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Translational Regenerative Medicine
1st Edition - December 1, 2014
Editors: Anthony Atala, Julie Allickson
Language: English
Hardback ISBN:9780124103962
9 7 8 - 0 - 1 2 - 4 1 0 3 9 6 - 2
eBook ISBN:9780124104570
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.
Provides formulaic coverage of the landscape, process development, manufacturing, challenges, evaluation, and regulatory aspects of the most promising regenerative medicine clinical applications
Covers clinical aspects of regenerative medicine related to skin, cartilage, tendons, ligaments, joints, bone, fat, muscle, vascular system, hematopoietic /immune system, peripheral nerve, central nervous system, endocrine system, ophthalmic system, auditory system, oral system, respiratory system, cardiac system, renal system, hepatic system, gastrointestinal system, genitourinary system
Identifies effective, proven tools and metrics to identify and pursue clinical and commercial regenerative medicine
researchers working in different phases of the development of regenerative medicine tools including cell banking, stem cells and cell therapy, biomaterials, and tissue engineering; and scientific administrators and grad students in biotechnology fields
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
No. of pages: 606
Language: English
Edition: 1
Published: December 1, 2014
Imprint: Academic Press
Hardback ISBN: 9780124103962
eBook ISBN: 9780124104570
AA
Anthony Atala
Anthony Atala, MD, is the G. Link Professor and Director of the Wake Forest Institute for Regenerative Medicine, and the W. Boyce Professor and Chair of Urology. Dr. Atala is a practicing surgeon and a researcher in the area of regenerative medicine. Fifteen applications of technologies developed in Dr. Atala's laboratory have been used clinically. He is Editor of 25 books and 3 journals. Dr. Atala has published over 800 journal articles and has received over 250 national and international patents. Dr. Atala was elected to the Institute of Medicine of the National Academies of Sciences, to the National Academy of Inventors as a Charter Fellow, and to the American Institute for Medical and Biological Engineering.
Dr. Atala has led or served several national professional and government committees, including the National Institutes of Health working group on Cells and Developmental Biology, the National Institutes of Health Bioengineering Consortium, and the National Cancer Institute’s Advisory Board. He is a founding member of the Tissue Engineering Society, Regenerative Medicine Foundation, Regenerative Medicine Manufacturing Innovation Consortium, Regenerative Medicine Development Organization, and Regenerative Medicine Manufacturing Society.
Affiliations and expertise
Professor, Wake Forest Institute for Regenerative Medicine, USA
JA
Julie Allickson
Affiliations and expertise
Director, Translational Research, Wake Forest School of Medicine, Winston-Salem, NC USA
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