The pharmacopeia is full of drugs that seem to have outlived their usefulness or never found wide application: Long-used medications known to be safe that have been superseded by drugs that are considered more efficacious; newer drugs that, while highly effective, were found to cause severe adverse events in some people. By use of genetic methods, it may be possible to ‘repurpose’ some of these medications for other indications. If good genetic markers of safety and efficacy can be established, such repurposed drugs could be helpful for targeted populations, where acceptable risk:benefit ratios can be more easily achieved. Systematic efforts along these lines are now being initiated in the National Center for Advancing Translational Sciences (NCATS). NCATS is a new entity within the NIH that aims to catalyze the generation of innovative methods and technologies to enhance the development, testing, and implementation of diagnostic tests and therapeutic agents across a wide range of human ills [www.ncats.nih.gov/].
