Did you ever wonder whether doctors want cures, or just treatments?Did you know ... This book reviews recent key, hard-won successes and findings from recent biomedical research. Written by one of the most ardent defenders of the public trust in science, it provides an accessible, detailed look at successes in translational biomedical and clinical research. The author provides an optimistic, forward-looking view for the possibility of change for the public good, cutting through the controversy and gets to very core of each topic. The public can be optimistic about the future of medicine, but only if they learn the facts of these advances, and learn what their doctors should be expected to know.Highly referenced, and filled with interviews from experts and people directly involved in the research behind the new facts in each chapter, this book is a rich source of information on advances in biomedicine that you will want to share with your family & friends.

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.

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.

Many voluntary health organizations fund translational research. An increasing number of these organizations are looking at venture philanthropy as a critical way to advance their missions of helping patients and working to cure disease. A wide range of participants gathered on October 3, 2008 at the Beckman Center of the National Academies of Science for a workshop titled "Venture Philanthropy Strategies Used by Patient Organizations to Support Translational Research." Participants with experience in venture philanthropy shared their experiences and lessons learned in order to improve efficiency and effectiveness in translational research.

The Environmental and Genetic Causes of Autism delves deep into the full body of past and current research to reveal how genetic predispositions and environmental factors can combine to produce the conditions autism and autism spectrum disorders (ASD). To make this groundbreaking volume, Dr. James Lyons-Weiler combed through the past fifty years of published research on autism, exploring subjects such as genetic variation, mechanisms of neurotoxicity of metals and pesticides, and the central and combined roles of each in causing autism. Lyons-Weiler provides a major overview of all aspects of the condition of autism, reviews changes in diagnoses and treatments, and explains how genetic information can be used to tailor effective treatments, and sometimes reversals, of the symptoms. He also presents practical forward-looking suggestions on how to design future studies to facilitate the discovery of biomarkers for autism risk and how to classify the full range of autism spectrum disorders. Autism is considered one of the most mystifying conditions of our day, and alarmed scientists, doctors, politicians, and parents are desperately trying to understand why the condition is escalating. According to the CDC, rates in the United States have risen from an estimated one in two thousand children in 1980, to one in sixty-eight in 2012, and a new National Health Interview Survey shows a rate of one in forty-five. By the time you read this book, that number may have changed yet again. While most autism researchers focus on either environmental or genetic causes of autism, Lyons-Weiler’s opus demonstrates that to fully understand the condition and to finally put its rate on the decrease, it is essential to pay attention to the science showing how the two classes of factors interact.

Translational Medicine: Optimizing Preclinical Safety Evaluation of Biopharmaceuticals provides scientists responsible for the translation of novel biopharmaceuticals into clinical trials with a better understanding of how to navigate the obstacles that keep innovative medical research discoveries from becoming new therapies or even making it to clinical trials. The book includes sections on protein-based therapeutics, modified proteins, oligonucleotide-based therapies, monoclonal antibodies, antibody–drug conjugates, gene and cell-based therapies, gene-modified cell-based therapies, combination products, and therapeutic vaccines. Best practices are defined for efficient discovery research to facilitate a science-based, efficient, and predictive preclinical development program to ensure clinical efficacy and safety. Key Features: Defines best practices for leveraging of discovery research to facilitate a development program Includes general principles, animal models, biomarkers, preclinical toxicology testing paradigms, and practical applications Discusses rare diseases Discusses "What-Why-When-How" highlighting different considerations based upon product attributes. Includes special considerations for rare diseases About the Editors Joy A. Cavagnaro is an internationally recognized expert in preclinical development and regulatory strategy with an emphasis on genetic medicines.. Her 40-year career spans academia, government (FDA), and the CRO and biotech industries. She was awarded the 2019 Arnold J Lehman Award from the Society of Toxicology for introducing the concept of science-based, case-by-case approach to preclinical safety evaluation, which became the foundation of ICH S6. She currently serves on scientific advisory boards for advocacy groups and companies and consults and lectures in the area of preclinical development of novel therapies. Mary Ellen Cosenza is a regulatory toxicology consultant with over 30 years of senior leadership experience in the biopharmaceutical industry in the U.S., Europe, and emerging markets. She has held leadership position in both the American College of Toxicology (ACT) and the International Union of Toxicology (IUTOX) and is also an adjunct assistant professor at the University of Southern California where she teaches graduate-level courses in toxicology and regulation of biologics.

