Abstract
Over the last two decades, a new therapeutic paradigm has emerged which has changed the way debilitating diseases may be treated in the future. Instead of using small-molecule drugs and devices to ameliorate the symptoms of disease, clinicians may harness the therapeutic power of cells to regenerate and cure diseases which currently represent a major unmet medical need. Advancements in the scientific knowledge of stem cell biology, along with highly encouraging preclinical proof-of-concept studies, in the last several years have served as a launch pad for testing such therapeutics in humans with life-threatening diseases. However, translating basic research findings into human therapy has not been straightforward and has presented many scientific, clinical, and regulatory challenges for scientists and clinicians. In this article, we provide a guidance framework for investigators for the design of early-phase clinical studies using stem cell-based therapeutics. Furthermore, important trial parameters and design features which must be considered before regulatory submission of such studies are highlighted.
Highlights
Stem cell therapy has enormous potential to alleviate human suffering and to provide solutions to conditions with a current unmet medical need
The number of clinical indications for use of these cells and the powerful therapeutic properties have produced a groundswell of interest by physicians around the world to translate scientific discoveries into patient benefit
To maximize the efficiency of translating discoveries into practice, physicians and scientists must understand how regulatory agencies assess new applications. These agencies must assess the current evidence of the potential for safely testing a novel therapy to determine the risk regarding its use in humans
Summary
Stem cell therapy has enormous potential to alleviate human suffering and to provide solutions to conditions with a current unmet medical need. The basis for evaluating the rigor of the safety studies in animals stems directly from the data associated with the reproducibility of the purity, stability, and potency of the cells generated by the protocol.
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