1st Edition

Handbook of Biomarkers and Precision Medicine

ISBN 9781498762588
Published May 1, 2019 by Chapman and Hall/CRC
631 Pages 100 Color Illustrations

USD $299.95

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Book Description

"The field of Biomarkers and Precision Medicine in drug development is rapidly evolving and this book presents a snapshot of exciting new approaches. By presenting a wide range of biomarker applications, discussed by knowledgeable and experienced scientists, readers will develop an appreciation of the scope and breadth of biomarker knowledge and find examples that will help them in their own work." -Maria Freire, Foundation for the National Institutes of Health

Handbook of Biomarkers and Precision Medicine provides comprehensive insights into biomarker discovery and development which has driven the new era of Precision Medicine. A wide variety of renowned experts from government, academia, teaching hospitals, biotechnology and pharmaceutical companies share best practices, examples and exciting new developments. The handbook aims to provide in-depth knowledge to research scientists, students and decision makers engaged in Biomarker and Precision Medicine-centric drug development.


  • Detailed insights into biomarker discovery, validation and diagnostic development with implementation strategies
  • Lessons-learned from successful Precision Medicine case studies
  • A variety of exciting and emerging biomarker technologies
  • The next frontiers and future challenges of biomarkers in Precision Medicine

Claudio Carini, Mark Fidock and Alain van Gool are internationally recognized as scientific leaders in Biomarkers and Precision Medicine. They have worked for decades in academia and pharmaceutical industry in EU, USA and Asia. Currently, Dr. Carini is Honorary Faculty at Kings’s College School of Medicine, London, UK. Dr. Fidock is Vice President of Precision Medicine Laboratories at AstraZeneca, Cambridge, UK. Prof.dr. van Gool is Head Translational Metabolic Laboratory at Radboud university medical school, Nijmegen, NL.

Table of Contents


Maria Freire

  1. What is a Biomarker and their Role in Drug Development?

  2. Definitions and conceptual framework of Biomarkers in Precision Medicine

    Claudio Carini, Mark D Fidock, Alain J van Gool

    In search of predictive biomarkers in cancer immunotherapy: Establishment of collaborative network for the validation process

    Giuseppe Masucci, Paola A Ascierto, Lisa Villabona

    Integrating personalized medicine in the healthcare: Payers and reimbursements

    Dominic Galante, Marylou Buyse

    Regulatory strategies to accelerate the implementation of biomarkers and companion diagnostics (CDX)

    F Owen Fields

  3. Biomarkers in Preclinical Sciences
  4. Lost in Translation – the challenges with the use of animal models in translational research
    Attila Seyhan,Claudio Carini

    Application of pharmacokinetic and pharmacodynamic modelling of biomarkers of efficacy in

    Translational research

    Neil Benson

    The DIAMONDS platform: Providing Decision Support for Toxicological Assessment based on Integration

    of Structural, Toxicological and Biomarker information

    Rob H Stierum, E Dinant Kroese, Jack TWE Vogels, Harrie Buist, Danyel GJ Jennen, Eugene P van Someren

    Predicting the (non)clinical toxicity of novel drug targets: an in silico perspective
    Jennifer Venhorst, Lars Verschuren, Annemette V Thougaard, Jorrit J Hornberg, Tanja Rouhani Rankouhi

    Unwanted Immunogenicity Responses to Biotherapeuitcs

    Shobha Purushothama, Boris Gorovits

    Biomarker use for advanced screening of drug-induced kidney injury

    Martijn J Wilmer, Frans GM Russel

    Soluble biomarkers for Cardiotoxicity

    Amy Pointon and Nick Edmunds

  5. Biomarkers in Translational Medicine
  6. Lost in Translation- Bridging the Pre-Clinical and Clinical Worlds Concepts, Examples, and Successes and Failure in Translational Medicine

    Attila Seyhan and Claudio Carini

    Necessary Characterization of Biomarkers to be "Fit for Purpose"

    Suso Platero 

    Crucial role of high quality biosamples in biomarker development

    Cornelia Stumptner, Karine Sargsyan, Penelope Kungl, Kurt Zatloukal

    Informatics solution for biomarker discovery and personalized medicine in clinical care
    Jan Willem Boiten, Rita Azevedo, Marinel Cavelaars, André Dekker, Remond JA Fijneman, Arnoud van der Maas, Morris Swertz, Jeroen AN Beliën, Gerrit A Meijer

