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Table of Contents
Intro
Preface
Contents
Contributors
About the Editor
1: The Art of Stem Cell-Based Therapy
1.1 The Dawn of a New Era
1.2 Lessons from a Past Disease
1.3 Potency Assays for Modern Disease Intervention
1.4 From Viral Vaccination to Safe Therapy with Cells
1.5 From Stem Cell Safety and Efficacy to Potency
1.6 Complementing Stem Cell-Based Therapy Art with Standard Operating Procedures
References
2: Potency Assay Development: A Keystone for Clinical Use
2.1 Potency Assays as Part of Cell-Based ATMP Quality Control Testing
2.1.1 What Is a Potency Assay?
2.1.2 Regulatory Requirements for a Potency Assay
2.2 Approach to Develop a Potency Assay for Cell-Based ATMPs
2.2.1 General Considerations
2.2.2 Progressive Implementation of a Potency Assay
2.3 Clinical Value of Potency Assays
2.3.1 Relationship Between Potency and Clinical Efficacy
2.3.2 Potency and Defining an Effective Dose for ATMPs
2.4 Potency Assay in Product and Process Development
2.4.1 Stability Studies
2.4.2 Comparability Studies
2.4.3 Compatibility Studies
2.5 State of the Art of Potency Assays for Cell-Based ATMPs
2.5.1 Examples of Potency Assays for Cell-Based ATMPs
2.5.2 Challenges to Potency Assay Development for ATMPs
2.6 Conclusion
References
3: Potency Assays: The 'Bugaboo' of Stem Cell Therapy
3.1 Potency - What a Cell Can Do
3.2 Relevance of Potency Assays
3.3 Regulatory Requirements
3.3.1 EMA Regulatory Requirements
3.3.2 FDA Regulatory Requirements
3.3.3 Japanese FDA Regulatory Requirements
3.4 Development of Potency Assays
3.4.1 Use of Surrogate Markers
3.4.2 Autologous and Allogeneic Products
3.4.3 Standardisation of Assays
3.4.4 Further Considerations
3.5 The Quality Target Product Profile
3.6 Final Remarks
References
4: Identifying Biomarkers for Osteogenic Potency Assay Development
4.1 Skeletal Stem Cell for Conservative Bone Healing
4.2 The Challenge of hBM-MSC Donor-Specific Heterogeneity
4.3 Telomerised MSC
Scalable Clonal Populations with Consistent Bone-Forming Potential
4.4 Comparative Analysis of Gene Expression, microRNA, Morphological Phenotypes and Cell Membrane or Secreted Proteins
4.5 Compelling Pathways for Functional Attributes in Osteogenic Potency Assays
4.6 Lessons Learned for Bone Repair ATMP Development
References
5: Potency Assay Considerations for Cartilage Repair, Osteoarthritis and Use of Extracellular Vesicles
5.1 Articular Cartilage
5.2 Cell-Based Treatment of Cartilage Defects
5.2.1 Potency Assays Used for Autologous Chondrocyte Implantation
5.2.2 Mechanism of Action (MoA) of MSC-Based Cartilage Defect Repair
5.2.3 MoA: Differentiation Versus Paracrine Signalling
5.2.4 Cell Fate of MSCs Used to Treat Cartilage Defects
Preface
Contents
Contributors
About the Editor
1: The Art of Stem Cell-Based Therapy
1.1 The Dawn of a New Era
1.2 Lessons from a Past Disease
1.3 Potency Assays for Modern Disease Intervention
1.4 From Viral Vaccination to Safe Therapy with Cells
1.5 From Stem Cell Safety and Efficacy to Potency
1.6 Complementing Stem Cell-Based Therapy Art with Standard Operating Procedures
References
2: Potency Assay Development: A Keystone for Clinical Use
2.1 Potency Assays as Part of Cell-Based ATMP Quality Control Testing
2.1.1 What Is a Potency Assay?
2.1.2 Regulatory Requirements for a Potency Assay
2.2 Approach to Develop a Potency Assay for Cell-Based ATMPs
2.2.1 General Considerations
2.2.2 Progressive Implementation of a Potency Assay
2.3 Clinical Value of Potency Assays
2.3.1 Relationship Between Potency and Clinical Efficacy
2.3.2 Potency and Defining an Effective Dose for ATMPs
2.4 Potency Assay in Product and Process Development
2.4.1 Stability Studies
2.4.2 Comparability Studies
2.4.3 Compatibility Studies
2.5 State of the Art of Potency Assays for Cell-Based ATMPs
2.5.1 Examples of Potency Assays for Cell-Based ATMPs
2.5.2 Challenges to Potency Assay Development for ATMPs
2.6 Conclusion
References
3: Potency Assays: The 'Bugaboo' of Stem Cell Therapy
3.1 Potency - What a Cell Can Do
3.2 Relevance of Potency Assays
3.3 Regulatory Requirements
3.3.1 EMA Regulatory Requirements
3.3.2 FDA Regulatory Requirements
3.3.3 Japanese FDA Regulatory Requirements
3.4 Development of Potency Assays
3.4.1 Use of Surrogate Markers
3.4.2 Autologous and Allogeneic Products
3.4.3 Standardisation of Assays
3.4.4 Further Considerations
3.5 The Quality Target Product Profile
3.6 Final Remarks
References
4: Identifying Biomarkers for Osteogenic Potency Assay Development
4.1 Skeletal Stem Cell for Conservative Bone Healing
4.2 The Challenge of hBM-MSC Donor-Specific Heterogeneity
4.3 Telomerised MSC
Scalable Clonal Populations with Consistent Bone-Forming Potential
4.4 Comparative Analysis of Gene Expression, microRNA, Morphological Phenotypes and Cell Membrane or Secreted Proteins
4.5 Compelling Pathways for Functional Attributes in Osteogenic Potency Assays
4.6 Lessons Learned for Bone Repair ATMP Development
References
5: Potency Assay Considerations for Cartilage Repair, Osteoarthritis and Use of Extracellular Vesicles
5.1 Articular Cartilage
5.2 Cell-Based Treatment of Cartilage Defects
5.2.1 Potency Assays Used for Autologous Chondrocyte Implantation
5.2.2 Mechanism of Action (MoA) of MSC-Based Cartilage Defect Repair
5.2.3 MoA: Differentiation Versus Paracrine Signalling
5.2.4 Cell Fate of MSCs Used to Treat Cartilage Defects