Therapeutic Development In The Absence Of Predictive Animal Models Of Nervous System Disorders


Therapeutic Development In The Absence Of Predictive Animal Models Of Nervous System Disorders
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Therapeutic Development In The Absence Of Predictive Animal Models Of Nervous System Disorders


Therapeutic Development In The Absence Of Predictive Animal Models Of Nervous System Disorders
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Author : National Academies of Sciences, Engineering, and Medicine
language : en
Publisher: National Academies Press
Release Date : 2017-06-24

Therapeutic Development In The Absence Of Predictive Animal Models Of Nervous System Disorders written by National Academies of Sciences, Engineering, and Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2017-06-24 with Medical categories.


Compared with other disease areas, central nervous system (CNS) disorders have had the highest failure rate for new compounds in advanced clinical trials. Most CNS drugs fail because of efficacy, and the core issue underlying these problems is a poor understanding of disease biology. Concern about the poor productivity in neuroscience drug development has gained intensity over the past decade, amplified by a retraction in investment from the pharmaceutical industry. This retreat by industry has been fueled by the high failure rate of compounds in advanced clinical trials for nervous system disorders. In response to the de-emphasis of CNS disorders in therapeutic development relative to other disease areas such as cancer, metabolism, and autoimmunity, the National Academies of Sciences, Engineering, and Medicine initiated a series of workshops in 2012 to address the challenges that have slowed drug development for nervous system disorders. Motivated by the notion that advances in genetics and other new technologies are beginning to bring forth new molecular targets and identify new biomarkers, the Academies hosted the third workshop in this series in September 2016. Participants discussed opportunities to accelerate early stages of drug development for nervous system disorders in the absence of animal models that reflect disease and predict efficacy. This publication summarizes the presentations and discussions from the workshop.



Improving And Accelerating Therapeutic Development For Nervous System Disorders


Improving And Accelerating Therapeutic Development For Nervous System Disorders
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Author : Institute of Medicine
language : en
Publisher: National Academies Press
Release Date : 2014-02-06

Improving And Accelerating Therapeutic Development For Nervous System Disorders written by Institute of Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014-02-06 with Medical categories.


Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials.



Improving The Utility And Translation Of Animal Models For Nervous System Disorders


Improving The Utility And Translation Of Animal Models For Nervous System Disorders
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Author : Forum on Neuroscience and Nervous System Disorders
language : en
Publisher: National Academies Press
Release Date : 2013-04-08

Improving The Utility And Translation Of Animal Models For Nervous System Disorders written by Forum on Neuroscience and Nervous System Disorders and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2013-04-08 with Medical categories.


Nervous system diseases and disorders are highly prevalent and substantially contribute to the overall disease burden. Despite significant information provided by the use of animal models in the understanding of the biology of nervous system disorders and the development of therapeutics; limitations have also been identified. Treatment options that are high in efficacy and low in side effects are still lacking for many diseases and, in some cases are nonexistent. A particular problem in drug development is the high rate of attrition in Phase II and III clinical trials. Why do many therapeutics show promise in preclinical animal models but then fail to elicit predicted effects when tested in humans? On March 28 and 29, 2012, the Institute of Medicine Forum on Neuroscience and Nervous System Disorders convened the workshop "Improving Translation of Animal Models for Nervous System Disorders" to discuss potential opportunities for maximizing the translation of new therapies from animal models to clinical practice. The primary focus of the workshop was to examine mechanisms for increasing the efficiency of translational neuroscience research through discussions about how and when to use animal models most effectively and then best approaches for the interpretation of the data collected. Specifically, the workshop objectives were to: discuss key issues that contribute to poor translation of animal models in nervous system disorders, examine case studies that highlight successes and failures in the development and application of animal models, consider strategies to increase the scientific rigor of preclinical efficacy testing, explore the benefits and challenges to developing standardized animal and behavioral models. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary also identifies methods to facilitate development of corresponding animal and clinical endpoints, indentifies methods that would maximize bidirectional translation between basic and clinical research and determines the next steps that will be critical for improvement of the development and testing of animal models of disorders of the nervous system.



