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Diana E. Pankevich, Theresa M. Wizemann,
and Bruce M. Altevogt, Rapporteurs
Forum on Neuroscience and
Nervous System Disorders
Board on Health Sciences Policy
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THE NATIONAL ACADEMIES PRESS • 500 Fifth Street, NW • Washington, DC 20001
NOTICE: The workshop that is the subject of this summary was approved by the
Governing Board of the National Research Council, whose members are drawn from the
councils of the National Academy of Sciences, the National Academy of Engineering,
and the Institute of Medicine.
This project was supported by contracts between the National Academy of Sciences and
the Alzheimer’s Association; CeNeRx Biopharma; the Department of Health and Human
Services’ National Institutes of Health (NIH, Contract No. N01-OD-4-2139) through the
National Eye Institute, National Institute of Mental Health, National Institute of
Neurological Disorders and Stroke, National Institute on Aging, National Institute on
Alcohol Abuse and Alcoholism, National Institute on Drug Abuse, and NIH Blueprint for
Neuroscience Research; Department of Veterans Affairs; Eli Lilly and Company; Fast
Forward, LLC; Foundation for the National Institutes of Health; GE Healthcare, Inc.;
GlaxoSmithKline, Inc.; Johnson & Johnson Pharmaceutical Research and Development,
LLC; Lundbeck Research USA; Merck Research Laboratories; The Michael J. Fox
Foundation for Parkinson’s Research; the National Science Foundation (Contract No.
OIA-0753701); One Mind for Research; Pfizer Inc.; the Society for Neuroscience; and
Wellcome Trust. The views presented in this publication are those of the editors and
attributing authors and do not necessarily reflect the view of the organizations or agencies
that provided support for this project.
International Standard Book Number-13: 978-0-309-26633-8
International Standard Book Number-10: 0-309-26633-5
Additional copies of this report are available from the National Academies Press, 500
Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624-6242 or (202) 334-3313;
http://www.nap.edu.
For more information about the Institute of Medicine, visit the IOM home page at:
www.iom.edu.
Copyright 2013 by the National Academy of Sciences. All rights reserved.
Printed in the United States of America
The serpent has been a symbol of long life, healing, and knowledge among almost all
cultures and religions since the beginning of recorded history. The serpent adopted as a
logotype by the Institute of Medicine is a relief carving from ancient Greece, now held by
the Staatliche Museen in Berlin.
Suggested citation: IOM (Institute of Medicine). 2013. Improving the utility and
translation of animal models for nervous system disorders: Workshop summary.
Washington, DC: The National Academies Press.
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The National Academy of Sciences is a private, nonprofit, self-perpetuating
society of distinguished scholars engaged in scientific and engineering research,
dedicated to the furtherance of science and technology and to their use for the
general welfare. Upon the authority of the charter granted to it by the Congress
in 1863, the Academy has a mandate that requires it to advise the federal
government on scientific and technical matters. Dr. Ralph J. Cicerone is
president of the National Academy of Sciences.
The National Academy of Engineering was established in 1964, under the
charter of the National Academy of Sciences, as a parallel organization of
outstanding engineers. It is autonomous in its administration and in the selection
of its members, sharing with the National Academy of Sciences the
responsibility for advising the federal government. The National Academy of
Engineering also sponsors engineering programs aimed at meeting national
needs, encourages education and research, and recognizes the superior
achievements of engineers. Dr. Charles M. Vest is president of the National
Academy of Engineering.
The Institute of Medicine was established in 1970 by the National Academy of
Sciences to secure the services of eminent members of appropriate professions
in the examination of policy matters pertaining to the health of the public. The
Institute acts under the responsibility given to the National Academy of Sciences
by its congressional charter to be an adviser to the federal government and, upon
its own initiative, to identify issues of medical care, research, and education.
Dr. Harvey V. Fineberg is president of the Institute of Medicine.
The National Research Council was organized by the National Academy of
Sciences in 1916 to associate the broad community of science and technology
with the Academy’s purposes of furthering knowledge and advising the federal
government. Functioning in accordance with general policies determined
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of both the National Academy of Sciences and the National Academy
of Engineering in providing services to the government, the public, and the
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by both Academies and the Institute of Medicine. Dr. Ralph J. Cicerone and
Dr. Charles M. Vest are chair and vice chair, respectively, of the National
Research Council.
www.national-academies.org
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PLANNING COMMITTEE ON IMPROVING
TRANSLATION OF ANIMAL MODELS FOR NERVOUS
SYSTEM DISRODERS1
RICHARD J. HODES (Co-Chair), National Institute on Aging
STEVEN M. PAUL (Co-Chair), Weill Cornell Medical College
TIMOTHY COETZEE, National Multiple Sclerosis Society
MARK A. GEYER, University of California, San Diego
WALTER J. KOROSHETZ, National Institute for Neurological
Disorders and Stroke
ALAN I. LESHNER, American Association for the Advancement of
Science
GERARD J. MAREK, Abbott Laboratories
WILLIAM Z. POTTER, Foundation for the National Institutes of
Health
SHARON ROSENZWEIG-LIPSON, IVS Pharma Consulting
TODD SHERER, The Michael J. Fox Foundation for Parkinson’s
Research
DAVID SHURTLEFF, National Institute of Drug Abuse
JUDY SIUCIAK, Foundation for the National Institutes of Health
STEVIN ZORN, Lundbeck USA
IOM Staff
BRUCE M. ALTEVOGT, Project Director
DIANA E. PANKEVICH, Program Officer
ELIZABETH K. THOMAS, Senior Program Assistant (until November
2012)
___________________________
1
Institute of Medicine planning committees are solely responsible for organizing the
workshop, identifying topics, and choosing speakers. The responsibility for the published
workshop summary rests with the workshop rapporteurs and the institution.
v
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FORUM ON NEUROSCIENCE AND NERVOUS SYSTEM
DISORDERS1
STEVEN HYMAN (Chair), The Broad Institute
SUSAN AMARA, Society for Neuroscience
MARC BARLOW, GE Healthcare, Inc.
