Improving and Accelerating Therapeutic Development for Nervous System Disorders - read free eBook in online reader or directly download on the web page. Select files or add your book in reader. Download and read online ebook Improving and Accelerating Therapeutic Development for Nervous System Disorders write by Institute of Medicine. This book was released on 2014-02-06. Improving and Accelerating Therapeutic Development for Nervous System Disorders available in PDF, EPUB and Kindle. 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.
Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Bloodâ¬"Brain Barrier
Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Bloodâ¬"Brain Barrier - read free eBook in online reader or directly download on the web page. Select files or add your book in reader. Download and read online ebook Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Bloodâ¬"Brain Barrier write by National Academies of Sciences, Engineering, and Medicine. This book was released on 2018-06-29. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Bloodâ¬"Brain Barrier available in PDF, EPUB and Kindle. Despite substantial advances in developing treatments for the serious illnesses that affect people worldwide, there remains a tremendous unmet need in the treatment of complex neurologic diseases, including neuropsychiatric and neurodegenerative disorders. Chief among the challenges that have hindered the development of therapeutics for central nervous system (CNS) disorders is the bloodâ€"brain barrier (BBB). The Forum on Neuroscience and Nervous System Disorders of the National Academies of Sciences, Engineering, and Medicine convened a workshop to explore the challenges associated with the BBB that have thus far stymied development of CNS drugs, examine new technologies that could address these challenges, and highlight potential opportunities for moving the field forward. 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 - read free eBook in online reader or directly download on the web page. Select files or add your book in reader. Download and read online ebook Improving the Utility and Translation of Animal Models for Nervous System Disorders write by Institute of Medicine. This book was released on 2013-04-25. Improving the Utility and Translation of Animal Models for Nervous System Disorders available in PDF, EPUB and Kindle. 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.
Advancing Gene-Targeted Therapies for Central Nervous System Disorders
Advancing Gene-Targeted Therapies for Central Nervous System Disorders - read free eBook in online reader or directly download on the web page. Select files or add your book in reader. Download and read online ebook Advancing Gene-Targeted Therapies for Central Nervous System Disorders write by National Academies of Sciences, Engineering, and Medicine. This book was released on 2019-12-01. Advancing Gene-Targeted Therapies for Central Nervous System Disorders available in PDF, EPUB and Kindle. On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.
Drug Repositioning
Drug Repositioning - read free eBook in online reader or directly download on the web page. Select files or add your book in reader. Download and read online ebook Drug Repositioning write by Joel Dudley. This book was released on 2017-07-14. Drug Repositioning available in PDF, EPUB and Kindle. Nervous system diseases represent a major health concern worldwide. Although important financial and professional investment, their etiology and pathophysiology still remain mostly elusive. Moreover, the clinical need of disease-modifying therapies is still unmet. In the last decades, traditional R&D has failed in identifying new effective therapies in many medical areas and drug repositioning has recently emerged as a promising alternative strategy to de novo drug discovery to improve and accelerate therapeutic development. For the first time, Drug Repositioning: Approaches and Applications for Neurotherapeutics reviews history and advances in drug repositioning, with a special focus on therapeutics for nervous system diseases. International experts from Academia, Industry and Non-profit organisations will provide different views on drug repositioning advantages, challenges and specific applications, which will be covered for nervous system diseases including Alzheimer's, Parkinson's, Huntington's diseases, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, ischemic stroke, and psychiatric disorders. This book provides a balanced overview and synthesis of drug repositioning concept, methods and applications for neurotherapeutics. It represents a valuable resource for students, scientists and clinicians working in academic settings, industry and government agencies within the fields of neuroscience, pharmacology, neurology, pharmaceutical sciences, drug discovery and development.
