Suicide Gene Therapy: Methods and Reviews

556 Pages·2004·4.299 MB·English

by  Ion Niculescu-Duvaz, Caroline J. Springer (auth.), Caroline J. Springer (eds.)

Suicide Gene Therapy
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™
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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™
Suicide Gene Therapy
Methods and Reviews
Edited by
Caroline J. Springer
Cancer Research UK Centre for Cancer Therapeutics
at the Institute of Cancer Research, Sutton, Surrey, UK
© 2004 Humana Press Inc.
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ISSN: 1543-1894
E-ISBN: 1-59259-429-8
Printed in the United States of America. 10 9 8 7 6 5 4 3 2 1
Library of Congress Cataloging-in-Publication Data
Suicide gene therapy : methods and reviews / edited by Caroline J. Springer.
p. ; cm. -- (Methods in molecular medicine, ISSN 1543-1894)
Includes bibliographical references and index.
ISBN 0-89603-971-4 (alk. paper)
1. Cancer--Gene therapy. [DNLM: 1. Neoplasms--therapy. 2. Gene Therapy--methods. QZ 266 S948
2004] I. Springer, Caroline J. II. Series.
RC271.G45S85 2004
616.99'4042--dc21
2003014330
Preface
Gene therapy has expanded rapidly over the last decade. The number of clinical trials
reported by 2001 included 532 protocols and 3436 patients. Phase I trials predominate with
359 trials of 1774 patients versus Phase II (57 trials with 507 patients) and Phase III (3
trials of 251 patients). The disease overwhelmingly targeted by gene therapy is cancer:
involving 331 trials with 2361 patients. Despite the somewhat disappointing results of
clinical trials to date, gene therapy offers tremendous promise for the future of cancer
therapy.
The area of gene therapy is vast, and both malignant and nonmalignant cells can be
targeted. Suicide Gene Therapy: Methods and Reviewscovers gene therapy that targets
malignant cells in a treatment that has become known as “suicide gene therapy.” Basically,
this approach uses the transduction of cancer cells with a gene for a foreign enzyme that,
when expressed, is able to activate a nontoxic prodrug into a highly cytotoxic drug able to
kill the cancer cell population. This is a major area in cancer gene therapy—in 2001 this
technique was represented by 52 clinical protocols with a total of 567 patients. Additional
trials used multiple gene therapy protocols that also involved suicide gene therapy (83 with
497 patients), indicating that the interest in this area is considerable.
Suicide Gene Therapy: Methods and Reviewsaims to cover comprehensively, both in
theoretical and practical terms, the rapidly evolving area of suicide gene therapy for cancer.
A multidisciplinary approach to this topic is presented here, focusing always on the state-
of-the-art—from basic research to clinical practice. Suicide Gene Therapy: Methods and
Reviewsis the first book on this topic to integrate theory and practice. The reader will find
an extensive review of the theoretical background of suicide gene therapy, covering all
major aspects, including the design and use of vectors for gene transduction, various
enzyme and prodrug systems, the mechanistic analysis of the bystander effect, the design
and synthesis of prodrugs, immunological implications, and the clinical impact. The reader
will find also the basic methodology used to explore, study, and expand this area.
Finally, I’d like to emphasize that in my opinion suicide gene therapy represents an area
where outstanding progress has been made and that provides new hope for the treatment of
cancer, especially cancer of solid tumors.
Caroline J. Springer
Acknowledgments
My sincere thanks to the authors: you were all a pleasure to work with. Also thanks
to Ms. Regan Barfoot, who worked tirelessly to bring the book to fruition. This book
would not have been possible without grants from Cancer Research UK (SP2330/0201
and SP2330/0102).
