Advances in Delivery Science and Technology
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1 Advances in Delivery Science and Technology Series Editor Michael J. Rathbone More information about this series at
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3 Kenneth A. Howard Thomas Vorup-Jensen Dan Peer Editors Nanomedicine
4 Editors Kenneth A. Howard Department of Molecular Biology and Genetics Interdisciplinary Nanoscience Center (inano) Aarhus University Aarhus C, Denmark Thomas Vorup-Jensen Department of Biomedicine Biophysical Immunology Laboratory Aarhus University Aarhus C, Denmark Dan Peer Department of Cell Research & Immunology and Department of Material Science and Engineering Laboratory of Precision NanoMedicine Tel-Aviv University Tel-Aviv, Israel ISSN ISSN (electronic) Advances in Delivery Science and Technology ISBN ISBN (ebook) DOI / Library of Congress Control Number: Controlled Release Society 2016 This work is subject to copyright. All rights are reserved by the Publisher, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting, reproduction on microfilms or in any other physical way, and transmission or information storage and retrieval, electronic adaptation, computer software, or by similar or dissimilar methodology now known or hereafter developed. The use of general descriptive names, registered names, trademarks, service marks, etc. in this publication does not imply, even in the absence of a specific statement, that such names are exempt from the relevant protective laws and regulations and therefore free for general use. The publisher, the authors and the editors are safe to assume that the advice and information in this book are believed to be true and accurate at the date of publication. Neither the publisher nor the authors or the editors give a warranty, express or implied, with respect to the material contained herein or for any errors or omissions that may have been made. Printed on acid-free paper This Springer imprint is published by Springer Nature The registered company is Springer Science+Business Media LLC New York
5 Editor Kenneth A. Howard would like to dedicate this book to his sister Jacqueline Teer.
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7 Preface Nanomedicine is set to meet medical challenges such as early and rapid diagnosis, targeted and effective drug delivery, and novel ways of providing organ and tissue replacement that work towards new personalized medicines. Utilizing properties occurring at the nanoscale for unique medical effects as well as understanding and controlling cellular molecular processes and clinical translation are key elements in nanomedicine. These skills are strongly bound by the threads that connect nanoscience, physics, material science, immunology, molecular biology, pharmacology, pharmaceutics, and medicine. The book Nanomedicine sets to capture this broad spectrum of disciplines whilst still maintaining focus on the key elements that establishes it as a bona fi de unique field. The inclusion of adequate background information for understanding the basic principles makes this text an excellent learning tool for students as well as a useful reference for more senior scientists and other practitioners in the field. The opening chapter defines the current field of nanomedicine by addressing the constituent key elements and describing major nanomedicine initiatives and programs, as well as product landscape to understand the development process and the impact of nanomedicine. The following three chapters focus on immune recognition with emphasis on the response by the innate immune system, notably complement activation of relevance in removal of nanomedicines as well as in disease pathogenesis. Chapter 2 focuses on complement factor inhibitors developed by structural biology determination. Chapter 3 addresses processes leading to nanoparticleinduced innate immune activation and discusses consequences for nanomedicinebased therapeutics. Chapter 4 focuses on protein polyvalent interactions with molecular recognition patterns at the nanoscale which is relevant in design criteria for the reduction of immune recognition of particulate nanomedicines. The next section focuses on nanomedicine-based diagnostics. Chapter 5 describes microfluidics-based tools for single cancer cell analysis and the use of nanotechnology for improved signal responses. Nanotheranostics and in vivo imaging modalities are addressed in Chapter 6. Chapters 7 11 describe a range of nanoscale nanopharmaceuticals. Chapter 7 gives an overview of a range of nanocarriers used for cancer treatment with a focus on targeting approaches. The ability to influence interaction vii
8 viii Preface with the extracellular and intracellular biological environment by particle geometry is the focus of Chapter 8. Nano-drug delivery systems for delivery of protein\peptide drugs to the brain are described in Chapter 9, and polymer-based DNA delivery to dendritic cells for cancer immunotherapy in Chapter 10. The section concludes with Chapter 11 on the use of the biopolymer silk in nanomedicine. The clinical translation of nanomedicines is dependent on safety and regulatory approval, addressed in Chapter 12. The final section describes nanomedicine-based tissue regeneration and cell therapy with the application of nanotechnology for medical implant drug release in Chapter 13, and Chapter 14 describes guided cellular responses by surface cues for nanomedicine applications. The book Nanomedicine is relevant both as a textbook of an emerging field and as an essential read that gives insight and clarity to this interdisciplinary subject.
