Cell therapy for stroke: remaining issues to address before embarking on clinical trials

Background and purpose: Stroke remains a significant clinical unmet condition, with only 3% of ischemic patient population benefiting from the thrombolytic drug tissue plasminogen activator largely because of the drug's narrow 3-hour therapeutic window. Extending the stroke therapeutic window will greatly impact on treatment, care, and management of patients. Summary of Review- Cell therapy is appealing in this regard as it widens the stroke treatment opportunity by targeting the neurorestorative phase (ie, several hours to days and even weeks or months after stroke). Although compelling preclinical evidence reveals that transplantation of stem/progenitor cells is safe and effective in animal models of stroke, the laboratory data need to be evaluated on their translational relevance for clinical application. In addressing this issue, I borrow heavily from the conference proceedings of the 2007 STEPS (Stem Cell Therapeutics as an Emerging Paradigm in Stroke).

Conclusions: Translational research guidelines are being adapted by academic institutes, industry, National Institutes of Health (NIH), and Food and Drug Administration (FDA), and adhering to these preclinical criteria will provide the basis for advancing cell therapy in stroke from the laboratory to the clinic.