Translational Regenerative Medicine

1st Edition - December 1, 2014
Editors: Anthony Atala, Julie Allickson
Language: English
Hardback ISBN:
9 7 8 - 0 - 1 2 - 4 1 0 3 9 6 - 2
eBook ISBN:
9 7 8 - 0 - 1 2 - 4 1 0 4 5 7 - 0

Translational Regenerative Medicine is a reference book that outlines the life cycle for effective implementation of discoveries in the dynamic field of regenerative medici… Read more

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Translational Regenerative Medicine is a reference book that outlines the life cycle for effective implementation of discoveries in the dynamic field of regenerative medicine. By addressing science, technology, development, regulatory, manufacturing, intellectual property, investment, financial, and clinical aspects of the field, this work takes a holistic look at the translation of science and disseminates knowledge for practical use of regenerative medicine tools, therapeutics, and diagnostics. Incorporating contributions from leaders in the fields of translational science across academia, industry, and government, this book establishes a more fluid transition for rapid translation of research to enhance human health and well-being.

Dedication
Chapter 1. The Landscape of Cell Tissues and Organs
- I. The Regenerative Medicine Field
- II. Conclusions
- Disclaimer
Section I. Cell Banking
- Chapter 2. Landscape of Cell Banking
  - I. Introduction: The Field of Cell Banking
  - II. Allogeneic and Autologous Cell Banking
  - III. Recruitment
  - IV. Regulations and Determination of Regulatory Guidance
  - V. Registration and Donor Eligibility Screening/Testing
  - VI. Current Good Tissue Practice (cGTP)
  - VII. Ethics (Personal Data, Health Information, Genetic Analysis, Privately Banked Cells)
  - VIII. Financial Aspects
  - IX. The Role of Banking in Regenerative Medicine
  - X. Conclusion
- Chapter 3. Cell Banking: Process Development and Cell Preservation
  - I. Introduction
  - II. Product Characterization and Release Specifications
  - III. Standard Life Cycle of Banked Cell Therapy Product
  - IV. CT Facility
  - V. Cleaning and Disinfection
  - VI. Environmental Monitoring
  - VII. Equipment
  - VIII. Critical Reagents and Supplies Management
- Chapter 4. Clinical Development of Placental Mesenchymal Stromal Cells
  - Conclusion
- Chapter 5. Translation of Regenerative Medicine Products Into the Clinic in the United States: FDA Perspective
  - I. Introduction and Chapter Overview
  - II. Brief Legislative History of FDA
  - III. Roles of Laws, Regulations, and Guidance
  - IV. FDA Organizational Structure and Jurisdictional Processes
  - V. Approval Mechanisms and Clinical Studies
  - VI. Meetings with Industry, Professional Groups, and Sponsors
  - VII. Regulations and Guidance of Special Interest for Regenerative Medicine
  - VIII. Preclinical Development Plan
  - IX. Clinical Development Plan
  - X. Special Topic 1: Current Good Manufacturing Practices
  - XI. Special Topic 2: Regulation of Minimally Manipulated, Unrelated Allogeneic Cord Blood
  - XII. Special Topic 3: Animal Cell-Based Products for Veterinary Applications
  - XIII. Use of Standards in Regenerative Medicine
  - XIV. Advisory Committee Meetings
  - XV. FDA Regulatory Science Research Initiatives and Critical Path
  - XVI. Other Communication Efforts
  - XVII. Conclusion
- Chapter 6. Newborn Stem Cell Banking Business Models
  - I. Introduction
  - II. Cell Biobanking History
  - III. Public NSC Biobanking
  - IV. Family NSC Biobanking
  - V. Operational Execution and Risk Management
  - VI. Unit Quality
  - VII. Unit Storage Maintenance
  - VIII. Risk Mitigation Strategies
  - IX. Market Potential
  - X. Competition
  - XI. Scalability
  - XII. Intrinsic Soundness of the Business Model
  - XIII. Hybrid NSC Biobanking Business Models
  - XIV. The Future of NSC Biobanking
  - Acronym
Section II. Stem Cells and Cell Therapy
- Chapter 7. Cell Therapy Landscape: Autologous and Allogeneic Approaches
