ABSTRACT

"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.

Features:

  • 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.

part 1|1 pages

What Is a Biomarker and Its Role in Drug Development?

chapter 1.1|6 pages

Definitions and Conceptual Framework of Biomarkers in Precision Medicine

ByClaudio Carini, Attila A. Seyhan, Mark D. Fidock, Alain J. van Gool

chapter 1.2|11 pages

In Search of Predictive Biomarkers in Cancer Immunotherapy

Establishment of Collaborative Networks for the Validation Process
ByGiuseppe Masucci, Paolo A. Ascierto, Lisa Villabona

chapter 1.3|8 pages

Integrating Personalized Medicine in the Health Care

Payers and Reimbursements
ByDominic Galante, Marylou Buyse

part 2|1 pages

Biomarkers in Preclinical Sciences

chapter 2.1|8 pages

Lost in Translation

The Challenges with the Use of Animal Models in Translational Research
ByAttila A. Seyhan

chapter 2.3|10 pages

The DIAMONDS Platform

Providing Decision Support for Toxicological Assessment Based on Integration of Structural, Toxicological, and Biomarker Information
ByRob H. Stierum, E. Dinant Kroese, Jack W. T. E. Vogels, Harrie Buist, Danyel G. J. Jennen, Eugene P. van Someren

chapter 2.4|13 pages

Predicting the Safety of Drug Targets

An In Silico Perspective
ByJennifer Venhorst, Lars Verschuren, Annemette V. Thougaard, Jorrit J. Hornberg, Tanja Rouhani Rankouhi

chapter 2.5|6 pages

Unwanted Immunogenicity Responses to Biotherapeutics

ByShobha Purushothama, Boris Gorovits

chapter 2.6|7 pages

Biomarker Use for Advanced Screening of Drug-Induced Kidney Injury

ByMartijn J. Wilmer, Frans G. M. Russel

chapter 2.7|10 pages

Soluble Biomarkers for Drug-Induced Cardiotoxicity

ByAmy Pointon, Nick Edmunds

part 3|1 pages

Biomarkers in Translational Medicine

chapter 3.1|9 pages

Lost in Translation

Bridging the Preclinical and Clinical Worlds Concepts, Examples, Successes, and Failures in Translational Medicine
ByAttila A. Seyhan, Claudio Carini

chapter 3.3|7 pages

Crucial Role of High Quality Biosamples in Biomarker Development

ByCornelia Stumptner, Karine Sargsyan, Penelope Kungl, Kurt Zatloukal

chapter 3.4|8 pages

Informatics Solutions for Biomarker Discovery and Personalized Medicine in Clinical Care

ByJan-Willem Boiten, Rita Azevedo, Marinel Cavelaars, André Dekker, Remond J. A. Fijneman, Arnoud van der Maas, Jeroen A. M. Beliën, Gerrit A. Meijer

chapter 3.5|7 pages

Systems Biology Approaches to Identify New Biomarkers

ByLars Kuepfer, Andreas Schuppert

chapter 3.6|6 pages

The Need for Global Collaboration in Translational Medicine

ByFlorence Bietrix, Antoni L. Andreu, Giovanni Migliaccio, Anton E. Ussi

part 4|1 pages

Biomarker-Informed Clinical Trials

chapter 4.1|4 pages

Overview of Biomarker Discovery and Statistical Considerations

ByWeidong Zhang

chapter 4.2|6 pages

Overview of Classical Design in Biomarker-Informed Clinical Trials

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

chapter 4.3|6 pages

Novel Biomarker Guided Clinical Trial Designs

A Practical Review
ByBo Huang, Jing Wang, Robert A. Beckman, Sandeep Menon, Weidong Zhang

chapter 4.4|4 pages

Outsourcing Biomarkers in Clinical Trials

Advantages and Disadvantages
ByRobert Holt

chapter 4.5|5 pages

Case Studies for Biomarker-Based Clinical Trials

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

chapter 4.6|5 pages

Informational Designs and Potential Applications to Rare Disease

ByRobert A. Beckman, Cong Chen

part 5|1 pages

The Road Ahead

chapter 5.1|9 pages

Paving a New Era in Patient Centric Care through Innovations in Precision Medicine