Principles of Translational Science in Medicine: From Bench to Bedside, Second Edition, provides an update on major achievements in the translation of research into medically relevant results and therapeutics. The book presents a thorough discussion of biomarkers, early human trials, and networking models, and includes institutional and industrial support systems. It also covers algorithms that have influenced all major areas of biomedical research in recent years, resulting in an increasing numbers of new chemical/biological entities (NCEs or NBEs) as shown in FDA statistics. The book is ideal for use as a guide for biomedical scientists to establish a systematic approach to translational medicine. - Provides an in-depth description of novel tools for the assessment of translatability of trials to balance risk and improve projects at any given stage of product development - New chapters deal with translational issues in the fastest growing population (the elderly), case studies, translatability assessment tools, and advances in nanotherapies - Details IPR issues of translation, especially for public-private-partnerships - Contains contributions from world leaders in translational medicine, including the former NIH director and authorities from various European regulatory institutions

An ideal health care system relies on efficiently generating timely, accurate evidence to deliver on its promise of diminishing the divide between clinical practice and research. There are growing indications, however, that the current health care system and the clinical research that guides medical decisions in the United States falls far short of this vision. The process of generating medical evidence through clinical trials in the United States is expensive and lengthy, includes a number of regulatory hurdles, and is based on a limited infrastructure. The link between clinical research and medical progress is also frequently misunderstood or unsupported by both patients and providers. The focus of clinical research changes as diseases emerge and new treatments create cures for old conditions. As diseases evolve, the ultimate goal remains to speed new and improved medical treatments to patients throughout the world. To keep pace with rapidly changing health care demands, clinical research resources need to be organized and on hand to address the numerous health care questions that continually emerge. Improving the overall capacity of the clinical research enterprise will depend on ensuring that there is an adequate infrastructure in place to support the investigators who conduct research, the patients with real diseases who volunteer to participate in experimental research, and the institutions that organize and carry out the trials. To address these issues and better understand the current state of clinical research in the United States, the Institute of Medicine's (IOM) Forum on Drug Discovery, Development, and Translation held a 2-day workshop entitled Transforming Clinical Research in the United States. The workshop, summarized in this volume, laid the foundation for a broader initiative of the Forum addressing different aspects of clinical research. Future Forum plans include further examining regulatory, administrative, and structural barriers to the effective conduct of clinical research; developing a vision for a stable, continuously funded clinical research infrastructure in the United States; and considering strategies and collaborative activities to facilitate more robust public engagement in the clinical research enterprise.

The aim of the book was to produce the most comprehensive examination of a pandemic that has ever been attempted. By cataloging the full extent of the Zika pandemic, this book will be the most complete history and epistemic contextualization ever attempted to date. The work should function as the primary source for students, researchers, and scholars who need information about the Zika pandemic. This book examines the technical literature, digital and popular literature, and online materials to fully contextualize this event and provide a bona fide record of this event and its implications for the future. It is somewhat serendipitous that while this work was underway, we are going through another pandemic. One of the primary lessons we did not learn by Zika was pandemic events will return repeatedly, and we need to learn from each one of them to prepare the planet for the next one. Just because Zika seemed to have died out does not make it less important. We were lucky that the virus evolved into what seemed to be a less virulent version of itself, and the vector mosquitoes were concentrated elsewhere. Finally, this book represents a tour de force in scholarship involving nearly 4,000 sources of information and does not shy from a detailed examination of the controversies, conspiracies, and long-term consequences when we avoid learning from outbreaks, such as Zika.

Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.