  - I. Introduction
  - II. Comparison of Commercial Potential between Autologous and Allogeneic Cell Therapy
  - III. Future Product Commercialization: Will Allogeneic or Autologous Cell Therapy Dominate?
  - IV. Conclusion
  - List of Abbreviations
- Chapter 8. Stem Cells and Cell Therapy: Autologous Cell Manufacturing
  - I. Autologous Therapy
  - II. Bone Marrow Aspiration
  - III. Manufacturing
  - IV. Allogeneic Therapy
  - V. Manufacturing
  - VI. Summary
- Chapter 9. Overview: Challenges of Process Development for Cellular Therapy
- Chapter 10. Tissue Engineering: Propagation and Potency Evaluation
  - I. Introduction: The State of Stem Cell Potency Evaluation
  - II. Developing a Reliable Potency Assay with Clinical Relevance
  - III. The Future of Stem Cell Potency Enumeration
Section III. Biomaterials in Regenerative Medicine
- Chapter 11. Biomaterials in Preclinical Approaches for Engineering Skeletal Tissues
  - I. Introduction to Skeletal Tissue Engineering (STE)
  - II. Biomaterials for Translational Regenerative Medicine
  - III. Could Bioreactors Be the Missing Link for Biomechanic Function?
  - IV. Scale-Up and Ready to Go Systems
  - V. Future Outcomes/Challenges
  - List of Acronyms and Abbreviations
- Chapter 12. Biomaterials in Regenerative Medicine: Considerations in Early Process Development
  - I. Introduction
  - II. Assembling a Design Team
  - III. Identifying an Unmet Problem
  - IV. Biomaterial-Specific Considerations
  - V. Regulatory Challenges
  - VI. Conclusion
  - VII. Key Points
  - List of Acronyms and Abbreviations
- Chapter 13. Biomaterials in Regenerative Medicine: Challenges in Technology Transfer from Science to Process Development
  - I. Introduction
  - II. Transfer of Biomaterials Technology from Laboratory to Commercial Production: Technical Considerations
  - III. Transfer of Technology from Science to Commercial Production: Current Challenges
  - IV. Options for Enabling Successful Transition of Technology from Science to Commercialization to Clinical Use
  - V. Additional Considerations for Process Development for Biomaterial-Based Products
  - VI. Summary
  - Further Reading
- Chapter 14. Paracrine Regulation from Tissue Engineered Constructs
  - I. Introduction
  - II. The Development of Endothelial Cell–Based Paracrine Tissue Engineering Solutions
  - III. Designing Paracrine Tissue Engineering Constructs
  - IV. Implications of Paracrine Tissue Engineering in Clinical Study Design
  - V. Conclusion
- Chapter 15. Creating Commercial Value from Biomaterials
  - I. Facing Reality: An Introduction to Translational Medicine and Commercialization
  - II. What Features Do You Really Need? Developing the Biomaterial
  - III. What Will You Sell? Choosing a Business Model
  - IV. Divide and Conquer: A Case Study in Licensing Focused Fields of Use
  - V. The Translational Imperative: Deliver Simplicity
Section IV. Tissue Engineering
- Chapter 16. Manufacturing of Regenerative Medicine Products
  - I. Introduction
  - II. Manufacturing Process
  - III. Manufacturing Facilities and Process Equipment
  - IV. Cost of Goods
  - V. Good Manufacturing Practices, Good Tissue Practices, and Quality Systems
  - VI. Conclusions
- Chapter 17. Regulatory Aspects
  - I. Introduction
  - II. Regulatory Path
  - III. Manufacturing Considerations
  - IV. Preclinical Considerations
  - V. Clinical Trial Design Considerations
  - VI. Developmental Challenges
  - VII. Conclusions
- Chapter 18. Global Design for Clinical Trials
  - I. Tissue Engineering and Regenerative Medicine
  - II. Follow-Up Studies on Clinical Trials of Tissue Engineering
  - III. Topics on Scaffold
  - IV. Global Design for Clinical Trials of Tissue Engineering
Section V. Enabling Tools
- Chapter 19. Biomarkers
  - I. Introduction
  - II. Disease- and Drug-Related Biomarkers
  - III. Biomarkers in Drug Development
  - IV. Biomarker Requirements
  - V. Biomarker Classification and Application
