gene therapy of cancer, methods and protocols

Methods and Protocols Methods and Protocols Gene Therapy of Cancer M E T H O D S I N M O L E C U L A R M E D I C I N E TM Humana Press Edited by Wolfgang Walther Ulrike Stein Gene Therapy of Cancer Edited by Wolfgang Walther Ulrike Stein Humana Press Gene Therapy of Cancer M E T H O D S I N M O L E C U L A R M E D I C I N E TM John M. Walker, S ERIES E DITOR 40. Diagnostic and Therapeutic Antibodies, edited by Andrew J. T. George and Catherine E. Urch, 2000 39. Ovarian Cancer: Methods and Protocols, edited by John M. S. Bartlett, 2000 38. Aging Methods and Protocols, edited by Yvonne A. Barnett and Christopher P. Barnett, 2000 37. Electrically Mediated Delivery of Molecules to Cells, edited by Mark J. Jaroszeski, Richard Heller, and Richard Gilbert, 2000 36. Septic Shock Methods and Protocols, edited by Thomas J. Evans, 2000 35. Gene Therapy of Cancer: Methods and Protocols, edited by Wolfgang Walther and Ulrike Stein, 2000 34. Rotavirus Methods and Protocols, edited by James Gray and Ulrich Desselberger, 2000 33. Cytomegalovirus Protocols, edited by John Sinclair, 2000 32. Alzheimer’s Disease: Methods and Protocols, edited by Nigel M. Hooper, 1999 31. Hemostasis and Thrombosis Protocols: Methods in Molecular Medicine, edited by David J. Perry and K. John Pasi, 1999 30. Vascular Disease: Molecular Biology and Gene Therapy Protocols, edited by Andrew H. Baker, 1999 29. DNA Vaccines: Methods and Protocols, edited by Douglas B. Lowrie and Robert Whalen, 1999 28. Cytotoxic Drug Resistance Mechanisms, edited by Robert Brown and Uta Böger-Brown, 1999 27. Clinical Applications of Capillary Electrophoresis, edited by Stephen M. Palfrey, 1999 26. Quantitative PCR Protocols, edited by Bernd Kochanowski and Udo Reischl, 1999 25. Drug Targeting, edited by G. E. Francis and Cristina Delgado, 1999 24. Antiviral Methods and Protocols, edited by Derek Kinchington and Raymond F. Schinazi, 1999 23. Peptidomimetics Protocols, edited by Wieslaw M. Kazmierski, 1999 22. Neurodegeneration Methods and Protocols, edited by Jean Harry and Hugh A. Tilson, 1999 21. Adenovirus Methods and Protocols, edited by William S. M. Wold, 1998 20. Sexually Transmitted Diseases: Methods and Protocols, edited by Rosanna Peeling and P. Frederick Sparling, 1999 19. Hepatitis C Protocols, edited by Johnson Yiu-Nam Lau, 1998 18. Tissue Engineering, edited by Jeffrey R. Morgan and Martin L. Yarmush, 1999 17. HIV Protocols, edited by Nelson Michael and Jerome H. Kim, 1999 16. Clinical Applications of PCR, edited by Y. M. Dennis Lo, 1998 Humana Press Totowa, New Jersey Edited by Wolfgang Walther Max-Delbrück-Center for Molecular Medicine, Berlin, Germany and Ulrike Stein Max-Delbrück-Center for Molecular Medicine, Berlin, Germany M E T H O D S I N M O L E C U L A R M E D I C I N E TM Gene Therapy of Cancer Methods and Protocols © 2000 Humana Press Inc. 999 Riverview Drive, Suite 208 Totowa, New Jersey 07512 All rights reserved. No part of this book may be reproduced, stored in a retrieval system, or transmitted in any form or by any means, electronic, mechanical, photocopying, microfilming, recording, or otherwise without written permission from the Publisher. Methods in Molecular Medicine ™ is a trademark of The Humana Press Inc. All authored papers, comments, opinions, conclusions, or recommendations are those of the author(s), and do not necessarily reflect the views of the publisher. This publication is printed on acid-free paper. 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The fee code for users of the Transactional Reporting Service is: [0-89603-714-2 (hardcover) 089603-843-2 (combbound)/00 $10.00 + $00.25]. Printed in the United States of America. 10 9 8 7 6 5 4 3 2 1 Library of Congress Cataloging in Publication Data Main entry under title: Methods in molecular medicine ™ . Gene therapy of cancer : methods and protocols / edited by Wolfgang Walther and Ulrike Stein. p. cm. (Methods in molecular medicine ; 35) Includes bibliographical references and index. ISBN 0-89603-714-2 (hard : alk. paper). ISBN 0-89603-843-2 (comb : alk. paper) 1. Cancer Gene therapy Laboratory manuals. I. Walther, Wolfgang. II. Stein, Ulrike. II. Series [DNLM: 1. Neoplasms therapy. 