Clinical trials in neuroprotection. 23-25 January 2003, Key Biscayne, Florida, USA

Many common neurological and psychiatric disorders are accompanied by extensive neuronal loss, either acutely or chronically. These include stroke, head trauma, spinal cord injury, epilepsy, perinatal and perioperative hypoxia-ischaemia, Alzheimer's and Parkinson's diseases, amyotrophic lateral sclerosis (ALS) and even some aspects of schizophrenia and depression. Despite considerable research effort, no agent has been found that can protect the brain from any of these neurodegenerative conditions. 'Clinical Trials in Neuroprotection,' sponsored by the University of Pennsylvania, was held to address this situation in a unique way. Rather than focus on one neurodegenerative condition, the conference brought together researchers from a variety of neuroprotection areas. They reviewed basic mechanisms of neuronal injury, the animal models used in a variety of paradigms and the design and results of clinical trials in many neuroprotection areas. The presentations and extensive discussions focused on common issues encountered and lessons learned, and on developing innovative strategies for clinical evaluation of potential neuroprotective agents. Conference participants came to a general consensus that some mechanisms were the same for all conditions, models need to be improved, new clinical trial paradigms should consider drug combinations and/or sequential administration of neuroprotective agents, new biomarkers should be developed and a 'proof-of-principle' trial would be widely valuable and should be developed.