Cancer Vaccines From Research to Clinical Practice Cancer Vaccines From Research to Clinical Practice Edited by Adrian Bot, MD, PhD Chief Scientifi c Offi cer, Kite Pharma, Inc., Los Angeles, California, USA Mihail Obrocea, MD Head, Clinical Development–Oncology, Abbott Biotherapeutics Corporation, Redwood City, California, USA and Francesco Marincola, MD Chief, Infectious Disease and Immunogenetics Section, Department of Transfusion Medicine, Clinical Center; Associate Director, Trans-NIH Center for Human Immunology, National Institutes of Health; Director, CC/CHI FOCIS Center of Excellence; President Elect, Society for the Immunotherapy of Cancer; President Elect, International Society for Translational Medicine, Bethesda, Maryland, USA Published in 2011 by Informa Healthcare, Telephone House, 69-77 Paul Street, London EC2A 4LQ, UK. Simultaneously published in the USA by Informa Healthcare, 52 Vanderbilt Avenue, 7th Floor, New York, NY 10017, USA. 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ISBN-13: 978-1-84184-829-7 Orders may be sent to: Informa Healthcare, Sheepen Place, Colchester, Essex CO3 3LP, UK Telephone: +44 (0)20 7017 6682 Email: [email protected] Website: http://informahealthcarebooks.com Library of Congress Cataloging-in-Publication Data Cancer vaccines : from research to clinical practice / edited by Adrian Bot, Mihail Obrocea, Francesco Marincola. p. ; cm. Includes bibliographical references and index. ISBN 978-1-84184-829-7 (hardback : alk. paper) 1. Cancer vaccines. I. Bot, Adrian. II. Obrocea, Mihail. III. Marincola, Francesco M. [DNLM: 1. Cancer Vaccines--therapeutic use. QZ 266] RC271.I45C42 2011 616.99’4061--dc23 2011017121 For corporate sales please contact: [email protected] For foreign rights please contact: [email protected] For reprint permissions please contact: [email protected] Typeset by Exeter Premedia Services Private Ltd., Chennai, India Printed and bound in the United Kingdom. The editors dedicate this book to all translational researchers, drug developers, clinicians, and entrepreneurs, who never gave up on the concept of active immunotherapy for cancer as a viable and promising option for oncology patients, for the present and future. Contents Contributors ix Foreword xiv Preface xv 1. Introduction: Cancer vaccines—mechanism and a clinical overview 1 Antoni Ribas and Adrian Bot 2. Revisiting the paradigm on the putative need for antigen-specifi c responses in cancer 9 Gail D. Sckisel , Julia K. Tietze, and William J. Murphy VACCINE PLATFORMS FOR GENITO-URINARY CANCERS, WITH POTENTIAL APPLICABILITY TO OTHER TUMORS 3. Development of novel immune interventions for genito-urinary cancers 25 Neeraj Agarwal and Nicholas J. Vogelzang 4. Autologous cellular immunotherapy in late-stage prostate cancer: The development history of Sipuleucel-T (PROVENGE®) 44 David L. Urdal and Mark W. Frohlich 5. Design, development, and translation of poxvirus-based vaccines for cancer 56 Benedetto Farsaci, Anna Kwilas, and James W. Hodge VACCINES FOR SOLID CANCERS AND HEMATOLOGICAL MALIGNANCIES 6. Of mice and men (and dogs!): The fi rst approved cancer therapy vaccine 78 Philip J. Bergman and Jedd D. Wolchok 7. Recombinant protein vaccination for antigen-specifi c cancer immunotherapy 87 Pedro de Sousa Alves and Vincent Brichard 8. Antigen-targeted synthetic vaccines for metastatic cancer 103 Zhiyong Qiu, David C. Diamond, Kent A. Smith, Dar Rosario, Sabrina Miles, Mihail Obrocea, Thomas M. Kundig, and Adrian Bot 9. Clinical perspectives in cancer vaccines for hematological diseases 126 Maurizio Chiriva-Internati, Leonardo Mirandola, Marjorie Jenkins, Martin Cannon, Everardo Cobos, and W. Martin Kast 10. Epitope-based vaccines for cancer 140 Vy Phan-Lai, Denise L. Cecil, Gregory E. Holt, Daniel R. Herendeen, Forrest Kievit, Miqin Zhang, and Mary L. Disis