New Vaccine Technologies

325 Pages·2001·2.117 MB·English

by  Ronald W. Ellis

MEDICAL INTELLIGENCE UNIT 26
Ronald W. Ellis
New Vaccine
E
L
L
IS
Technologies
MIU
26
N
e
w
V
a
c
c
in
e
T
e
c
h
n
o
lo
g
ie
s
M
EDICAL
I
NTELLIGENCE
U 26
NIT
New Vaccine
Technologies
Ronald W. Ellis, Ph.D.
BioChem Pharma
(to become Shire Biologics after soon-expected merger)
Northborough, Massachusetts, U.S.A.
LANDES BIOSCIENCE EUREKAH.COM
GEORGETOWN, TEXAS AUSTIN, TEXAS
U.S.A. U.S.A.
N V T
EW ACCINE ECHNOLOGIES
Medical Intelligence Unit
Eurekah.com
Landes Bioscience
designed by Lana K. Moore
Copyright ©2001 Eurekah.com
All rights reserved.
No part of this book may be reproduced or transmitted in any form or by any means, electronic or
mechanical, including photocopy, recording, or any information storage and retrieval system,
without permission in writing from the publisher.
Printed in the U.S.A.
Please address all inquiries to the Publishers:
Eurekah.com / Landes Bioscience, 810 South Church Street
Georgetown, Texas, U.S.A. 78626
Phone: [phone]; FAX: [phone]
www.Eurekah.com
www.landesbioscience.com
ISBN 1-58706-050-7 (hard cover version)
ISBN 1-58706-080-9 (soft cover version)
While the authors, editors and publisher believe that drug selection and dosage and the specifications
and usage of equipment and devices, as set forth in this book, are in accord with current recommend-
ations and practice at the time of publication, they make no warranty, expressed or implied, with
respect to material described in this book. In view of the ongoing research, equipment development,
changes in governmental regulations and the rapid accumulation of information relating to the
biomedical sciences, the reader is urged to carefully review and evaluate the information provided
herein.
Library of Congress Cataloging-in-Publication Data
New Vaccine Technologies / [edited by] Ronald W. Ellis
p.;cm.-- (Biotechnology intelligence unit)
Includes bibliographical references and index.
ISBN 1-58706-050-7 (alk. paper)
1. Vaccines--Biotechnology. I. Ellis, Ronald W. II. Series.
[DNLM: 1. Vaccines. 2. Biotechnology. QW 805 N5324 2001]
TP248.65.V32N49 2001
615'372--dc21 00-063630
D
EDICATION
Dedicated to my wife Danielle and children Jacob and Miriam for their
love, patience and support, and to the memory of my father.
CONTENTS
Preface .................................................................................................XI
1. New Technologies for Making Vaccines.................................................1
Ronald W. Ellis
Introduction..........................................................................................1
Live Vaccines.........................................................................................4
Subunit/Inactivated Vaccines ................................................................8
DNA (Nucleic Acid) Vaccines.............................................................14
Formulation of Antigens .....................................................................15
Conclusion..........................................................................................16
2. Clinical Issues for New Technologies...................................................21
Luc Hessel
Introduction........................................................................................21
Definition of the Medical Needs.........................................................22
Moving from Preclinical to Clinical Development...............................22
Demonstration of the Proof-of-Principle.............................................23
Design and Implementation of a Clinical Development Plan..............23
Continuous Assessment of Safety and Efficacy ....................................27
Specific Issues......................................................................................28
Conclusion..........................................................................................30
3. Regulatory Issues..................................................................................33
Marion F. Gruber, Paul G. Richman and Julianne C. M. Clifford
Introduction........................................................................................33
Federal Regulations Pertaining to Vaccines..........................................33
Premarketing Phase.............................................................................35
Postmarketing Phase............................................................................41
4. In-Licensing Issues and Vaccine Technologies......................................44
Dale R. Spriggs
Introduction........................................................................................44
Why Do Companies In-License?.........................................................44
How Do Companies Evaluate Licensing Opportunities?.....................46
How Can an Inventor Make the Technology More Attractive? ...........48
Framework of a Licensing Agreement..................................................49
What Does the Future Hold?..............................................................50
CODA................................................................................................50
5. Live Vaccines........................................................................................51
Alan R. Shaw
Smallpox Vaccine................................................................................51
Japanese Encephalitis Vaccines............................................................52
Yellow Fever Vaccines .........................................................................53
Poliovirus Vaccines..............................................................................54
Measles Vaccines.................................................................................57
Rubella Vaccines .................................................................................59
Mumps Vaccines.................................................................................61
Trivalent Measles-Mumps-Rubella Vaccines .......................................64
Quadrivalent Measles-Mumps-Rubella-Varicella Vaccines ..................67
Varicella Vaccines................................................................................67
Rotavirus Vaccines ..............................................................................70
Live Attenuated Influenza Vaccines.....................................................73
6. Recombinant Live Attenuated Viral Vaccines.......................................90
Richard R. Spaete
Advantages and Concerns Associated with the Use
of Live Attenuated Vaccines ............................................................90
Vaccine Efforts for Herpesviruses........................................................91
Prospects for the Future.......................................................................97
7. Live Viral Vectors...............................................................................101
Elizabeth B. Kauffman, Michel Bublot, Russell R. Gettig,
Keith J. Limbach, Steven E Pincus and Jill Taylor
DNA Viruses.....................................................................................102
RNA Viruses.....................................................................................114
8. Inactivated Virus Vaccines..................................................................134
Andrew D. Murdin, Benjamin Rovinski, Suryaprakash Sambhara
Introduction......................................................................................134
Current Inactivated Virus Vaccines...................................................134
Issues Affecting the Use of Inactivated Virus Vaccines.......................137
Conclusion........................................................................................146
9. Live Attenuated Bacterial Vaccines.....................................................152
Kevin P. Killeen and Victor J DiRita
Introduction......................................................................................152
Types of Vaccines..............................................................................152
Live Attenuated Bacterial Vaccines....................................................155
