Repurposing drugs to treat neurological diseases

Excerpt

The development of new drugs can be expensive: on average, it costs over $2 billion to take a medication from inception to clinical practice. Drug discovery is particularly difficult in neuropsychiatric diseases as targets are often poorly defined, inaccessible, and not easily assayed. Furthermore, many neurological diseases progress slowly over many years, often requiring clinical trials examining efficacy to be large and have complex end points. However, one way of expediting drug development is to repurpose drugs from their original use to a new indication. Multiple examples exist in clinical medicine: sildenafil was an ineffective angina drug repurposed for erectile dysfunction; minoxidil was a hypertension drug repurposed for hair loss. These unexpected therapeutic discoveries were made serendipitously, and often as side-effects in clinical trials. Unbiased, high-throughput screens are now being used systematically to test libraries of clinically approved drugs in areas of medicine away from their usual indications. These screens can generate novel therapeutic avenues, shed light on molecular pathology, and lead directly to clinical trials. …