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 Mathews, David M. Blass, Hilary Bok, Joseph T. Coyle, Patrick Duggan, Ruth R Faden, Julia Finkel, John D. Gearhart, Argye Hillis-Trupe, Ahmet Hoke, Richard Johnson, Michael V 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 D. Smith, Davor Solter, Hongjun Song, Jeremy Sugarman, Angelo Vescovi, Wise Young, Henry T. Greely, Richard J. Traystman

Research output: Contribution to journalArticle

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 languageEnglish (US)
Pages (from-to)1-9
Number of pages9
JournalJournal of Cerebral Blood Flow and Metabolism
Volume29
Issue number1
DOIs
StatePublished - Jan 2009

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Nervous System
Animal Models
Safety
Regenerative Medicine
Lenses
Clinical Trials
Therapeutics

Keywords

  • Clinical trials
  • Modeling
  • Neurologic conditions
  • Stem and progenitor cells

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine
  • Clinical Neurology
  • Neurology

Cite this

The role of animal models in evaluating reasonable safety and efficacy for human trials of cell-based interventions for neurologic conditions. / Regenberg, Alan; Mathews, Debra J; Blass, David M.; Bok, Hilary; Coyle, Joseph T.; Duggan, Patrick; Faden, Ruth R; Finkel, Julia; Gearhart, John D.; Hillis-Trupe, Argye; Hoke, Ahmet; Johnson, Richard; Johnston, Michael V; Kahn, Jeffrey; Kerr, Douglas; King, Patricia; Kurtzberg, Joanne; Liao, S. Matthew; McDonald, John W.; McKhann, Guy; Nelson, Karin B.; Rao, Mahendra; Siegel, Andrew W.; Smith, Kirby D.; Solter, Davor; Song, Hongjun; Sugarman, Jeremy; Vescovi, Angelo; Young, Wise; Greely, Henry T.; Traystman, Richard J.

In: Journal of Cerebral Blood Flow and Metabolism, Vol. 29, No. 1, 01.2009, p. 1-9.

Research output: Contribution to journalArticle

Regenberg, A, Mathews, DJ, Blass, DM, Bok, H, Coyle, JT, Duggan, P, Faden, RR, Finkel, J, Gearhart, JD, Hillis-Trupe, A, Hoke, A, Johnson, R, Johnston, MV, Kahn, J, Kerr, D, King, P, Kurtzberg, J, Liao, SM, McDonald, JW, McKhann, G, Nelson, KB, Rao, M, Siegel, AW, Smith, KD, 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
Regenberg, Alan ; Mathews, Debra J ; Blass, David M. ; Bok, Hilary ; Coyle, Joseph T. ; Duggan, Patrick ; Faden, Ruth R ; Finkel, Julia ; Gearhart, John D. ; Hillis-Trupe, Argye ; Hoke, Ahmet ; Johnson, Richard ; Johnston, Michael V ; Kahn, Jeffrey ; Kerr, Douglas ; King, Patricia ; Kurtzberg, Joanne ; Liao, S. Matthew ; McDonald, John W. ; McKhann, Guy ; Nelson, Karin B. ; Rao, Mahendra ; Siegel, Andrew W. ; Smith, Kirby D. ; Solter, Davor ; Song, Hongjun ; Sugarman, Jeremy ; Vescovi, Angelo ; Young, Wise ; Greely, Henry T. ; Traystman, Richard J. / The role of animal models in evaluating reasonable safety and efficacy for human trials of cell-based interventions for neurologic conditions. In: Journal of Cerebral Blood Flow and Metabolism. 2009 ; Vol. 29, No. 1. pp. 1-9.
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