Foreword

Objectives
Target Audience
Scope and Content of Report
Methodology

Chapter One: Introduction

What Exactly Is a Stem Cell?
History of the Technology
Corporate Development of Stem Cell Technologies
When Will There Be Payoff?
Stem Cell Background
• Stem Cell Terminology
• Stem Cell Medical Science

Regenerative Biology
Challenges in Stem Cell Research
• Immune Response
• Can Stem Cells Be Effective in Regenerating Tissues?

Technical Challenges in Stem Cell Research
• Embryonic Stem Cells
• Adult Stem Cells

Comparisons of Embryonic and Adult Stem Cells
• Similarities: Adult and Embryonic Stem Cells
• Differences: Adult and Embryonic Stem Cells
• What Is Known About Human Pluripotent Stem Cells?

Adult Stem Cells
• What Is Known About Adult Stem Cells?

NIH Funding for Stem Cell Research

Chapter Two: Demographics and Disease Modalities

Potential Disease Modalities
Summary of Important Similarities and Differences Between Adult and Embryonic Stem Cells
Actual and Potential Uses of Human Adult Stem Cells
• Potential Uses of Human ES Cells (Plus Certain Nonhematopoietic Uses of Adult Stem Cells)
• Using Human Embryonic Stem Cells for Therapeutic Transplants
• Special Considerations Regarding the Therapeutic Application of Human ES Cells
• Other Potential Uses of Human Embryonic Stem Cells
• Conclusions

Chapter Three: Stem Cell Products In Research And Development

The Potential for Stem Cell Products
Areas of Product Development
Neural, Brain, and Spinal Stem Cell Applications
Liver Stem Cell Applications
Pancreas Stem Cell Applications
Cardiac Stem Cell Applications
Renal Stem Cell Applications
Blood Stem Cell Applications
Retinal Stem Cell Applications
Diabetes—Stem Cell Applications
Orthopedics—Stem Cell Applications
Other Stem Cell Applications
Indications

Chapter Four: Stem Cell Companies

Introduction
• Companies Involved in Stem Cell Product Development
• Source and Type of Stem Cells
• Therapeutic Area Targeted by Stem Cell Technology
• Safety Issues and Cellular Therapies

Select Stem Cell Company Profiles
• Aastrom Biosciences, Inc.
• Bioheart, Inc.
• BioTransplant, Inc.
• Cell Based Delivery, Inc.
• Diacrin, Inc.
• Geron Corporation, Inc.
• ImClone Systems Incorporated
• Incara Pharmaceuticals, Inc.
• Islet Technology
• MorphoGen Pharmaceuticals, Inc.
• NeoTherapeutics, Inc.
• NeuralStem Biopharmaceuticals, Ltd.
• Nexell Therapeutics, Inc.
• Osiris Therapeutics, Inc.
• ReNeuron, Inc.
• SELECT Therapeutics, Inc./Cell Science Therapeutics, Inc.
• Stem Cell Pharmaceuticals Inc.
• StemCells, Inc.
• Titan Pharmaceuticals, Inc.

Chapter Five: Academic Organizations In Stem Cell Research Activities

Introduction
Highlights of Recent Academic Research Activities
Academic and Government Organizations Listed by Area of Activity
Literature Review of Academic and Non-Commercial Organizations Involved with Stem Cells
• Hematopoietic Stem Cell Transplantation: mostly stem cell support institutions that published clinical trials or research involving stem cells (2000-2001)

Chapter Six: Stem Cell Research Suppliers and Enabling Technologies

Introduction
Stem Cell Supply Research Activity
Effectiveness of Research Products
Challenges to the Supply Industry
Activities of Suppliers in the Field
Media Development Support for Stem Cell Research
Acquisition Activity
Established Suppliers
Companies on the NIH Stem Cell Registry
• Sixty-Four Stem-Cell Lines Eligible for Funding

