Gene Therapy for Cancer PDF
Preview Gene Therapy for Cancer
G T C ENE HERAPY FOR ANCER C D D D ANCER RUG ISCOVERY AND EVELOPMENT B A. T , S E EVERLY EICHER ERIES DITOR Transforming Growth Factor-(cid:71) in Cancer Combination Cancer Therapy: Modulators Therapy, Volume II: Cancer Treatment and Potentiators, edited by Gary K. and Therapy,edited by Sonia Jakowlew, Schwartz, 2005 2007 Cancer Chemoprevention,Volume 2:Strategies Transforming Growth Factor-(cid:71) in Cancer for Cancer Chemoprevention, edited by Gary Therapy, Volume 1: Basic and Clinical J. Kelloff, Ernest T. Hawk, and Caroline C. Biology, edited by Sonia Jakowlew, 2007 Sigman, 2005 Microtubule Targets in Cancer Therapy, Death Receptors in Cancer Therapy, edited by edited byAntonio T. Fojo, 2007 Wafik S. El-Deiry, 2005 Cytokines in the Genesis and Treatment of Cancer Chemoprevention,Volume 1:Promising Cancer,edited by Michael A. Caligiuri, Cancer Chemopreventive Agents, edited by Michael T. Lotze, and Frances R. Balkwill, Gary J. Kelloff, Ernest T. Hawk, and 2007 Caroline C. Sigman, 2004 Regional Cancer Therapy, edited by Peter M. Proteasome Inhibitors in Cancer Therapy, Schlag and Ulrike Stein, 2007 edited by Julian Adams, 2004 Gene Therapy for Cancer, edited by Kelly K. Nucleic Acid Therapeutics in Cancer, edited Hunt, Stephan A. Vorburger, and Stephen by Alan M. Gewirtz, 2004 G. Swisher, 2007 DNA Repair in Cancer Therapy, edited by Deoxynucleoside Analogs in Cancer Therapy, Lawrence C. Panasci and Moulay A. Alaoui- edited by Godefridus J. Peters, 2006 Jamali, 2004 Cancer Drug Resistance, edited by Beverly A. Hematopoietic Growth Factors in Oncology: Teicher, 2006 Basic Science and Clinical Therapeutics, Histone Deacetylases: Transcriptional Regula- edited by George Morstyn, MaryAnn tion and Other Cellular Functions, edited Foote, and Graham J. Lieschke, 2004 byEric Verdin, 2006 Handbook of Anticancer Pharmacokinetics Immunotherapy of Cancer, edited by Mary and Pharmacodynamics, edited by William L. Disis, 2006 D. Figg and Howard L. McLeod, 2004 Biomarkers in Breast Cancer: Molecular Anticancer Drug Development Guide: Preclinical Diagnostics for Predicting and Monitoring Screening, Clinical Trials, and Approval, Therapeutic Effect, edited by Giampietro Second Edition, edited by Beverly A. Teicher Gasparini and Daniel F. Hayes, 2006 and Paul A. Andrews, 2004 Protein Tyrosine Kinases: From Inhibitors to Handbook of Cancer Vaccines, edited by Useful Drugs, edited by Doriana Fabbro Michael A. Morse, Timothy M. Clay, and and Frank McCormick, 2005 Kim H. Lyerly, 2004 Bone Metastasis: Experimental and Clinical Drug Delivery Systems in Cancer Therapy, Therapeutics, edited by Gurmit Singh and edited by Dennis M. Brown, 2003 Shafaat A. Rabbani, 2005 Oncogene-Directed Therapies, edited by The Oncogenomics Handbook,edited by Janusz Rak, 2003 William J. LaRochelle and Richard A. Cell Cycle Inhibitors in Cancer Therapy: Shimkets, 2005 Current Strategies, edited by Antonio Camptothecins in Cancer Therapy,edited by Giordano and Kenneth J. Soprano, 2003 Thomas G. Burke and Val R. Adams, 2005 Chemoradiation in Cancer Therapy, edited byHak Choy, 2003 G T C ENE HERAPY FOR ANCER Edited by K K. H , ELLY UNT MD The University of Texas M. D. Anderson Cancer Center, Houston, TX S A. V , , TEPHAN ORBURGER MD MCR Inselspital Bern, University of Bern, Bern, Switzerland S G. S , TEPHEN WISHER MD The University of Texas M. D. Anderson Cancer Center, Houston, TX © 2007 Humana Press Inc. 999 Riverview Drive, Suite 208 Totowa, New Jersey 07512 www.humanapress.com 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. All articles, comments, opinions, conclusions, or recommendations are those of the author(s), and do not necessarily reflect the views of the publisher. Due diligence has been taken by the publishers, editors, and authors of this book to assure the accuracy of the information published and to describe generally accepted practices. The