Anthony Boutros El-Khoueiry, MD

Anthony El-Khoueiry, MD, is an assistant professor of clinical medicine at the Keck School of Medicine of USC and director of clinical translation at the Southern California Clinical and Translational Science Institute (SC CTSI). He has extensive experience in clinical and translational research through his roles as medical director of the Clinical Investigations Support Office and director of the phase I drug development program at the USC Norris Comprehensive Cancer Center. He is the recipient of the National Cancer Institute Cancer Investigator Team Leadership Award in 2011.

Dr. El-Khoueiry has established a national reputation in clinical research-related hepatobiliary (liver, gall bladder and bile duct) and pancreatic cancers. He is a member of the National Cancer Institute Hepatobiliary Cancers Task Force and chair of the Southwest Oncology Group hepatobiliary cancers subcommittee. In addition, he is the principal investigator of several phase I, including first-in-human, clinical trials aimed at developing novel therapies for solid tumors. Dr. El-Khoueiry works with several basic scientists at USC on joint translational projects designed to identify molecular predictors of outcome and survival for patients with advanced gastrointestinal cancers.

Dr. El-Khoueiry completed his medical degree, residency in internal medicine and fellowship in hematology and oncology at USC. He unravels scientific and non-scientific mysteries in four languages: English, Arabic, French and Spanish.

• USC: Dean’s Scholar, 1998
• Division of Hematology: Researcher of the Year, 2003
• Southwest Oncology Group: Young Investigator Training Award, 2004
• National Cancer Institute: Clinical Investigator Team Leadership Award, 2011
   – 2012
• Golden Key National Honor Society, University of California, Irvine

• Immunosuppressive mechanisms responsible for development of non-viral liver cancer and control of its response to immune checkpoint inhibitors
  NIH · U01AA027681 · Sep 25, 2018 – Aug 31, 2023 · Role: Co-Principal Investigator
• NCTN – Network Lead Academic Participating Site: USC
  NIH · UG1CA180830 · Apr 17, 2014 – Feb 29, 2032 · Role: Co-Principal Investigator
• NCI NCTN- Network Lead Academic Participating Site: USC
  NIH · U10CA180830 · Apr 17, 2014 – Feb 28, 2019 · Role: Co-Principal Investigator