Central Nervous System Diseases


Central Nervous System Diseases
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Author : Dwaine F. Emerich
language : en
Publisher: Springer Science & Business Media
Release Date : 1999-11-24

Central Nervous System Diseases written by Dwaine F. Emerich and has been published by Springer Science & Business Media this book supported file pdf, txt, epub, kindle and other format this book has been release on 1999-11-24 with Medical categories.


Prominent experimentalists critically review the animal models widely used in developing powerful new therapies for central nervous system diseases. Coverage includes novel uses of animal models of Alzheimer's, Parkinson's, and Huntington's diseases, and studies of aging. Techniques that rely heavily on behavioral analyses, as well as models developed from infusions of neurotoxins and from advances in molecular biology, are thoroughly explicated, as are models developed for more acute neurological conditions, including traumatic brain injury and stroke. Comprehensive and authoritative, Central Nervous System Diseases: Innovative Animal Models from Lab to Clinic offers neuroscientists, pharmacologists, and interested clinicians a unique survey of the most productive animal models of the leading neurological diseases currently employed to develop today's innovative drug therapies.



Handbook Of Innovations In Central Nervous System Regenerative Medicine


Handbook Of Innovations In Central Nervous System Regenerative Medicine
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Author : António Salgado
language : en
Publisher: Elsevier
Release Date : 2020-06-10

Handbook Of Innovations In Central Nervous System Regenerative Medicine written by António Salgado and has been published by Elsevier this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-06-10 with Medical categories.


Handbook of Innovations in CNS Regenerative Medicine provides a comprehensive overview of the CNS regenerative medicine field. The book describes the basic biology and anatomy of the CNS and how injury and disease affect its balance and the limitations of the present therapies used in the clinics. It also introduces recent trends in different fields of CNS regenerative medicine, including cell transplantation, bio and neuro-engineering, molecular/pharmacotherapy therapies and enabling technologies. Finally, the book presents successful cases of translation of basic research to first-in-human trials and the steps needed to follow this path. Areas such as cell transplantation approaches, bio and neuro-engineering, molecular/pharmacotherapy therapies and enabling technologies are key in regenerative medicine are covered in the book, along with regulatory and ethical issues. Describes the basic biology and anatomy of the CNS and how injury and disease affect its balance Discusses the limitations of present therapies used in the clinics Introduces the recent trends in different fields of CNS regenerative medicine, including cell transplantation, bio and neuro-engineering, molecular/pharmacotherapy therapies, and enabling technologies Presents successful cases of translation of basic research to first-in-human trials, along with the steps needed to follow this path



Biomarkers Of Neuroinflammation


Biomarkers Of Neuroinflammation
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Author : National Academies of Sciences, Engineering, and Medicine
language : en
Publisher: National Academies Press
Release Date : 2018-03-16

Biomarkers Of Neuroinflammation written by National Academies of Sciences, Engineering, and Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-03-16 with Medical categories.


Neuroinflammation is a burgeoning area of interest in academia and biopharma, with a broadly acknowledged role in many central nervous system (CNS) disorders. However, there is little agreement on the pathophysiological mechanisms that underlie the manifestations of neuroinflammation in the CNS compartment and how neuroinflammation operates as a driver and also as a consequence of disease in the brain. Moreover, another unclear area is how to translate increased understanding of the mechanisms that underlie neuroinflammation and its manifestations in the CNS to therapeutics. To address these gaps in understanding mechanisms and how to translate that understanding into therapeutics, the Forum on Neuroscience and Nervous System Disorders of the National Academies of Sciences, Engineering, and Medicine convened a workshop on March 20-21, 2017, bringing together key leaders in the field from industry, academia, and governmental agencies to explore the role and mechanisms of neuroinflammation in a variety of CNS diseases. The workshop also considered strategies to advance the identification and validation of biomarkers of neuroinflammation that could accelerate development of therapies, bringing much-needed treatments to patients with disorders ranging from neuroinflammatory diseases such as multiple sclerosis (MS) to neuropsychiatric disorders such as depression. This publication summarizes the presentations and discussions from the workshop.



Neuroscience Trials Of The Future


Neuroscience Trials Of The Future
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Author : National Academies of Sciences, Engineering, and Medicine
language : en
Publisher: National Academies Press
Release Date : 2016-12-07

Neuroscience Trials Of The Future written by National Academies of Sciences, Engineering, and Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2016-12-07 with Medical categories.


