From Clone to Clinic PDF
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FROM CLONE TO CLINIC Developments in Biotherapy VOLUME 1 FROM CLONE TO CLINIC Edited by D. J. A. CROMMELIN Professor of Pharmacology, University of Utrecht, Utrecht, The Netherlands and H. SCHELLEKENS Senior Research Fellow, TNO, Rijswijk. The Netherlands SPRINGER SCIENCE+BUSINESS MEDIA, B.V. Library of Congress Cataloging in Publication Data From clone to clinic I edited by D.J.A. Crommel in and H. Schellekens. p. cm. -- <Developments in blotherapy ; v. 1) ISBN 978-94-010-5683-0 ISBN 978-94-011-3780-5 (eBook) DOI 10.1007 /978-94-011-3780-5 1. Pharmaceutical b1otechnology. 2. Monoclonal ant1bodies. 1. Crommelin. D. J. A. (Daan J. A.) II. Schelleken, Huub. III. Series. [DNLM: 1. Antibodies, Monoclonal--therapeutic use. 2. Biolog1cal Products--standards. 3 . Clon lng, Molecular. 4. DNA, RecoNb1nant. 5. Genetic Engineering . OW 575 F931J RS380.F76 1990 615·.7--dc20 DNLM/DLC for Library of Congres s 90-5267 ISBN 978-94-010-5683-0 Printed on acid-free paper AlI Rights Reserved © 1990 by Springer Science+Business Media Dordrecht Origina1ly published by Kluwer Academic Publishers in 1990 Softcover reprint of the hardcover Ist edition 1990 No part of the material protected by this copyright notice may be reproduced or utilized in any form or by any means, electronic or mechanical, including photocopying, recording or by any information storage and retrieval system, without written permission from the copyright owner. Contents Preface IX List of Contributors Xl PART ONE: REGISTRATION, TOXICITY AND QUALITY CONTROL 1. Toxicity testing of rDNA products A.D. Dayan 2. Experiences with the EC "high tech" procedure HJ.M. van de Donk and I. Hegger 13 3. The regulation of pharmaceuticals: philosophy and principles J.C. Petricciani 29 4. Quality control of vaccines J.H. Peetermans 29 5. Regulatory affairs and biotechnology in Europe: the CPMP "high tech" and multi- state procedures B. Tryzelaar 37 6. Formulating biotechnology products T. Mundorf, M. Bichler and G. Flotzinger 45 7. Development of analytical methods for monitoring the stability of antibody forma- tion by hybridoma cells in continuous culture systems J.M. Coco Martin, P. Koolwijk, D.E. Martens, J.W. Oberink, C.A.M. van der Velden-de Groot and E.c. Beuvery 57 PART TWO: MONOCLONAL ANTIBODIES 8. Toward human monoclonal antibodies D.R Burton, M.A. Persson, W.D. Huse and E.S. Golub 67 9. Biodistribution, binding and intemalisation of monoclonal antibodies to human ovarian carcinoma cells L.G. Poe1s, O.C. Boerman, L. Massuger, R Claessens, U.K. Nlissander, Q. van Hoesse1, C. Thomas, P. Kenemans and P. Jap 73 10. Pharmacokinetics and tissue distribution ofIndium-lll-labe1ed OV -TL 3 F(ab')2 in ovarian cancer patients L.F.A.G. Massuger, RA.MJ. Claessens, L.G. Poe1s, RH.M. Verheijen, Ch.P.T. Schijf, F.H.M. Corstens and P. Kenemans 83 11. Characterization of human monoclonal antibodies specific for the rabies virus L. Edelman and M. Lafon 89 12. Biochemical and immunological evaluation of an anti-fibrin monoclonal antibody complex containing T2Gls Fab' intended for imaging venous thrombi P.-H. Lai, J. Brown, L.A. Epps, K. George, A. Sautter, C. Coonley, M. vi Plucinsky, S. Buttrum, W.C. Bogard, Jr., RT. Dean and H.I. Berger 95 13. Monoclonal antibodies in radioimmunoscintigraphy. Some hurdles between clone and clinic D. Blok, R.I.1. Feitsman and E.K.I. Pauwels 105 14. Clinical relevance of the tumor marker CA 15.3 in the management of cancer patients G.G. Bon, P. Kenemans, C.A. Yedema, G.I. van Kamp, H.W. Nijman and 1. Hilgers 111 15. Practical applications of monoclonal antibodies against polymorphic epithelial mucin in the differential diagnosis of human tumors J. Hilgers and P. Kenemans 123 16. Selection of monoclonal anti-digoxin antibodies suitable for monitoring of cardiac glycosides S.N. Tyutyulkova, S.D. Kyurkchiev and I.R Kehayov 135 17. Diagnostic use of anti-modified nucleoside monoclonal antibodies M. Mizugaki, K. Itoh and N. Ishida 143 18. Monoclonal antibodies radiolabeled with different radioisotopes for biodistribution and radioimmunodetection of tumor xenografts in the nude rat M.W.A. de Jonge, J.G.W. Kosterink, Yu Yan bin, 1.W.M. Bulte, R.A.M. Ken- gen, T.H. The and L. de Leij 149 19. Bispecific monoclonal antibody (BIAB)-retargeted cellular therapy for local treatment of cancer patients L. de Leij, M.W.A. de Jonge, 1. ter Haar, H. Spakman, E. de Vries, P. Willemse, N.H. Mulder, H. Berendsen, M. Elias, C. Smit