2. Gene Therapy methods. QZ 266 G3259 2000] RC271.G45G484 2000 616.99'4042 dc21 DNLM/DLC for Library of Congress 99-38474 CIP Preface v Since the discovery of the molecular structure of genes and the unveiling of the molecular basis of numerous human diseases, scientists have been fasci- nated with the possibility of treating certain diseases by transducing foreign DNA into the affected cells. Initially, it was proposed that the foreign DNA could either replace defective nonfunctional genes, or code for therapeutic proteins. This concept has evolved into the rapidly growing field of gene therapy. Even though surgery, radiotherapy, and chemotherapy are widely avail- able and routinely used for cancer treatment, these therapies fail to cure approximately 50 percent of cancer patients. Therefore, since it is a disease characterized by aberrant gene expression, cancer has been a target of gene therapy research since the inception of this treatment modality. Numerous cancer gene therapy strategies are currently being investigated, including gene replacement therapy, the regulation of gene expression to modulate immuno- logical responses to tumors, the direct killing of tumor cells, and direct inter- ference with tumor growth. In this context, gene transfer systems, tumor-specific expression vectors, and novel therapeutic genes have been extensively stud- ied. All these strategies aim for the selective destruction of human malignant disease while circumventing the destruction of nonmalignant cells and tissues thereby minimizing toxicity to the patient. Rapid progress in the field of cancer gene therapy, exemplified by the vast number of publications in this area, creates a challenging situation for scientists and clinicians who need to be cognizant of the most recent advances in gene transfer techniques. This volume of Gene Therapy of Cancer: Meth- ods and Protocols in the Methods in Molecular Medicine series will provide researchers with a broad array of methods used to study cancer gene therapy in both the laboratory and clinical trials. Moreover, several chapters are included to provide short overviews of specialized gene therapy strategies for the treat- ment of particular malignancies. Gene Therapy of Cancer: Methods and Protocols does not provide com- prehensive reviews of all methodologies currently used for gene therapy of cancer. Rather the topics we have selected consist of approved procedures, vi Preface Wolfgang Walther Ulrike Stein current trends, and representative strategies in cancer gene therapy using dif- ferent classes of therapeutic genes, suppressor genes, antisense oligonucle- otides, ribozymes, viral- and nonviral-vector systems, and tumor targeting approaches at the preclinical and, more importantly, at the clinical level. For cancer gene therapy to be successful in the treatment of human cancers, exten- sive preclinical evaluation is essential. Therefore, the first part of this book discusses relevant experiments from preclinical studies followed by clinical gene therapy protocols in the second part. Gene Therapy of Cancer: Methods and Protocols should provide practi- cal guidance for basic and clinical researchers, as well as graduate and post- graduate students working in the exciting and emerging field of gene therapy. Contents Preface v Contributors xi PART I. EXPERIMENTAL APPROACES TO CANCER GENE THERAPY A: Immunotherapy/Tumor Vaccination 1 Immunizing Potential of Cytokine-Transduced Tumor Cells Mario P. Colombo and Monica Rodolfo 3 2 Particle-Mediated Gene Transfer into Dendritic Cells: A Novel Strategy for the Induction of Immune Responses against Tumor Antigens Thomas Tüting and Andreas Albers 27 3Cancer Gene Therapy with Heat Shock Protein-65 Gene Katalin V. Lukacs and Artit Nakakes 49 4Recombinant Vaccinia Virus MVA for Generation and Analysis of T Cell Responses Against Tumor Associated Antigens Ingo Drexler, Karl Heller, Marion Ohlmann, Volker Erfle, and Gerd Sutter 57 B: Suicide Gene Therapy 5 Selection of Cytochrome P450 Genes for Use in Prodrug Activation-Based