9 Acknowledgements The editors would like to express their sincere gratitude to all the authors for the time and effort they have given in the preparation of the excellent chapters contained in the book. We are grateful to the CRS and especially Series Editor Michael Rathbone for giving us the opportunity to put together a book we feel is needed in the field. Our special thanks also go to our publisher Springer and their staff members Carolyn Honour, Renata Hutter, Sarah McCabe, Jacob Rosati, and Michelle Feng He, all who worked with both timely attention and patience during the preparation of this book. ix
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11 Contents 1 Nanomedicine: Working Towards Defining the Field... 1 Kenneth A. Howard 2 Complement Regulators and Inhibitors in Health and Disease: A Structural Perspective Laure Yatime, Goran Bajic, Janus Asbjørn Schatz-Jakobsen, and Gregers Rom Andersen 3 The Art of Complement: Complement Sensing of Nanoparticles and Consequences S. Moein Moghimi, Kiana C. Trippler, and Dmitri Simberg 4 The Nanoscience of Polyvalent Binding by Proteins in the Immune Response Thomas Vorup-Jensen 5 Microfluidics-based Single Cell Analytical Platforms for Characterization of Cancer Emil Laust Kristoffersen, Morten Leth Jepsen, Birgitta R. Knudsen, and Yi-Ping Ho 6 Nanotheranostics and In-Vivo Imaging Brandon Buckway and Hamidreza Ghandehari 7 Targeting Cancer Using Nanocarriers Dalit Landesman-Milo, Shahd Qassem, and Dan Peer 8 The Importance of Particle Geometry in Design of Therapeutic and Imaging Nanovectors Matthew J. Ware, Jenolyn F. Alexander, Huw D. Summers, and Biana Godin xi
12 xii Contents 9 Delivery of Peptides and Proteins to the Brain Using Nano-Drug Delivery Systems and Other Formulations David Stepensky 10 Polymer-Based DNA Delivery Systems for Cancer Immunotherapy Ayelet David and Adi Golani-Armon 11 The Use of Silk in Nanomedicine Applications Raymond Chiasson, Moaraj Hasan, Qusai Al Nazer, Omid C. Farokhzad, and Nazila Kamaly 12 Nanotoxicology and Regulatory Affairs Christiane Beer 13 The Application of Nanotechnology for Implant Drug Release Morten Østergaard Andersen 14 Guided Cellular Responses by Surface Cues for Nanomedicine Applications Ryosuke Ogaki, Ole Zoffmann Andersen, and Morten Foss Index
13 Contributors Jenolyn F. Alexander Department of Nanomedicine, Houston Methodist Research Institute, Houston, TX, USA Qusai Al Nazer Laboratory of Nanomedicine and Biomaterials, Brigham and Women s Hospital, Harvard Medical School, Boston, MA, USA Gregers Rom Andersen Department of Molecular Biology and Genetics, Aarhus University, Aarhus C, Denmark Morten Østergaard Andersen The Maersk Mc-Kinney Moller Institute and Department of Chemical Engineering, Biotechnology and Environmental Technology, Faculty of Engineering, University of Southern Denmark, Odense, Denmark Ole Zoffmann Andersen Interdisciplinary Nanoscience Center (inano), Aarhus University, Aarhus C, Denmark Goran Bajic Department of Molecular Biology and Genetics, Aarhus University, Aarhus C, Denmark Christiane Beer Department of Public Health, Aarhus University, Aarhus C, Denmark Brandon Buckway Department of Pharmaceutics and Pharmaceutical Chemistry, University of Utah, Salt Lake City, UT, USA Utah Center for Nanomedicine, Nano Institute of Utah, University of Utah, Salt Lake City, UT, USA Huntsman Cancer Institute, University of Utah, Salt Lake City, UT, USA Raymond Chiasson Laboratory of Nanomedicine and Biomaterials, Brigham and Women s Hospital, Harvard Medical School, Boston, MA, USA Ayelet David Department of Clinical Biochemistry and Pharmacology, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel xiii
14 xiv Contributors Omid C. Farokhzad Laboratory of Nanomedicine and Biomaterials, Brigham and Women s Hospital, Harvard Medical School, Boston, MA, USA Morten Foss Interdisciplinary Nanoscience Center (inano), Aarhus University, Aarhus C, Denmark Hamidreza Ghandehari Department of Pharmaceutics and Pharmaceutical Chemistry, University of Utah, Salt Lake City, UT, USA Utah Center for Nanomedicine, Nano Institute of Utah, University of Utah, Salt Lake City, UT, USA Huntsman Cancer Institute, University of Utah, Salt Lake City, UT, USA Department of Bioengineering, University of Utah, Salt Lake City, UT, USA Biana Godin Department of Nanomedicine, Houston Methodist Research Institute, Houston, TX, USA Adi Golani-Armon Department of Clinical Biochemistry and Pharmacology, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel Moaraj Hasan Laboratory of Nanomedicine and Biomaterials, Brigham and Women s Hospital, Harvard Medical School, Boston, MA, USA Yi-Ping Ho Department of Molecular Biology and Genetics, Interdisciplinary Nanoscience Center (inano), Aarhus University, Aarhus C, Denmark Kenneth A. Howard Department of Molecular Biology and Genetics, Interdisciplinary Nanoscience Center (inano), Aarhus University, Aarhus C, Denmark Morten Leth Jepsen Department of Molecular Biology and Genetics, Interdisciplinary Nanoscience Center (inano), Aarhus University, Aarhus C, Denmark Nazila Kamaly Laboratory of Nanomedicine and Biomaterials, Brigham and Women s Hospital, Harvard Medical School, Boston, MA, USA Department of Micro- and Nanotechnology, Technical University of Denmark, Lyngby, Denmark Birgitta R. Knudsen Department of Molecular Biology and Genetics, Interdisciplinary Nanoscience Center (inano), Aarhus University, Aarhus C, Denmark Emil Laust Kristoffersen Department of Molecular Biology and Genetics, Interdisciplinary Nanoscience Center (inano), Aarhus University, Aarhus C, Denmark Dalit Landesman-Milo Laboratory of NanoMedicine, Department of Cell Research and Immunology, George S. Wise Faculty of Life Sciences, Tel Aviv University, Tel Aviv, Israel
15 Contributors xv Department of Materials Sciences and Engineering, The Iby and Aladar Fleischman Faculty of Engineering, Tel Aviv University, Tel Aviv, Israel Center for Nanoscience and Nanotechnology, Tel Aviv University, Tel Aviv, Israel S. Moein Moghimi School of Medicine, Pharmacy and Health, Durham University, Stockton-on-Tees, TS17 6BH, UK Ryosuke Ogaki Interdisciplinary Nanoscience Center (inano), Aarhus University, Aarhus C, Denmark Dan Peer Department of Cell Research & Immunology and Department of Material Science and Engineering Laboratory of Precision NanoMedicine, Tel-Aviv University, Tel Aviv, Israel Shahd Qassem Laboratory of NanoMedicine, Department of Cell Research and Immunology, Tel Aviv University, Tel Aviv, Israel Department of Materials Sciences and Engineering, The Iby and Aladar Fleischman Faculty of Engineering, Tel Aviv University, Tel Aviv, Israel Center for Nanoscience and Nanotechnology, Tel Aviv University, Tel Aviv, Israel Janus Asbjørn Schatz-Jakobsen Department of Molecular Biology and Genetics, Aarhus University, Aarhus C, Denmark Dmitri Simberg Department of Pharmaceutical Sciences, The Skaggs School of Pharmacy and Pharmaceutical Sciences, University of Colorado Denver, Aurora, CO, USA David Stepensky Department of Clinical Biochemistry and Pharmacology, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel Huw D. Summers Centre for Nanohealth, College of Engineering, Swansea University, Swansea, UK Kiana C. Trippler Department of Pharmaceutical Sciences, The Skaggs School of Pharmacy and Pharmaceutical Sciences, University of Colorado Denver, Aurora, CO, USA Thomas Vorup-Jensen Department of Biomedicine, Biophysical Immunology Laboratory, Aarhus University, Aarhus C, Denmark Matthew J. Ware Centre for Nanohealth, College of Engineering, Swansea University, Swansea, UK Department of Nanomedicine, Houston Methodist Research Institute, Houston, TX, USA Laure Yatime DIMNP UMR5235, University of Montpellier, Montpellier, France
