Stem Cells Come of Age

Summary

Commercialization of stem cells can potentially help to treat an astounding variety of medical conditions. After a slow start, the stem cell age is finally poised to begin, as numerous factors converge to catapult stem cell technology into the medical mainstream. This report considers:

• The current state of stem cell science and technology
• Supplies and services
• Major applications of stem cell science
• Sources of funding, regulatory hurdles, and the commercial outlook
• IP challenges, public perception, bioethical concerns, and diversity in policies

Stem cell science is on the precipice of becoming big business. These enigmatic cells lie at the heart of a fledgling technology with great clinical promise. Stem Cells Come of Age analyzes the current state of stem cell science and technology, and explores some of the non-scientific issues that are part of the emerging, complex, stem cell picture. We begin by tracing the experiments that set the stage for today's stem cell technology, as well as examining the state of ""stemness"" and factors outside the genome that affect cell fate. Human embryonic stem (ES) and induced pluripotent stem (iPS) cells are contrasted-while each can become any cell type of the body (i.e., are pluripotent), each has its own niche.

We consider the technological landscape, continuing our look at ES and iPS cells with a description of many types of multipotent, or ""adult,"" stem cells, which are more restricted in their fates, and how we might use them. We cover the care and feeding of stem cells-their isolation, derivation, culture, and characterization. We explore how stem cells fit into tissue engineering, and then consider suppliers of reagents, devices, and materials.

Stem Cells Come of Age highlights three major applications of stem cell science. In drug discovery and development, stem cells are being used to identify novel drug targets, describe the earliest inklings of disease, and reveal disease subtypes. ES and iPS cells will be particularly valuable in the second major arena for stem cell science, recapitulating pathogenesis in vitro. In the third area, therapeutics, we take a closer look at three therapeutic goals: treating the failing heart, cancer, and eye diseases.

We present a commercial outlook, looking at a mix of areas that are critical to the translation of stem cell science from bench to bedside. Pharma is just beginning to fund stem cell research at academic centers and biotech companies, and venture capitalists are on the lookout for researchers who can succinctly pitch a stem cell-based product or service.

Expert roundtables and exclusive interviews offer the voices of those in the field of stem cell research. These conversations reveal how the people who invented the enabling technologies and have done groundbreaking experiments think and share ideas. Finally, we profile a handful of companies that work with stem cells or supply the tools of the trade, selected for their diversity.

