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

TitleThe role of animal models in evaluating reasonable safety and efficacy for human trials of cell-based interventions for neurologic conditions.
Publication TypeJournal Article
Year of Publication2009
AuthorsRegenberg 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 MS, 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
JournalJournal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism
Volume29
Issue1
Pagination1-9
Date Published2009 Jan
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.

DOI10.1002/mrm.21712
Alternate JournalJ. Cereb. Blood Flow Metab.