Book contents
- Alzheimer’s Disease Drug Development
- Alzheimer’s Disease Drug Development
- Copyright page
- Dedication
- Contents
- Contributors
- Foreword
- Acknowledgments
- Section 1 Advancing Alzheimer’s Disease Therapies in a Collaborative Science Ecosystem
- Section 2 Non-clinical Assessment of Alzheimer’s Disease Candidate Drugs
- 7 Role of Animal Models in Alzheimer’s Disease Drug Development
- 8 Use of Induced Pluripotent Stem Cell-Derived Neuronal Disease Models from Patients with Familial Early-Onset Alzheimer’s Disease in Drug Discovery
- 9 Preclinical Longitudinal In Vivo Biomarker Platform for Alzheimer’s Disease Drug Discovery
- 10 Biobanking and Biomarkers in the Alzheimer’s Disease Drug-Development Ecosystem
- Section 3 Alzheimer’s Disease Clinical Trials
- Section 4 Imaging and Biomarker Development in Alzheimer’s Disease Drug Discovery
- Section 5 Academic Drug-Development Programs
- Section 6 Public–Private Partnerships in Alzheimer’s Disease Drug Development
- Section 7 Funding and Financing Alzheimer’s Disease Drug Development
- Index
- References
8 - Use of Induced Pluripotent Stem Cell-Derived Neuronal Disease Models from Patients with Familial Early-Onset Alzheimer’s Disease in Drug Discovery
from Section 2 - Non-clinical Assessment of Alzheimer’s Disease Candidate Drugs
Published online by Cambridge University Press: 03 March 2022
Book contents
- Alzheimer’s Disease Drug Development
- Alzheimer’s Disease Drug Development
- Copyright page
- Dedication
- Contents
- Contributors
- Foreword
- Acknowledgments
- Section 1 Advancing Alzheimer’s Disease Therapies in a Collaborative Science Ecosystem
- Section 2 Non-clinical Assessment of Alzheimer’s Disease Candidate Drugs
- 7 Role of Animal Models in Alzheimer’s Disease Drug Development
- 8 Use of Induced Pluripotent Stem Cell-Derived Neuronal Disease Models from Patients with Familial Early-Onset Alzheimer’s Disease in Drug Discovery
- 9 Preclinical Longitudinal In Vivo Biomarker Platform for Alzheimer’s Disease Drug Discovery
- 10 Biobanking and Biomarkers in the Alzheimer’s Disease Drug-Development Ecosystem
- Section 3 Alzheimer’s Disease Clinical Trials
- Section 4 Imaging and Biomarker Development in Alzheimer’s Disease Drug Discovery
- Section 5 Academic Drug-Development Programs
- Section 6 Public–Private Partnerships in Alzheimer’s Disease Drug Development
- Section 7 Funding and Financing Alzheimer’s Disease Drug Development
- Index
- References
Summary
Incorporation of familial early-onset Alzheimer’s disease (EOAD) patient-based induced pluripotent stem cell (iPSC)-derived neuronal cell models into the AD drug discovery and preclinical development processes, provides for a tremendous technological advance, with implications extending from enabling a far more thorough preclinical pharmacological evaluation, using human patient-derived cellular model systems to assess efficacy against established, clinically relevant disease-associated biomarkers, including the evaluation of the effects on disease-associated endotypes, to unveiling previously unknown, pathologically-relevant pathways and identifying novel and potentially druggable therapeutic targets. This chapter discusses the status of promising disease-modifying therapeutics for AD, including the discovery and preclinical development of a clinically relevant series of small molecules and how familial EOAD patient-based iPSC-derived neuronal cell models have been critically utilized to dramatically improve this arduous yet necessary process.
Keywords
- Type
- Chapter
- Information
- Alzheimer's Disease Drug DevelopmentResearch and Development Ecosystem, pp. 95 - 105Publisher: Cambridge University PressPrint publication year: 2022
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