Cancer Gene Therapy Jodi E. D. Hecht and David J. Waxman 77 6Construction of P450-Expressing Tumor Cell Lines Using Retroviruses Jodi E. D. Hecht, Youssef Jounaidi, and David J. Waxman 85 7 In Vitro Methods for Evaluation of P450 -Based Anticancer Gene Therapy Jodi E. D. Hecht and David J. Waxman 95 8 Tumor Models for Evaluation of P450 Gene Therapy In Vivo Jodi E. D. Hecht, Pamela Schreiber Schwartz, and David J. Waxman 107 vii C: Anti-Oncogene and Suppressor Gene Therapy 9 Intracellular Single-Chain Antibodies for Gene Therapy Guadalupe Bilbao, Jesus Gomez-Navarro, Keizo Kazano, Juan Luis Contreras, and David T. Curiel 121 10 Combined Adenoviral Transfer of Tumor Suppressor and Cell-Cycle Genes for Tumor-Cell Apoptosis Karsten Brand, Volker Sandig, and Michael Strauss 151 D: Antisense Gene Therapy 11 Inhibition of Cell Growth by Antisense Oligonucleotides Targeting the Growth-Related Protein Kinase c-raf Doriano Fabbro, B. P. Monia, K H. Altmann, and Thomas Geiger 167 12 IGF-1 Antisense Strategies for Cancer Treatment Yue Xin Pan and Donald D. Anthony 189 E: Ribozyme Gene Therapy 13 Anti- MDR1 Ribozyme Gene Therapy Takao Ohnuma, Hiroyuki Kobayashi, and Fu-Sheng Wang 207 14 Anti-c- erb B2 Ribozyme for Gene Therapy of Breast Cancer Toshiya Suzuki, Masami Bessho, and Kevin J. Scanlon 247 15 Anti-K- ras Ribozyme Adenoviral Vector for Gene Therapy of Non-Small Cell Lung Cancer Yu-An Zhang, John Nemunaitis, and Alex W. Tong 261 F: Delivery Systems and Tumor Targeting 16 Green Fluorescent Protein Retroviral Vector: Generation of High-Titer Producer Cells and Virus Supernatant Wolfgang Uckert, Lene Pedersen, and Walter Günzburg 275 17 HSV-1 Vectors for Gene Therapy of Experimental CNS Tumors Ulrich Herrlinger, Andreas Jacobs, Manish Aghi, Deborah E. Schuback, and Xandra O. Breakefield 287 18 Intratumoral Injection of Naked DNA Jingping Yang 313 19 Cationic Liposome Gene Transfer Kyonghee Kay Son 323 20 In Vivo Particle-Mediated Gene Transfer for Cancer Therapy Alexander L. Rakhmilevich and Ning-Sun Yang 331 viii Contents Contents ix 21 Gene Targeting to Hepatomas (AFP) Shotaro Tsuruta, Akio Ido, and Shigenobu Nagataki 345 22 Adenovirus-Mediated Targeted Gene Therapy for Breast Cancer and for Purging Hematopoietic Stem-Cell Sources Ling Chen 361 23 Chemotherapy-Inducible Vector for Gene Therapy of Cancer Wolfgang Walther, Ulrike Stein, Robert H. Shoemaker, and Peter M. Schlag 371 G: Alternative Approaches in Cancer Gene Therapy 24 Oncolytic Adenoviral Vectors Ramon Alemany and Wei-Wei Zhang 395 25 Genetically Modified Clostridium for Gene Therapy of Tumors Mary E. Fox, Marilyn J. Lemmon, Amato J. Giaccia, Nigel P. Minton, and J. Martin Brown 413 26 Tumor-Targeted Salmonella : Strain Development and Expression of the HSV-tK Effector Gene David Bermudes, Brooks Low, and John M. Pawelek 419 Part II. Clinical Protocols for Cancer Gene Therapy A: Immunotherapy/Tumor Vaccination 27 Ex Vivo Cytokine Gene Transfer in Melanomas by Using Particle Bombardment Dirk Schadendorf 439 28 Intratumoral Gene Transfer of the HLA-B7 Gene Into Colon Carcinoma Metastases Evanthia Galanis and Joseph Rubin 453 29 Hybrid Cell Vaccination in Patients with Metastatic Melanoma Uwe Trefzer, Guido Weingart, Wolfram Sterry, and Pete Walden 469 B: Suicide Gene Therapy 30 Retroviral Transfer of the Herpes Simplex Virus-Thymidine Kinase ( HSV-tK ) Gene for the Treatment of Cancer Rajagopal Ramesh, Anupama Munshi, Aizen J. Marrogi, and Scott M. Freeman 479 31 Gene Therapy for Treatment of Brain Tumors ( HSV-tK In Vivo Gene Transfer): A Case Study Friedrich Weber, Frank Floeth, and Hans Bojar 499 [...]...x Contents 32 Gene Therapy of Glioblastoma Multiforme with a Bicistronic Retroviral Vector Expressing Human IL-2 and HSV-tk Giorgio Palù, Massimo Pizzato, Roberta Bonaguro, and Frederico Colombo 511 33 Intratumoral Gene Transfer of the Cytosine Deaminase Gene for the Treatment of Breast Cancer Hardev S Pandha and Nicholas R Lemoine 523 C: Anti-Oncogene and Suppressor Gene Therapy 34 Adenovirus-Mediated... 