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

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Bench to Bedside: Translating Pre-clinical Research Into Clinical Trials for Childhood Brain Tumors

Bench to Bedside: Translating Pre-clinical Research Into Clinical Trials for Childhood Brain Tumors
Author:
Publisher: Frontiers Media SA
Total Pages: 108
Release: 2023-09-05
Genre: Medical
ISBN: 2832532926
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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.

The CTSA Program at NIH

The CTSA Program at NIH
Author: Institute of Medicine
Publisher: National Academies Press
Total Pages: 179
Release: 2013-10-09
Genre: Medical
ISBN: 0309284740
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In 2006 the National Institutes of Health (NIH) established the Clinical and Translational Science Awards (CTSA) Program, recognizing the need for a new impetus to encourage clinical and translational research. At the time it was very difficult to translate basic and clinical research into clinical and community practice; making it difficult for individual patients and communities to receive its benefits. Since its creation the CTSA Program has expanded, with 61 sites spread across the nation's academic health centers and other institutions, hoping to provide catalysts and test beds for policies and practices that can benefit clinical and translation research organizations throughout the country. The NIH contracted with the Institute of Medicine (IOM) in 2012 to conduct a study to assess and provide recommendations on appropriateness of the CTSA Program's mission and strategic goals and whether changes were needed. The study was also address the implementation of the program by the National Center for Advancing Translational Sciences (NCATS) while exploring the CTSA's contributions in the acceleration of the development of new therapeutics. A 13-member committee was established to head this task; the committee had collective expertise in community outreach and engagement, public health and health policy, bioethics, education and training, pharmaceutical research and development, program evaluation, clinical and biomedical research, and child health research. The CTSA Program at NIH: Opportunities for Advancing Clinical and Translational Research is the result of investigations into previous program evaluations and assessments, open-session meetings and conference class, and the review of scientific literature. Overall, the committee believes that the CTSA Program is significant to the advancement of clinical and translational research through its contributions. The Program would benefit from a variety of revisions, however, to make it more efficient and effective.

Pediatric Neuro-oncology

Pediatric Neuro-oncology
Author: Katrin Scheinemann
Publisher: Springer
Total Pages: 0
Release: 2024-09-22
Genre: Medical
ISBN: 9783031620164
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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.

The Role of NIH in Drug Development Innovation and Its Impact on Patient Access

The Role of NIH in Drug Development Innovation and Its Impact on Patient Access
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
Total Pages: 103
Release: 2020-01-27
Genre: Medical
ISBN: 0309498511
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To explore the role of the National Institutes of Health (NIH) in innovative drug development and its impact on patient access, the Board on Health Care Services and the Board on Health Sciences Policy of the National Academies jointly hosted a public workshop on July 24â€"25, 2019, in Washington, DC. Workshop speakers and participants discussed the ways in which federal investments in biomedical research are translated into innovative therapies and considered approaches to ensure that the public has affordable access to the resulting new drugs. This publication summarizes the presentations and discussions from the workshop.

Modern Methods of Clinical Investigation

Modern Methods of Clinical Investigation
Author: Institute of Medicine
Publisher: National Academies Press
Total Pages: 241
Release: 1990-02-01
Genre: Medical
ISBN: 0309042860
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The very rapid pace of advances in biomedical research promises us a wide range of new drugs, medical devices, and clinical procedures. The extent to which these discoveries will benefit the public, however, depends in large part on the methods we choose for developing and testing them. Modern Methods of Clinical Investigation focuses on strategies for clinical evaluation and their role in uncovering the actual benefits and risks of medical innovation. Essays explore differences in our current systems for evaluating drugs, medical devices, and clinical procedures; health insurance databases as a tool for assessing treatment outcomes; the role of the medical profession, the Food and Drug Administration, and industry in stimulating the use of evaluative methods; and more. This book will be of special interest to policymakers, regulators, executives in the medical industry, clinical researchers, and physicians.

Critical Thinking in Clinical Research

Critical Thinking in Clinical Research
Author: Felipe Fregni
Publisher: Oxford University Press
Total Pages: 480
Release: 2018-03-13
Genre: Medical
ISBN: 0199324514
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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.

Biomarkers for Traumatic Brain Injury

Biomarkers for Traumatic Brain Injury
Author: Svetlana Dambinova
Publisher: Royal Society of Chemistry
Total Pages: 247
Release: 2012
Genre: Medical
ISBN: 1849733899
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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.

Hormones and Aging

Hormones and Aging
Author: Gerald Litwack
Publisher: Academic Press
Total Pages: 658
Release: 2021-03-09
Genre: Science
ISBN: 0323855490
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Hormones and Aging, Volume 115 in the Vitamins and Hormones series, highlights advances in the field, with this new volume presenting timely topics, including hypothalamic aging and hormones, endocannabinoids and aging-inflammation, neuroplasticity, mood and pain, the impact of hormones and bone loss across the menopause transition, and much more. Provides the authority and expertise of leading contributors from an international board of authors Presents the latest release in the Vitamins and Hormones series Includes the latest information on Hormones and Aging

Diseases of the Nervous System

Diseases of the Nervous System
Author: Harald Sontheimer
Publisher: Elsevier
Total Pages: 502
Release: 2021-05-20
Genre: Medical
ISBN: 0128213965
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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

Gene Therapy of the Central Nervous System: From Bench to Bedside

Gene Therapy of the Central Nervous System: From Bench to Bedside
Author: Michael G. Kaplitt
Publisher: Gulf Professional Publishing
Total Pages: 380
Release: 2006
Genre: Medical
ISBN: 9780123976321
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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.