contributors herein have carefully checked to ensure that the drug selections and dosages set forth in this text are accurate and in accord with the standards accepted at the time of publication. Notwithstanding, as new research, changes in government regulations, and knowledge from clinical experience relating to drug therapy and drug reactions constantly occurs, the reader is advised to check the product information provided by the manufacturer of each drug for any change in dosages or for additional warnings and contraindications. This is of utmost importance when the recommended drug herein is a new or infrequently used drug. It is the responsibility of the treating physician to determine dosages and treatment strategies for individual patients. Further it is the responsibility of the health care provider to ascertain the Food and Drug Administration status of each drug or device used in their clinical practice. The publisher, editors, and authors are not responsible for errors or omissions or for any consequences from the application of the information presented in this book and make no warranty, express or implied, with respect to the contents in this publication. Cover design by Carlotta Craig Cover illustration: Figure 3 from Chapter 17, “Nonviral Genetic Vaccines for Cancer,” by Steven R. Little, Daniel G. Anderson, and Robert Langer This publication is printed on acid-free paper. 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Printed in the United States of America. 10 9 8 7 6 5 4 3 2 1 E-ISBN 1-59745-222-X 13-digit ISBN 978-1-58829-472-2 Library of Congress Cataloging-in-Publication Data Gene therapy for cancer / edited by Kelly K. Hunt, Stephan A. Vorburger, Stephen G. Swisher. p. ; cm. — (Cancer drug discovery and development) Includes bibliographical references and index. ISBN 1-58829-472-2 (alk. paper) 1. Cancer—Gene therapy. I. Hunt, Kelly K. II. Vorburger, Stephan A. III. Swisher, Stephen G. IV. Series. [DNLM: 1. Neoplasms—genetics. 2. Neoplasms—therapy. 3. Gene Therapy—methods. QZ 266 G3253 2007] RC271.G45G463 2007 616.99’40694—dc22 2006012653 P REFACE The possibility of treating cancer, a disease defined by genetic defects, by introducing genes targeting these very alterations has led to an immense interest in gene therapy for cancer. Although incremental successes have been realized, enthusiasm for gene therapy has declined due to an increasing number of obstacles. These obstacles include vector systems that do not reach systemic metastases, therapeutic genes with redundant mecha- nisms allowing for cellular resistance, and toxicities in clinical trials leading to premature closure of these studies. Different tactics to overcome or circumvent these obstacles have catalyzed the development of a wide range of gene therapy approaches. Thus far, almost two-thirds of gene therapy trials have focused on cancer. This reflects the concept that gene therapy approaches for the treatment of cancer do not necessarily require long-term expression of the gene as is necessary for the treatment of primary genetic defects like hemophilia or juvenile diabetes. Unlike the treatment of genetic defects, where expres- sion of the corrected gene needs to be strong, permanent and, sometimes regulated, tactics to treat tumors can be based on temporary and locally limited effects. In addition, cancer cells have different properties than normal cells and this allows for targeting gene therapy to specific cells, a major advantage over current antitumor therapies, which are also toxic to normal cells and tissues. Gene Therapy for Cancercovers the current ideas and technology of gene therapy, as well as the demanding task of bringing it to and applying it in clinical trials. The book is divided into three major parts: (1) Vectors used in gene therapy against cancer, (2) targets and specific approaches for the therapy of cancer, and (3) clinical applications of cancer