Stem cells offer tremendous promise for advancing health and medicine. Whether being used to replace damaged cells and organs or else by supporting the body's intrinsic repair mechanisms, stem cells hold the potential to treat such debilitating conditions as Parkinson's disease, diabetes, and spinal cord injury. Clinical trials of stem cell treatments are under way in countries around the world, but the evidence base to support the medical use of stem cells remains limited. Despite this paucity of clinical evidence, consumer demand for treatments using stem cells has risen, driven in part by a lack of available treatment options for debilitating diseases as well as direct-to-consumer advertising and public portrayals of stem cell-based treatments. Clinics that offer stem cell therapies for a wide range of diseases and conditions have been established throughout the world, both in newly industrialized countries such as China, India, and Mexico and in developed countries such as the United States and various European nations. Though these therapies are often promoted as being established and effective, they generally have not received stringent regulatory oversight and have not been tested with rigorous trials designed to determine their safety and likely benefits. In the absence of substantiated claims, the potential for harm to patients - as well as to the field of stem cell research in general - may outweigh the potential benefits.

To explore these issues, the Institute of Medicine, the National Academy of Sciences, and the International Society for Stem Cell Research held a workshop in November 2013. Stem Cell Therapies summarizes the workshop. Researchers, clinicians, patients, policy makers, and others from North America, Europe, and Asia met to examine the global pattern of treatments and products being offered, the range of patient experiences, and options to maximize the well-being of patients, either by protecting them from treatments that are dangerous or ineffective or by steering them toward treatments that are effective. This report discusses the current environment in which patients are receiving unregulated stem cell offerings, focusing on the treatments being offered and their risks and benefits. The report considers the evidence base for clinical application of stem cell technologies and ways to assure the quality of stem cell offerings.

Contents

• THE NATIONAL ACADEMIES
• PLANNING COMMITTEE
• BOARD ON HEALTH SCIENCES POLICY
• BOARD ON LIFE SCIENCES
• Reviewers
• Acknowledgments
• Abbreviations and Acronyms
• 1. Introduction and Themes of the Workshop
  • BACKGROUND
  • THEMES OF THE WORKSHOP
  • ORGANIZATION OF THE REPORT
• 2. Stem Cell Therapies—Knowns and Unknowns
  • STEM CELLS AS REGULATED THERAPEUTICS
  • CLINICAL OFFERINGS USING STEM CELLS
• 3. Patients' Experiences
  • MEDICAL TOURISM: PATIENTS SEEKING STEM CELL TREATMENTS
  • PATIENTS SEEKING UNPROVEN OR UNREGULATED TREATMENTS
  • ONE PATIENT'S EXPERIENCES
• 4. Comparative Regulatory and Legal Frameworks
  • REGULATORY AND LEGAL FRAMEWORKS IN THE UNITED STATES
  • REGULATORY AND LEGAL FRAMEWORKS IN ITALY
  • REGULATORY AND LEGAL FRAMEWORKS IN JAPAN
  • REGULATORY AND LEGAL FRAMEWORKS IN MEXICO
  • REGULATORY AND LEGAL FRAMEWORKS IN CHINA
  • SUGGESTED IMPROVEMENTS FOR REGULATION
• 5. The Roles of Professional Societies
  • THE SOCIETY FOR ASSISTED REPRODUCTIVE TECHNOLOGY
  • THE INTERNATIONAL SOCIETY FOR STEM CELL RESEARCH
  • THE INTERNATIONAL SOCIETY FOR CELLULAR THERAPY
• 6. Moving Forward
  • WHAT IS THE MOST IMPORTANT PROBLEM?
  • WHAT NEEDS TO BE KNOWN?
  • WHAT COULD BE DONE?
  • A CONTINUED DIALOGUE
• References
• APPENDIXES
  • A Workshop Agenda
  • B Speaker Biographical Sketches
  • C Statement of Task
  • D Registered Attendees
  • E Glossary

Rapporteurs: Adam C. Berger, Sarah H. Beachy, and Steve Olson.

This project was supported by contracts between the National Academy of Sciences and the Alliance for Regenerative Medicine (unnumbered contract); California Institute for Regenerative Medicine (unnumbered contract); The Ellison Medical Foundation (Contract No. OP-CW-0405-13); Howard Hughes Medical Institute (unnumbered contract); International Society for Stem Cell Research (unnumbered contract); and U.K. Academy of Medical Sciences (unnumbered contract). The project was also sponsored by the Presidents' Committee Funds of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine. The views presented in this publication do not necessarily reflect the views of the organizations or agencies that provided support for the activity.

Suggested citation:

IOM (Institute of Medicine) and NAS (National Academy of Sciences). 2014. Stem cell therapies: Opportunities for ensuring the quality and safety of clinical offerings. Washington, DC: The National Academies Press.

NOTICE: The workshop that is the subject of this workshop summary was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine.