Biological Barriers to Protein Delivery PDF
Preview Biological Barriers to Protein Delivery
Biological Barriers to Protein Delivery Pharmaceutical Biotechnology Series Editor: Ronald T. Borchardt The UniversityofKansas Lawrence, Kansas Volume I PROTEIN PHARMACOKINETICS AND METABOLISM Edited by Bobbe L. Ferraiolo, Marjorie A. Mohler, and Carol A. Gloff Volume 2 STABILITY OF PROTEIN PHARMACEUTICALS, Part A: Chemical and Physical Pathways ofProtein Degradation Edited by Tim 1. Ahern and Mark C. Manning Volume 3 STABILITY OF PROTEIN PHARMACEUTICALS, Part B: In Vivo Pathways ofDegradation and Strategies for Protein Stabilization Edited by Tim 1. Ahern and Mark C. Manning Volume 4 BIOLOGICAL BARRIERS TO PROTEIN DELIVERY Edited by Kenneth L. Audus and Thomas 1. Raub Volume 5 STABILITY AND CHARACTERIZATION OF PROTEIN AND PEPTIDE DRUGS: Case Histories Edited by Y. John Wang and Rodney Pearlman Biological Barriers to Protein Delivery Edited by Kenneth L. Audus University of Kansas Lawrence, Kansas and Thomas J. Raub The Upjohn Company Kalamazoo, Michigan Springer Science+Business Media, LLC Llbrary of Congress Cataloglng-ln-Publlcatlon Data Biological barrlers to protein delivery I edited by Kenneth L. Audus and Thomas J. Raub. p. cm. -- IPharmaceutlcal biotechnology ; v. 4) Includes bibliographical references and index. ISBN 978-1-4613-6256-2 ISBN 978-1-4615-2898-2 (eBook) DOI 10.1007/978-1-4615-2898-2 1. Protein drugs--Physiological transport. 2. Drug dellvery systems. 1. Audus. Kenneth L. II. Raub. Thomas J. III. Series. RS431.P75B56 1993 615' .3--dc20 92-38532 CIP ISBN 978-1-4613-6256-2 © 1993 Springer Science+Business Media New York Originally published by Plenum Press New York in 1993 Softcover reprint of the hardcover Ist edition 1993 AII 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 Contributors Maria Susana Bahia • Department ofInternal Medicine, School of Medicine, Yale University, New Haven, Connecticut 06520 AjayK. Banga • Department ofPharmacal Sciences, School ofPharmacy, Au burn University, Auburn, Alabama 36849 Laurence T. Baxter • Department of Radiation Oncology, Harvard Medical School, Steele Laboratory, Massachusetts General Hospital, Boston, Massa chusetts 02114 Richard C. Boucher • Division of Pulmonary Diseases, Department of Medi cine, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 Richard D. Broadwell • Division of Neurological Surgery, Department of Sur gery, and Department ofPathology, University ofMaryland School ofMedi cine, Baltimore, Maryland 21201 Sandra A. Brockman • Department of Biological Sciences, Carnegie Mellon University, Pittsburgh, Pennsylvania 15213 Marcelino Cereijido • Department of Physiology and Biophysics, Center for Research and Advanced Studies, Mexico City, Mexico fie W Chien • ControlledDrug-DeliveryResearchCenter, CollegeofPharmacy, Rutgers-The State University ofNew Jersey, Piscataway, New Jersey 08855 Ruben Gerardo Contreras • Department ofPhysiology and Biophysics, Center for Research and Advanced Studies, Mexico City, Mexico v vi Contributors David C. Dahl • Department of Medicine, University of Minnesota, and Re gional Kidney Disease Program, Hennepin County Medical Center, Minne apolis, Minnesota 55415 M. R. Garcia-Villegas • Department ofPhysiology and Biophysics, Center for Research and Advanced Studies, Mexico City, Mexico LorenzaGonzalez-Mariscal • DepartmentofPhysiologyand Biophysics, Center for Research and Advanced Studies, Mexico City, Mexico Steen H. Hansen • Structural Cell Biology Unit, Department ofAnatomy, The PanumInstitute,UniversityofCopenhagen,DK-2200CopenhagenN,Denmark NigelM. Hooper • DepartmentofBiochemistryand MolecularBiology, Univer sity ofLeeds, Leeds LS2 9JT, United Kingdom Rakesh K. Jain • Department ofRadiation Oncology, Harvard Medical School, Steele Laboratory, Massachusetts General Hospital, Boston, Massachusetts 02114 ConradE. Johanson • Cerebrospinal fluid Research Laboratory, Departmentof Clinical Neurosciences, Program in Neurosurgery, Brown UniversitylRhode Island Hospital, Providence, Rhode Island 02902 Larry G. Johnson • Division ofPulmonary Diseases, Department ofMedicine, The University ofNorth Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 Michael W Konrad • The deVlarninck Institute, Lafayette, California 94549 Jean-Pie"e Kraehenbuhl • Swiss Institute for Cancer Research and Institute of Biochemistry, University ofLausanne, CH-1066 Epalinges, Switzerland Asrar B. Malik • Department of Physiology and Cell Biology, The Albany Medical College ofUnion University, Albany, New York 12208 Dirk K. F. Meijer • Department ofPharmacology and Therapeutics, University Centre for Pharmacy, Groningen, The Netherlands HansPMerkle • DepartmentofPharmacy,SwissFederalInstituteofTechnology, CH-8092 Zurich, Switzerland ShozoMuranishi • DepartmentofBiopharmaceutics,KyotoPharmaceuticalUni versity, Misasagi, Yamashina-ku, Kyoto, 607, Japan Contributors vii RobertF. Murphy • DepartmentofBiologicalSciences,CarnegieMellonUniver sity, Pittsburgh, Pennsylvania 15213 MarianR. Neutra • GastrointestinalCellBiologyLaboratory,Children'sHospital andDepartmentofPediatrics, HarvardMedicalSchool,Boston,Massachusetts 02115 HiroakiOkada • DDSResearchLaboratories,ResearchPharmaceuticalDivision, Takeda Chemical Industries, Ltd., Osaka, 532, Japan SjurOlsnes • InstituteforCancerResearch,NorwegianRadiumHospital,Monte bello, 0310 Oslo 3, Norway RalphRabkin • DepartmentofMedicine, Nephrology Section, StanfordUniver sity School of Medicine, Stanford, and Palo Alto Department of Veterans Affairs Medical Center, Palo Alto, California 94304 Octavio Ruiz • Department ofPhysiology and Biophysics, Center for Research and Advanced Studies, Mexico City, Mexico Kirsten Sandvig • Institute for Cancer Research, Norwegian Radium Hospital, Montebello, 0310 Oslo 3, Norway Alma Siflinger-Birnboim • Department of Physiology and Cell Biology, The Albany Medical College of Union University, Albany, New York 12208 Bo van Deurs • Structural Cell Biology Unit, Department of Anatomy, The PanumInstitute,UniversityofCopenhagen,DK-2200CopenhagenN,Denmark Gregor J. M. Wolany • Department of Pharmacy, Swiss Federal Institute of Technology, CH-8092 Zurich, Switzerland AkiraYamamoto • DepartmentofBiopharmaceutics, KyotoPharmaceuticalUni versity, Misasagi, Yamashina-ku, Kyoto, 607, Japan Kornelia Ziegler • Department of Pharmacology and Toxicology, Justus-Liebig University, Giessen, Germany Preface to the Series Amajorchallengeconfrontingpharmaceuticalscientistsinthefuturewillbe to design successfuldosageforms forthe nextgeneration ofdrugs. Manyof these drugs will be complex polymers ofamino acids (e.g., peptides, pro teins), nucleosides (e.g., antisense molecules), carbohydrates (e.g., polysac charides), orcomplex lipids. Throughrationaldrugdesign, syntheticmedicinalchemistsareprepar ing very potent and very specific peptides and antisense drug candidates. These molecules are being developed with molecular characteristics that permitoptimalinteractionwiththespecificmacromolecules(e.g.,receptors, enzymes, RNA, DNA)that mediate theirtherapeuticeffects. Rationaldrug design does not necessarily mean rational drug delivery, however, which strivestoincorporateintoa moleculethemolecularpropertiesnecessaryfor optimaltransferbetweenthepointofadministrationandthepharmacologi cal targetsitein the body. Like rationaldrugdesign, molecularbiologyishavingasignificantim pact on the pharmaceutical industry. For the first time, it is possible to producelargequantitiesofhighlypure proteins, polysaccharides, and lipids for possiblepharmaceuticalapplications. Likepeptidesandantisense mole cules,thedesignofsuccessfuldosageformsforthesecomplexbiotechnology productsrepresentsa majorchallengeto pharmaceutical scientists. Development ofan acceptable drug dosage