To elucidate neuroprotective effect of granulocyte colony-stimulating factor (G-CSF) for acute spinal cord injury (SCI), we performed experimental studies and early phase of clinical trials. The results of experimental studies showed that G-CSF exerts neuroprotective effects for acute SCI via mobilization of bone marrow-derived cells into injured spinal cord, suppression of neuronal apoptosis, suppression of inflammatory cytokine up-regulation, suppression of oligodendrocyte apoptosis and promotion of angiogenesis. Next we moved to clinical trial. In a phase I/IIa trial, no adverse events were observed. Then, we conducted a non-randomized, non-blinded, comparative trial, which suggested the efficacy of G-CSF for promoting neurological recovery. We are now preparing a phase III trial to confirm G-CSF treatment efficacy for acute SCI. The current trial will include cervical SCI within 48 hours after injury. Patients will be randomly assigned to G-CSF and placebo groups and evaluated by double blinded manner. Our primary endpoint is changes in American Spinal Injury Association motor scores from baseline to 3 months. Each group will include 44 patients (88 total patients). After completion of this clinical trial, pharmaceutical approval will be applied for health insurance publication. G-CSF-mediated neuroprotection is a promising candidate for a novel therapeutic approach for SCI.
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