Erythropoietin (EPO) has been used in hundreds of thousands of patients to treat anemia, but has been completely ignored as a clinical neuroprotective agent. However, recent evidence suggests that EPO is a more potent neurotrophic factor than the traditional nerve growth factors such as GDNF in a wide range of clinical situations. Furthermore, EPO is remarkably effective in protecting and developing dopaminergic neurons and in protecting dopaminergic neuron loss in a mouse model of Parkinson's disease. Taken together with its long safety record, these data suggest that EPO deserves immediate evaluation as a novel treatment for Parkinson's disease. The proposed studies are designed to simultaneously evaluate the mechanisms underlying the potent neuroprotective effects of EPO and determine its optimal, clinically relevant delivery strategy in anticipation of rapid translation to human studies.
