Notably, MA242, an MDM2 inhibitor discovered in our lab, has unique mechanisms of action different from the existing MDM2 inhibitors under preclinical and clinical investigations and shows significant antitumor activity in preclinical pancreatic cancer (Wang et al., 2018) and hepatocellular carcinoma (HCC) (Wang et al., 2019b) models. The gene discussed is MDM2; the disease is hepatocellular carcinoma.