  - VI. Discovery of Molecular Biomarkers
  - VII. Surrogate End Points and Potential Disadvantages
  - VIII. Biomarker in Regenerative Medicine
  - XI. Quality Management
  - X. Personalized Medicine
- Chapter 20. Translational Animal Models for Regenerative Medicine Research
  - I. Introduction
  - II. Brief History of Translational Animal Models for Regenerative Medicine Research with an Emphasis on Hematopoietic and Immune System Regeneration
  - III. Musculoskeletal Tissue Engineering
  - IV. Soft Tissue Regeneration
  - V. Ocular and Brain Repair and Regeneration
  - VI. Heart Muscle and Vascular Regeneration
  - VII. Lung Regeneration
  - VIII. Engineered Intestinal, Liver, and Pancreas Regeneration
  - IX. Urogenital Repair and Bladder Tissue Engineering
  - X. Future Approaches: Stem Cells, Reprogrammed Cells, and Immunodeficient and Humanized Mouse Models for Tissue Regeneration
  - XI. Pros and Cons of Translational Animal Models for Regenerative Medicine Research
  - Acronyms and Abbreviations
- Chapter 21. Translational Imaging for Regenerative Medicine
  - I. Introduction
  - II. Contrast Agents
  - III. Ultrasound
  - IV. X-ray CT
  - V. Nuclear Imaging
  - VI. Magnetic Resonance Imaging
  - VII. Multimodal Imaging
  - VIII. Summary
Section VI. Clinical Aspects of Regenerative Medicine
- Chapter 22. Skin and Skin Appendage Regeneration
  - I. Introduction
  - II. Epidermis
  - III. Hair Follicle and Sebaceous Gland
  - IV. Sweat Gland
  - V. Future Challenges in Skin Regeneration
- Chapter 23. Clinical Aspects of Regenerative Medicine: Tendon, Ligament, and Joint
  - I. Introduction
  - II. Platelet-Rich Plasma
  - III. Bone Marrow Concentrate
  - IV. Discussion
  - V. Practical Considerations
  - VI. Summary
- Chapter 24. Bone Regeneration
  - I. Clinical Importance of Bone Healing
  - II. Basic Biology of Bone Healing
  - III. Bone Regeneration
  - IV. Current and Future Repair Strategies
  - V. Conclusions
- Chapter 25. Regenerative Medicine Therapies Using Adipose-Derived Stem Cells
  - I. Adipose-Derived Stem Cells for Therapy
  - II. Regulatory Process
  - III. Current Clinical Trials and Evolving Potential
  - IV. Methods of Lipoharvest
  - V. Methods of SVF Isolation: Automated versus Manual
  - VI. Flow Cytometry Analysis
  - VII. Concluding Remarks
  - List of Abbreviations
- Chapter 26. Transplantation of Myogenic Cells in Duchenne Muscular Dystrophy Patients: Clinical Findings
  - I. Introduction
  - II. Cells with Myogenic Capacity: Candidates for Transplantation
  - III. Gene Complementation
  - IV. Neoformation of Myofibers
  - V. Generation of Donor-Derived Satellite Cells
  - VI. Control of Acute Rejection
  - VII. Potential Future Development in the Control of Acute Rejection
  - VIII. The Importance of the Method of Cell Injection
  - IX. Potential Future Developments in the Method of Cell Injection
  - X. Conclusions
- Chapter 27. Regeneration of the Vascular System
  - I. Introduction
  - II. Tissue Neovascularization
  - III. Bioengineered Blood Vessels
  - IV. Summary
- Chapter 28. Hematopoiesis in Regenerative Medicine
  - I. Introduction/Historical Perspective
  - II. HSC Transplantation for Hematologic Diseases/Disorders
  - III. Alternate Sources of HSC
  - IV. HSC Transplantation to Induce Immunological Tolerance and Treat Autoimmunity
  - V. HSC Transplantation for Diseases of Nonhematopoietic Organs/Tissues
  - VI. Summary
- Chapter 29. The Application and Future of Neural Stem Cells in Regenerative Medicine
  - I. Establishment of Neural Stem Cells and Induction of Pluripotent Cells for Transplantation
  - II. Future Roles of Stem Cell Research
  - List of Abbreviations and Acronyms
- Chapter 30. Central Nervous System
  - I. Introduction
  - II. Regenerative Strategies in the Injured CNS
  - III. The Current Landscape of Clinical Trials in the CNS
  - IV. Strategies to Address Clinical Challenges for Regenerative Medicine in the CNS
