Treatment of human brain disease with erythropoietin (EPO) in order to achieve neuroprotection and/or neuroregeneration represents a totally new frontier in translational neuroscience. Rather than specifically targeting the cause of a particular disease entity, EPO nonspecifically influences components of the "final common pathway" that determine disease severity and progression in a number of entirely different brain diseases. EPO acts in an antiapoptotic, anti-inflammatory, antioxidant, neurotrophic, angiogenetic, stem cell-modulatory fashion. Importantly, it appears to influence neural plasticity. Most likely due to these properties, EPO has been found by many investigators to be protective or regenerative and to improve cognitive performance in various rodent models of neurological and psychiatric disease. The "Göttingen-EPO-stroke trial" has provided first promising data on humans for a neuroprotective therapy of an acute brain disease. Experimental EPO treatment to improve cognitive function in patients with schizophrenia represents a novel neuroregenerative strategy for a chronic brain disease. An exploratory trial in chronic progressive multiple sclerosis as an example of an inflammatory disease of the nervous system yielded first positive results of EPO treatment on both motor function and cognition. These promising results are just the beginning and will hopefully stimulate further work along these lines.