Novel Therapeutics from Modern Biotechnology: From Laboratory to Human Testing / Edition 1

by Dale L. Oxender, Leonard E. Post

Overview

A cover story of Business Week Magazine in January 1984 stated "Biotech Comes of Age". In February 1986, Venture Magazine had a cover article entitled "The Biotech Revolution is Here". This article went on to say "New Genetic Technologies Will Transform Our Lives". These announcements were made many years after the first biotechnology companies, such as Genentech, Cetus, Amgen and Biogen, were formed-to commercialize the "New Biology". . At the time of writing this book, there are over 1300 biotech companies developing new technologies or identifying potential biotech drugs. Most of these companies were started in the height of the "high-technology hype", although companies are still forming as the technology advances. A more recent survey showed only a relatively small number of Food and Drug Administration (FDA) approvals among over several hundred biotechnology products now in clinical trial. One could ask why it has taken so long to produce biotechnology products. Part of the reason is that each new class of biotech products brings with it a set of problems that need to be solved before they enter clinical trials. These problems are often unique to biotechnology products, such as peptides, proteins, monoclonal antibodies, nucleic acids and cellular therapies.

Product Details

ISBN-13:	9783540650256
Publisher:	Springer Berlin Heidelberg
Publication date:	12/22/1998
Series:	Handbook of Experimental Pharmacology , #137
Edition description:	1999
Pages:	248
Product dimensions:	6.10(w) x 9.25(h) x (d)

Table of Contents

1 Overview of Regulatory Expectations for Introducing Novel Therapies into Clinical Trials.- A. Introduction.- B. Roles of Regulatory Scientists.- C. Product Development and Availability.- D. Data Requirements.- E. Manufacturing.- F. Preclinical Safety Testing.- G. Case-By-Case Approach.- H. Testing Goals.- I. Study Design.- J. Defining Exposure.- K. Product-Specific Concerns.- L. Accessibility of Preclinical Safety Data.- M. Clinical Studies.- N. Summary.- References.- 2 Preparation of Clinical Trial Supplies of Biopharmaceuticals.- A. Introduction.- B. Preclinical Studies.- C. Clinical Supplies.- D. Purification of rDNA-Derived Anti-RSV MAb.- E. Product Quality Issues.- F. Process Design and Validation.- G. Process Economics and the Future of Chromatography.- H. Conclusions.- References.- 3 Proteins as Drugs: Analysis, Formulation and Delivery.- A. Introduction.- B. The Analysis of Protein Pharmaceuticals.- C. Formulation.- D. Delivery.- References.- 4 Strategies for Dealing With the Immunogenicity of Therapeutic Proteins.- A. Introduction.- B. Case Histories of Protein Therapeutic Development.- C. Strategies Under Development for Increasing the Therapeutic Value of Proteins and Peptides.- D. Choosing the Proper Strategy for a Protein Therapeutic.- E. The Future of Protein Therapeutics.- References.- 5 Targeted Toxin Hybrid Proteins.- A. Introduction.- B. Preclinical Development of Anti-Tac(Fv) Toxins.- C. Preclinical Development of Inerleukin 6-PE4E.- D. Summary.- References.- 6 SB 209763: A Humanized Monoclonal Antibody for the Prophylaxis and Treatment of Respiratory Syncytial Virus Infection.- A. Introduction.- B. Early Challenges in the Development of SB 209763.- C. Preclinical Evaluation Prior to Testing in Humans.- D. Challenges for the Early Clinical Development of SB 209763.- E. Conclusion.- References.- 7 Preclinical Development of Antisense Therapeutics.- A. Introduction.- B. Pharmacology of Antisense Oligodeoxynucleotides.- C. Pharmacokinetics and Toxicity of Oligodeoxynucleotide Therapeutics.- D. Chemistry, Manufacture and Control of Phosphorothioate Oligodeoxynucleotide Drugs.- E. Formulation and Drug Delivery of Oligodeoxynucleotides.- F. Summary.- References.- 8 Formulation and Delivery of Nucleic Acids.- A. Introduction.- B. Formulation of DNA.- C. Delivery to Target Cells.- D. Cell Entry.- E. Endosomal Release.- F. Nuclear Localization.- G. Gene Expression.- References.- 9 Safe, Efficient Production of Retroviral Vectors.- A. Introduction.- B. Vectors.- C. Production of Retroviral Vectors.- D. Downstream Processing.- E. GMP Production of Retroviral Vectors.- F. In-Process Assays.- G. Quality Control.- H. Safety.- I. Summary and Conclusions.- References.- 10 Clinical Systems for the Production of Cells and Tissues for Human Therapy.- A. Introduction.- B. Cell Therapy and Tissue Engineering.- C. Critical Requirements for Ex Vivo Cell Production.- D. Cell-Culture Devices and Procedures.- E. Applications for On-Site Delivery of Therapeutic Cell Production.- F. Summary.- References.