Concerns about Competence of Approved Cell Lines
Organization Profiles: Organizations with NIH-Listed Stem Cell Lines
Stem Cell Products Background
• Bone Marrow Stroma
• Mesenchymal Stem Cells
• Stem Cell Factor
• CFU Assays
• Liquid Cell Culture Media
• Isolation of Stem Cells
• Clonogenic Progenitor Assays, In Vitro Tests

Chapter Seven: Legislative, Social, and Political Issues

Concerns Regarding Origins of Stem Cells
• Human Face to the Debates
• Political Rulings

Potential for Irresponsible Research
• Human Cloning
• Scientists Create Human Embryos to Harvest Stem Cells
• Risks Associated with Stem Cells in Clinical Applications
• Risks Associated with Human Cloning
• Private Sector Cloning

Impact of Restrictions on Embryonic Stem Cells
Alternative Sources of Stem Cells
• Sources of Human Embryonic Stem Cells
• Sources of Other Human Stem Cells
• Limitations of Adult Stem Cells

Events in Stem Cell Legislation and Ethics
• Public Opinion on Stem Cell Issues
• Moral Issues—Opposing Views
• Summary of NBAC Review: Ethical Issues Associated with Human Stem Cell
• Research
• National Institutes of Health Guidelines for Research Using Human Pluripotent Stem Cells
• August 2001 Bush Administration Federal Funding Limitations

International Stem Cell Rules and Legislation
• European Group on Ethics in Science and New Technologies
• Country-Specific Legislation and Guidelines

Chapter Eight: Strategic Issues

The Investment Climate
• Funding to Survive Clinical Trials
• Licensing Stem Cell Technologies
• Technology Partnerships
• Stem Cell Licensing Issues

US Regulatory Issues
• Federal Agencies Involved in Stem Cell Regulation
• FDA Regulatory Overview
• Cell and Tissue Regulations
• Implementation of the New Regulations
• Standard Methods
• FDA Market Approvals
• Process for Approval of Drugs and Biologics
• Process for Approval of Medical Devices
• Regulatory Issues Specific to Stem Cell Products

Regulatory Issues in Europe and Japan
• Marketing Outside the United States

Reimbursement
Enabling Technologies
• Growing or Culturing Stem Cells
• Patents and Stem Cells

Appendix A: Stem Cells: A Primer

National Institutes of Health
• What is a Stem Cell?
• How are pluripotent stem cells derived?
• Potential Applications of Pluripotent Stem Cells
• Adult Stem Cells
• Summary

Appendix B: Stem Cell Researchers and Links

Coalition for the Advancement of Medical Research

Appendix C: Stem Cell Glossary

Key Terms in Stem Cell Technology
General Glossary

List of Exhibits

Table 1-1: Timeline of Human Embryonic Stem Cell Research
Table 2-1: Diseases for Which Unrelated Marrow Donor Transplants Have Been Performed
Table 4-1: Companies, Contact Details, and Descriptions
Table 4-2: Cord blood and Therapeutic Stem Cell Services Companies
Table 5-1. Stem Cells Academic/Government Organizations
Table 6-1: Companies and Research Laboratories with Stem Cell Lines
Table 6-2. Select Organizations Providing Cell Lines
Table 6-3. Cell Culture Systems: Media, Sera and Cell Growth Systems for Stem Cell Reagents: Stem Cell Factors/Cytokines, Antibodies, Conjugates, Growth Factors, Kits, Services
Table 7-1: Events in Stem Cell Policy and Ethics
Table 7-2: Results of Poll asking "Do You Support Federal Funding for Stem-Cell Research?"
Table 7-3: Organizations Involved in Bioethics
Table 7-4: Summary of September 1999 NBAC Recommendations on Stem Cell Research
Table B-1: Academic and Government Laboratories Involved in Stem Cell Research Sorted by Organization
Table B-2: Researchers at Commercial Stem Cell Organizations Contributing to 7/01 NIH Report
Table B-3: Stem Cell Web Links