Emerging Protein Biotherapeutics PDF

Preface

Over the last two decades, development of protein biotherapeutics has dramatically increased and has become an essential part of modern medical treatments for cancers and autoimmune diseases. Protein biotherapeutics are expected to be the major part of the pharmaceutical market in the next few years. The current estimates of different genes in the human genome indicate that there are 25,000 to 40,000 functional genes. Together with alternative splicing of these genes and posttranslational modification of proteins, human genes have the potential to code for a large number of functionally distinct protein molecules. As more and more novel protein molecules are being linked with the underlying disease mechanisms, these estimates pose an immense challenge to modern medicine. On the other hand, these estimates represent a tremendous opportunity in terms of harnessing the potential of targeting these proteins to develop protein biotherapeutics to alleviate disease.

Protein biotherapeutics have several distinct advantages over small-molecule drugs, and serve highly specific and complex biologic functions that cannot be mimicked by simple chemical compounds. Because the action of protein biotherapeutics is highly specific, their interference with normal biologic processes and induction of adverse effects is very limited and usually well tolerated. In addition, protein biotherapeutics have the potential for far-reaching patent protection due to the unique nature of functions of these molecules. This aspect is particularly attractive for biopharmaceutical and biotechnology companies because it may provide them with assurance for return on the large amount of investment typically needed to develop protein biotherapeutics. Together, these advantages make protein biotherapeutics not only very attractive when compared with small-molecule drugs but also help to propel the field further.

Targeting protein antigens has been the most highly investigated area of basic medical research for over two decades. These investigations have benefited from the enormous growth in our understanding of mechanisms of disease progression and the explosion in the knowledge involved in regulation of normal and pathological immune response. In addition, much has been learned about the molecular mechanisms of tumor progression, escape of tumor cells from host surveillance mechanisms, and from discovery of the novel protein molecules important in tumor growth. These new findings are shifting currently established paradigms and providing a foundation for novel drug design efforts. This exemplary growth in our knowledge has provided us with several new protein biotherapeutics that have advanced to human clinical trials, while many more are still being tested in preclinical settings.

As in other rapidly evolving fields, protein biotherapeutics is still an area of intensive research. Progress made in this area is not necessarily congruent and is often difficult to simulate into a cogent whole. A concise account of advances in this area in a readily available format is urgently needed. The aim of Emerging Protein Biotherapeutics is to make readily available the major research important in the exploitation of protein targets for developing therapeutic strategies for human diseases, in a single volume. Under the auspices of Taylor & Francis Group/CRC Press, I have undertaken the task to concisely consolidate current knowledge of key protein targets important in autoimmunity and cancers focusing on both basic aspects and their clinical application.

In this volume, a number of leading scientists in the autoimmunity and cancer fields have attempted to cover many aspects of biology of protein targets, ranging from the in vivo role in the disease process to various strategies to exploit the development of these targets for therapeutic use. Relevant background information, a discussion of the clinical implications of biology regulated by these protein targets, an account of preclinical and clinical testing of various candidates, and a useful bibliography is provided in each chapter. This consolidated information will help the reader to make more detailed analysis and will make the study of protein biotherapeutics accessible at all levels of expertise. This book offers researchers an efficient and rational way to organize the theoretical and experimental knowledge currently available and presents a framework that is readily applicable to develop strategies for targeting proteins for clinical applications. This book will also be highly valuable for basic researchers to understand the disease process.

I express my sincere thanks to all of my contributors for their excellent effort and undertaking this project with such enthusiasm, to Judith E. Spiegel for commissioning me to edit this volume, and to Patricia Roberson and the staff of Taylor & Francis Group for help with publication coordination.

I anticipate that many researchers will be inspired by the comprehensive work presented here and will contribute to this field by further investigations. Looking ahead, I predict that this field will gain much more importance in the future because of the rising awareness of use of protein biotherapeutics and their effect on the disease process. In coming years researchers studying protein biotherapeutics will play an important role in the development of therapeutic strategies to target many human diseases including cancers, autoimmune disease, and others. The information provided in this book has broad implications and will be of value to biologists, immunologists, cancer biologists, biotechnology and pharmaceutical scientists, researchers in basic medical sciences, and clinicians as well.