  - V. Conclusions and Outlook
- Chapter 31. In situ Tissue Engineering Bone Regeneration in Jaw Reconstruction
  - I. The Biology of In situ Tissue Engineering
  - II. How the Three Components of the Tissue Engineering Triangle Work to Regenerate Bone
  - III. The Clinical Technique Required
  - IV. The Prepared In situ Tissue Engineered Bone Graft
  - V. Surgery to Place an In situ Tissue Engineered Graft
  - VI. Surgery for Mandibular Reconstruction
  - VII. Surgery for Maxillary Reconstruction
  - VIII. Biologic Activity within an ISTE
  - IX. Outcome Analysis: A Three-Cohort Study
  - X. Conclusion
  - List of Acronyms and Abbreviations
- Chapter 32. Regenerative Medicine for Diseases of the Respiratory System
  - I. Introduction
  - II. Regenerative Medicine
  - III. Tissue Engineering
  - IV. Upper Airways: Nasopharynx Vocal Cords and Larynx
  - V. Trachea
  - VI. Lungs
  - VII. Cell Therapy
  - VIII. Clinical Translation and Its Barriers
  - IX. Mesenchymal Stromal Cells
  - X. Mononuclear Cells
  - XI. Endothelial Progenitor Cells
  - XII. ESCS and iPSCs
  - XIII. Other Cell Types
  - XIV. Pharmacological Intervention
  - XV. Conclusion
- Chapter 33. Renal System
  - I. Introduction
  - II. Structure and Function of the Kidney
  - III. Acute and Chronic Kidney Disease
  - IV. Evidence of Normal Kidney Repair in Humans
  - V. Regenerative Strategies for Kidney Repair
  - VI. Cell Therapy for Renal Failure
  - VII. Tissue Engineering Approaches for Renal Failure
  - VIII. Conclusion
- Chapter 34. Translational Regenerative Medicine–Hepatic Systems
  - I. Introduction: Hepatic Systems
  - II. Liver Diseases
  - III. Therapies for Liver Diseases
  - IV. Liver Transplantation
  - V. Cellular Therapies
  - VI. Gene Therapy Treatments for Liver Disease
  - VII. Liver Bioengineering
  - VIII. Liver Assist Devices
  - IX. Future Directions
  - X. Concluding Remarks
- Chapter 35. Advances in Neo-Innervation of the Gut
  - I. Enteric Nervous System: Development and Functions
  - II. Enteric Nervous System Disorders: Descriptions and Clinical Treatments
  - III. Enteric Neural Cell Transplantation for Treatment of Aganglionic Disorders
  - IV. Utilizing Tissue Engineering to Improve Enteric Neural Cell Therapy
  - V. Conclusion
- Chapter 36. Genitourinary System
  - I. Introduction
  - II. Genitourinary System Regeneration
  - III. Conclusions and Future Outlook
- Chapter 37. Clinical Aspects of Regenerative Medicine: Immune System
  - I. Regenerating the Immune System by Entire or Partial Replacement
  - II. Engineering the Immune System with Specialized Component Therapies
  - III. Mesenchymal Stem Cells: Specialized Component Therapy Affecting Both Immune Responses and Regeneration
  - IV. Conclusions
Section VII. Translational Aspects of Regenerative Medicine
- Chapter 38. Development of Appropriate Imaging Methods to Trace Cell Fate, Engraftment, and Cell Survival
  - I. Introduction
  - II. Imaging Approaches
  - III. Magnetic Resonance Imaging
  - IV. Positron Emission Tomography Imaging
  - V. Optical Imaging
  - VI. Emerging Approaches
  - VII. Selecting a Modality
  - VIII. Validation
  - IX. Conclusions
- Chapter 39. Gap Analysis to Target Therapies
  - I. Introduction
  - II. The Pathway
  - III. Preclinical Data Packages
  - IV. Preclinical Expectations
  - V. Product Manufacturing
  - VI. Clinical Protocol
  - VII. Special Concerns for Cell and Gene Therapy Trials
- Chapter 40. Funding for the Translation of Regenerative Medicines
  - I. Introduction
  - II. Investment Justification
  - III. Global Funding of Regenerative Medicine
  - IV. Private Funding of Regenerative Medicine
  - V. Stimulating Private Investment
  - VI. Creative Government Financing Mechanisms
  - VII. Conclusion
Index

Life Sciences

Physical Sciences & Engineering

Social Sciences & Humanities

Health

Translational Regenerative Medicine

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Anthony Atala

Julie Allickson