Contents

• Chapter 1
• Introduction
  • 1.1. The State of Stem Cell Science and Technology
  • Types of Stem Cells
  • Stem Cell R&d
  • 1.2. Stem Cells in Normal Development
  • from Fertilized Ovum to Multicellular Organism
  • Defining Characteristics of Stem Cells
  • 1.3. from Stem Cell Science to Technology
  • Early Thoughts on Reprogramming and Cloning
  • The Origin of Embryonic Stem (Es) Cells
  • The State of Stemness
  • Plurinet, The Regulatory Network behind Pluripotency
  • Epigenetics and Micrornas
  • Induced Pluripotent Stem (Ips) Cells
  • The Future: Hes or Ips Cells?
  • Medical Tourism: Protecting Healthcare Consumers
• Chapter 2
• The Technological Landscape
  • 2.1. Adult (Tissue-Specific) Stem Cells
  • Reproductive Structures and Prenatal Tissues
  • Postnatal Tissues
  • Hematopoietic Stem Cells (Hscs)
  • Mesenchymal Stem Cells (Mscs)
  • Perivascular Stem Cells (Pericytes)
  • Neural Stem and Progenitor Cells
  • 2.2. Tools and Technologies in Context
  • Culturing Es Cells
  • Characterizing Es Cells
  • Maintaining or Differentiating Es Cells
  • Umbilical Cord Stem Cells to Treat Bronchopulmonary Dysplasia
  • 2.3. Tissue Engineering
  • Different Tissues, Different Challenges
  • Cells + Ecm
  • Hydrogels
  • 2.4. Supplies and Services
  • The Care and Feeding of Stem Cells: Media, Markers, and Matrix
  • Cells for Sale (or Free)
• Chapter 3
• Applications
  • 3.1. Drug Discovery and Development
  • Identifying New-and Not So New-drug Targets
  • Targeting The Pre-Manifest Stage of Disease
  • Revealing Disease Subtypes
  • Toxicology
  • 3.2. Stem Cells to Recapitulate Disease
  • 3.3. Therapeutics
  • 3.4. A Trio of Therapeutics
  • The failing Heart
  • Cancer Stem Cells
  • The Eye
• Chapter 4
• Commercial Outlook
  • 4.1. Funding
  • Pharma Comes on Board
  • Venture Capital
  • 4.2. Regulatory Hurdles
  • 4.3. Marketing Concerns
  • 4.4. Intellectual Property
  • Human Es Cells: The Warf Patents
  • Adult Stem Cells: Patenting Neurospheres
  • Ips Cells: Donor Sources
  • 4.5. Public Perception of Stem Cell Technology
  • 4.6. Hype and False Hope Breed Medical Tourism
  • 4.7. Bioethical Concerns
  • Medical Tourism Revisited
  • Protecting Cell Donors
  • Stem Cell Banks
  • 4.8. A Policy Patchwork
  • Global Policy
  • The United States: A Closer Look
• Chapter 5
• Expert Interviews
  • 5.1. Expert Roundtable 1: Induced Pluripotent (Ips) Cells
• Participants: George Daley, Md, Phd, past President of Isscr and Associate in Medicine at Children's Hospital Boston; Shinya Yamanaka, Md, Phd, Senior Investigator, Gladstone Institute of Cardiovascular Disease and The L.K. Whittier Foundation Investigator in Stem Cell Biology and Professor of Anatomy at The University of California, San Francisco and Director, Center for Ips Cell Research and Application and Professor, Institute for Frontier Medical Sciences, Kyoto University, Japan; Kathrin Plath, Phd, Assistant Professor at Ucla; Rudolf Jaenisch, Md, Member of The Whitehead Institute and A Professor of Biology at Mit (Cambridge, Ma); Junying Yu, Phd, Assistant Scientist in James Thomson's Lab at The University of Wisconsin in Madison; Sir Ian Wilmut, Phd, Director of The Centre for Regenerative Medicine at The Queen's Medical Research Institute, University of Edinburgh.
  • 5.2. Expert Roundtable 2: Stem Cells-from Bench to Bedside
• Participants: Katherine A. High, Md, Professor of Pediatrics at The University of Pennsylvania School of Medicine and Director of The Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia; Giulio Cossu, Md, Director of The Stem Cell Research Institute, San Raffaele Scientific Institute, Milan; Judy Lieberman, Md, Phd, Senior Investigator at The Immune Disease Institute, Professor of Pediatrics, and Director, Division of Aids, All at Harvard Medical School; Alok Srivastava, Md, Head of The Department of Hematology at Christian Medical College (Vellore, India).
  • 5.3. Interview with Dennis Steindler, Phd
  • Executive Director, The Evelyn F. and William L. Mcknight Brain Institute, University of Florida in Gainesville
  • 5.4. Interview with Sally Temple, Phd
  • Scientific Director, The New York Neural Stem Cell Institute
  • Professor, Albany Medical College and The University at Albany
  • 5.5. Interview with Amy Wagers, Phd
  • Investigator, Joslin Diabetes Center
  • Assistant Professor of Pathology, Harvard Medical School
• Chapter 6
• Selected Company Profiles
  • 6.1. Cellartis
  • 6.2. Cryo-Cell
  • 6.3. Geron
  • 6.4. Novocell
  • 6.5. Osiris Therapeutics
  • 6.6. Primegen Biotech
  • 6.7. Stem Cell Sciences
  • 6.8. Stemcells
  • 6.9. Vet-Stem Regenerative Veterinary Medicine
  • References
  • Glossary of Selected Terms
  • Company Index with Web Addresses
• Figures
  • Figure 1.1. Stem Cell Dividing
  • Figure 4.1. Tumor Neurosphere
• Tables
  • Table 1.1. Selected Clinical Trials for Stem Cell Treatments
  • Table 1.2. A Comparison of Basic Stem Cell Types
  • Table 1.3. Reprogramming Strategies
  • Table 1.4. Transgenes Used to Derive Human Ips Cells
  • Table 1.5. Pluripotency Genes Expressed in Both Es and Ips Cells
  • Table 1.6. A Snapshot of Stem Cell Companies
  • Table 2.1.The Medical Trash Heap: Sources of Adult Stem Cells
  • Table 2.2. Some Characteristics of Hes Cells
  • Table 2.3. Some Hes Cell Markers
  • Table 2.4. Some Compounds Required to Maintain Es Cells
  • Table 2.5. Steps in Obtaining and Processing Stem Cells
  • Table 2.6. Hydrogels Used in Tissue Engineering
  • Table 2.7. Steps in Using Hydrogels
  • Table 2.8. Surface Markers of Human Stem Cell Types
  • Table 3.1. First Cell Lines at Harvard Stem Cell Institute's Ips Core
  • Table 3.2. Selected Case and Pilot Studies with Promising Results
  • Table 3.3. A Wish-List for Stem Cell Therapies
  • Table 3.4. Some Cancer Stem Cell Markers
  • Table 4.1. Numbers of Potential Patients for Stem Cell Therapies
  • Table 4.2. Some Organizations That Fund Human Stem Cell Research
  • Table 4.3. Patents for Es Cells
  • Table 4.4. The UK Stem Cell Bank Regulatory Framework
  • Table 4.5. Members of The International Stem Cell Forum
  • Table 4.6. A Brief History of US Human Es Cell Policy
  • Table 4.7. A Brief History of Spanish Human Es Cell Policy