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

Author: National Academies of Sciences, Engineering, and Medicine

Publisher: National Academies Press

Published: 2017-06-24

Total Pages: 95

ISBN-13: 0309455138

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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.


Book Synopsis Therapeutic Development in the Absence of Predictive Animal Models of Nervous System Disorders by : National Academies of Sciences, Engineering, and Medicine

Download or read book Therapeutic Development in the Absence of Predictive Animal Models of Nervous System Disorders written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2017-06-24 with total page 95 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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 the Utility and Translation of Animal Models for Nervous System Disorders

Improving the Utility and Translation of Animal Models for Nervous System Disorders

Author: Institute of Medicine

Publisher: National Academies Press

Published: 2013-04-25

Total Pages: 111

ISBN-13: 0309266335

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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.


Book Synopsis Improving the Utility and Translation of Animal Models for Nervous System Disorders by : Institute of Medicine

Download or read book Improving the Utility and Translation of Animal Models for Nervous System Disorders written by Institute of Medicine and published by National Academies Press. This book was released on 2013-04-25 with total page 111 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Improving and Accelerating Therapeutic Development for Nervous System Disorders

Improving and Accelerating Therapeutic Development for Nervous System Disorders

Author: Institute of Medicine

Publisher: National Academies Press

Published: 2014-02-06

Total Pages: 107

ISBN-13: 0309292492

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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.


Book Synopsis Improving and Accelerating Therapeutic Development for Nervous System Disorders by : Institute of Medicine

Download or read book Improving and Accelerating Therapeutic Development for Nervous System Disorders written by Institute of Medicine and published by National Academies Press. This book was released on 2014-02-06 with total page 107 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

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

Author: National Academies of Sciences, Engineering, and Medicine

Publisher: National Academies Press

Published: 2017-05-24

Total Pages: 95

ISBN-13: 0309455162

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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.


Book Synopsis Therapeutic Development in the Absence of Predictive Animal Models of Nervous System Disorders by : National Academies of Sciences, Engineering, and Medicine

Download or read book Therapeutic Development in the Absence of Predictive Animal Models of Nervous System Disorders written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2017-05-24 with total page 95 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Central Nervous System Diseases

Central Nervous System Diseases

Author: Dwaine F. Emerich

Publisher: Springer Science & Business Media

Published: 1999-11-24

Total Pages: 511

ISBN-13: 1592596916

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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.


Book Synopsis Central Nervous System Diseases by : Dwaine F. Emerich

Download or read book Central Nervous System Diseases written by Dwaine F. Emerich and published by Springer Science & Business Media. This book was released on 1999-11-24 with total page 511 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Neuroscience Trials of the Future

Neuroscience Trials of the Future

Author: National Academies of Sciences, Engineering, and Medicine

Publisher: National Academies Press

Published: 2016-11-07

Total Pages: 111

ISBN-13: 0309442583

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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.


Book Synopsis Neuroscience Trials of the Future by : National Academies of Sciences, Engineering, and Medicine

Download or read book Neuroscience Trials of the Future written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2016-11-07 with total page 111 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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

Author: Jules J. Berman

Publisher: Academic Press

Published: 2018-01-26

Total Pages: 398

ISBN-13: 0128143940

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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.


Book Synopsis Precision Medicine and the Reinvention of Human Disease by : Jules J. Berman

Download or read book Precision Medicine and the Reinvention of Human Disease written by Jules J. Berman and published by Academic Press. This book was released on 2018-01-26 with total page 398 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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

Author: Jules J. Berman

Publisher: Academic Press

Published: 2020-07-03

Total Pages: 308

ISBN-13: 012821371X

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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


Book Synopsis Logic and Critical Thinking in the Biomedical Sciences by : Jules J. Berman

Download or read book Logic and Critical Thinking in the Biomedical Sciences written by Jules J. Berman and published by Academic Press. This book was released on 2020-07-03 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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

Animal Models of Cognitive Impairment

Animal Models of Cognitive Impairment

Author: Edward D. Levin

Publisher: CRC Press

Published: 2006-06-22

Total Pages: 395

ISBN-13: 1420004336

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The costs associated with a drug's clinical trials are so significant that it has become necessary to validate both its safety and efficacy in animal models prior to the continued study of the drug in humans. Featuring contributions from distinguished researchers in the field of cognitive therapy research, Animal Models of Cognitive Impairmen


Book Synopsis Animal Models of Cognitive Impairment by : Edward D. Levin

Download or read book Animal Models of Cognitive Impairment written by Edward D. Levin and published by CRC Press. This book was released on 2006-06-22 with total page 395 pages. Available in PDF, EPUB and Kindle. Book excerpt: The costs associated with a drug's clinical trials are so significant that it has become necessary to validate both its safety and efficacy in animal models prior to the continued study of the drug in humans. Featuring contributions from distinguished researchers in the field of cognitive therapy research, Animal Models of Cognitive Impairmen

Transgenic Neuroscience Research

Transgenic Neuroscience Research

Author: National Academies of Sciences, Engineering, and Medicine

Publisher: National Academies Press

Published: 2019-04-03

Total Pages: 91

ISBN-13: 0309488761

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To examine the promise, concerns, and challenges related to neuroscience research using genetically modified nonhuman primates, the National Academies of Sciences, Engineering, and Medicine hosted a public workshop on October 4, 2018, bringing together an international group of experts and stakeholders representing academia, industry, laboratory animal management, disease-focused foundations, and federal agencies. The workshop was designed to explore the current state and future promise of research using genetically modified nonhuman primate models of disease to understand the complex functions of the brain that control behavior, movement, and cognition in both health and disease states. This publication summarizes the presentations and discussions from the workshop.


Book Synopsis Transgenic Neuroscience Research by : National Academies of Sciences, Engineering, and Medicine

Download or read book Transgenic Neuroscience Research written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2019-04-03 with total page 91 pages. Available in PDF, EPUB and Kindle. Book excerpt: To examine the promise, concerns, and challenges related to neuroscience research using genetically modified nonhuman primates, the National Academies of Sciences, Engineering, and Medicine hosted a public workshop on October 4, 2018, bringing together an international group of experts and stakeholders representing academia, industry, laboratory animal management, disease-focused foundations, and federal agencies. The workshop was designed to explore the current state and future promise of research using genetically modified nonhuman primate models of disease to understand the complex functions of the brain that control behavior, movement, and cognition in both health and disease states. This publication summarizes the presentations and discussions from the workshop.