The Challenge of Interpreting Alanine Aminotransferase Elevations in Clinical Trials of New Drug Candidates

Bardoxolone methyl has shown promise in improving kidney function associated with type 2 diabetes. However, this drug also caused elevations in serum alanine aminotransferase (ALT) in some patients, raising liver safety concerns. In this issue, Lewis et al. present data supporting these elevations as an on-target effect of the drug, but the evidence provided will probably not carry much weight with regulators. This example highlights the urgent need for improved liver safety biomarkers.