Bench To Bedside Translating Pre Clinical Research Into Clinical Trials For Childhood Brain Tumors

umors arising in the brain are the most common solid cancers in children. They are the major cause of childhood cancer deaths. Indeed, more children die from brain tumors than any other disease. Despite improvement in cure rates towards the end of the 20th century, survival statistics have remained unchanged over the past two decades and remain at a level well below that of other childhood cancers, such as leukemia. Also, survivors have a high risk of significant permanent adverse side effects that require a lifetime of clinical management, significantly impacting health systems and quality of life for patients. The lack of advancements in childhood brain tumor treatment was due to deficiencies in knowledge about the underlying biological causes. However, pediatric neuro-oncology has undergone an exciting and dramatic transformation during the past 10 years, driven by advances in genomic technology and international collaboration. Armed with this new knowledge, there is a tremendous opportunity to personalize the treatment by developing novel therapeutic approaches that are tailored to each molecular subtype of these devastating brain cancers to improve the cure rate while minimizing toxicities.

This textbook covers all the steps in manufacturing a biomedical product from bench to bedside. It specifically focuses on quality assurance and management and explains the different good practice principles in the various phases of product development as well as how to fulfill them: Good laboratory practice, good manufacturing practice and good clinical practice. It provides readers with the know-how to design biomedical experiments to ensure quality and integrity, to plan and conduct standard preclinical studies and to assure the quality of the final manufactured biomedical products. Importantly, it also addresses ethical concerns and considerations. The book discusses the guidelines and ethical considerations for preclinical and clinical studies, to allow readers to identify safety concerns regarding biomedical products and to improve pre-clinical studies for the development of better products. This textbook is a valuable guide for biomedical students (B.Sc., M.S., and Ph.D. students) in the field of molecular medicine, medical biotechnology, stem cell research and related areas, as well as for professionals such as quality control staff, tissue bankers, policy-makers and health professionals.

Due to injuries sustained in sports and in combat, interest in traumatic brain injury (TBI) has never been greater. This book will fulfill a gap in understanding of what is occurring in the brain following injury that can subsequently be detected in biological fluids and imaging.

One of the most crucial skills a clinician, scientist, or student can learn is to create, conduct, and interpret the conclusions of a clinical study. Critical Thinking in Clinical Research teaches these fundamentals in four distinct sections, called "units": the first unit focuses on issues surrounding the design of a study such as population, question selection, randomization, and blinding; Unit 2 presents statistical methods such as analyzing data collected, how to present and discuss the data concisely; the third unit covers practical aspects such as methodology, organizational considerations, principles of trial conduct and reporting; and the final unit delves into study designs, providing the advantages and drawbacks of each design style. Each chapter begins with a short introduction, followed by a hypothetical case that challenges the reader to make decisions, to consider pros and cons of specific approaches, and to evaluate options based on specific conditions. Knowing how to critically read and understand scientific papers and to collect, analyze, and interpret research data, which they in turn can then present in their own scientific manuscript makes this book the perfect resource for anyone looking to contribute to the wealth of scientific and medical inquiry.

This book provides a comprehensive coverage of the state of the art in precision medicine in stroke. It starts by explaining and giving general information about precision medicine. Current applications in different strokes types (ischemic, haemorrhagic) are presented from diagnosis to treatment. In addition, ongoing research in the field (early stroke diagnosis and estimation of prognosis) is extensively discussed. The final part provides an in-depth discussion of how different interdisciplinary areas like artificial intelligence, molecular biology and genetics are contributing to this area. Precision Medicine in Stroke provides a practical approach to each chapter, reinforcing clinical applications and presenting clinical cases. This book is intended for all clinicians that interact with stroke patients (neurologists, internal medicine doctors, general practitioners, neurosurgeons), students and basic researchers.

The study of the brain continues to expand at a rapid pace providing fascinating insights into the basic mechanisms underlying nervous system illnesses. New tools, ranging from genome sequencing to non-invasive imaging, and research fueled by public and private investment in biomedical research has been transformative in our understanding of nervous system diseases and has led to an explosion of published primary research articles. Diseases of the Nervous System, Second Edition, summarizes the current state of basic and clinical knowledge for the most common neurological and neuropsychiatric conditions. In a systematic progression, each chapter covers either a single disease or a group of related disorders ranging from static insults to primary and secondary progressive neurodegenerative diseases, neurodevelopmental illnesses, illnesses resulting from nervous system infection and neuropsychiatric conditions. Chapters follow a common format and are stand-alone units, each covering disease history, clinical presentation, disease mechanisms and treatment protocols. Dr. Sontheimer also includes two chapters which discuss common concepts shared among the disorders and how new findings are being translated from the bench to the bedside. In a final chapter, he explains the most commonly used neuroscience jargon. The chapters address controversial issues in current day neuroscience research including translational research, drug discovery, ethical issues, and the promises of personalized medicine. This new edition features new chapters on Pain and Addiction to highlight the growing opioid crisis and the ethical issue of prescriptions drug abuse. This book provides an introduction for course adoption and an introductory tutorial for students, scholars, researchers and medical professionals interested in learning the state of the art concerning our understanding and treatment of diseases of the nervous system. Each chapter includes suggested further readings and/or journal club recommendations. 2016 PROSE Award winner of the Best Textbook Award in Biological and Life Sciences Provides a focused tutorial introduction to the core diseases of the nervous system Includes comprehensive introductions to Stroke, Epilepsy, Alzheimer's Disease, Parkinson's Disease, Huntington's Disease, ALS, Head and Spinal Cord Trauma, Multiple Sclerosis, Brain Tumors, Depression, Schizophrenia and many other diseases of the nervous system Covers more than 40 diseases from the foundational science to the best treatment protocols Includes discussions of translational research, drug discovery, personalized medicine, ethics, and neuroscience New Edition features two new chapters on Pain and Addiction

While the first edition of this book provided a succinct introduction to pediatric neuro-oncology, biological knowledge of childhood CNS tumors has “exploded” over the past few years and a new edition of this textbook is needed to keep it up-to-date. This updated edition will include chapters on cancer predisposition in children with brain tumors, gliomas, embryonal brain tumors, ependymoma, CNS-GCT, targeted therapies in pediatric brain tumors, and long-term sequelae. New developments covered include the following: - Techniques like DNA methylation have improved the diagnostic process, and have led to an integrated diagnosis of histology, ICH and methylation. - Tumor pathways have been detected, which defines more subgroups within a tumor entity, and results in more individualized treatment for the patient. - Therapeutic options outside the standard combination of surgery, chemotherapy, and radiation have either been implemented within the last years, or are currently under consideration. This book will be aimed at pediatric oncologists and neurooncologists, neurosurgeons, radiation oncologists. Chapters detailing quality of life and supportive care will make this 2nd edition a useful resource for nurses, social workers, physiotherapists, and occupational therapists alike.

Few areas of biomedical research provide greater opportunities to capitalize upon the revolution in genomics and molecular biology than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy in the Brain: From Bench to Bedside represents the definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped to create the field as well as those who are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects which would be of interest to anyone who is currently using or contemplating exploring gene therapy for neurobiological applications. Basic gene transfer technologies are discussed, with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells. Numerous research applications are reviewed, particularly in complex fields such as behavioral neurobiology. Several preclinical areas are also covered which are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis. Among the most exciting advances in recent years has been the use of neurological gene therapy in human clinical trials, including Parkinson's disease, Canavan disease and Batten disease. Finally, readers will find "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use. This work provides up-to-date information and key insights into those gene therapy issues which are important to both scientists and clinicians focusing upon the brain and central nervous system.