Using novel technologies and models to identify biomarkers and explore therapeutic strategies for neurological disorders PDF Download

Author: Dongdong Qin
Publisher: Frontiers Media SA
ISBN: 2832521126
Category : Science
Languages : en
Pages : 116

View

Book Description

Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309455162
Category : Medical
Languages : en
Pages : 95

View

Book Description
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.

Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309178282
Category : Medical
Languages : en
Pages : 128

View

Book Description
Biomarkers, or biological markers, are quantitative measurements that offer researchers and clinicians valuable insight into diagnosis, treatment and prognosis for many disorders and diseases. A major goal in neuroscience medical research is establishing biomarkers for disorders of the nervous system. Given the promising potential and necessity for neuroscience biomarkers, the Institute of Medicine Forum on Neuroscience and Nervous System Disorders convened a public workshop and released the workshop summary entitled Neuroscience Biomarkers and Biosignatures: Converging Technologies, Emerging Partnerships. The workshop brought together experts from multiple areas to discuss the most promising and practical arenas in neuroscience in which biomarkers will have the greatest impact. The main objective of the workshop was to identify and discuss biomarker targets that are not currently being aggressively pursued but that could have the greatest near-term impact on the rate at which new treatments are brought forward for psychiatric and neurological disorders.

Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309292492
Category : Medical
Languages : en
Pages : 107

View

Book Description
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.

Author: Bernhard Baune
Publisher: Academic Press
ISBN: 0128131772
Category : Medical
Languages : en
Pages : 594

View

Book Description
Personalized Psychiatry presents the first book to explore this novel field of biological psychiatry that covers both basic science research and its translational applications. The book conceptualizes personalized psychiatry and provides state-of-the-art knowledge on biological and neuroscience methodologies, all while integrating clinical phenomenology relevant to personalized psychiatry and discussing important principles and potential models. It is essential reading for advanced students and neuroscience and psychiatry researchers who are investigating the prevention and treatment of mental disorders. - Combines neurobiology with basic science methodologies in genomics, epigenomics and transcriptomics - Demonstrates how the statistical modeling of interacting biological and clinical information could transform the future of psychiatry - Addresses fundamental questions and requirements for personalized psychiatry from a basic research and translational perspective

Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309442583
Category : Medical
Languages : en
Pages : 111

View

Book Description
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.

Author: Ying Wang
Publisher: Frontiers Media SA
ISBN: 2832539351
Category : Medical
Languages : en
Pages : 141

View

Book Description
Over the last few years, new high-throughput biotechnologies are revolutionizing our ways to utilize human biospecimens for understanding atherosclerotic disease. These recent advances allow deep profiling of individual cells at the genomics, epigenomics, transcriptomics and proteomics levels, or even simultaneous detection of various combinations of ‘Omics’ in the same cell. Additionally, novel methods to integrate data at different levels from tissue sections and dissociated tissues are the emerging trends in large and institutional biobank studies. Growing literature has shown the value of such sequencing and bioinformatic strategies in shedding light on (1) how risk genes, as identified by the Genome-Wide Association Study, contribute to atherogenesis (genotype to phenotype), and (2) how features of atherosclerotic lesions affect patient response in clinical trials (phenotype to the clinical outcome). The hybrid of cutting-edge biotechnologies and bioinformatic approaches helps us maximize biobank resources to accelerate bench-to-bedside research.

Author: António Salgado
Publisher: Elsevier
ISBN: 0128180854
Category : Medical
Languages : en
Pages : 712

View

Book Description
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

Author: Michael Mahler
Publisher: Academic Press
ISBN: 032385432X
Category : Science
Languages : en
Pages : 302

View

Book Description
Precision Medicine and Artificial Intelligence: The Perfect Fit for Autoimmunity covers background on artificial intelligence (AI), its link to precision medicine (PM), and examples of AI in healthcare, especially autoimmunity. The book highlights future perspectives and potential directions as AI has gained significant attention during the past decade. Autoimmune diseases are complex and heterogeneous conditions, but exciting new developments and implementation tactics surrounding automated systems have enabled the generation of large datasets, making autoimmunity an ideal target for AI and precision medicine. More and more diagnostic products utilize AI, which is also starting to be supported by regulatory agencies such as the Food and Drug Administration (FDA). Knowledge generation by leveraging large datasets including demographic, environmental, clinical and biomarker data has the potential to not only impact the diagnosis of patients, but also disease prediction, prognosis and treatment options. - Allows the readers to gain an overview on precision medicine for autoimmune diseases leveraging AI solutions - Provides background, milestone and examples of precision medicine - Outlines the paradigm shift towards precision medicine driven by value-based systems - Discusses future applications of precision medicine research using AI - Other aspects covered in the book include regulatory insights, data analytics and visualization, types of biomarkers as well as the role of the patient in precision medicine

Author: Joao Quevedo
Publisher: Academic Press
ISBN: 012819183X
Category : Medical
Languages : en
Pages : 472

View

Book Description
The Neurobiology of Bipolar Disorder: Road to Novel Therapeutics combines the basic neurobiology of bipolar disorder with discussions of the most recent advances in research, including the interacting pathways implicated in the pathophysiology of bipolar disorder, genetic approaches and the pharmacogenomics of bipolar disorder. The basic foundational understanding of the neurobiology underlying the disorder, along with a comprehensive summary of the most recent advances in research combine to aid advanced students and researchers in their understanding of bipolar disorder management using novel and fast-acting pharmaceutical and neuromodulatory approaches. Aids readers in understanding bipolar disorder in the context of NIMH Research Domain Criteria (RDoC) recommendations Covers a range of existing and potential pharmacologic and non-pharmacologic treatment options, from lifestyle adjustments to novel therapeutics Synthesizes a discussion of the cellular and molecular mechanisms underlying symptoms with clinical aspects of bipolar disorder