Introduction to Clinical applications of stem cells at the National Institutes of Health: a meeting held on 24 May 2004 at the Clinical Center of the National Institutes of Health, Bethesda, Maryland, USA

Abstract

Over the past 5 years, there has been an explosion of interest world-wide in expanding cell therapy applications to many aspects of clinical medicine. This has been fueled by still controversial reports of stem cell ‘plasticity’ or the ability of an already committed primitive cell in one tissue to redirect its potential and daughter cells along a completely different pathway; the identification of stem and progenitor cells in many adult tissues and demonstration in animal models that they can be manipulated to contribute to tissue regeneration; and progress in understanding and manipulating of human embryonic stem cells. The intramural research programs of many of the institutes comprising the National Institutes of Health (NIH) include scientists and clinicians at the forefront of these activities. The NIH Clinical Center, due to its long tradition of support of innovative clinical research and its extraordinary cell processing unit within the Department of Transfusion Medicine, is a national leader in translating basic laboratory advances into innovative cell therapies for the treatment of human disease. At this juncture in time, as many investigators in disciplines new to the use of cellular therapies plan clinical applications, we organized a 1-day symposium to share scientific insights and experience with clinical stem cell protocols, as well as delineate critical issues required for moving the stem cell field forward into clinical applications. Emphasis was placed on identification of key challenges to the field, including practical issues such as scale-up of cellular products from a laboratory bench or a mouse to good manufacturing processing for human application, and compliance with the ever-expanding regulatory framework for using celland gene-based therapies in humans. Our intent was to create a community of investigators who will come together for scientific collaborations and institutional planning in order for the NIH Clinical Center to be poised to implement the myriad of new cellular therapy protocols that will come forward in the next several years. We began with presentations from hematology/oncology and stem cell transplantation investigators, who defined and characterized stem and progenitor cells and relayed their extensive clinical experience in novel and complex allogeneic stem cell and T-cell therapies in humans. Next, we moved to discussions from investigators studying mesenchymal stem cells to regenerate cartilage and bone, likely to move into the clinic in the near future. Then, we focused on the cardiovascular system, in which high-profile and often controversial basic and clinical studies have rapidly advanced. We finished our survey with presentations from investigators studying regenerative approaches to neurologic and endocrine disorders, areas that are particularly rich from a basic science perspective at the NIH. Shifting gear, we then focused on the more practical issues of tracking administered cells in vivo via novel imaging technology, producing cell therapy products for clinical administration, and the regulatory process required for clinical protocol approval. We hope this symposium, and the issue of Cytotherapy in which these presentations are summarized, will be of use to the intramural NIH community and investigators world-wide in the development of cell-based therapies to treat human disease.