Live Attenuated Mycobacterium Bovis
(BCG Tuberculosis Vaccine).........................................................155
Immune Correlates of Protection ......................................................158
Live Attenuated Salmonella typhi (Typhoid Fever Vaccines
and Vaccine Candidates)...............................................................158
Live Attenuated Shigella Sp (Shigellosis Vaccine Candidates)............161
Live Virulence-Attenuated Vibrio cholerae (Cholera Vaccines)...........164
Animal Model...................................................................................165
Vaccine Efforts..................................................................................165
10. Live Attenuated Bacterial Vectors.......................................................172
Sims K. Kochi and Kevin P. Killeen
Introduction......................................................................................171
Salmonella Vectors............................................................................172
bacille Calmette-Guérin ....................................................................175
Vibrio Vectors...................................................................................176
Listerial Vectors.................................................................................178
Next Generation Bacterial Vectors ....................................................179
DNA Delivery...................................................................................180
Summary...........................................................................................182
11. Protein-Based Vaccines ......................................................................186
Sheena M. Loosmore, Gavin R. Zealey and Raafat E.F. Fahim
Introduction......................................................................................186
Pediatric Vaccines..............................................................................186
Adult Vaccines ..................................................................................189
Vaccines Against Nosocomial Infections............................................194
Cancer Vaccines................................................................................194
Vaccines Against Autoimmune Diseases............................................197
Current Technologies........................................................................198
Emerging Technologies.....................................................................199
Summary...........................................................................................201
12. Peptide Vaccines ................................................................................214
Damu Yang, Gregory E. Holt, Michael P. Rudolf, Markwin P. Velders,
Remco M. P. Brandt, Eugene D. Kwon and W. Martin Kast
Introduction......................................................................................214
Molecular Basis for the Development of Peptide Vaccines.................214
Advantages and Disadvantages of Peptide-Based Vaccines.................215
Adjuvants and Delivery Systems........................................................215
Design of Peptide Vaccines: Synthetic Peptides as B-Cell Vaccines....216
Peptide-Based T-Cell Vaccines. Identification of Peptide Epitopes
Recognized by T Cells...................................................................217
Synthetic Peptides as T-Cell Vaccines ...............................................217
Recombinant Vaccines Expressing T-Cell Epitopes...........................218
Adoptive Cellular Therapy ................................................................219
Summary and Perspectives ................................................................220
13. Polysaccharide Vaccines .....................................................................227
Stephen Freese
Polysaccharide Immunity..................................................................227
Issues in Designing a Conjugate........................................................227
Issues in Making a Conjugate............................................................229
Applications ......................................................................................232
14. DNA Vaccines....................................................................................240
Daniel E. McCallus, Catherine J. Pachuk, Shaw-guang Lee
and C. Satishchandran
Introduction......................................................................................240
Gene Expression................................................................................241
Mechanisms of Immunostimulation..................................................242
Routes of Administration ..................................................................245
Intracellular Delivery of DNA Vaccines ............................................248
Safety of Nucleic Acid Vaccines.........................................................251
Future Directions of DNA Vaccines..................................................253
Summary...........................................................................................256
15. Plant-Derived Vaccines......................................................................263
Amanda M. Walmsely and Charles J. Arntzen
Introduction......................................................................................263
Mucosal Vaccines..............................................................................263
Production of Plant-Derived Vaccines...............................................264
Plant-Derived Vaccines .....................................................................265
Summary...........................................................................................270
Future Use of Plant-Delivered Vaccines ............................................270
16. Biological Aspects and Prospects for Adjuvants
and Delivery Systems .........................................................................274
Bror Morein and Ke-Fei Hu
Introduction......................................................................................274
Innate Immunity: The Gateway to an Acquired Immune Response ..277
APCs Instruct the Acquired Immune System ....................................280
Immune Modulation is Based on Cross-Talk Between Innate
Immunity and Helper T Cells.......................................................281
The Collaboration Between the Complement System and B Cells:
Roles for Adjuvants .......................................................................282
Immune Modulation For CTL..........................................................283
Delivery Systems ...............................................................................284
Vaccines for Newborns and Elderly Require
Suitable Strong Adjuvants .............................................................285
The Present Situation and Future Aspects of Adjuvants
and Delivery Systems.....................................................................286
17. Transcutaneous Immunization...........................................................292
Gregory M. Glenn
Introduction......................................................................................292
Barriers and Targets for TCI .............................................................292
Adjuvants and TCI............................................................................294
Immune Responses to Transcutaneous Immunization.......................295
Mucosal Responses............................................................................296
Diversity of Antigens.........................................................................298
Delivery Options Using Transcutaneous Immunization....................299
Optimization for Enhancement of the Immune Response.................299
Human Studies .................................................................................302
Conclusions.......................................................................................302
Index..................................................................................................305
EDITOR
Ronald W. Ellis, Ph.D.
