Cell-Based Therapeutics in Stroke: An Industry Perspective

Abstract

Although stroke is the second leading cause of death worldwide, more than 1000 published preclinical studies and more than 100 clinical trials have failed to confirm even a single neuroprotective agent for the treatment of this devastating disease. The solution for future drug development of stroke therapeutics must be truly transformational, considering less emphasis on animal models of neuroprotection, greater emphasis on mechanisms that are more pleiotropic and clinical trials that address critical, often neglected, aspects of study design, such as population homogeneity, endpoints, and outcomes specifically related to baseline deficits and ensuring that the timing of endpoint measurement provides a sufficient epoch of time to demonstrate improved outcome. Cell therapy may provide the field with the disruptional catalyst to address the shortcomings of the previous several decades, assuming that our understanding of the potential benefit of cell therapy in a therapeutic role can advance more in parallel with our technical capabilities for their utilization. The great promise of stem cell therapy to repair, replace, and/or redirect endogenous stem cells provides an exciting alternative therapeutic strategy for the treatment of stroke and many other diseases with an unmet medical need.