Editorial Reviews

Reviewer: Thomas L. McDonald, PhD (University of Nebraska Medical Center)
Description: This book is an overview of problems and issues confronted in developmental work related to the use of biologicals for therapeutic purposes. The topics and examples provided cover a broad range of common problems associated with biotechnology product development, but more importantly, the editors and contributors provide and document how these problems were approached and solved. The book is well referenced and indexed for ease in navigation.
Purpose: The purpose is to introduce basic, common problems and considerations that must be addressed in developing a "biotech" therapeutic prior to its entry into clinical trials. This book is an excellent resource, in particular with the litany of references that document approaches to solutions regarding biologicals as therapeutics.
Audience: The audience is not indicated by the editors, but from my point of view as a research scientist and president of a University Technology Licensing Corporation, I would have greatly benefited from the information provided here if it had been published ten years ago. I would consider this book a "must read" for scientists in applied research, graduate students, and all personnel in technology transfer/intellectual property offices.
Features: The editors provide an introductory overview of the problems that are associated with introducing biological material into a living being for a therapeutic benefit. Discussions of complex issues related to production, toxicity, immunogenicity, and targeted delivery are a highlight. Although the depth of discussion on each of the topics is somewhat limited, the contributors compensate by providing sufficient references for further reading. The subject index is well designed and very helpful in navigating through the text. I found several of the illustrations, in particular those in the chapter on "Formulation and Delivery of Nucleic Acids," useful.
Assessment: This is a wonderful blend of presenting the problems that must be addressed when considering the use of a biological product as a therapeutic, and strategic approaches that companies have used to solve them. The strategies exemplified here are beyond just mere examples as they create an awareness of confounding issues that would be useful resources to scientists performing basic research in related areas. Although I greatly enjoyed the content of the book, the price seems very high to me.

Doody's Review Service

Reviewer:This is a wonderful blend of presenting the problems that must be addressed when considering the use of a biological product as a therapeutic, and strategic approaches that companies have used to solve them. The strategies exemplified here are beyond just mere examples as they create an awareness of confounding issues that would be useful resources to scientists performing basic research in related areas. Although I greatly enjoyed the content of the book, the price seems very high to me.
Description:
Purpose:This book is an overview of problems and issues confronted in developmental work related to the use of biologicals for therapeutic purposes. The topics and examples provided cover a broad range of common problems associated with biotechnology product development, but more importantly, the editors and contributors provide and document how these problems were approached and solved. The book is well referenced and indexed for ease in navigation.
Audience:The purpose is to introduce basic, common problems and considerations that must be addressed in developing a "biotech" therapeutic prior to its entry into clinical trials. This book is an excellent resource, in particular with the litany of references that document approaches to solutions regarding biologicals as therapeutics.
Features:The audience is not indicated by the editors, but from my point of view as a research scientist and president of a University Technology Licensing Corporation, I would have greatly benefited from the information provided here if it had been published ten years ago. I would consider this book a "must read" for scientists in applied research, graduate students, and all personnel in technology transfer/intellectual property offices.
Assessment:The editors provide an introductory overview of the problems that are associated with introducing biological material into a living being for a therapeutic benefit. Discussions of complex issues related to production, toxicity, immunogenicity, and targeted delivery are a highlight. Although the depth of discussion on each of the topics is somewhat limited, the contributors compensate by providing sufficient references for further reading. The subject index is well designed and very helpful in navigating through the text. I found several of the illustrations, in particular those in the chapter on "Formulation and Delivery of Nucleic Acids," useful.