BioChem Pharma
(to become Shire Biologics after soon-expected merger)
Northborough, Massachussetts, U.S.A.
Chapter 1
CONTRIBUTORS
Charles J. Arntzen Russell R. Gettig
Boyce Thompson Institute for Plant Arbovirus Unit
Research Griffin Lab
Ithaca, New York, U.S.A. Slingerlands, New York, U.S.A.
Chapter 15 Chapter 7
Remco M. P. Brandt Gregory M. Glenn
Cardinal Bernardin Cancer Center IOMAI Corporation
Loyola University Chicago Washington, District of Columbia,
Maywood, Illinois, U.S.A. U.S.A.
Chapter 12 Chapter 17
Michel Bublot Marion Gruber
Arbovirus Unit Office of Vaccines Research and Review
Griffin Lab Division of Vaccines and Related
Slingerlands, New York, U.S.A. Products Applications
Chapter 7 Rockville, Maryland, U.S.A.
Chapter 3
Julianne C.M. Clifford
Office of Vaccines Research and Review Luc Hessel
Division of Vaccines and Related Medical Department
Products Applications Pasteur Merieux MSD
Rockville, Maryland, U.S.A. Lyon Cedex, France
Chapter 3 Chapter 2
Victor J. DiRita Gregory E. Holt
Department Microbiology and Cardinal Bernardin Cancer Center
Immunology Loyola University Chicago
Unit for Laboratory Animal Medicine Maywood, Illinois, U.S.A.
University of Michigan Medical School Chapter 12
Ann Arbor, Michigan, U.S.A.
Chapter 9 Ke-Fei Hu
Department of Virology
Rafaat Fahim National Veterinary Institute
Connaught Laboratories, Ltd. Uppsala, Sweden
Willowdale, Ontario, Canada Chapter 16
Chapter 11
W. Martin Kast
Stephen Freese Cardinal Bernardin Cancer Center
Wyeth-Lederle Vaccines Loyola University Chicago
West Henrietta, New York, U.S.A. Maywood, Illinois, U.S.A.
Chapter 13 Chapter 12
Elizabeth Kauffman Andrew Murdin
Arbovirus Unit Aventis Pasteur Ltd.
Griffin Lab Willowdale, Ontario, Canada
Slingerlands, New York, U.S.A. Chapter 8
Chapter 7
Catherine J. Pachuk
Kevin Killeen Wyeth-Lederle Vaccines and Pediatrics
AVANT Immunotherapeutics Inc. Malvern, Pennsylvania, U.S.A.
Needham, Massachusetts, U.S.A. Chapter 14
Chapter 9, 10
Steven E Pincus
Sims K. Kochi Arbovirus Unit
AVANT Immunotheraputics, Inc. Griffin Lab
Needham, Massachusetts, U.S.A. Slingerlands, New York, U.S.A.
Chapter 10 Chapter 7
Eugene D. Kwon Paul C. Richman
Cardinal Bernardin Cancer Center Office of Vaccines Research and Review
Loyola University Chicago Division of Vaccines and Related
Maywood, Illinois, U.S.A. Products Applications
Chapter 12 Rockville, Maryland, U.S.A.
Chapter 3
Shaw-guang Lee
Wyeth-Lederle Vaccines and Pediatrics Benjamin Rovinski
Malvern, Pennsylvania, U.S.A. Aventis Pasteur Ltd.
Chapter 14 Willowdale, Ontario, Canada
Chapter 8
Keith J. Limbach
Arbovirus Unit Michael P. Rudolf
Griffin Lab Cardinal Bernardin Cancer Center
Slingerlands, New York, U.S.A. Loyola University Chicago
Chapter 7 Maywood, Illinois, U.S.A.
Chapter 12
Sheena M. Loosmore
Connaught Laboratories, Ltd. Suryaprakash Sambhara
Willowdale, Ontario, Canada Aventis Pasteur Ltd.
Chapter 11 Willowdale, Ontario, Canada
Chapter 8
Daniel McCallus
Wyeth-Lederle Vaccines and Pediatrics C. Satishchandran
Malvern, Pennsylvania, U.S.A. Wyeth-Lederle Vaccines and Pediatrics
Chapter 14 Malvern, Pennsylvania, U.S.A.
Chapter 14
Bror Morein
Department of Virology Alan Shaw
National Veterinary Institute Virus and Cell Biology
Uppsala, Sweden Merck Research Laboratory
Chapter 16 West Point, Pennsylvania, U.S.A.
Chapter 5