form is a complex process requiring strong interactions between scientists from many different divi sionsinapharmaceuticalcompany, includingdiscovery, development,and manufacturing. Theserieseditor, theeditorsoftheindividualvolumes,and thepublisherhopethatthisnewserieswillbeparticularlyhelpfultoscientists in thedevelopmentareasofa pharmaceuticalcompany(e.g., drug metabo lism, toxicology, pharmacokinetics and pharmacodynamics, drug delivery, ix x PrefacetotheSeries preformulation, formulation, andphysicalandanalyticalchemistry). Inad dition,wehopethisserieswillhelptobuildbridgesbetweenthedevelopment scientistsand scientists in discovery (e.g., medicinal chemistry, pharmacol ogy, immunology, cell biology, molecular biology) and in manufacturing (e.g., processchemistry,engineering). Thedesignofsuccessfuldosageforms forthenextgenerationofdrugswillrequirenotonlyahighlevelofexpertise byindividualscientists, butalsoa highdegreeofinteractionbetweenscien tists in thesedifferentdivisions ofa pharmaceutical company. Finally,everyoneinvolvedwiththisserieshopesthatthesevolumeswill alsobeusefultotheeducatorswhoaretrainingthenextgenerationofphar maceutical scientists. Inaddition to havinga high level ofexpertise intheir respective disciplines, these young scientistswill need to have the scientific skills necessary to communicate with their peers in other scientific disciplines. RONALDT. BORCHARDT SeriesEditor Preface The application ofnative proteins or polypeptides as therapeutically useful drugs, e.g., insulin, factorvm, hasbeeninpracticefor severaldecades; however, withthe recent advent ofrecombinant DNA technology, the number ofprotein and peptide drugsanddrugcandidateshasincreasedexponentially. Currently,eightrecombinant proteins arecommerciallyavailable: humangrowthhormone, insulin, interferon-a, tissue plasminogen activator, erythropoietin, granulocytecolony stimulating factor, factor vm, and hepatitis B vaccine. In 1988, it was estimated that over 250 companies worldwide were developing nearly 100recombinant proteinsI as human and veterinary products. Today, there are 132 biotechnology-based drugs and vac cinesindevelopment with 21oftheseawaitingFDAapprova12; this numberis much largerwithmany nearFDAapproval. Inaddition, nonrecombinantproteins, suchas antibodies, and peptides are being used and developed for therapeutic purposes. These peptide and protein products ofbiotechnology present a unique spectrum of problemsforthepharmaceuticalscientistaswellas thepharmacologist. Besidesthe fact that these molecules are often involved in many biological responses and activitieswhicharenotfullyunderstood,thesizeandcomplexphysiochemicalnature ofthesemoleculesresultinsignificantdeliverylimitationsenforcedbyphysiological phenomena. These biological barriers are a composite of cellular linings, e.g., epitheliaandendothelia,metabolism, immunology,clearance, andthephysicallaws thatgovernsolutediffusion. Consequently, the traditional approaches to drug deliv ery are not likely to be successful. Rational approaches to developmentofdelivery strategiesforproteinsevolvefrom anunderstandingofthenatureofthesebiological barriers at a fundamental level. The scope of this volume is a current and critical review of information regarding peptide and protein transport and metabolism as it relates to delivery of endogenous(physiologicalligands)andrecombinantproteinstomammalianorgans, tissues, and cells. Although not always possible due to the paucity ofinformation available, each chapteremphasizes mechanisms oftransport including quantitative evidenceandstructure/function(cause/effect)relationships. Thisvolumeisintended ICopsey, D. N., and Delnatte, S. Y. 1., Genetically Engineered Human Therapeutic Drugs, Stockton, New York, 1988. 2Pharm. Manufact. Assn., Products in the Pipeline, BiolTechnology9:947-949, 1991. xi
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