Can molecular and cellular neuroprotection be translated into therapies for patients?: yes, but not the way we tried it before.

BACKGROUND AND PURPOSE The concept of neuroprotection is based on sound scientific data derived in preclinical studies. However, candidate neuroprotectants have never been successfully translated to patients. METHODS A review of past approaches to cellular and molecular neuroprotection, preclinical neuroprotection studies, and clinical approaches was undertaken. RESULTS Although there is no evidence for fundamental barriers in biological principles that limit the translation of promising therapies to humans, ample evidence exists as to a lack of rigor in preclinical studies, obstacles posed by the complexities of acute ischemic stroke syndromes, and regulatory barriers. Alternative methods to translating stroke drugs may require trials in restricted stroke indications in well-defined patient populations. CONCLUSIONS The translational gap between cellular and molecular neuroscience and patient therapy may be bridged by first developing therapies for narrow stroke indications. A single success may stimulate further research, funding, and a capacity to generalize initial results to broader stroke populations.