Unpicking a clinical trial death

Biotrail building in Rennes, France

Source: © Kevin Niglaut/ABACA/PA Images

A new study shows up off-target effects of a drug that proved deadly, and highlights the pitfalls of drug discovery

The case of BIA 10-2474 is a terrifying one. It’s an experimental inhibitor of the enzyme fatty acid amide hydrolase (FAAH), a protein target with a number of potential uses in pain relief, anxiety, and several other conditions. Many readers will remember, though, that the compound was given to human volunteers in early 2016 with catastrophic results. One person died, and several endured severe neurological injuries, even though no such effects had been noted in animal studies. Since then, the question has been how this happened, and how it could have been allowed to happen at all.