VACCINE DEVELOPMENT: TRIAL DESIGN AND IMMUNE ASSAYS 11. Emerging clinical trial design concepts for therapeutic cancer vaccines 150 Cristina Musselli, Leah Isakov, and Kerry Wentworth CONTENTS 12. T-cell immune monitoring assays to guide the development of new cancer vaccines 156 Cedrik M. Britten, Sylvia Janetzki, Cécile Gouttefangeas, Marij J. P. Welters, Michael Kalos, Christian Ottensmeier, Axel Hoos, and Sjoerd H. van der Burg 13. A biomarker-based, systems biology approach guiding the development of active immunotherapies and immune monitoring 167 Glenda Canderan, Peter Wilkinson, John Schatzle, Mark Cameron, and Rafi ck-Pierre Sekaly EMERGING NEW VACCINE STRATEGIES, TARGETS, AND ADJUVANTS 14. Targeting regulatory T cells and other strategies to enable cancer vaccines 182 Christopher Paustian, Shawn M. Jensen, Sarah Church, Sachin Puri, Chris Twitty, Hong-Ming Hu, Brendan D. Curti, Walter J. Urba, Raj K. Puri, and Bernard A. Fox 15. Molecular targeting of cancer stem cells 202 Zhenhua Li, Debraj Mukherjee, Jang-Won Lee, and John S. Yu 16. RNA in cancer vaccine therapy 217 Smita Nair, David Boczkowski, Scott Pruitt, and Johannes Urban 17. Induction of innate immunity by nucleic acids: A potential adjuvant for cancer vaccines? 232 Bo Jin and Anthony E. T. Yeo NEXT-GENERATION HYBRID ACTIVE/PASSIVE IMMUNOTHERAPIES 18. Passive immunotherapy by T cell–engaging bispecifi c antibodies 250 Patrick A. Baeuerle and Benno Rattel 19. Antibodies to peptide–HLA complexes have potential application for cancer diagnosis and therapy 263 Jon A. Weidanz and William H. Hildebrand Index 277 viii Contributors Neeraj Agarwal Assistant Professor of Medicine, University of Utah, Huntsman Cancer Institute, Salt Lake City, UT, USA Patrick A. Baeuerle Senior Vice President, Chief Scientifi c Offi cer, Micromet AG, Munich, Germany Philip J. Bergman Chief Medical Offi cer, BrightHeart Veterinary Centers, Armonk, NY, USA; Adjunct Associate, Memorial Sloan-Kettering Cancer Center, New York, NY, USA David Boczkowski Department of Surgery, Duke University Medical Center, Durham, NC, USA Adrian Bot Chief Scientifi c Offi cer, Kite Pharma, Inc., Los Angeles, CA, USA Vincent Brichard Vice President, Cancer Immunotherapeutics, GlaxoSmithKline Biologicals, Rixensart, Belgium Cedrik M. Britten Johannes Gutenberg–University Mainz, Department of Internal Medicine III, Division of Experimental Oncology, and Ribological GmbH, Mainz, Germany Mark Cameron Vaccine and Gene Therapy Institute, Port Saint Lucie, FL, USA Glenda Canderan Vaccine and Gene Therapy Institute, Port Saint Lucie, FL, USA Martin Cannon Department of Microbiology and Immunology, University of Arkansas for Medical Sciences, Little Rock, AR, USA Denise L. Cecil Senior Fellow, Department of Medicine, University of Washington, Seattle, WA, USA Maurizio Chiriva-Internati Director of Basic Research, Director of Translational Research, Division of Hematology/Oncology, Texas Tech University Health Sciences Center and Southwest Cancer Treatment and Research Center, Lubbock, TX, USA Sarah Church Laboratory of Molecular and Tumor Immunology, Providence Cancer Center, Portland, OR, USA Everardo Cobos Division of Hematology/Oncology, Texas Tech University Health Sciences Center and Southwest Cancer Treatment and Research Center, Lubbock, TX, USA Brendan D. Curti Clinical Research, Robert W. Franz Cancer Research Center, Earle A. Chiles Research Institute, Providence Cancer Center, Providence Portland Medical Center, Portland, OR, USA Pedro Miguel de Sousa Alves Senior Scientist, Cancer Immunotherapeutics, GlaxoSmith- Kline Biologicals, Rixensart, Belgium David C. Diamond MannKind Corporation, Valencia, CA, USA Mary L. Disis Professor, Division of Oncology, University of Washington; Seattle Cancer Care Alliance, Fred Hutchinson Cancer Research Center, Seattle, WA, USA