gene therapy. The delivery of an antitumor gene, a toxic agent, or an immunostimulating drug selectively to tumor cells is one of the most crucial steps in achieving successful cancer gene therapy. We have dedicated a considerable portion of Gene Therapy for Cancerto a description of the various aspects of gene delivery including vehicles (vectors), their characteristics, and production methods. Knowledge of the specific strategies and targets for the treatment of cancer has increased dramatically over the past decade. These range from methods that induce immediate cancer cell death through expression of genes that trigger the cell-death program or by reactivating pathways that render mutated cells susceptible to antitumor agents. Additional methods run the gamut from the correction of underlying defects at molecular levels to activation of the immune system or the tumor microenvironment. Understanding the basic underlying oncogenic changes allows for development of vectors engineered to exploit these gene mutations through selective spread of the vector in tumor cells with the specific changes. Background knowledge, technical details, and preclinical and clinical results are pro- vided by specialists in each of these approaches. Probablymore so than in any other antitumor therapy, bringing gene therapy approaches to the clinic is a difficult task burdened by numerous regulations and limited by scarce funding opportunities. We have asked experts in clinical gene therapy trials to discuss the v vi Preface trials and tribulations of realizing advances in gene therapy at the preclinical level to the benefit of patients with cancer. The readers will gain significant insight into these difficulties and learn how to overcome the obstacles on the way from the laboratory to the bedside. Gene therapy approaches and results that have reached the stage of clinical testing are described by their principal investigators. WithGene Therapy for Cancerwe have sought to provide a comprehensive and in- depth view of currently available techniques for cancer gene therapy, including their limitations and the potential for future advances. This should prove to be a valuable resource for both researchers and clinicians in the field. With this approach we hope to provide an oppor- tunity for clinicians and researchers to communicate their perspectives, allowing for increased collaboration and perhaps more rapid advances in this challenging field. We would like to thank all the specialists who dedicated their valuable time to provide the most important and exciting advances in cancer gene therapy for this book. We are deeply grateful to our families for their continuous support, endless patience, and under- standing. Kelly K. Hunt, MD Stephan A. Vorburger, MD Stephen G. Swisher, MD C ONTENTS Preface...............................................................................................................v Contributors......................................................................................................ix I V ECTORS 1 Adenovector-Mediated Cancer Gene Therapy .......................................3 Bingliang Fang and Jack A. Roth 2 Efficacy, Toxicity, and Immunogenicity of Adenoviral Vectors.........23 Dmitry M. Shayakhmetov 3 Lentiviral and Retroviral Vector Systems ............................................39 Renata Stripecke and Noriyuki Kasahara 4 Vaccinia and Pox-Virus.........................................................................73 Sricharan Chalikonda and David L. Bartlett 5 Herpes Simplex Virus as a Therapy for Cancer ...................................87 Ian J. Mohr 6 Alphavirus Vectors for Gene Therapy Applications..........................109 Kenneth Lundstrom 7 Vesicular Stomatitis Virus and RNA Viruses as Gene Therapy Vectors.................................................................121 Glen N. Barber 8 Parvovirus Vectors ..............................................................................141 Selvarangan Ponnazhagan 9 Nonviral Vector Systems.....................................................................157 Pui-yan Lee and Leaf Huang II G T A ENE HERAPY PPROACHES 10 Oncogenes, Tumor Suppressor Genes, and Apoptosis-Inducing Genes Utilized in Cancer Gene Therapy.........................................173 Lidong Zhang and Bingliang Fang 11 Gene Silencing Therapy Against Cancer............................................185 Chao-Zhong Song 12 Tumor Targeting–Retargeted Adenovirus..........................................197 Masato Yamamoto and David T. Curiel 13 Oncolytic Herpes Simplex for Gene Therapy in Preclinical and Clinical Trials.....................................................213 Richard H. Pin, Maura Reinblatt, Yuman Fong, and William R. Jarnagin vii viii Contents 14 Cytokine Gene Therapy for Genitourinary Cancer ............................223 Terry L. Timme, Tetsuo Fujita, Hongyu Wang, Koji Naruishi, Dov Kadmon, Robert J. Amato, Brian J. Miles, Gustavo Ayala, Thomas M. Wheeler, Bin S. Teh, E. Brian Butler, and Timothy C. Thompson 15 Combination of Gene Therapy with Radiation...................................243 Anupama Munshi and Raymond E. Meyn 16 Gene Transfer for Chemoprotection and Enrichment of Hematopoietic Stem Cells...........................................................257 Justin C. Roth and Stanton L. Gerson 17 Nonviral Genetic Vaccines for Cancer ...............................................281 Steven R. Little, Daniel G. Anderson, and Robert Langer 18 Gene Therapy Targeted at Angiogenesis and Lymphangiogenesis for Cancer Treatment.......................................................................301 Anita T. Tandle and Steven K. Libutti 19 Tumor-Specific Replicating Adenoviruses: (cid:41) 24 for Human Gliomas.................................................................331 Giacomo G. Vecil, Juan Fueyo, and Frederick F. Lang III C A LINICAL PPLICATIONS 20 Problems, Side Effects, and Disappointments in Clinical Cancer Gene Therapy....................................................351 Ta-Chiang Liu and David H. Kirn 21 Trials and Tribulations in Developing Clinical Trials of Gene Therapy: E1A for Breast or Ovarian Cancer....................387 Naoto T. Ueno, Judith K. Wolf, David M. Gershenson, Gabriel N. Hortobagyi, and Mien-Chie Hung 22 Phase I Clinical Experience with Intravenous Administration of PV701, an Oncolytic Virus .........................................................399 Andrew L. Pecora and Robert M. Lorence 23 MDA-7/IL-24 as a Multi-Modality Therapy for Cancer....................413 Sunil Chada, Dora Bocangel, Abujiang Pataer, Abner M. Mhashilkar, Satoshi Inoue, Ryo Miyahara, Jack A. Roth, Elizabeth A. Grimm, Stephen G. Swisher, Kelly K. Hunt, and Rajagopal Ramesh 24 Development of Therapeutic Genes for Breast Cancer Treatment....435 Mien-Chie Hung and Duen-Hwa Yan 25 Steps in a Translational Cancer Gene Therapy Trial..........................447 Urs W. von Holzen and Stephen G. Swisher Index ....................................................................................................461 C ONTRIBUTORS ROBERT J. AMATO • Scott Department of Urology, Baylor College of Medicine, Houston, TX DANIEL G. ANDERSON • Chemical Engineering Department, Massachusetts Institute of Technology, Cambridge, MA GUSTAVO AYALA • Department of Pathology, Baylor College of Medicine, Houston, TX GLEN N. BARBER • Department of Microbiology and Immunology, University of Miami School of Medicine, Miami, FL DAVID L. BARTLETT • Division of Surgical Oncology, University of Pittsburgh, Pittsburgh, PA DORA BOCANGEL • Introgen Therapeutics, Houston, TX E. BRIAN BUTLER • Department of Radiology, Baylor College of Medicine, Houston, TX SUNIL CHADA • Introgen Therapeutics, Houston, TX SRICHARAN CHALIKONDA • Division of Surgical Oncology, University of Pittsburgh, Pittsburgh, PA