v
Contents
Preface ..............................................................................................................v
Contributors .....................................................................................................xi
1 Introduction to the Background, Principles,
and State of the Art in Suicide Gene Therapy
Ion Niculescu-Duvaz and Caroline J. Springer....................................1
2 Introduction to Vectors for Suicide Gene Therapy
Caroline J. Springer.............................................................................29
3 Construction of VNP20009: A Novel, Genetically Stable
Antibiotic-Sensitive Strain of Tumor-Targeting Salmonella
for Parenteral Administration in Humans
Kenneth Brooks Low, Martina Ittensohn, Xiang Luo, Li-Mou Zheng,
Ivan King, John M. Pawelek, and David Bermudes........................47
4 Nonreplicating DNA Viral Vectors for Suicide Gene Therapy:
The Adenoviral Vectors
Masato Yamamoto and David T. Curiel.............................................61
5 Replication-Selective Oncolytic Adenoviruses
Gunnel Halldén, Stephen H. Thorne, Jingping Yang,
and David H. Kirn...........................................................................71
6 Retroviral Vectors for Suicide Gene Therapy
Colin Porter.........................................................................................91
7 Nonviral Liposomes
Andrew D. Miller..............................................................................107
8 Peptide- and Polymer-Based Gene Delivery Vehicles
Richard Brokx and Jean Gariépy......................................................139
9 Design of Prodrugs for Suicide Gene Therapy
Dan Niculescu-Duvaz, Ion Niculescu-Duvaz,
and Caroline J. Springer...............................................................161
10 Cytochrome P450-Based Gene Therapies for Cancer
E. Antonio Chiocca and David J. Waxman.......................................203
11 Tumor Sensitization to Purine Analogs by E. coli PNP
Kimberly V. Curlee, William B. Parker, and Eric J. Sorscher...........223
12 Enzyme–Prodrug Systems: Carboxylesterase/CPT-11
Mary K. Danks and Philip M. Potter.................................................247
vii
viii Contents
13 Enzyme–Prodrug Systems:
Thymidine Phosphorylase/5'-Deoxy-5-Fluorouridine
Alexandre Evrard, Joseph Ciccolini, Pierre Cuq,
and Jean-Paul Cano......................................................................263
14 Methods to Improve Efficacy in Suicide Gene Therapy Approaches:
Targeting Prodrug-Activating Enzymes Carboxypeptidase G2
and Nitroreductase to Different Subcellular Compartments
Silke Schepelmann, Robert Spooner, Frank Friedlos,
and Richard Marais.......................................................................279
15 Extracellular β-Glucuronidase for Gene-Directed
Enzyme–Prodrug Therapy
Sabine Brüsselbach...........................................................................303
16 Enhancement of Suicide Gene Prodrug Activation
by Random Mutagenesis
Jean-Emmanuel Kurtz and Margaret E. Black...................................331
17 Combination Suicide Gene Therapy
Wolfgang Uckert, Brian Salmons, Christian Beltinger,
Walter H. Günzburg, and Thomas Kammertöns..........................345
18 Immune Response to Suicide Gene Therapy
Shigeki Kuriyama, Hirohisa Tsujinoue, and Hitoshi Yoshiji.............353
19 Targeting Cancer With Gene Therapy Using Hypoxia as a Stimulus
Gabi U. Dachs, Olga Greco, and Gillian M. Tozer..........................371
20 Radiation-Activated Antitumor Vectors
Simon D. Scott and Brian Marples...................................................389
21 In Vitro and In Vivo Models for the Evaluation of GDEPT:
Quantifying Bystander Killing in Cell Cultures and Tumors
William R. Wilson, Susan M. Pullen, Alison Hogg,
Stephen M. Hobbs, Frederik B. Pruijn, and Kevin O. Hicks...........403
22 Suicide Gene Therapy in Liver Tumors
Long R. Jiao, Roman Havlik, Joanna Nicholls,
Steen Lindkaer Jensen, and Nagy A. Habib..................................433
23 Clinical Trials With GDEPT:
Cytosine Deaminase and 5-Fluorocytosine
Nicola L. Brown and Nicholas R. Lemoine.......................................451
24 The Nitroreductase/CB1954 Enzyme–Prodrug System
Nicola K. Green, David J. Kerr, Vivien Mautner,
Peter A. Harris, and Peter F. Searle.............................................459
Contents ix
25 Side Effects of Suicide Gene Therapy
Marjolijn M. van der Eb, Bertie de Leeuw, Alex J. van der Eb,
and Rob C. Hoeben......................................................................479
26 Antibody-Directed Enzyme–Prodrug Therapy
R. Barbara Pedley, Surinder K. Sharma, Robert E. Hawkins,
and Kerry A. Chester....................................................................491
27 Bioreductive Prodrugs for Cancer Therapy
Beatrice Seddon, Lloyd R. Kelland, and Paul Workman..................515
