Anthony B. El-Khoueiry

Anthony B. El-Khoueiry, MD is Professor of Clinical Medicine and Associate Director for Clinical Research at USC Norris Comprehensive Cancer Center, where he leads the Gastrointestinal Oncology Program. He is internationally recognized for leading the CheckMate 040 trial—the landmark dose-escalation and expansion study of nivolumab (anti-PD-1) in advanced hepatocellular carcinoma—which was the first major immune checkpoint inhibitor trial in HCC and demonstrated durable objective responses in sorafenib-naive and sorafenib-pretreated patients, securing an accelerated FDA approval for nivolumab in 2017. Dr. El-Khoueiry subsequently led CheckMate 040 expansion cohorts evaluating nivolumab plus ipilimumab, which demonstrated a 32% ORR and durable responses in sorafenib-pretreated HCC, leading to a second FDA approval specifically for this combination in 2020. His research encompasses the immunobiology of the liver tumor microenvironment, mechanisms of primary and acquired resistance to checkpoint inhibitors in HCC, and next-generation combination strategies. He has been a leading trial investigator for multiple hepatobiliary malignancies and is a respected educator and mentor in GI oncology.