• BEACON-HCC: Best evidence and north american consensus on treatment allocation for hepatocellular carcinoma. Hepatology. 2026 Aug 07.. View in PubMed
• A first-in-human, open-label multicentre Phase 1 study of the orally administered E7386 in patients with selected advanced neoplasms. Br J Cancer. 2026 Sep; 135(5):736-746.. View in PubMed
• A Phase 1b Study of Botensilimab and Balstilimab in Treatment-Refractory Hepatocellular Carcinoma. Liver Cancer. 2026 Mar 30.. View in PubMed
• Phase 1 Study of Rezatapopt, a p53 Reactivator, in TP53 Y220C-Mutated Tumors. N Engl J Med. 2026 Feb 26; 394(9):872-883.. View in PubMed
• Botensilimab (Fc-enhanced anti-CTLA-4 antibody) plus balstilimab (anti-PD-1 antibody) in patients with treatment-refractory ovarian cancer. J Immunother Cancer. 2025 Dec 23; 13(12).. View in PubMed
• Assessing the Content and Quality of Online Patient Education Material on Clinical Trials From NCI-Designated Comprehensive Cancer Centers. J Natl Compr Canc Netw. 2025 Dec 18; 24(1).. View in PubMed
• Outcomes of Patients With Solid Tumor Treated on Phase I Clinical Trials Within a Comprehensive Cancer Center’s Private and Safety-Net Hospitals. JCO Oncol Pract. 2025 Dec 03.. View in PubMed
• Radiomic signatures to estimate survival in patients with advanced hepatocellular carcinoma treated with sorafenib: Cancer and Leukemia Group B 80802 (Alliance). ESMO Open. 2025 Dec; 10(12):105848.. View in PubMed
• Final Results from a First-in-Human Phase I Study of the Dual Isocitrate Dehydrogenase (IDH) 1/2 Inhibitor, LY3410738, in Advanced Solid Tumors Harboring IDH1 or IDH2 Mutations. Clin Cancer Res. 2025 Dec 01; 31(23):4920-4932.. View in PubMed
• A multicenter dose-defining/expansion phase 1b study of first-line regorafenib plus pembrolizumab in patients with advanced hepatocellular carcinoma. Hepatology. 2026 Jun 01; 83(6):1433-1445.. View in PubMed
• Safety and efficacy of intratumourally administered INT230-6 in adult patients with advanced solid tumours: results from an open-label phase 1/2 dose escalation study. EBioMedicine. 2025 Nov; 121:105980.. View in PubMed
• Navigating second-line therapy after immunotherapy in advanced HCC☆. JHEP Rep. 2026 Jan; 8(1):101630.. View in PubMed
• Characterizing Kupffer Cell Production of CD5 Antigen-Like and Its Function on Regulating Migration of Natural Killer T Cells. Am J Pathol. 2025 10; 195(10):1839-1853.. View in PubMed
• Isoform specific regulation of osteopontin by AKT2 in hepatocytes and livers. Cell Signal. 2025 Aug; 132:111799.. View in PubMed
• First-in-Human Phase I Study of a CD16A Bispecific Innate Cell Engager, AFM24, Targeting EGFR-Expressing Solid Tumors. Clin Cancer Res. 2025 Apr 01; 31(7):1257-1267.. View in PubMed
• TIMEPOINT, a phase 1 study combining MTL-CEBPA with pembrolizumab, supports the immunomodulatory effect of MTL-CEBPA in solid tumors. Cell Rep Med. 2025 Apr 15; 6(4):102041.. View in PubMed
• Proinflammatory macrophages release CXCL5 to regulate T cell function and limit effects of αPD-1 in steatosis-driven liver cancer. JHEP Rep. 2025 Jun; 7(6):101385.. View in PubMed
• EphB4-ephrin-B2 are targets in castration resistant prostate cancer. Br J Cancer. 2025 May; 132(8):679-689.. View in PubMed
• Botensilimab (Fc-enhanced anti-cytotoxic lymphocyte-association protein-4 antibody) Plus Balstilimab (anti-PD-1 antibody) in Patients With Relapsed/Refractory Metastatic Sarcomas. J Clin Oncol. 2025 04 10; 43(11):1358-1368.. View in PubMed
• Transarterial chemoembolisation combined with lenvatinib plus pembrolizumab versus dual placebo for unresectable, non-metastatic hepatocellular carcinoma (LEAP-012): a multicentre, randomised, double-blind, phase 3 study. Lancet. 2025 01 18; 405(10474):203-215.. View in PubMed
• SWOG S1815: A Phase III Randomized Trial of Gemcitabine, Cisplatin, and Nab-Paclitaxel Versus Gemcitabine and Cisplatin in Newly Diagnosed, Advanced Biliary Tract Cancers. J Clin Oncol. 2025 02 10; 43(5):536-544.. View in PubMed
• A phase I dose-escalation study of LRP5/6 antagonist BI 905677 in patients with advanced solid tumors. ESMO Open. 2024 Nov; 9(11):103729.. View in PubMed
• Atezolizumab Plus Chemotherapy With or Without Bevacizumab in Advanced Biliary Tract Cancer: Clinical and Biomarker Data From the Randomized Phase II IMbrave151 Trial. J Clin Oncol. 2025 02 10; 43(5):545-557.. View in PubMed
