Abstract
Stroke remains the leading cause of disability and among the top five causes of death around the world. While recanalization therapies, first approved about two decades ago for ischemic stroke, have changed the nihilistic approach that prevailed in the past, their impact remains small. Hence, there is a critical need to translate novel stroke therapies to reduce the burden of ischemic stroke. There is a strong biologic rationale for cell-based therapy with a variety of stem cells in ischemic stroke, given that the brain’s repair process after stroke is driven by endogenous stem cells. Over the last 15 years, numerous preclinical studies of cell-based therapy for ischemic stroke show consistent promise of safety and efficacy. While these studies utilize a variety of cells and different routes of cell administration, the intra-arterial route of cell delivery has several attractive clinical attributes including minimally invasive and targeted delivery of cells to area of brain injury. This route of delivery of cells has been the focus of our laboratory and here we review the preclinical data and the recent rise in published early clinical trials of cell-based therapy in ischemic stroke. These clinical trial data in their sum total suggest robust safety of this approach and set the stage for the next phase of clinical trials to establish its clinical efficacy in this devastating disease.