    Systems Biology Approaches to Identify New Biomarkers

    Lars Kuepfer, Andreas Schuppert

    The need for global collaboration in translational medicine

    Florence Bietrix, Antoni L Andreu, Giovanni Migliaccio, Anton E Ussi

  7. Biomarker-informed Clinical Trials
  8. Overview of biomarker discovery and statistical considerations

    Overview of classical design in biomarker informed clinical trials

    Bo Huang, Jing Wang, Robert A. Beckman, Sandeep Menon and Weidong Zhang

    Novel biomarker guided clinical trials design: A practical view

    Bo Huang, Jing Wang, Robert A. Beckman, Sandeep Menon and Weidong Zhang

    Outsourcing Biomarker Clinical Trials: Advantages and Disadvantages

    Robert Holt

    Case studies for biomarker based clinical trials

    Bo Huang, Jing Wang, Robert A. Beckman, Sandeep Menon and Weidong Zhang

    Informational designs and potential applications to Rare Disease

    Robert Beckman, Cong Chen

  9. The road ahead: Identification of the hurdles and solutions to the implementation of Precision Medicine in healthcare
  10. Paving a new era in patient centric care through innovations in Precision Medicine

    Mark Fidock, Glen Hughes, Alain J van Gool, Claudio Carini

    Integrating Molecular Testing into Early Stage Clinical Applications

    Ectopic gene deregulations and chromosome conformations: integrating novel molecular testing into clinical applications, from leukemias to gliomas"

    Francis Hector Grand, Matthew Salter, Ewan Hunter, Alexandre Akoulitchev

    Challenges and Opportunities of Next Generation Sequencing Companion Diagnostics

    Benoit Destenaves

    Implementing companion diagnostics

    Martina Kaufmann

    Publishing biomedical research findings - what are the odds?

    Andreas Scherer

    Computational Biology: Modeling from Molecule to Disease and Personalized Medicine

    Nina Kusch, Lisa Turnhoff, Andreas Schuppert

    Expectations for Quality Control: Challenges with multi-plex assays

    John L Allinson

  11. Lessons from the past and pioneers of the future
  12. Development of Mariviroc and the Companion Diagnostic HIV Tropism Assay

    Elna van der Ryst, James Demarest, Jayvant Heera, Mike Westby,Charles Knisch

    Case studies: Infertility and Anti-Müllerian Hormone

    Martin Hund, Joan-Carles Arce

    Factor Xa: Case Study

    Boris Gorovits

    Delivering Companion Diagnostics for Complex Biomarkers: The BRCA Companion Diagnostic Story for LYNPARZATM (Olaparib)

    Maria C M Orr and Simon P Dearden

    Delivering the personalized medicine promise: best practice recommendations for designing biomarker development processes

    Karen KY Lam, Mary Zacour, Ji-Young V Kim, Zsuzsanna Hollander, Rhonda Wideman, Raymond T Ng, Scott J Tebbutt, Bruce M McManus

    Development, Launch and Adoption of a Pharmacodiagnostic: Planning Versus Reacting for Pharmaceutical Companies

    Mike Montalto, George Green

    Biomarker Consortia to drive BM discovery and clinical applications in the UK – lessons learned from RA-MAP

    John Isaacs, Sarah Brockbank, Ayako Pedersen Andrew Cope

    Measuring the Success of US biomarker Consortia

    Menetski Joseph

    The value of public-private partnerships in the Netherlands

    Hanna L Groen, Nico LU van Meeteren

  13. Paving a new era of personalized medicine: Emerging technologies
  14. A new era of personalized medicine: emerging technologies - functional proteomics, companion diagnostics and precision cancer medicine

    Michela Perani, Peter Parker

    The impact of epigenetics on the future of personalized medicine

    Jane Mellor

    Incorporating predictive imaging biomarkers in clinical trials for personalised healthcare

    John C Waterton

    Innovation in Metabonomics to improve personalized healthcare

    Nelma Pértega-Gomez and Massimo Loda

    Ultra-sensitive immunoassays to detect low abundant protein biomarkers

    Paul Wheeler

    Glycomics as an innovative approach for personalized medicine

    Stefanie Holst, Nurulamin Abu Bakar, Monique van Scherpenzeel, Manfred Wuhrer, Dirk J Lefeber

    miRNA as novel biomarkers of health and disease

    Laura García Bermejo

    Targeted proteomics for absolute quantification of protein biomarkers in serum and tissues
    Coşkun Güzel, Christoph Stingl, Frank Klont, Roel Tans, Esther Willems, Rainer Bischoff, Alain J van Gool, Theo M Luider, the Biomarker Development Center Consortium

    Integrated omics for tissue-based mapping of the human proteome

    Cecilia Lindskog

    Proteogenomics and multi-omics data integration for personalized medicine

    Peter Horvatovich, Corry-Anke Brandsma, Frank Suits, Rainer Bischoff, Victor Guryev

