The role of animal models in evaluating reasonable safety and efficacy for human trials of cell-based interventions for neurologic conditions.
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.
Regenberg
Alan
A
Johns Hopkins Berman Institute of Bioethics, Johns Hopkins University, Baltimore, Maryland 21201, USA. aregenbe@jhsph.edu
Mathews
Debra J H
DJ
Blass
David M
DM
Bok
Hilary
H
Coyle
Joseph T
JT
Duggan
Patrick
P
Faden
Ruth
R
Finkel
Julia
J
Gearhart
John D
JD
Hillis
Argye
A
Hoke
Ahmet
A
Johnson
Richard
R
Johnston
Michael
M
Kahn
Jeffrey
J
Kerr
Douglas
D
King
Patricia
P
Kurtzberg
Joanne
J
Liao
S Matthew
SM
McDonald
John W
JW
McKhann
Guy
G
Nelson
Karin B
KB
Rao
Mahendra
M
Siegel
Andrew W
AW
Smith
Kirby
K
Solter
Davor
D
Song
Hongjun
H
Sugarman
Jeremy
J
Vescovi
Angelo
A
Young
Wise
W
Greely
Henry T
HT
Traystman
Richard J
RJ
D000818:Animals; D002986:Clinical Trials as Topic; D006801:Humans; D023421:Models, Animal; D008959:Models, Neurological; D012307:Risk Factors; D017751:Safety Management