ByMark D. Fidock, Glen Hughes, Alain J. van Gool, Claudio Carini

chapter 5.2|9 pages

Integrating Molecular Testing into Early Stage Clinical Applications

ByJonathan I. Wilde

chapter 5.3|9 pages

Ectopic Gene Deregulations and Chromosome Conformations

Integrating Novel Molecular Testing into Clinical Applications, from Leukemias to Gliomas
ByFrancis Hector Grand, Matthew Salter, Ewan Hunter, Alexandre Akoulitchev

chapter 5.5|7 pages

Implementing Companion Diagnostics

ByMartina Kaufmann

chapter 5.6|14 pages

Publishing Biomedical Research Findings

What are the Odds?
ByAndreas Scherer

chapter 5.7|8 pages

Computational Biology

Modeling from Molecule to Disease and Personalized Medicine
ByNina Kusch, Lisa Turnhoff, Andreas Schuppert

chapter 5.8|8 pages

Expectations for Quality Control

Challenges with Multiplex Assays
ByJohn L. Allinson

part 6|1 pages

Lessons from the Past and Pioneers of the Future

chapter 6.1|12 pages

Development of Maraviroc and the Companion Diagnostic HIV Tropism Assay

ByElna van der Ryst, James Demarest, Jayvant Heera, Mike Westby, Charles Knirsch

chapter 6.2|12 pages

Case Studies

Infertility and Anti-Müllerian Hormone
ByMartin Hund, Joan-Carles Arce

chapter 6.3|6 pages

Factor Xa

Case Study
ByBoris Gorovits

chapter 6.4|14 pages

Delivering Companion Diagnostics for Complex Biomarkers

The BRCA Companion Diagnostic Story for LYNPARZA− (Olaparib)
ByMaria C. M. Orr, Simon P. Dearden

chapter 6.5|7 pages

Delivering the Personalized Medicine Promise

Best Practice Recommendations for Designing Biomarker Development Processes
ByKaren K. Y. Lam, Mary Zacour, Ji-Young V. Kim, Zsuzsanna Hollander, Rhonda Wideman, Raymond T. Ng, Scott J. Tebbutt, Bruce M. McManus

chapter 6.6|8 pages

Development, Launch, and Adoption of a Pharmacodiagnostic

Planning versus Reacting for Pharmaceutical Companies
ByMichael C. Montalto, George A. Green

chapter 6.7|7 pages

Biomarker Consortia to Drive BM Discovery and Clinical Applications in the UK

Lessons Learned from RA-MAP
ByJohn D. Isaacs, Sarah Brockbank, Ayako Pedersen, Andrew P. Cope

chapter 6.8|7 pages

Measuring the Success of U.S. Biomarker Consortia

ByJoseph P. Menetski

chapter 6.9|6 pages

The Value of Public–Private Partnerships in the Netherlands

ByHanna L. Groen, Nico L. U. van Meeteren

part 7|1 pages

Paving a New Era of Personalized Medicine

chapter 7.1|8 pages

A New Era of Personalized Medicine

Emerging Technologies—Functional Proteomics, Companion Diagnostics, and Precision Cancer Medicine
ByMichela Perani, Peter J. Parker

chapter 7.2|10 pages

The Impact of Epigenetics on the Future of Personalized Medicine

ByJane Mellor

chapter 7.4|9 pages

Innovation in Metabonomics to Improve Personalized Health Care

ByNelma Pértega-Gomes, Massimo Loda

chapter 7.5|11 pages

Predictive Cancer Biomarkers

Current Practice and Future Challenges
ByVladimira Koudelakova, Marian Hajduch

chapter 7.6|11 pages

Glycomics as an Innovative Approach for Personalized Medicine

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

chapter 7.7|3 pages

miRNAs as Novel Biomarkers for Health and Disease

ByMaría Laura García Bermejo

chapter 7.8|7 pages

Targeted Proteomics for Absolute Quantification of Protein Biomarkers in Serum and Tissues

