The Role of Animal Models in Evaluating Reasonable Safety and Efficacy for Human Trials of Cell-based Interventions for Neurologic Conditions

Overview

Journal J Cereb Blood Flow Metab

Publisher Sage Publications

Specialties Cardiology & Vascular Diseases
Endocrinology
Neurology

Date 2008 Aug 30

PMID 18728679

Citations 14

Authors

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

Affiliations

Soon will be listed here.

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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