The disastrous 1993 trial of fialuridine (FIAU), in which five patients died, prompted calls for rigorous new guidelines for drug testing, and a debate between the FDA and researchers over who was at fault in the tragedy. Now publication of an analysis of the Phase II study by NIH researchers in the New England Journal of Medicine has pinpointed the probable cause of the nucleoside analog's liver toxicity in the unique structure of the compound.
FIAU, which was being tested in 15 patients with chronic hepatitis B, caused severe hepatotoxicity in seven patients. Of those seven, five