ByCoşkun Güzel, Christoph Stingl, Frank Klont, Roel Tans, Esther Willems, Rainer Bischoff, Alain J. van Gool, Theo M. Luider

chapter 7.9|7 pages

Integrated Omics for Tissue-Based Mapping of the Human Proteome

ByCecilia Lindskog

chapter 7.10|10 pages

Proteogenomics and Multi-omics Data Integration for Personalized Medicine

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

chapter 7.11|7 pages

Sequencing Approaches for Personalized Cancer Therapy Selection in Pathology

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

chapter 7.12|6 pages

Mass Spectrometry Imaging

Enabling Comprehensive Local Analysis of Molecular Biomarkers in Tissue for Personalized Medicine
ByTiffany Porta, Steven W. M. Olde Damink, Ron M. A. Heeren

chapter 7.13|7 pages

Insights of Personalized Medicine from the Gut Microbiome

Lessons Learned from Celiac Disease
ByGloria Serena, Anna Sapone, Alessio Fasano

part 8|1 pages

The Next Frontiers of Therapeutic Target Areas

chapter 8.1|15 pages

Precision Mucosal Sampling and Biomarkers in Allergic Rhinitis and Asthma

ByAkhilesh Jha, Ryan S. Thwaites, Nehmat Singh, Trevor T. Hansel

chapter 8.2|12 pages

Stratified Medicine in Autoimmune Diseases

ByJohn A. Reynolds, Ian N. Bruce

chapter 8.3|20 pages

Biomarkers of Systemic Lupus Erythematosus

ByHiroshi Kato, Andras Perl

chapter 8.4|13 pages

Advances in Cardiovascular Diagnostics

Use of Cardiac Biomarkers in Clinical Research and Care of the Patient
ByMark B. Effron, Zahoor Khan

chapter 8.5|6 pages

Biomarkers for Psychiatric Disease

An Overview
ByRoy H. Perlis

chapter 8.6|9 pages

Progressing Towards Precision Psychiatry

Current Challenges in Applying Biomarkers in Psychiatry
ByVictor L. Kallen, Brisa S. Fernandes

chapter 8.7|9 pages

Identification of Biomarkers for the Diagnosis, Prediction, and Progression of Alzheimer’s Disease

ByBennett P. Greenwood, Vladimir Tolstikov, Paula P. Narain, Jeremy Chaufty, Viatcheslav R. Akmaev, Vivek Vishnudas, Stephane Gesta, Eric J. Nestler, Rangaprasad Sarangarajan, Niven R. Narain, Michael A. Kiebish

chapter 8.8|13 pages

Biomarkers in Lung Cancer

ByGeorge Santis, Richard Lee, Crispin T. Hiley

part 9|1 pages

Lessons Learned

chapter 9.1|12 pages

Translational Science in Modern Pharmaceutical R&D

The Influence of Organizational Structures on Successes and Failures
ByPeter M. A. Groenen

chapter 9.2|6 pages

Biomarker Qualification and Companion Diagnostics

Failure and Success of Regulatory Processes
ByFederico Goodsaid

chapter 9.3|9 pages

Not All Biomarkers Are Worthwhile

An Unsuccessful Use and Pitfalls to Avoid
ByAbdel-Baset Halim

chapter 9.4|17 pages

Research Reproducibility

The Case for Standards
ByLeonard P. Freedman, Raymond H. Cypess

chapter 9.5|12 pages

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

ByAmrit Singh, Casey P. Shannon, Kim-Anh Lê Cao, Scott J. Tebbutt

chapter 9.6|6 pages

Can Health Care Be Made More Affordable by the Use of Biomarkers?

ByReinier L. Sluiter, Wietske Kievit, Gert Jan van der Wilt

chapter 9.7|9 pages

Omics Driven Medicine for Democratizing Health Care Delivery and Health Economics

ByMichael A. Kiebish, Viatcheslav R. Akmaev, Vivek Vishnudas, Stephane Gesta, Rangaprasad Sarangarajan, Niven R. Narain