Neurotherapeutics in the Era of Translational Medicine

Richard A. Smith, Brian K. Kaspar, Clive N. Svendsen

• Publisher: Academic Press
• Year: 2020
• eBook ISBN:
  9780128172742
• Topic: Sciences biologiques
• Subtopic: Neurosciences

For the first time in history, there is now hope for treating neurological disorders that had previously been considered untreatable. The remarkable confluence of events that has heralded this is the focus of Neurotherapeutics in the Era of Translational Medicine. This anthology, written by many of the prominent scientists and researchers in the field of biotechnology, recounts the breathtaking advances that are revolutionizing treatment for disorders such as amyotrophic lateral sclerosis?, spinal muscular atrophy, multiple sclerosis, Parkinson's disease, myasthenia gravis, migraine, and glioblastoma. The "story behind the story" of these translational efforts is told, with authors depicting the ups and downs encountered on the path of their drug discovery and development effort. In parallel with this path, advances in identifying novel biomarkers and disease models are summarized, as are contemporary issues focusing on clinical trial design, bioethics, innovative funding strategies, and collaborations between government and academia in an effort to facilitate breakthrough treatments. The book is written by members of the biotech and pharmaceutical ecosystem for those who belong to it and aspire to become part of it. - Comprehensive review on the progress of translational research in neurotherapeutics for neurologic disorders - Discusses important issues in clinical trials such as design and ethical issues - Written for neuroscientists, neurologists and pharmacologists

Table of contents

• Cover image
• Title page
• Table of Contents
• Copyright
• Contributors
• Preface
• Prologue
• Chapter 1: Development of a molecular therapy for the SOD1 familial variant of ALS
• Chapter 2: Unraveling molecular biology of C9ORF72 repeat expansions in amyotrophic lateral sclerosis-frontotemporal dementia: Implications for therapy
• Chapter 3: Translating molecular therapies for spinal muscular atrophy
• Chapter 4: From uncovering the mechanism of transthyretin aggregation to the drug tafamidis for ameliorating neurodegeneration and cardiomyopathy
• Chapter 5: A novel treatment for Parkinson's disease and ALS: Combined cell and gene therapies
• Chapter 6: Parkinson's disease: Genetic-driven therapeutic approaches
• Chapter 7: Neural stem cell-mediated brain tumor therapy
• Chapter 8: Advances in migraine therapeutics: The role of calcitonin gene-related peptide
• Chapter 9: The impact of translational research on the development of therapeutic agents for multiple sclerosis
• Chapter 10: Advances in treating myasthenia gravis
• Chapter 11: The endocannabinoid system as a target for the treatment of neurological disorders
• Chapter 12: Identification of biomarkers for diagnosing and monitoring therapy in the treatment of neurologic disorders
• Chapter 13: 3D Alzheimer's disease in a dish: Implications for drug discovery
• Chapter 14: Improving clinical trial efficiency with machine learning models of disease progression
• Chapter 15: Positron emission tomography imaging agents for evaluating the pathologic features of Alzheimer's disease and drug development
• Chapter 16: Repurposing FDA-approved pharmaceuticals: The development of Nuedexta
• Chapter 17: Bioethics in the era of translational medicine
• Chapter 18: Government efforts to promote the development of neurotherapeutics for the treatment of neurological diseases
• Chapter 19: Innovative funding models for neurotherapeutics
• Index