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.
1. Cover image - p. page_i 2. Title page - p. page_i 3. Table of Contents - p. page_i 4. Copyright - p. page_iv 5. Contributors - p. page_xi 6. Preface - p. page_xv 7. Prologue - p. page_xix 8. Chapter 1: Development of a molecular therapy for the SOD1 familial variant of ALS - p. page_1 9. Chapter 2: Unraveling molecular biology of C9ORF72 repeat expansions in amyotrophic lateral sclerosis-frontotemporal dementia: Implications for therapy - p. page_19 10. Chapter 3: Translating molecular therapies for spinal muscular atrophy - p. page_49 11. Chapter 4: From uncovering the mechanism of transthyretin aggregation to the drug tafamidis for ameliorating neurodegeneration and cardiomyopathy - p. page_65 12. Chapter 5: A novel treatment for Parkinson's disease and ALS: Combined cell and gene therapies - p. page_105 13. Chapter 6: Parkinson's disease: Genetic-driven therapeutic approaches - p. page_135 14. Chapter 7: Neural stem cell-mediated brain tumor therapy - p. page_161 15. Chapter 8: Advances in migraine therapeutics: The role of calcitonin gene-related peptide - p. page_181 16. Chapter 9: The impact of translational research on the development of therapeutic agents for multiple sclerosis - p. page_203 17. Chapter 10: Advances in treating myasthenia gravis - p. page_243 18. Chapter 11: The endocannabinoid system as a target for the treatment of neurological disorders - p. page_265 19. Chapter 12: Identification of biomarkers for diagnosing and monitoring therapy in the treatment of neurologic disorders - p. page_291 20. Chapter 13: 3D Alzheimer's disease in a dish: Implications for drug discovery - p. page_311 21. Chapter 14: Improving clinical trial efficiency with machine learning models of disease progression - p. page_333 22. Chapter 15: Positron emission tomography imaging agents for evaluating the pathologic features of Alzheimer's disease and drug development - p. page_367 23. Chapter 16: Repurposing FDA-approved pharmaceuticals: The development of Nuedexta - p. page_413 24. Chapter 17: Bioethics in the era of translational medicine - p. page_437 25. Chapter 18: Government efforts to promote the development of neurotherapeutics for the treatment of neurological diseases - p. page_449 26. Chapter 19: Innovative funding models for neurotherapeutics - p. page_457 27. Index - p. page_477
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.
1. Cover image - p. page_i 2. Title page - p. page_i 3. Table of Contents - p. page_i 4. Copyright - p. page_iv 5. Contributors - p. page_xi 6. Preface - p. page_xv 7. Prologue - p. page_xix 8. Chapter 1: Development of a molecular therapy for the SOD1 familial variant of ALS - p. page_1 9. Chapter 2: Unraveling molecular biology of C9ORF72 repeat expansions in amyotrophic lateral sclerosis-frontotemporal dementia: Implications for therapy - p. page_19 10. Chapter 3: Translating molecular therapies for spinal muscular atrophy - p. page_49 11. Chapter 4: From uncovering the mechanism of transthyretin aggregation to the drug tafamidis for ameliorating neurodegeneration and cardiomyopathy - p. page_65 12. Chapter 5: A novel treatment for Parkinson's disease and ALS: Combined cell and gene therapies - p. page_105 13. Chapter 6: Parkinson's disease: Genetic-driven therapeutic approaches - p. page_135 14. Chapter 7: Neural stem cell-mediated brain tumor therapy - p. page_161 15. Chapter 8: Advances in migraine therapeutics: The role of calcitonin gene-related peptide - p. page_181 16. Chapter 9: The impact of translational research on the development of therapeutic agents for multiple sclerosis - p. page_203 17. Chapter 10: Advances in treating myasthenia gravis - p. page_243 18. Chapter 11: The endocannabinoid system as a target for the treatment of neurological disorders - p. page_265 19. Chapter 12: Identification of biomarkers for diagnosing and monitoring therapy in the treatment of neurologic disorders - p. page_291 20. Chapter 13: 3D Alzheimer's disease in a dish: Implications for drug discovery - p. page_311 21. Chapter 14: Improving clinical trial efficiency with machine learning models of disease progression - p. page_333 22. Chapter 15: Positron emission tomography imaging agents for evaluating the pathologic features of Alzheimer's disease and drug development - p. page_367 23. Chapter 16: Repurposing FDA-approved pharmaceuticals: The development of Nuedexta - p. page_413 24. Chapter 17: Bioethics in the era of translational medicine - p. page_437 25. Chapter 18: Government efforts to promote the development of neurotherapeutics for the treatment of neurological diseases - p. page_449 26. Chapter 19: Innovative funding models for neurotherapeutics - p. page_457 27. Index - p. page_477
