Initial attempts to translate the results of promising basic research into novel therapies for the treatment of diseases in man often leads to disappointment as the effects seen in the pre-clinical modeling do not translate to benefits in the clinical trials. The use of autologous bone marrow derived progenitor cells to repair the heart following ischaemic injury has been suggested by some as an example of promising basic research that has failed to show similar promise when applied to man. Results of experiments in animal models of acute and chronic myocardial ischaemia suggested that delivery of autologous bone marrow derived cells to the site of injury could lead to a dramatic recovery of cardiac function with the demonstration of various mechanisms ranging from transdifferentiation of cells to the promotion of angiogenesis described. It is not surprising that these positive results (with little in the way of negative findings published) caught the attention of clinical academics given the acute need for new treatment strategies for patients with cardiovascular disease and a rapid process of translation into clinical studies occurred. Now, several years later the results of the clinical trials in which only small improvements in cardiac function have been demonstrated in response to autologous bone marrow cell therapy indicate to some that this is yet another example of a therapeutic approach identified by basic research that has been lost in translation. Before this view is widely accepted and efforts moves away from this area of research it is important to carefully consider the significance of the results of the clinical trials and importantly the relevance of the tools that have been used to measure physiological end-points that have been used as markers of success. The ongoing ‘REGENERATE’ series of clinical trials will be used to highlight these issues and the importance of defining success in a way that is clinically relevant.
University of Oxford (2011) Proc Physiol Soc 23, SA9
Research Symposium: Bone marrow stem cell therapy – a clinical viewpoint
A. Mathur1
1. Queen Mary, University of London, London, United Kingdom.
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Where applicable, experiments conform with Society ethical requirements.