Biopharmaceutical Drug Design and Development PDF
Preview Biopharmaceutical Drug Design and Development
Biopharmaceutical Drug Design and Development 8 iopharmaceutical Drug Design and Development Edited by Susanna Wu-Pong Virginia Commonwealth University, Richmond, VA and Yongyut Rojanasakul West Virginia University, Morgantown, WV Foreword by Joseph R. Robinson Springer Science+Business Media, LLC © 1999 Springer Science+Business Media New York Originally published by Humana Press Inc. in 1999 Softcover reprint of the hardcover 1s t edition 1999 AII rights reserved. No part ofthis book may be reproduced, stored in a retrieval system, or transmitted in any form or by any means, electronic, mechanical, photocopying, microfilming, recording, or other wise without written permission from the Publisher. AII 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. ~ ANSI Z39 .48-1984 (American Standards Institute) Permanence of Paper for Printed Library Materials. Source for cover illustration: Methods in Molecular Biology, Volume 62, Recombinant Gene Expression Protocols, edited by Rocky S. Tuan: Fig. 3 from Chapter 20, "Expression ofExogenous Genes inXenopus Oocytes, Eggs, and Embryos," by Koichiro Shiokawa, Chie Koga, Yazuru lto, and Mikihito Shibata. Cover design by Patricia F. Cleary. 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Reporting Service is: [0-89603-691-X/99 $10.00 + $00.25]. 10 9 8 7 6 5 4 3 2 1 Library of Congress Cataloging in Publication Data Main entry under title: Biopharmaceutical drug design and development/edited by Susanna Wu-Pong and Y ongyut Rojanasakul. p. cm.- Includes index. ISBN 978-1-4757-4644-0 ISBN 978-1-59259-705-5 (eBook) DOI 10.1007/978-1-59259-705-5 \. Pharmaceutical biotechnology. 2. Gene therapy. 3. Biotechnology. 4. Molecular biology. 1. Wu-Pong, Susanna. II. Rojanasakul, Yongyut. [DNLM: 1. Biotechnology-methods. 2. Molecular Biology. 3. Genetic Engineering. QH 453 M7186 1999] RS380M65 1999 d615.07'.19-dc21 DNLM/DLC for Library of Congress 99-53441 CIP Foreword New discoveries in biology are occurring at an incredible rate, and with these discoveries arise nearly unimaginable opportunities in every area of human existence. Imagine the excitement surround ing the "penicillin project" and the subsequent rapid development of anti-infective agents that took place in the 1940s and 1950s. Fast for ward to the world today and our ability to treat life-threatening infec tions. This is but one small piece in the present kaleidoscope of new therapeutic agents. In fact, the world of science, biology, and medi cine is changing so quickly that it is difficult for scientists and medi cal practitioners to stay abreast of their fields and confidently anticipate that their education and training will sustain them over a three- to four-decade career without considerable continuing educa tion and training. For the pharmaceutical scientist responsible for the discovery and development of therapeutic agents based on advances in biotechnology, it is imperative to quickly come up to speed and stay at the forefront of developments, which is no easy task for those not specifically trained in this area. Biopharmaceutical Drug Design and Development, edited by Susanna Wu-Pong and Yongyut Rojanasakul, cuts a potentially wide swath in terms of its intended audience. It clearly is a primer for those not trained in the area, or for those who wish to be brought into the mainstream of drug discovery and development in the world of bio technology. It is also a useful teaching text for such medical special ties as pharmacy, nursing, and biotechnology. Biopharmaceutical Drug Design and Development is an emi nently readable book that fills a current void by bridging the highly specialized world where new medicines are emerging from biotech nology, as well as the more traditional small-molecule drug world. The issues and concems associated with gaining approval, manufac- V vi Foreword turing, and selling biotechnology products are, in many respects, con siderably different from those encountered with their small-molecule counterparts. The book also makes apparent the great difficulties en countered in trying to target these molecules both spatially and tem porally in the human body. All in all, Biopharmaceutical Drug Design and Development gives a wonderful short history of the background in biotechnology required to understand the field, as well as a view of the issues and problems in the world of biotechnology product development. Joseph R. Robinson, PHD School ofP harmacy University of Wisconsin-Madison Preface In the four decades since the historical discovezy of the structure of DNA by Watson and Crick, a revolution in biomedical technology has oc curred. Previous to this momentous event, the use oftherapeutic quantities of human proteins, the creation of novel protein and nucleic acid drugs, and the delivezy oftherapeutic genes were only science fiction fantasies. Today, such once-incredible technologies are either used daily in the treatment of dis eases, including diabetes or heart attacks, or in developments slated for FDA approval as early as the next decade. The technological hasis underlying these remarkable advances is biotechnology, i.e., the use of cells, enzymes, DNA, or other biologica! materials to produce a commercially useful product. The rapid expansion of biotechnology since the 1950s has resulted in the development of an array of technologies and methodologies that are ex trernely sensitive, powerful, and accurate. On one hand, the explosion in the field ofbiotechnology has produced 17 new, FDA-approved recombinant or synthetic protein drugs since 1986, with hundreds more currently in clinical trials. On