MARK BEAR, Massachusetts Institute of Technology
KATJA BROSE, Neuron
DANIEL BURCH, PPDi
C. THOMAS CASKEY, Baylor College of Medicine
TIMOTHY COETZEE, Fast Forward, LLC
EMMELINE EDWARDS, NIH Neuroscience Blueprint
MARTHA FARAH, University of Pennsylvania
RICHARD FRANK, GE Healthcare, Inc.
DANIEL GESCHWIND, University of California, Los Angeles
HANK GREELY, Stanford University
MYRON GUTMANN, National Science Foundation
RICHARD HODES, National Institute on Aging
STUART HOFFMAN, U.S. Department of Veterans Affairs
THOMAS INSEL, National Institute of Mental Health
PHILLIP IREDALE, Pfizer Global Research and Development
DANIEL JAVITT, Nathan S. Kline Institute for Psychiatric Research
FRANCES JENSEN, University of Pennsylvania School of Medicine
STORY LANDIS, National Institute of Neurological Disorders and Stroke
ALAN LESHNER, American Association for the Advancement of
Science
HUSSEINI MANJI, Johnson & Johnson Pharmaceutical Research and
Development, Inc.
DAVID MICHELSON, Merck Research Laboratories
RICHARD MOHS, Lilly Research Laboratories
JONATHAN MORENO, University of Pennsylvania
ATUL PANDE, GlaxoSmithKline, Inc.
STEVEN PAUL, Weill Cornell Medical College
TODD SHERER, The Michael J. Fox Foundation for Parkinson’s
Research
PAUL SIEVING, National Eye Institute
1
Institute of Medicine forums and roundtables do not issue, review, or approve
individual documents. The responsibility for the published workshop summary rests with
the workshop rapporteurs and the institution.
vii
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JUDITH SIUCIAK, Foundation for the National Institutes of Health
MARC TESSIER-LAVIGNE, The Rockefeller University
WILLIAM THIES, Alzheimer’s Association
NORA VOLKOW, National Institute on Drug Abuse
KENNETH WARREN, National Institute on Alcohol Abuse and
Alcoholism
JOHN WILLIAMS, Wellcome Trust
STEVIN ZORN, Lundbeck USA
CHARLES ZORUMSKI, Washington University School of Medicine
IOM Staff
BRUCE M. ALTEVOGT, Forum Director
DIANA E. PANKEVICH, Program Officer
ANDREW POPE, Director, Board on Health Sciences Policy
viii
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Reviewers
This workshop summary has been reviewed in draft form by
individuals chosen for their diverse perspectives and technical expertise,
in accordance with procedures approved by the National Research
Council’s Report Review Committee. The purpose of this independent
review is to provide candid and critical comments that will assist the
institution in making its published workshop summary as sound as
possible and to ensure that the workshop summary meets institutional
standards for objectivity, evidence, and responsiveness to the study
charge. The review comments and draft manuscript remain confidential
to protect the integrity of the process. We wish to thank the following
individuals for their review of this workshop summary:
Nathalie Breysee, Lundbeck Research, USA
Malcolm MacLeod, University of Edinburgh
David Shurtleff, National Institute on Drug Abuse
Rae Silver, Columbia University
Mark Tricklebank, Eli Lilly and Company
Bart van der Worp, University Medical Center Utrecht
Although the reviewers listed above have provided many construc-
tive comments and suggestions, they did not see the final draft of
the workshop summary before its release. The review of this workshop
summary was overseen by Floyd E. Bloom, The Scripps Research Insti-
tute. Appointed by the Institute of Medicine, he was responsible for mak-
ing certain that an independent examination of this workshop summary
was carried out in accordance with institutional procedures and that all
review comments were carefully considered. Responsibility for the final
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content of this workshop summary rests entirely with the authors and the
institution.
x
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Contents
1 INTRODUCTION AND OVERVIEW 1
2 EVALUATION OF CURRENT ANIMAL MODELS 9
3 TRANSLATION FROM ANIMAL MODELS TO THE
CLINIC: CASE EXAMPLES FROM NEUROSCIENCE
RESEARCH 21
4 PERSPECTIVES ON STANDARDIZATION 33
5 PERSPECTIVES ON CORRESPONDING ANIMAL
AND CLINICAL ENDPOINTS 45
6 ADDRESSING THE TRANSLATIONAL DISCONNECT 55
7 SUMMARY OF WORKSHOP TOPICS 67
APPENDIXES
A References 77
B Workshop Agenda 83
C Registered Attendees 93
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