Mitochondrial Transplantation in Ischemic Stroke: Insights from a First-in-Human Brain Trial

This review evaluates autologous mitochondrial transplantation as a novel therapeutic strategy for cerebral ischemia, focusing on its evolution, methodological advances, and therapeutic potential. By integrating historical perspectives with recent advances, we discuss the progress and challenges of mitochondrial transplantation in mitigating ischemic injury, particularly in the context of ischemic-reperfusion injury (IRI).

Human trials in cardiac ischemia have established safety and effectiveness of mitochondrial transplantation, setting a precedent for application in cerebral ischemia. Our Phase 1 trial for cerebral ischemia involves defining safety endpoints and integrating transplant into the standard of care workflow for ischemic stroke treatment. These steps are essential for translation to clinical practice and demonstrate the promising but complex path ahead.

The clinical translation of mitochondrial transplantation as a therapeutic for cerebral ischemia requires further research to confirm safety and demonstrate efficacy in this setting.