Translating stem cell research to the clinic: a primer on translational considerations for your first stem cell protocol
O’Brien, Timothy Creane, Michael Windebank, Anthony J. Terzic, Andre Dietz, Allan B.
O’Brien, Timothy
Creane, Michael
Windebank, Anthony J.
Terzic, Andre
Dietz, Allan B.
Publication Date
2015-08-22
Keywords
Type
Article
Downloads
Citation
O’Brien, Timothy; Creane, Michael; Windebank, Anthony J. Terzic, Andre; Dietz, Allan B. (2015). Translating stem cell research to the clinic: a primer on translational considerations for your first stem cell protocol. Stem Cell Research & Therapy 6 ,
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.
Funder
Publisher
Springer Nature
Publisher DOI
Rights
Attribution-NonCommercial-NoDerivs 3.0 Ireland