    Sequencing Approaches for Personalized Cancer Therapy Selection in Pathology

    Wendy de Leng, Manon Huibers, John Hinrichs, Edwin Cuppen, Stefan Willems

    Mass spectrometry imaging: enabling comprehensive spatial analysis of molecular biomarkers in tissue for personalized medicine

    Tiffany Porta, Steven WM Olde Damink, Ron MA Heeren

    Insights for personalized medicine from the gut microbiome: Lesson learned from celiac disease

    Gloria Serena, Anna Sapone, Alessio Fasano

  15. The next frontiers of therapeutic target areas: Autoimmunity, inflammation, respiratory, metabolic, cardiovascular and neurological diseases
  16. Precision Mucosal Sampling and Biomarkers in Allergic Rhinitis and Asthma

    Ashillesh Jha, Ryan S Thwaites, Nehmat Singh, Trevor Hansel

    Stratified medicine in autoimmune diseases

    John A Reynolds, Ian Bruce

    Biomarkers of Systemic Lupus Erythematosus

    Hiroshi Kato, Andras Perl

    Advances in cardiovascular Diagnostics – Use of cardiac biomarkers in clinical research and care of the patient

    Mark Effron and Zahoor Khan

    Biomarkers for psychiatric disease: an overview

    Roy Perlis

    Progressing towards precision psychiatry: current challenges in applying biomarkers in psychiatry
    Victor L Kallen, Brisa S Fernandes

    Identification of Biomarkers for the diagnosis, prediction and progression of Alzheimer’s Disease

    Bennett P Greenwood, Vladimir Tolstikov, Paula P Narain, Jeremy Chaufty, Viatcheslav R Akmaev, Vivek Vishnudas, Stephane Gesta, Rangaprasad Sarangarajan, Niven R Narain, Mike Kiebish

    Biomarkers in Lung Cancer

    George Santis, Richard Lee and Crispin T Hiley

  17. Lessons Learned: What have we learned and what is the next challenge?

          Translational science in modern pharmaceutical R&D: the influence of organizational

          structures on successes and failures

          Peter MA Groenen

          Biomarkers Qualification and Companion Diagnostics- Failure and Success of

          regulatory Process

         Federico Goodsaid

         Not all Biomarkers Are Worthwhile: An Unsuccessful Use and Pitfalls to Avoid

         Abdel Halim

         Research Reproducibility: The Case for Standards

         Leonard P Freedman, Raymond H Cypess

        Can Big Data Analytics Recapitulate Biology? A Survey of Multi-omics Data

         Integration Approaches 
         Armit Singh, Casey P Shannon, Kim-Anh Lê Cao, and Scott J Tebbutt

         Can healthcare be made more affordable by the use of biomarkers?

         Rene L Sluiter, Wietske Kievit, Gert Jan van der Wilt

        Omics driven medicine for democratizating healthcare delivery and health economics

         Mike Kiebish, Viatcheslav R Akmaev, Vivek Vishnudas, Stephane Gesta, Rangaprasad

        Sarangarajan, Niven R

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Claudio Carini, MD, PhD, FRCPath, has been involved in the application of biomarkers in precision medicine for the past 18 years. He is currently appointed as H. Faculty in the Department of Cancer and Pharmaceutical Sciences, Faculty of Life Sciences & Medicine at King’s College, School of Medicine, London. He is a member of the F-NIH Biomarkers Consortium Steering Committees for Cancer, Inflammation & Immunity, and Neuroscience. He is also a senior member of the Scientific Advisory Boards for several biotechnology companies and a member of the New York Academy of Sciences. Claudio was formerly the Global Head of Clinical Immunology and Biomarkers at Pfizer Inc., where he led precision medicine activities across multiple therapeutic areas and facilitated international collaborations with prestigious academic institutions. He also held senior roles in pharmaceutical companies (Wyeth, Novartis, and Roche) and faculty positions at Harvard and Johns Hopkins Medical School. He is a strong believer of open innovation networks, and in his roles he has been collaborating extensively with different stakeholders to speed up the clinical application of scientific research through public–private partnerships. He has served in several national and international scientific boards: F-NIH Inflammation/Immunity; MRC, Inflammation/ Immunology Initiative—Rheumatoid Arthritis Consortium; MRC, "Stratified Medicine, SLE Consortium; IMI, ABIRISK, EU/EFPIA"; The PML Consortium. Claudio has over 200 publications in national and international peer-reviewed journals. He has also co-edited three books in the field of Biomarkers and Precision Medicine: Biomarkers in Drug Development: A Handbook of Practice Application and Strategy, (2010), Clinical and Statistical Considerations in Personalized Medicine, (2014), and Handbook of Biomarkers & Precision Medicine (2019 in press).