Sibinga, W. de Lau, W. Tax and T.H. The 159 20. Enhanced binding of t-PA to fibrin using bispecific monoclonal antibodies R. Bos, M. Otter and W. Nieuwenhuizen 167 21. Recent developments and perspectives on the future of human and murine monoclonal antibodies in the diagnosis and treatment of cancer R.P. McCabe, M.V. Haspel, J.A. Carrasquillo, RG. Steis, N. Pomato, R. Sub- ramanian, E.M. Paris and M.G. Hanna, Jr. 175 22. Pharmacokinetics and safety of a human IGM antibody, HA-IA R.C. Straube, C.R. Smith, I.E. Allen, C.P. Dating, C. Kilgarriff, 1.B. Cornett and S.D. Bolmer 189 23. Development and clinical experience with humanised monoclonal antibodies G. Hale, M.I.S. Dyer, M.R. Clark and H. Waldmann 195 24. A method for the transformation of hybridoma cell lines with improved efficiency: its use in the production of bispecific monoclonal antibodies R. Bos, E. van den Berg and W. Nieuwenhuizen 201 25. To an optimal design of an airlift bioreactor for the cultivation of hybridomas D.E. Martens, J. Coco Martin, C.A.M. van der Velden-de Groot, E.C. Beuvery, C.D. de Gooijer and J. Tramper 209 vii PART THREE: MARKETED PRODUCTS 26. Experience with marketed biotech products: rt-PA L. Nelles and D. Collen 219 27. Clinical trial of recombinant human IL-2 in the treatment of Mycobacterium Avium complex infection H. Toba, I. Tsuyuguchi, H. Kimura, H. Fujiwara, S. Hanamoto, H. Kawasumi and S. Kishimoto 227 28. Use of recombinant human erythropoietin in anemic dialysis patients I.C. Egrie 233 29. Experience with biosynthetic growth hormone J.M. Wit 239 PART FOUR: NEW PRODUCTS 30. Recombinant follicle stimulating hormone. I. Construction, selection and charac- terization of a cell line P. van Wezenbeek, J. Draaijer, F. van Meel and W. Olijve 245 31. Recombinant human follicle stimulating hormone. II. Biochemical and biological characteristics W. de Boer and B. Mannaerts 253 32. Heterologous expression of human Interleukin-3 R.W. van Leen, PJ. Lemson and J.G. Bakhuis 261 33. Purification of recombinant human Interleukin-3 from Bacillus Licheniformis B. Noordam, R.F.W.C. van Beckhoven, R. van Lambalgen and N.L.M. Persoon 269 34. The acid- and thermolabile interferon alpha: a SUbtype, or a new cell inhibitor? L. Borecky, P. Kontsek, V. Lackovic, I. Mistn'kova and M. Novak 275 35. The IFNy receptor as tool for the discovery of new immunomodulatory drugs L. Ozmen, M. Fountoulakis, D. Stuber and G. Garotta 283 36. Cloned receptors and transfected cell lines in the design of new drugs: muscarinic cholinergic receptors W. Sadee, S. Maeda, I. Lameh and R.I. Cone 291 PART FIVE: DRUG DELIVERY 37. Structural analysis of carbohydrate chains of native and recombinant-DNA glycoproteins J.P. Kamerling, K. Hard and J.F.G. Vliegenthart 295 38. The role of protein structure in surface tension kinetics A.-P. Wei, J.N. Herron and J.D. Andrade 305 39. Antigen carriers: a success determining factor for subunit vaccines? G.F.A. Kersten, E.C. Beuvery and DJ.A. Crommelin 315 40. Intranasal delivery of insulin: absorption enhancement by the fusidate derivative STDHF in rabbits and rats and effects on human nasal ciliary movement in vitro viii J. Verhoef, W.AJJ. Hennens, MJ.M. Deurloo, S.G. Romeijn, N.G.M. Schipper and P.W.H.M. Merkus 323 41. Improved oral peptide delivery by means ofmucoadhesion C.-M. Lehr, J.A. Bouwstra, J.J. Tukker, J. Verhoef, A.G. de Boer and H.E. Junginger 329 42. Delivery of therapeutic peptides and proteins M. Mackay 335 43. Compatibility studies of a soluble T4 receptor with a microinfusion pump for continuous intravenous therapy A.L. Shorter, M.B. Seaman, D. Dunleavy, P. Smialkowski and D. Schrader 343 44. Immunoliposome-mediated delivery of chemotherapeutics U.K. Nassander, G. Stonn, P.A. Steerenberg, W.H. de Jong, G. de Groot, L.G. Poels, Q.G.C.M. van Hoesel and DJ.A. Crommelin 357 45. A novel approach for the selection and detection of cells transfected with adenosine deaminase expression vectors Q. Shen, V.W. van Beusechem, M.P.W. Einerhand and D. Valerio 367 46. Toward gene therapy in hemophilia A: introduction of factor VIII expression vectors into somatic cells R.C. Hoeben, M.P.W. Einerhand, SJ. Cramer, E. Briet, H. van Onnondt, D. Valerio and AJ. van der Eb 373 Preface This book contains a selection of the papers presented at the meeting "Between Clone and Clinic" which was organised in March 1990 in Amsterdam by the dutch Organisation for Applied Research, TNO, and the University of Utrecht. The scope of