Principles of Cancer Biotherapy 5th Edition Principles of Cancer Biotherapy 5th Edition Edited by Robert K. Oldham Hematology-Oncology Associates of Southeast Missouri Hospital Southeast Medical Plaza Cape Girardeau, MO USA Robert O. Dillman Hoag Cancer Center Newport Beach, CA USA University of California Irvine, CA USA Robert K. Oldham Robert O. Dillman Hematology-Oncology Associates Hoag Cancer Center of Southeast Missouri Hospital Newport Beach, CA Southeast Medical Plaza USA Cape Girardeau, MO University of California USA Irvine, CA USA ISBN 978-90-481-2277-6 e-ISBN 978-90-481-2289-9 DOI 10.1007/978-90-481-229-9 Springer Dordrecht Heidelberg London New York Library of Congress Control Number: 2009929387 First edition published 1987 by Raven Press Second edition published 1993 by Williams & Wilkins Third and Fourth edition published 1998 Kluwer Academic Publishers © Springer Science+Business Media B.V. 2009 No part of this work may be reproduced, stored in a retrieval system, or transmitted in any form or by any means, electronic, mechanical, photocopying, microfi lming, recording or otherwise, without written permission from the Publisher, with the exception of any material sup- plied specifi cally for the purpose of being entered and executed on a computer system, for exclusive use by the purchaser of the work. Printed on acid-free paper Springer is part of Springer Science+Business Media (www.springer.com) Preface The idea for the fi rst edition of Principles of Cancer those mediated by their active sites. There is increasing Biotherapy was formulated in the early 1980s. As the evidence that drug development will focus on the inter- founding director of the Biological Response Modifi ers action between the smaller active regions of these large Program for the National Cancer Institute from 1980- biological molecules and their receptors. This opens up 1984, one of us (rko) envisioned a textbook that would a broad fi eld of molecular design for extending and embody the principles of the then fl edgling fourth modal- improving the therapeutic activities of natural biological ity of cancer treatment - biotherapy. Contributing authors molecules. This has recently been extended to small were solicited in 1985, and the fi rst edition came off the molecules interacting with DNA/RNA (anti-sense), with presses in 1987. Principles represented the fi rst compre- enzymes such as tyrosine kinase and with growth and hensive textbook on the use of cancer biotherapy and vascular factor receptors. The last decade has been summarized the work done in this fi eld through 1986. extraordinarily productive in the development of new The second edition of Principles was published in anticancer drugs through chemical and biological manip- 1991 about the time biotherapy was more broadly recog- ulation of these natural molecules. Thus, the body itself nized as the fourth major cancer treatment modality. has become the “medicine cabinet” of the future. Subsequent textbooks by DeVita, Hellman and Rosenberg In the 1990s and beyond into this millennium, medi- [1] in 1991, Mitchell [2] in 1993 and Rosenberg [3] in cine will face extraordinary demands. While technol- 2000 validated the importance of this modality in ogy brings us tremendous opportunity, it also highlights cancer care. problems in our medical care system. Most new tech- This third edition was published in 1998 and con- nology is expensive and, as it comes from the laboratory fi rmed the tremendous progress that had been made in to the clinic, is by its very nature untried and unproven. the previous fi ve years using biologicals in cancer treat- Our medical care system involves a private and govern- ment. It was generally agreed that biopharmaceuticals ment insurance reimbursement system that favors pay- were producing major opportunities for new cancer ing for marginally effective medical care of the past therapies. Cancer biotherapy was emerging as a more rather than innovative medical treatments of the future. specifi c and targeted form of systemic cancer treatment. Such a system is inhibitory to the development of effec- Cancer growth control was also becoming an effective tive new anti-cancer medicines. method of treatment complementing cancer destruction To more rapidly and effi ciently exploit the opportuni- as mechanisms of cancer treatment and “cure”. ties in cancer biotherapy in the future, patients, employ- The fourth edition of Principles was published in ers, insurers, universities, and government must come 2003. It was