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Suggested Citation:"Front Matter." Institute of Medicine. 2013. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13530.
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IMPROVING THE UTILITY
AND TRANSLATION
OF ANIMAL MODELS
FOR NERVOUS SYSTEM
DISORDERS

WORKSHOP SUMMARY

Diana E. Pankevich, Theresa M. Wizemann,
and Bruce M. Altevogt, Rapporteurs


Forum on Neuroscience and
Nervous System Disorders

Board on Health Sciences Policy

INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES

THE NATIONAL ACADEMIES PRESS

Washington, D.C.

www.nap.edu

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13530.
×

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.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13530.
×

Knowing is not enough; we must apply.
Willing is not enough; we must do.

—Goethe

image

INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES

Advising the Nation. Improving Health.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13530.
×

THE NATIONAL ACADEMIES

Advisers of the Nation on Science, Engineering, and Medicine

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 by the Academy, the Council has become the principal operating agency of both the National Academy of Sciences and the National Academy of Engineering in providing services to the government, the public, and the scientific and engineering communities. The Council is administered jointly 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

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13530.
×

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)

________________________

1Institute 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.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13530.
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Suggested Citation:"Front Matter." Institute of Medicine. 2013. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13530.
×

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

___________________________

1Institute 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.

Page viii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2013. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13530.
×

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

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13530.
×

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 constructive 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 Institute. Appointed by the Institute of Medicine, he was responsible for making 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

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13530.
×

content of this workshop summary rests entirely with the authors and the institution.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13530.
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Nervous system diseases and disorders are highly prevalent and substantially contribute to the overall disease burden. Despite significant information provided by the use of animal models in the understanding of the biology of nervous system disorders and the development of therapeutics; limitations have also been identified. Treatment options that are high in efficacy and low in side effects are still lacking for many diseases and, in some cases are nonexistent. A particular problem in drug development is the high rate of attrition in Phase II and III clinical trials. Why do many therapeutics show promise in preclinical animal models but then fail to elicit predicted effects when tested in humans?

On March 28 and 29, 2012, the Institute of Medicine Forum on Neuroscience and Nervous System Disorders convened the workshop "Improving Translation of Animal Models for Nervous System Disorders" to discuss potential opportunities for maximizing the translation of new therapies from animal models to clinical practice. The primary focus of the workshop was to examine mechanisms for increasing the efficiency of translational neuroscience research through discussions about how and when to use animal models most effectively and then best approaches for the interpretation of the data collected. Specifically, the workshop objectives were to: discuss key issues that contribute to poor translation of animal models in nervous system disorders, examine case studies that highlight successes and failures in the development and application of animal models, consider strategies to increase the scientific rigor of preclinical efficacy testing, explore the benefits and challenges to developing standardized animal and behavioral models. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary also identifies methods to facilitate development of corresponding animal and clinical endpoints, indentifies methods that would maximize bidirectional translation between basic and clinical research and determines the next steps that will be critical for improvement of the development and testing of animal models of disorders of the nervous system.

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