DAVID T. CURIEL • Gene Therapy Center, University of Alabama, Birmingham, AL BINGLIANG FANG • Department of Thoracic and Cardiovascular Surgery–Research, The University of Texas M.D. Anderson Cancer Center, Houston, TX YUMAN FONG • Department of Surgery, Memorial Sloan-Kettering Cancer Center, New York, NY JUAN FUEYO • Department of Neuro-Oncology/Research, The University of Texas M. D. Anderson Cancer Center, Houston, TX TETSUO FUJITA • Scott Department of Urology, Baylor College of Medicine, Houston, TX DAVID M. GERSHENSON • Department of Gynecologic Oncology, The University of Texas M.D. Anderson Cancer Center, Houston, TX STANTON L. GERSON • Division of Hematology-Oncology, Case Western Reserve University School of Medicine, Cleveland, OH ELIZABETH A. GRIMM • Department of Experimental Therapuetics, The University of Texas M.D. Anderson Cancer Center, Houston, TX GABRIEL N. HORTOBAGYI • Department of Breast Medical Oncology, The University of Texas M.D. Anderson Cancer Center, Houston, TX LEAF HUANG • Center for Pharmacogenetics, Department of Pharmaceutical Science, University of Pittsburgh, Pittsburgh, PA MIEN-CHIE HUNG • Department of Molecular & Cellular Oncology, The University of Texas M.D. Anderson Cancer Center, Houston, TX KELLY K. HUNT • Department of Surgical Oncology, The University of Texas M.D. Anderson Cancer Center, Houston, TX ix x Contributors SATOSHI INOUE • Department of Thoracic and Cardiovascular Surgery, The University of Texas M. D. Anderson Cancer Center, Houston, TX WILLIAM R. JARNAGIN • Hepatobiliary Service, Department of Surgery, Memorial Sloan- Kettering Cancer Center, New York, NY DOV KADMON • Scott Department of Urology, Baylor College of Medicine, Houston, TX NORIYUKI KASAHARA • Department of Medicine, UCLA Geffen School of Medicine, Macdonald Research Laboratories, Los Angeles, CA DAVID H. KIRN • Department of Clinical Pharmacology, University of Oxford Medical School, Oxford, UK FREDERICK F. LANG • Department of Neurosurgery, The University of Texas M. D. Anderson Cancer Center, Houston, TX ROBERT S. LANGER • Chemical & Biomedical Engineering, Massachusetts Institute of Technology, Cambridge, MA PUI-YAN LEE • Center for Pharmacogenetics, Department of Pharmaceutical Science, University of Pittsburgh, Pittsburgh, PA STEVEN K. LIBUTTI • Surgery Branch, National Cancer Institute, Bethesda, MD STEVEN R. LITTLE • Departments of Chemical Engineering and Bioengineering, University of Pittsburgh, Pittsburgh, PA TA-CHIANG LIU • Molecular Neurosurgery Laboratory, Massachusetts General Hospital, Harvard Medical School, Boston, MA ROBERT M. LORENCE • Wellstat Biologics Corporation, Gaithersburg, MD KENNETH LUNDSTROM • BioXtal / Cancer Therapeutic Sciences, Switzerland RAYMOND E. MEYN, JR. • Department of Experimental Radiation Oncology, The University of Texas M. D. Anderson Cancer Center, Houston, TX ABNER M. MHASHILKAR • Introgen Therapeutics, Houston, TX BRIAN J. MILES • Scott Department of Urology, Baylor College of Medicine, Houston, TX RIO MIYAHARA • Department of Thoracic and Cardiovascular Surgery, The University of Texas M. D. Anderson Cancer Center, Houston, TX IAN J. MOHR • Department of Microbiology, New York University School of Medicine, New York, NY ANUPAMA MUNSHI • Department of Experimental Radiation Oncology, The University of Texas M. D. Anderson Cancer Center, Houston, TX KOJI NARUISHI • Scott Department of Urology, Baylor College of Medicine, Houston, TX ABUJIANG PATAER • Department of Thoracic and Cardiovascular Surgery, The University of Texas M.D. Anderson Cancer Center, Houston, TX ANDREW L. PECORA • The Cancer Center, Hackensack University Medical Center, Hackensack, NJ RICHARD H. PIN • Department of Surgery, Memorial Sloan-Kettering Cancer Center, New York, NY SELVARANGAN PONNAZHAGAN • Department of Pathology, University of Alabama at Birmingham, Birmingham, AL
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