35: Gene Therapy: Methods and Protocols Edited by: W Walther and U Stein © Humana Press, Inc., Totowa, NJ 3 4 Colombo and Rodolfo The events caused by the injection of cytokine-transduced tumors include both the early infiltration of granulocytes and then of macrophages and lymphocytes, and the release of secondary cytokines Tumor-cell debris derived from tumor destruction may represent the source of. .. JONG HO WON • Genetic Therapy Program of the Yale Cancer Center and the Medical Oncology Section, Department of Internal Medicine, Yale University School of Medicine, New Haven, CT LIAN HUA YIN • Genetic Therapy Program of the Yale Cancer Center and the Medical Oncology Section, Department of Internal Medicine, Yale University School of Medicine, New Haven, CT JINGPING YANG • Genetic Therapy, Gaithersburg,... PAWELEK • Departments of Dermatology and Pharmacology, Yale University School of Medicine, New Haven, CT LENE PEDERSEN • Department of Molecular and Structural Biology, University of Aarhus, Aarhus, Denmark Contributors xv XUE YAN PENG • Genetic Therapy Program of the Yale Cancer Center and the Medical Oncology Section, Department of Internal Medicine, Yale University School of Medicine, New Haven,... University of Alabama at Birmingham, Birmingham, AL MATTHEW COOPERBERG • Genetic Therapy Program of the Yale Cancer Center, and the Medical Oncology Section, Department of Internal Medicine, Yale University School of Medicine, New Haven, CT DAVID T CURIEL • Gene Therapy Program, University of Alabama at Birmingham, Birmingham, AL ALBERT DEISSEROTH • Department of Internal Medicine, Yale University School of. .. School of Medicine, Stanford, CA Contributors xiii JESUS GOMEZ-NAVARRO • Gene Therapy Program, University of Alabama at Birmingham, Birmingham, AL WALTER GÜNZBURG • Institute of Virology, University of Veterinary Sciences, Vienna, Austria XIANG YANG DAVID GUO • Genetic Therapy Program of the Yale Cancer Center and the Medical Oncology Section, Department of Internal Medicine, Yale University School of. .. PIZZATO • Institute of Microbiology, University of Padova Medical School, Padova, Italy ALEXANDER L RAKHMILEVICH • Department of Human Oncology, University of Wisconsin Medical School, Madison, WI RAJAGOPAL R AMESH • Department of Thoracic and Cardiovascular Surgery, M.D Anderson Cancer Center, Houston, TX DANIEL J RILEY • Department of Medicine, Division of Nephrology, University of Texas Health Center,... Colombo and Monica Rodolfo 1 Introduction 1.1 Cytokine -Gene- Modified Tumor Cell Vaccines for Cancer Immunotherapy: Concepts, Rationale, and Prospective The molecular definition of tumor antigens, costimulatory signals, and the possibility to genetically engineer tumor cells as well as simple protocols for efficient isolation and preparation of dendritic cells (DC) renew the interest in tumor immunotherapy... Rockville, MD SHRINAVASSAN SHRIMDKANDADA • Genetic Therapy Program of the Yale Cancer Center and the Medical Oncology Section, Department of Internal Medicine, Yale University School of Medicine, New Haven, CT KYONGHEE KAY SON • Department of Pharmaceutics, College of Pharmacy, Rutgers, The State University of New Jersey, Piscataway, NJ ULRIKE STEIN • Max-Delbrück-Center of Molecular Medicine, Robert-Rössle... because of genetic defects, cannot survive in conventional animal facilities and should be maintained in conditions aimed to prevent adventitious infections Handling requires knowledge of the proper methods for avoiding injury to the handler and to the animal Pain and distress should be avoided as far as possible, anesthesia should be used for all surgical procedures, and euthanasia by acceptable methods . Methods and Protocols Methods and Protocols Gene Therapy of Cancer M E T H O D S I N M O L E C U L A R M E D I C I N E TM Humana Press Edited by Wolfgang Walther Ulrike Stein Gene Therapy of. Heller, and Richard Gilbert, 2000 36. Septic Shock Methods and Protocols, edited by Thomas J. Evans, 2000 35. Gene Therapy of Cancer: Methods and Protocols, edited by Wolfgang Walther and Ulrike. treat- ment of particular malignancies. Gene Therapy of Cancer: Methods and Protocols does not provide com- prehensive reviews of all methodologies currently used for gene therapy of cancer. Rather