apparent that biotherapy and the use of bio- together and redefi ne the system of reimbursement to pharmaceuticals had not only become recognized as the maximize the patient’s opportunity for access to new fourth modality of cancer treatment, it was clear that and potentially effective cancer therapies. To simply biopharmaceuticals had become the dominant form of reimburse for old ineffective or marginally effective new cancer therapeutics which in the future will replace treatment is not the answer. Provisions must be made to less selective, more toxic forms of therapy. fund clinical research and afford these new approaches For years, the chemical manipulation of small mole- broader use at the bedside. We must develop methods to cules has been pursued in drug development. We now allow our patients access to the opportunities of the have all the tools for the biological manipulation of natu- future, while maintaining solid support for effective ral substances for therapeutic use. In fact, as we better therapies of the past. No longer is it acceptable to pay understand the interaction between biological molecules only for medical care that utilizes old technology, such and their receptors, it is clear that biological manipula- as chemotherapy, that is approved but only marginally tion and chemical manipulation are coming together to effective. Across the broad spectrum of human malig- bring molecular medicine to the bedside. Many biologi- nancies, most chemotherapeutic drugs are toxic and of cal molecules are large and have functions other than limited medical value. We must support clinical research v vi Preface in its efforts to bring newer methods of cancer treatment These chapters illustrate some of these new methods of to the clinic, methods that are less toxic and more thinking and illustrate new strategies for the treatment effective. and control of cancer. It is always diffi cult to move from We believe cancer biotherapy will ultimately replace past dogmas to future opportunities, but this fi fth edi- much of what we utilize today in cancer treatment. In tion of Principles of Cancer Biotherapy illustrates why it light of this view, we want to thank all the authors for is so important for researchers, regulators and clinicians their dedication to purpose in writing this fi fth edition of to explore and apply these new opportunities in cancer Principles. This book summarizes an evolving science biotherapy to the benefi t of our patients. and a rapidly changing medical practice. As we progress into the millennium, it now becomes possible to envision Robert K. Oldham, M.D. Robert O. Dillman MD a much more diversifi ed system of cancer research and treatment that will afford greater opportunities for our patients. As indicated in some of the chapters in Principles, References there is increasing evidence that our historical “kill and cure” outlook in cancer treatment is in need of modifi ca- 1. DeVita VT Jr, Hellman S, Rosenberg SA. Biologic Therapy of Cancer. J.B. Lippincott, 1991. tion. Some forms of cancer biotherapy use the strategy 2. Mitchell MS. Biological Approaches to Cancer Treatment. of tumor growth stabilization and control through con- McGraw-Hill, 1993. tinued biological therapy over a longer period of time, 3. Rosenberg SA. Principles and Practices of the Biologic Therapy of akin to the use of insulin in the treatment of diabetes. Cancer. J.B. Lippincott, 2000. Contents Preface v Robert K. Oldham Contributors ix 1. Cancer biotherapy: general principles 1 Robert K. Oldham 2. The pathogenesis of cancer metastasis: relevance to therapy 17 Sun-Jin Kim, Cheryl Hunt Baker, Yasuhiko Kitadai, Toru Nakamura, Toshio Kuwai, Takamitsu Sasaki, Robert Langley, and Isaiah J. Fidler 3. Developmental therapeutics and the design of clinical trials 41 Robert K. Oldham 4. Recombinant proteins and genomics in cancer therapy 53 Kapil Mehta, Bulent Ozpolat, Kishorchandra Gohil, and Bharat B. Aggarwal 5. Current concepts in immunology 85 Robert K. Oldham 6. Therapeutic approaches to cancer-associated immune suppression 101 Robert K. Oldham 7. Cancer vaccines 147 Kenneth A. Foon and Malek M. Safa 8. Cytokines 155 Walter M. Lewko and Robert K. Oldham 9. Interferons: therapy for cancer 277 David Goldstein, Robert Jones, Richard V. Smalley, and Ernest C. Borden 10. Monoclonal antibody therapy 303 Robert O. Dillman 11. Immunotoxins 407 Arthur E. Frankel, Jung-Hee Woo, and David M. Neville 12. Drug Immunoconjugates 451 Malek Safa, Kenneth A. Foon, and Robert K. Oldham 13. Targeted radionuclide therapy of cancer 463 John M. Pagel, Otto C. Boerman, Hazel B. Breitz, and Ruby Meredith vii viii Contents 14. Stem cell/bone marrow transplantation as biotherapy 497 Robert K. Oldham 15. Cellular immunotherapy (CI), where have we been and where are we going? 505 John R. Yannelli 16. Growth and differentiation factors as cancer therapeutics 527 Kapil Mehta, Robert K. Oldham, and Bulent Ozpolat 17. Granulocyte colony-stimulating factor: biology and clinical potential 569 MaryAnn Foote and George Morstyn 18. Granulocyte-macrophage colony-stimulating factor 581 Maryann Foote and George Morstyn 19. Cancer gene therapy 589 Donald J. Buchsbaum, C. Ryan Miller, Lacey R. Mcnally, and Sergey A. Kaliberov 20. Regulatory process for approval of biologicals for cancer therapy 613 Antonio J. Grillo-López 21.1. Cancer biotherapy: 2009 disease-related activity 631 Robert K. Oldham and Robert O. Dillman 21.2. Biological therapy of melanoma 633 Robert K. Oldham 21.3. Biological therapy of genitourinary cancer 645 Robert K. Oldham 21.4. Biological therapy of colon cancer 659 Robert O. Dillman 21.5. Biological therapy of breast cancer 669 Robert O. Dillman 21.6. Biological therapy of lung cancer 679 Robert O. Dillman 21.7. Biological therapy of B and T cell lymphoproliferative disorders 693 Robert O. Dillman 21.8. Biological therapy of multiple myeloma 711 Robert K. Oldham 21.9. Biological therapy of squamous cell cancers of the head and neck 713 Robert O. Dillman 21.10. Biological therapy of glioblastoma 723 Robert O. Dillman 22. Speculations for 2009 and beyond 733 Robert K. Oldham Index 739 Contributors Robert K. Oldham Toshio Kuwai Hematology-Oncology Associates of Southeast Department of Cancer Biology Missouri Hospital M. D. Anderson Cancer Center Southeast Medical Plaza The University of Texas Cape Girardeau, MO Houston, TX USA USA Robert O. Dillman Takamitsu Sasaki Hoag Cancer Center Department of Cancer Biology Newport Beach, CA M. D. Anderson Cancer Center USA The University of Texas University of California Houston, TX Irvine, CA USA USA Robert Langley Sun-Jin Kim Department of Cancer Biology Department of Cancer Biology M. D. Anderson Cancer Center M. D. Anderson Cancer Center The University of Texas The University of Texas Houston, TX Houston, TX USA USA Isaiah J. Fidler Cheryl Hunt Baker Department of Cancer Biology Department of Cancer Biology M. D. Anderson Cancer Center M. D. Anderson Cancer Center The University of Texas The University of Texas Houston, TX Houston, TX USA USA Kapil Mehta Yasuhiko Kitadai Department of Experimental Therapeutics Department of Cancer Biology M. D. Anderson Cancer Center M. D. Anderson Cancer Center The University of Texas The University of Texas Houston, TX Houston, TX USA USA Bulent Ozpolat Toru Nakamura Department of Experimental Therapeutics Department of Cancer Biology M. D. Anderson Cancer Center M. D. Anderson Cancer Center The University of Texas The University of Texas Houston, TX Houston, TX USA USA ix x Contributors Kishorchandra Gohil Jung-Hee Woo Department of Internal Medicine Cancer Research Institute of Scott &White The University of California Temple, TX Davis, CA USA USA David M. Neville Bharat B. Aggarwal Angimmune LLC, Department of Experimental Therapeutics Bethesda, MD M. D. Anderson Cancer Center USA The University of Texas Houston, TX John M. Pagel USA The Fred Hutchinson Cancer Research Center Kenneth A. Foon Seattle, WA Department of Hematological Malignancies USA Nevada Cancer Institute Las Vegas, NV Otto C. Boerman USA University Medical Center Nijmegen Malek M. Safa The Netherlands Division of Hematology / Oncology University of Cincinnati Hazel B. Breitz Cincinnati, OH Poniard Pharmaceuticals, Inc. USA Seattle, WA USA Walter M. Lewko Cancer Cellular Therapeutics, Inc. Ruby F. Meredith Cape Girardeau, MO Department of Radiation Oncology USA University of Alabama Birmingham Birmingham, Alabama David Goldstein USA Department of Medical Oncology Prince of Wales Hospital John R. Yannelli Randwick, Sydney Department of Microbiology, Australia Immunology and Molecular Genetics Markey Cancer Center Robert Jones University of Kentucky, Centre for Oncology and Applied Pharmacology School of Medicine CRUK Beatson Laboratories Lexington, Kentucky Glasgow, Scotland USA UK MaryAnn Foote Richard V. Smalley (deceased) Thousand Oaks, CA Synertron Inc. USA Madison, WI USA Dr George Morstyn Department of Microbiology Arthur E. Frankel Monash University Cancer Research Institute of Scott &White Victoria Temple, TX Australia USA