On March 3-4, 2016, the National Academies of Sciences, Engineering, and Medicine's Forum on Neuroscience and Nervous System Disorders held a workshop in Washington, DC, bringing together key stakeholders to discuss opportunities for improving the integrity, efficiency, and validity of clinical trials for nervous system disorders. Participants in the workshop represented a range of diverse perspectives, including individuals not normally associated with traditional clinical trials. The purpose of this workshop was to generate discussion about not only what is feasible now, but what may be possible with the implementation of cutting-edge technologies in the future.



Precision Medicine And The Reinvention Of Human Disease


Precision Medicine And The Reinvention Of Human Disease
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Author : Jules J. Berman
language : en
Publisher: Academic Press
Release Date : 2018-01-26

Precision Medicine And The Reinvention Of Human Disease written by Jules J. Berman and has been published by Academic Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-01-26 with Business & Economics categories.


Despite what you may have read in the popular press and in social media, Precision Medicine is not devoted to finding unique treatments for individuals, based on analyzing their DNA. To the contrary, the goal of Precision Medicine is to find general treatments that are highly effective for large numbers of individuals who fall into precisely diagnosed groups. We now know that every disease develops over time, through a sequence of defined biological steps, and that these steps may differ among individuals, based on genetic and environmental conditions. We are currently developing rational therapies and preventive measures, based on our precise understanding of the steps leading to the clinical expression of diseases. Precision Medicine and the Reinvention of Human Disease explains the scientific breakthroughs that have changed the way that we understand diseases, and reveals how medical scientists are using this new knowledge to launch a medical revolution. Clarifies the foundational concepts of Precision Medicine, distinguishing this field from its predecessors such as genomics, pharmacogenetics, and personalized medicine. Gathers the chief conceptual advances in the fields of genetics, pathology, and bioinformatics, and synthesizes a coherent narrative for the field of Precision Medicine. Delivers its message in plain language, and in a relaxed, conversational writing style, making it easy to understand the complex subject matter. Guides the reader through a coherent and logical narrative, gradually providing expertise and skills along the way. Covers the importance of data sharing in Precision Medicine, and the many data-related challenges that confront this fragile new field.



Logic And Critical Thinking In The Biomedical Sciences


Logic And Critical Thinking In The Biomedical Sciences
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Author : Jules J. Berman
language : en
Publisher: Academic Press
Release Date : 2020-07-03

Logic And Critical Thinking In The Biomedical Sciences written by Jules J. Berman and has been published by Academic Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-07-03 with Medical categories.


Science is not a collection of facts. Science is the process by which we draw inferences from facts. Volume I of Logic and Critical Thinking in the Biomedical Sciences invites readers to linger over a collection of common observations to see what inferences can be drawn, when one applies a bit of deductive logic. If we just think about what we observe, it is often possible to discover profound biomedical insights.Volumes 1 and 2 of Logic and Critical Thinking in the Biomedical Sciences are written for biomedical scientists and college-level students engaged in any of the life sciences, including bioinformatics and related data sciences. Provides a strong introduction to deductive methods that can be directly applied to the biomedical sciences Using hundreds of examples, shows how creative scientists draw important inferences from observations that are often ignored by their peers Discusses complex biological and medical concepts in a relaxed manner, intended to focus the reader’s attention on the deductive process, without dwelling excessively on details



Biomarkers To Enable Therapeutics Development In Neurodevelopmental Disorders


Biomarkers To Enable Therapeutics Development In Neurodevelopmental Disorders
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Author : Mustafa Sahin
language : en
Publisher:
Release Date : 2020

Biomarkers To Enable Therapeutics Development In Neurodevelopmental Disorders written by Mustafa Sahin and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020 with categories.


This eBook is a collection of articles from a Frontiers Research Topic. Frontiers Research Topics are very popular trademarks of the Frontiers Journals Series: they are collections of at least ten articles, all centered on a particular subject. With their unique mix of varied contributions from Original Research to Review Articles, Frontiers Research Topics unify the most influential researchers, the latest key findings and historical advances in a hot research area! Find out more on how to host your own Frontiers Research Topic or contribute to one as an author by contacting the Frontiers Editorial Office: frontiersin.org/about/contact.