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17 Editor s Biography Kenneth A. Howard is an Associate Professor and Group Leader at the Department of Molecular Biology and Genetics at the Interdisciplinary Nanoscience Center (inano), Aarhus University, Denmark. His research and teaching activities are focused on delivery science, nanomedicine, and RNA interference. Kenneth Alan Howard received a PhD in pharmaceutical science from the University of Nottingham, UK, and has held postdoctoral positions at the CRC Institute for Cancer Studies, University of Birmingham, UK, and the School of Pharmacy, University of London. Dr. Howard is an active member of the Controlled Release Society serving on the Board of Scientific Advisors (chair), Nominations Committee, and organizer of CRS Educational workshops RNA Interference Biology and Therapeutics and Albumin: The Next Generation Therapeutic. Dan Peer is a translational scientist and Professor that leads an NIH-funded lab in the Faculty of Life Science and the Faculty of Engineering at Tel Aviv University (TAU). He is also the Director of the Focal Technology Area (FTA) on Nanomedicines for Personalized Theranostics, a National Nanotechnology Initiative, and the Director of the Leona M. and Harry B. Helmsley Nanotechnology Research Fund. Prof. Peer s work was among the first to demonstrate systemic delivery of RNAi molecules using targeted nanocarriers to the immune system, and he pioneered the use of RNA interference (RNAi) for in vivo validation of new drug targets within the immune system. He is an editor of several books in the field of nanomedicine, Editor of Molecular and Cellular Therapies (Springer), Editor of Biology and Medicine in Nanotechnology (IOP), and an Associate Editor of the Journal of Controlled Release (Elsevier), Journal of Biomedical Nanotechnology, and Biochemistry ; he is on the Editorial Boards of the Biomedical Microdevices (Springer), Cancer Letters (Elsevier), Nanomedicine: Nanotechnology, Biology and Medicine (Elsevier), and Bioconjugate Chemistry (ACS). Prof. Peer is currently the President of the Israeli Chapter of the Controlled Release Society and a Member of the Israel Young Academy of Sciences and Humanities. xvii
18 xviii Editor s Biography Thomas Vorup-Jensen is head of the Biophysical Immunology Laboratory in the Department of Biomedicine and a member of the inano at Aarhus University. He received a PhD in medicine from Aarhus University on topics involving the discovery, characterization, and recombinant manufacture of molecules of the complement system. More recently, he was awarded from the same institution the Doctor of Medical Science degree following a dissertation on polyvalent interactions between molecules of the immune system and their targets, which includes certain nanomedicines. Following his work as Research Fellow in Pathology with Harvard Medical School and later employment with Aarhus University, his research has focused on understanding the nanoscience of protein ligand recognition. In addition to his academic efforts, Prof. Vorup-Jensen and his laboratory actively collaborate with industry partners to translate scientific findings into new treatments of clinical unmet needs, in particular focusing on autoimmune and infectious diseases. Prof. Vorup-Jensen is on the editorial boards of Frontiers in Pharmacology, Frontiers in Immunology, and Molecular and Cellular Therapies. Prof. Vorup-Jensen was recently elected member of the Danish Academy of Natural Sciences.
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