Gene Therapy of Cancer: Methods and Protocols

634 Pages·2000·15.064 MB·English

by  Mario P. Colombo, Monica Rodolfo (auth.), Wolfgang Walther, Ulrike Stein (eds.)

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 ETM
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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 ETM
John M. Walker, SERIES EDITOR
40. Diagnostic and Therapeutic 28. Cytotoxic Drug Resistance
Antibodies,edited by Andrew J. T. Mechanisms, edited by Robert Brown
George and Catherine E. Urch, 2000 and Uta Böger-Brown, 1999
39. Ovarian Cancer: Methods and
27. Clinical Applications of Capillary
Protocols, edited by John M. S.
Electrophoresis, edited by Stephen M.
Bartlett, 2000
Palfrey, 1999
38. Aging Methods and Protocols, edited
26. Quantitative PCR Protocols, edited
byYvonne A. Barnett and Christopher
byBernd Kochanowski and Udo
P. Barnett, 2000
Reischl, 1999
37. Electrically Mediated Delivery of
25. Drug Targeting, edited by G. E.
Molecules to Cells, edited by Mark J.
Francis and Cristina Delgado, 1999
Jaroszeski, Richard Heller, and
Richard Gilbert, 2000 24. Antiviral Methods and Protocols,
edited by Derek Kinchington
36. Septic Shock Methods and Protocols,
and Raymond F. Schinazi, 1999
edited by Thomas J. Evans, 2000
23. Peptidomimetics Protocols, edited by
35. Gene Therapy of Cancer: Methods
Wieslaw M. Kazmierski, 1999
and Protocols, edited by Wolfgang
Walther and Ulrike Stein, 2000 22. Neurodegeneration Methods and
Protocols, edited by Jean Harry
34. Rotavirus Methods and Protocols,
and Hugh A. Tilson, 1999
edited by James Gray and Ulrich
Desselberger, 2000 21. Adenovirus Methods and Protocols,
edited by William S. M. Wold, 1998
33. Cytomegalovirus Protocols, edited by
John Sinclair, 2000 20. Sexually Transmitted Diseases:
Methods and Protocols, edited by
32. Alzheimer’s Disease: Methods and
Rosanna Peeling and P. Frederick
Protocols,edited by Nigel M. Hooper, 1999
Sparling, 1999
31. Hemostasis and Thrombosis
19. Hepatitis C Protocols, edited by
Protocols:Methods in Molecular
Johnson Yiu-Nam Lau, 1998
Medicine,edited by David J. Perry
and K. John Pasi, 1999 18. Tissue Engineering, edited by
Jeffrey R. Morgan and Martin L.
30. Vascular Disease: Molecular Biology
Yarmush, 1999
and Gene Therapy Protocols, edited
byAndrew H. Baker, 1999 17. HIV Protocols, edited by Nelson
Michael and Jerome H. Kim, 1999
29. DNA Vaccines: Methods and
Protocols,edited by Douglas B. 16. Clinical Applications of PCR, edited
Lowrie and Robert Whalen, 1999 byY. M. Dennis Lo, 1998
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 ETM
Gene Therapy
of Cancer
Methods and Protocols
Edited by
Wolfgang Walther
Max-Delbrück-Center for Molecular Medicine, Berlin, Germany
and
Ulrike Stein
Max-Delbrück-Center for Molecular Medicine, Berlin, Germany
Humana Press Totowa, New Jersey
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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
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
inboth 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 Protocolsdoes 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,
v
vi Preface
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 Protocolsshould 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.
Wolfgang Walther
Ulrike Stein
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
3 Cancer Gene Therapy with Heat Shock Protein-65 Gene
Katalin V. Lukacs and Artit Nakakes.................................................49
4 Recombinant 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
6 Construction 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
viii Contents
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-erbB2 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
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