical Neurology
Cardiology and Cardiovascular Medicine

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10.1038/jcbfm.2008.98

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Regenberg, A., Mathews, D. J. H., Blass, D. M., Bok, H., Coyle, J. T., Duggan, P., Faden, R., Finkel, J., Gearhart, J. D., Hillis, A., Hoke, A., Johnson, R., Johnston, M., Kahn, J., Kerr, D., King, P., Kurtzberg, J., Liao, S. M., McDonald, J. W., ... Traystman, R. J. (2009). The role of animal models in evaluating reasonable safety and efficacy for human trials of cell-based interventions for neurologic conditions. Journal of Cerebral Blood Flow and Metabolism, 29(1), 1-9. https://doi.org/10.1038/jcbfm.2008.98

Regenberg, A, Mathews, DJH, Blass, DM, Bok, H, Coyle, JT, Duggan, P, Faden, R, Finkel, J, Gearhart, JD, Hillis, A, Hoke, A, Johnson, R, Johnston, M, Kahn, J, Kerr, D, King, P, Kurtzberg, J, Liao, SM, McDonald, JW, McKhann, G, Nelson, KB, Rao, M, Siegel, AW, Smith, K, Solter, D, Song, H, Sugarman, J, Vescovi, A, Young, W, Greely, HT & Traystman, RJ 2009, 'The role of animal models in evaluating reasonable safety and efficacy for human trials of cell-based interventions for neurologic conditions', Journal of Cerebral Blood Flow and Metabolism, vol. 29, no. 1, pp. 1-9. https://doi.org/10.1038/jcbfm.2008.98

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abstract = "Progress in regenerative medicine seems likely to produce new treatments for neurologic conditions that use human cells as therapeutic agents; at least one trial for such an intervention is already under way. The development of cell-based interventions for neurologic conditions (CBI-NCs) will likely include preclinical studies using animals as models for humans with conditions of interest. This paper explores predictive validity challenges and the proper role for animal models in developing CBI-NCs. In spite of limitations, animal models are and will remain an essential tool for gathering data in advance of first-in-human clinical trials. The goal of this paper is to provide a realistic lens for viewing the role of animal models in the context of CBI-NCs and to provide recommendations for moving forward through this challenging terrain.",

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author = "Alan Regenberg and Mathews, {Debra J.H.} and Blass, {David M.} and Hilary Bok and Coyle, {Joseph T.} and Patrick Duggan and Ruth Faden and Julia Finkel and Gearhart, {John D.} and Argye Hillis and Ahmet Hoke and Richard Johnson and Michael Johnston and Jeffrey Kahn and Douglas Kerr and Patricia King and Joanne Kurtzberg and Liao, {S. Matthew} and McDonald, {John W.} and Guy McKhann and Nelson, {Karin B.} and Mahendra Rao and Siegel, {Andrew W.} and Kirby Smith and Davor Solter and Hongjun Song and Jeremy Sugarman and Angelo Vescovi and Wise Young and Greely, {Henry T.} and Traystman, {Richard J.}",

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AU - Coyle, Joseph T.

AU - Duggan, Patrick

AU - Faden, Ruth

AU - Finkel, Julia

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AU - Johnson, Richard

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AU - Kerr, Douglas

AU - King, Patricia

AU - Kurtzberg, Joanne

AU - Liao, S. Matthew

AU - McDonald, John W.

AU - McKhann, Guy

AU - Nelson, Karin B.

AU - Rao, Mahendra

AU - Siegel, Andrew W.

AU - Smith, Kirby

AU - Solter, Davor

AU - Song, Hongjun

AU - Sugarman, Jeremy

AU - Vescovi, Angelo

AU - Young, Wise

AU - Greely, Henry T.

AU - Traystman, Richard J.

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AB - Progress in regenerative medicine seems likely to produce new treatments for neurologic conditions that use human cells as therapeutic agents; at least one trial for such an intervention is already under way. The development of cell-based interventions for neurologic conditions (CBI-NCs) will likely include preclinical studies using animals as models for humans with conditions of interest. This paper explores predictive validity challenges and the proper role for animal models in developing CBI-NCs. In spite of limitations, animal models are and will remain an essential tool for gathering data in advance of first-in-human clinical trials. The goal of this paper is to provide a realistic lens for viewing the role of animal models in the context of CBI-NCs and to provide recommendations for moving forward through this challenging terrain.

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The role of animal models in evaluating reasonable safety and efficacy for human trials of cell-based interventions for neurologic conditions

Abstract

Keywords

ASJC Scopus subject areas

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