this meeting was the development of biotechnological pharmaceuticals mainly made by recombinant DNA technology or monoclonal antibody techniques. All aspects concerning the development of the products after host cells producing them are obtained where discussed. The meeting was attended by twohundred specialists from all over the globe, including phar- macologists, toxicologists, registration experts, Quality Assurence managers, production en- gineers and physicians. Biotechnological pharmaceuticals are in general large and complex protein molecules. Bringing these products to the market poses other problems than encountered with the classical chemical drugs. The source of biotechnological pharmaceuticals are living cells. The function of cells are depend- ent on many factors and the stability of production may be a problem. Good Laboratory and Manufactory Practices with Quality Control (GLP and GMP) are of paramount importance and are discussed in a number of papers. The products of the new biotechnology are often highly specific and only active in the human species. Also the side effects can only be studied in the clinical setting. Even when the product is active in animals there is the problem of antigenicity. During treatment the animals will produce antibodies which neutralise the activity. So safety testing may prove difficult. Within the EEC there is a centralised registration of biotechnological pharmaceuticals. Submis- sion in a single european country within the community is sufficient. This donor country will submit the dossier in Brussels, where an evaluation by EEC experts is made. When approved in Brussels registration in individual countries is greatly facilitated and after 1992 mandatory. Two papers in the book discuss this european registration procedure. Although the techniques for the production of these new biotechnological pharmaceuticals are new, a number of products as OKT-3, insuline, hepatitis B vaccine, interferon, erythropoetine an human growth hormone are now on the market for several years. The problems encountered when developing these products are presented by representatives of the industries involved. Much is expected from the use of monoclonal antibodies alone or as carriers of other agents. To avoid antigenicity a lot of attention has focused on the development of human monoclonal antibodies and a number of papers give a tour d'horizon on this topic. Biotechnology products are by their size and relative instability difficult to get to their target organs. Development of drug delivery systems is urgently needed before most of these new drugs can be used successfully. A number of new opportunities in this area are presented in this book. This was the first time that all these experts convened and discussed the problems of the develop- ment of biotechnological pharmaceuticals comprehensively. Judging from the quality of the papers in this book the meeting was a success and deserves to be repeated. Daan Crommelin and Huub Schellekens July 1990 ix List of Contributors D.BLOK University Hospital Leiden, Division of Nuclear Medicine, Department of Diagnostic Radiology, Rijnsburgerweg 10, 2333 AA Leiden, The Netherlands Co-authors: R. 1. J. FEITSMA and E. K. J. PAUWELS G. G. BON Free University Hospital, Department of Obstetrics & Gynaecology, De Boelelaan 1117, 1081 HV Amsterdam, The Netherlands Co-authors: P. KENEMANS, CA. YEDEMA, G. J. VAN KAMP, H. W. NIJMAN and J. HILGERS L.BORECKY Institute of Virology, Slovak Academy of Sciences, Dti brawska 9, 84246 Bratislava, Czechos- lovakia Co-authors: P. KONTSEK, V. LACKOVIC, J. MISTRlKOV A and M. NovAK R.BOS Gaubius Institute TNO, P.O. Box 612, 2300 AP Leiden, The Netherlands Chapter 20 co-authors: M. OTTER and W. NIEUWENHUIZEN Chapter 24 co-authors: E. VA N DEN BERG and W. NIEUWENHUIZEN D.R.BURTON Department of Molecular Biology, Scripps Clinic & Research Foundation, 10666 N. Torrey Pines Road, La Jolla, CA 92037, U.S.A. Co-authors: M. A. PERSSON, W. D. HUSE and E. S. GOLUB J. M. COCO MARTIN National Institute for Public Health and Environmental Protection (RIVM), Department of Inactivated Viral Vaccines, P.O. Box 1,3720 BA Bilthoven, The Netherlands Co-authors: P. KOOLWlJK, D. E. MARTENS, J. W. OBERINK, C. A. M. VAN DER VELDEN-DE GROOT and E. C. BEUVERY D. J. A. CROMMELIN University of Utrecht, Faculty of Pharmacy, Department of Pharmaceutics, Croesestraat 79, 3522 AD Utrecht, The Netherlands xi
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