Index............................................................................................................543
Contributors
CHRISTIAN BELTINGER • University Children’s Hospital, Ulm, Germany,
DAVID BERMUDES • Vion Pharmaceuticals, New Haven, CT
MARGARET E. BLACK • Department of Pharmaceutical Sciences, Washington State
University, Pullman, WA
RICHARD BROKX • Department of Medical Biophysics, University of Toronto,
Toronto, Ontario, Canada
NICOLA L. BROWN • Cancer Research UK Molecular Oncology Unit, Imperial
College of Science, Technology and Medicine; Hammersmith Hospital, London,
United Kingdom
SABINE BRÜSSELBACH • Institute of Molecular Biology and Tumour Research (IMT),
Philipps-University Marburg, Marburg, Germany
JEAN-PAUL CANO • Department of Toxicology, Faculty of Pharmacy, University
of Montpellier, Montpellier, France
KERRY A. CHESTER• Cancer Research UK Targeting and Imaging Group,
Department of Oncology, Royal Free and University College Medical School,
London, United Kingdom
E. ANTONIO CHIOCCA • Molecular Neuro-Oncology Laboratory, Neurosurgery
Service, Massachusetts General Hospital; Harvard Medical School, Boston, MA
JOSEPH CICCOLINI • Department of Pharmacokinetics and Toxicokinetics, Faculty
of Pharmacy, University of Aix-Marseille, Marseille, France
PIERRE CUQ • Department of Toxicology, Faculty of Pharmacy, University
of Montpellier, Montpellier, France
DAVID T. CURIEL • Division of Human Gene Therapy, Departments of Medicine,
Pathology and Surgery, and the Gene Therapy Center, University of Alabama
at Birmingham, Birmingham, AL
KIMBERLY V. CURLEE • Department of Human Genetics, University of Alabama
at Birmingham, Birmingham, UK
GABI U. DACHS • Tumour Microcirculation Group, Gray Laboratory Cancer
Research Trust, Mount Vernon Hospital, Northwood, UK
MARY K. DANKS • Department of Molecular Pharmacology, St. Jude Children’s
Research Hospital, Memphis, TN
ALEX J. VANDER EB • Departments of Molecular Cell Biology and of Radiation
Genetics, Leiden University Medical Center, Leiden, The Netherlands
MARJOLIJN M. VANDER EB • Departments of Molecular Cell Biology and of Surgery,
Leiden University Medical Center, Leiden, The Netherlands,
ALEXANDRE EVRARD • Department of Toxicology, Faculty of Pharmacy, University
of Montpellier, Montpellier, France
xi
xii Contributors
FRANK FRIEDLOS • The Cancer Research UK Centre for Cancer Therapeutics, The
Institute of Cancer Research, London, United Kingdom
JEAN GARIÉPY • Department of Medical Biophysics, University of Toronto, Toronto,
Ontario, Canada
OLGA GRECO • Tumour Microcirculation Group, Gray Laboratory Cancer Research
Trust, Mount Vernon Hospital, Northwood, UK
NICOLA K. GREEN • Hybrid Systems Ltd., Littlemore Park, Oxford, United Kingdom
WALTER H. GÜNZBURG • Institute of Virology, University of Veterinary Sciences,
Vienna, Austria
NAGY A. HABIB • Liver Surgery Section, Division of Surgery, Anaesthetics,
and Intensive Care, Faculty of Medicine, Imperial College of Science
Technology and Medicine, London, United Kingdom
GUNNEL HALLDÉN • Viral and Genetic Therapy Programme, Cancer Research UK
and Imperial College School of Medicine, Hammersmith Hospital, London,
United Kingdom
PETER A. HARRIS • Kudos Pharmaceuticals Ltd., Cambridge, United Kingdom
ROMAN HAVLIK • Liver Surgery Section, Division of Surgery, Anaesthetics, and
Intensive Care, Faculty of Medicine, Imperial College of Science Technology
and Medicine, London, United Kingdom
ROBERT E. HAWKINS • Cancer Research UK Department of Medical Oncology,
Christie CRUK Research Centre, Christie Hospital NHS Trust, Manchester,
United Kingdom
KEVIN O. HICKS • Auckland Cancer Society Research Centre, The University
of Auckland, Auckland, New Zealand
STEPHEN M. HOBBS • Institute of Cancer Research, Sutton, Surrey, United Kingdom
ROB C. HOEBEN • Department of Molecular Cell Biology, Leiden University Medical
Center, Leiden, The Netherlands
ALISON HOGG • Auckland Cancer Society Research Centre, The University
of Auckland, Auckland, New Zealand
MARTINA ITTENSOHN • Vion Pharmaceuticals, New Haven, CT
STEEN LINDKAER JENSEN • Liver Surgery Section, Division of Surgery, Anaesthetics,
and Intensive Care, Faculty of Medicine, Imperial College of Science,
Technology, and Medicine, London, United Kingdom
LONG R. JIAO • Liver Surgery Section, Division of Surgery, Anaesthetics, and
Intensive Care, Faculty of Medicine, Imperial College of Science, Technology,
and Medicine, London, United Kingdom
THOMAS KAMMERTÖNS • Max-Delbrück-Center for Molecular Medicine, Berlin,
Germany
LLOYD R. KELLAND • Cancer Research UK Centre for Cancer Therapeutics, Institute
of Cancer Research, Sutton, Surrey, United Kingdom
DAVID J. KERR • Department of Clinical Pharmacology, University of Oxford,
Oxford, United Kingdom
IVAN KING • Vion Pharmaceuticals, New Haven, CT
DAVID H. KIRN • Department of Pharmacology, Oxford University Medical School,
Oxford, United Kingdom