Mark D. Fidock, PhD, is Vice President of Precision Medicine Laboratories within the IMED Biotech Unit, AstraZeneca, Cambridge, UK. He received his PhD from the University of East Anglia, UK, in Molecular Pharmacology. Mark is a trained molecular biologist/pharmacologist and gained extensive experience in drug development early in his career while at Pfizer Ltd, Sandwich, UK. He supported the development and approval of four new therapies and provided leadership for the Biomarker and Translational Medicine activities across multiple therapeutic areas. Mark joined AstraZeneca in 2013, appointed to the role of Head of Precision Medicine Laboratories and a member of the Precision Medicine and Genomics Leadership Team. He has overall accountability for all clinical testing activities that enables diagnostic development and regulatory approval. Mark leads a global multidisciplinary team providing strategy and direction in the development of Companion Diagnostic tests linked to targeted therapeutics across Oncology; Respiratory; and Cardiovascular, Renal, & Metabolism. These laboratory tests are deployed across both early- and late-phase clinical trials with the intent for global regulatory approval. By 2019 Mark had achieved >25 regulatory approved companion diagnostic tests across 3 major markets (USA, EU and Japan) linked to 4 AstraZeneca targeted therapies. Mark has a passion for innovative diagnostic science, driving research such as the use of artificial intelligence across tissue and molecular diagnostics to enable approaches that improve clinical testing outcomes with the ambition to change clinical care pathways. Mark has published more than 30 peer-reviewed articles in scientific journals and an active editor of the Journal of Precision Medicine. As an internationally recognized expert in Precision Medicine he holds an honorary position at the University of Cambridge and a member of the Institute of Translational and Stratified Medicine, Plymouth, scientific advisory board.

Alain J. van Gool, PhD, is a professor of Personalized Healthcare and heads the Translational Metabolic Laboratory at the Radboud university medical center, Nijmegen Netherlands, with a strong passion in the application of biomarkers in translational medicine and personalized healthcare. Being trained as biochemist and molecular biologist, Alain worked at pharmaceutical industry (Organon, Schering-Plough, MSD), academia, applied research institutes, and university medical centers in EU, USA, and Singapore. He has been leading technology-based laboratories, cross-functional expert teams, therapeutic project teams, and public–private consortia, many of which were focused on the discovery, development, and implementation of translational biomarkers in a variety of therapeutic areas. His technical expertise resides most strongly in molecular profiling through x-omics, analytical biomarker development, and applications in translational scientific research. Alain is a strong believer of public–private partnerships and thrives to work with specialists to translate research findings to applications. With that background, he currently also acts as Strategic Advisor to the Executive Board of Radboudumc, coordinates the Radboudumc Technology Centers, is Scientific Lead Technologies of DTL (the Dutch Techcenter for Life Sciences), is Chair Biomarker Platform of EATRIS (the European infrastructure for Translational Medicine), is co-initiator of Health-RI (the Netherlands Personalized Medicine and Health Research Infrastructure), and project leader and PI of the Netherlands X-omics Initiative, thus contributing to the organization and coordination of local, national, and European technology infrastructures. Alain has published 40 peer-reviewed articles in scientific journals and is a frequently invited keynote speaker at scientific conferences and public events. Complementing his daily work, he enjoys contributing as translational biomarker scientist to editorial boards of scientific journals, funding agencies, and scientific advisory boards of start-up enterpreneurs, multinational companies, diagnostic organizations, public–private consortia, and conference organizers.


"This handbook is hardcover and was published at CRC Press / Taylor and Francis Group in 2019. From the first time I opened it, I could notice that the printing and quality of paper is exceptional. The handbook has 631 pages and very nicely coloured figures, schemes and drawings, that are very easily understandable and very helpful in processing the text of the handbook. The field of biomarkers is approached in this book in a scholastic manner, starting from the first discovery and use of biomarkers to the regulatory approaches in the field of precision medicine, passing from preclinical studies, translational medicine, clinical trials, and implementation in practice. Several case-studies are presented related to HIV diagnosis, infertility, coagulation, BRCA identification, but also case-studies related to biomarker-based clinical trials... This handbook is complete, exact, succinct and unique, and gathers information from different perspectives, useful to different players from the field of biomarkers in precision medicine, making it a an excellent tool for learning. First of all, I would recommend this handbook to researchers from the medical and connected fields. It could help the development of this engaging field of personalized care due to highly accurate and relevant data presented by the authors. Secondly, I would recommend it to medical doctors from all specialties since it offers a deep and comprehensive approach into a field that only a few decades ago was hardly recognized and since the practice of medicine in most specialties during the following years will, for sure, include a personalized approach to
patients. Both these addressees, researchers and medical practitioners, can make a difference. Least but not last, I would say that even residents and medical students would benefit from the comprehensive approach that this book provides."
- Costela Serban, ISCB December 2019