Fig. 5: The journey from development to clinical application of venetoclax is a triumph of rational cancer therapy design.

Extensive experimental data identifying the prosurvival BCL-2 family members as potential therapeutic targets, focused preclinical work combining BH3 mimetics with standard-of-care therapies in AML, and highly promising, international clinical trials have led to FDA approval of venetoclax with HMA or LDAC in an impressively short timeframe.