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Exploratory clinical testing of neuroscience drugs

Nature Neuroscience volume 5, pages 1023–1025 (2002)Cite this article

Other articles in this special issue illustrate the remarkable promise of today's neuroscience for ameliorating the impact of nervous system diseases: a massive impact measurable in deaths, suffering, lost human potential and drained resources. At last we have the tools and understanding to begin to identify rational countermeasures against scourges that have long had names but not treatments. In just the past few years, the first drugs useful in the amelioration of stroke, multiple sclerosis, amyotrophic lateral sclerosis and Alzheimer's disease symptoms, as well as several new anticonvulsants, have been approved by U.S. and European regulatory agencies. New therapeutic strategies are emerging at a quickening pace, filling the research pipelines in biotechnology and pharmaceutical companies. In tantalizing view are drugs that may slow the progression of Alzheimer's or Parkinson's disease, reduce the brain's vulnerability to ischemic insults, alleviate depression, anxiety or pain refractory to current medications—and even help restore functions lost to spinal cord injury.

This ongoing revolution in the identification of novel therapeutic concepts is well matched to advances in the science of practical drug development. Medicinal chemists have learned how to hit identified molecular targets with precision and efficiency, drawing structural clues from crystal structures, molecular modeling and the high-throughput screening of compound libraries; resulting leads are filtered with the eye of experience and refined through combinatorial chemistry. Elaborate counterscreening ensures high levels of specificity, and candidate compounds are run through a gauntlet of assays designed to turn back those with potential for organ toxicity, carcinogenicity, teratogenicity, immunogenicity or interference with the metabolism of other drugs.

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  1. Merck Research Laboratories, WP42-212, 770 Sumneytown Pike, West Point, 19486, Pennsylvania, USA

    Dennis W. Choi

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  1. Dennis W. Choi
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Choi, D. Exploratory clinical testing of neuroscience drugs. Nat Neurosci 5 (Suppl 11), 1023–1025 (2002). https://doi.org/10.1038/nn930

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