Stem Cell Therapies: Opportunities for Ensuring the Quality and Safety of Clinical Offerings: Summary of a Joint Workshop
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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.
interpret that?” Clinics that have good laboratory practices, that are helping patients, and are publishing their results could be identified and certified. 52 STEM CELL THERAPIES However, if governments, regulators, and scientists are simply trying to shut every clinic down, patients will view them as part of the problem rather than part of the solution. WHAT NEEDS TO BE KNOWN? Van Bokkelen raised the issue of the size of the stem cell clinic industry and its potential impacts. Rough
is “long overdue for the development of a coherent national strategy and a national [or international] initiative” on stem cell therapies. It could marshal expertise and pool resources to drive the development of the field, he said. A CONTINUED DIALOGUE In his closing remarks, Fineberg emphasized the opportunity for a higher order of collaboration across the scientific, clinical, and patient communities. Continued dialogue and a clearer definition of needs could hasten the arrival of demonstrably
quality and safety of stem cell therapies. The staff also wish to thank the members of the planning committee for their work in developing an excellent workshop agenda. The project director would like to thank project staff, who worked diligently to develop both the workshop and the resulting summary. xiii Contents ABBREVIATIONS AND ACRONYMS 1 2 INTRODUCTION AND THEMES OF THE WORKSHOP Background, 3 Themes of the Workshop, 6 Organization of the Report, 7 STEM CELL THERAPIES—KNOWNS AND
Weill Medical College Christopher Scott Stanford University Aditi Sengupta Department of Continuing Medical Education Harvard Medical School Douglas Sipp RIKEN Center for Developmental Biology Susan Soloman New York Stem Cell Foundation Alan Spitzer MEDNAX National Medical Group Sally Temple Regenerative Research Foundation Kemi Tomobi Student National Medical Association Gil Van Bokkelen Athersys, Inc. Luciano Vidal Hospital Lanari David C. Vladeck Georgetown University School of Law John
claims], the numbers are big.” The home countries of patients are difficult to ascertain, but some evidence suggests that the majority of patients are traveling from developed countries to developing countries, Petersen said. Stem cell clinics are a global phenomenon, Caulfield said, with clinics spread around the world (Regenberg et al., 2009). A survey of 224 patients, most of whom were from the United States, Canada, and the United Kingdom, found that about 60 percent were male and about 44