the other hand, this explosion has resulted in a field whose technol ogy can only remotely be understood by the population at large, and only moderately conceptualized by even those with college or graduate degrees in science. Therefore, the goal ofthis book is to provide students and profession als in health care disciplines with a comprehensive account ofboth the neces sary background information and ali the recent advances in medical biotechnology needed to further the readers' awareness ofthis dynamic field. Biopharmaceutical Drug Design and Development opens with an in troduction to the biotechnology industry and a thorough review of the bio chemical and biologica! principles underlying the specific methods that comprise biotechnology. The structure ofDNA, RNA, and pro teins are reviewed, in addition to the basic concepts of gene structure and expression, cell membrane biology, and immunology. These topics are also discussed in detail throughout the book to the degree that they are relevant to understanding the principles and recent advances in biotechnology. vii viii Preface Next, the basic tools used in biotechnology are reviewed in detail, in cluding the scientific principles, methodology, uses, and limitations of each method. These methods include recombinantDNAtechnology, the polymerase chain reaction, monoclonal antibodies, and transgenic and gene transfer tech nologies. Other relevant analytical techniques that apply to nucleic acid or protein analysis are also discussed in this section. In addition, Biopharmaceutical Drug Design and Development pro vides an in-depth discussion of the application ofbiotechnology to medicine and phannacy. The unique nature ofthe biotechnology-derived products (lruge, hydrophilic, unstable nucleic acid or protein molecules; vaccines; cells; transgenic plants or animals) has resulted in both new treatment opportunities and a need for advances in several disciplines, including nucleic acid and protein chemistry, drug design, the biochemical hasis of diseases, drug deliv ery, fonnulation, scaleup, manufacturing, analysis, and quality control. The drug approval process and the clinical applications oft hese unique products are also addressed. The FDA objectives and requirements regarding biologica! product testing in preclinical and clinical trials, as well as postapproval considerations, are reviewed. Finally, for each approved and investigational therapy, the rationale, indications for use, and special storage and usage requirements are discussed in light of the unique nature of these products. Through this comprehensive, in-depth review and analysis ofbiotech nology, we expect that our readers will gain a much deeper appreciation for this remarkable field. Whether such an appreciation and understanding is used to enhance one's research or one's clinical practice, or simply used to raise awareness as a consumer ofbiotechnology-derived products, the phe nomenal nature and enonnous potential ofthis technology will become clear to the reader. Similarly, the hurdles that bar full realization of this technology will also become apparent. Biopharmaceutical Drog Design and Develop ment is intended to bring us closer to elucidating, prioritizing, and overcom ing these hurdles as scientists, clinicians, and consumers in the health care industry. Susanna Wu-Pong Yongyut Rojanasakul Contents Foreword ....................................................................................................... v Preface ........................................................................................................ vii Contributors ................................................................................................ xi CH. 1. An Overview of Biotechnology Susanna Wu-Pong ............................................................ 1 CH. 2. Basic Elements of Molecular Biology Yongyut Rojanasakul ..................................................... 21 CH. 3. Tools for Molecular Analysis Yongyut Rojanasakul and Sujatha Dokka ................... 3 7 CH. 4. Treatment Options Susanna Wu-Pong .......................................................... 51 CH. 5. Viral Vectors for Gene Therapy Kristoffer Valerie ............................................................ 69 CH. 6. Nonviral Gene Delivery Frank L. Sorgi and Hans Schreier .............................. J07 CH. 7. Oligonucleotides and Ribozymes Melanie Lysik and Susanna Wu-Pong ....................... 143 CH. 8. Design and Engineering of Proteins as Therapeutic Agents David R. Corey ............................................................. 187 CH. 9. Challenges and Issues in the Development of F ormulations of Protein Pharmaceuticals Steven J. Shire ............................................................... 205 CH. 1O . Protein Drug Delivery Udaya B. Kompella ...................................................... 239 CH. 11. Protein Drug Manufacturing Denny Lanfear, Randy Hassler, Karen Sitney, Craig Curless, Sugu Patro, Larry Tsai, and John Ogez ............................................................. 275 ix Contents X CH. 12. Antibodies Jerry T. Thompson and Kathleen D. Rigaut. .............. 329 CH. 13. Immunotherapy Robert Tam ................................................................... 3 4 9 CH. 14. Product Deve1opment and New Drug Approva1 Victoria G. Hale ........................................................... 3 75 CH. 15. Therapeutic U se of Recombinant Proteins Ronald M. Bukowski .................................................... 3 9 3 Index ................................................................................................. 429
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