• Reply to the Letter to the Editor “Reflections on the statistical methodology in nivolumab and ipilimumab therapy research” by S. Yang. Ann Oncol. 2024 Sep; 35(9):827-828.. View in PubMed
• Improved efficacy of pembrolizumab combined with soluble EphB4-albumin in HPV-negative EphrinB2 positive head neck squamous cell carcinoma. Oncotarget. 2024 07 10; 15:444-458.. View in PubMed
• Botensilimab plus balstilimab in relapsed/refractory microsatellite stable metastatic colorectal cancer: a phase 1 trial. Nat Med. 2024 Sep; 30(9):2558-2567.. View in PubMed
• COMPANION-002 A clinical trial of investigational drug CTX-009 plus paclitaxel vs paclitaxel in second line advanced BTC. Future Oncol. 2024; 20(30):2241-2248.. View in PubMed
• Nivolumab plus ipilimumab combination therapy in patients with advanced hepatocellular carcinoma previously treated with sorafenib: 5-year results from CheckMate 040. Ann Oncol. 2024 06; 35(6):537-548.. View in PubMed
• CALGB 80802 (Alliance): Impact of Sorafenib with and without Doxorubicin on Hepatitis C Infection in Patients with Advanced Hepatocellular Carcinoma. Cancer Res Commun. 2024 03 07; 4(3):682-690.. View in PubMed
• Nivolumab in sorafenib-naive and sorafenib-experienced patients with advanced hepatocellular carcinoma: 5-year follow-up from CheckMate 040. Ann Oncol. 2024 04; 35(4):381-391.. View in PubMed
• Exposure-response analysis for nivolumab plus ipilimumab combination therapy in patients with advanced hepatocellular carcinoma (CheckMate 040). Clin Transl Sci. 2023 08; 16(8):1445-1457.. View in PubMed
• Phase 1 study of GSK3368715, a type I PRMT inhibitor, in patients with advanced solid tumors. Br J Cancer. 2023 08; 129(2):309-317.. View in PubMed
• Profiling of Circulating Tumor Cells for Screening of Selective Inhibitors of Tumor-Initiating Stem-Like Cells. Adv Sci (Weinh). 2023 05; 10(14):e2206812.. View in PubMed
• Adjuvant Chemoradiation in Patients with Lymph Node-Positive Biliary Tract Cancers: Secondary Analysis of a Single-Arm Clinical Trial (SWOG 0809). Ann Surg Oncol. 2023 Mar; 30(3):1354-1363.. View in PubMed
• Association of Body Mass Index With the Safety Profile of Nivolumab With or Without Ipilimumab. JAMA Oncol. 2023 01 01; 9(1):102-111.. View in PubMed
• Nivolumab Plus Cabozantinib With or Without Ipilimumab for Advanced Hepatocellular Carcinoma: Results From Cohort 6 of the CheckMate 040 Trial. J Clin Oncol. 2023 03 20; 41(9):1747-1757.. View in PubMed
• Zanidatamab, a novel bispecific antibody, for the treatment of locally advanced or metastatic HER2-expressing or HER2-amplified cancers: a phase 1, dose-escalation and expansion study. Lancet Oncol. 2022 12; 23(12):1558-1570.. View in PubMed
• First-in-human study of an OX40 (ivuxolimab) and 4-1BB (utomilumab) agonistic antibody combination in patients with advanced solid tumors. J Immunother Cancer. 2022 10; 10(10).. View in PubMed
• DNMT and EZH2 inhibitors synergize to activate therapeutic targets in hepatocellular carcinoma. Cancer Lett. 2022 11 01; 548:215899.. View in PubMed
• Molecular pathogenesis and systemic therapies for hepatocellular carcinoma. Nat Cancer. 2022 04; 3(4):386-401.. View in PubMed
• Quality of life assessment of cabozantinib in patients with advanced hepatocellular carcinoma in the CELESTIAL trial. Eur J Cancer. 2022 06; 168:91-98.. View in PubMed
• Safety and efficacy of cabozantinib for patients with advanced hepatocellular carcinoma who advanced to Child-Pugh B liver function at study week 8: a retrospective analysis of the CELESTIAL randomised controlled trial. BMC Cancer. 2022 Apr 09; 22(1):377.. View in PubMed
• Nivolumab versus sorafenib in advanced hepatocellular carcinoma (CheckMate 459): a randomised, multicentre, open-label, phase 3 trial. Lancet Oncol. 2022 01; 23(1):77-90.. View in PubMed
• Outcomes Based on Plasma Biomarkers for the Phase 3 CELESTIAL Trial of Cabozantinib versus Placebo in Advanced Hepatocellular Carcinoma. Liver Cancer. 2022 Jan; 11(1):38-47.. View in PubMed
• Final Overall Survival Efficacy Results of Ivosidenib for Patients With Advanced Cholangiocarcinoma With IDH1 Mutation: The Phase 3 Randomized Clinical ClarIDHy Trial. JAMA Oncol. 2021 Nov 01; 7(11):1669-1677.. View in PubMed
• Efficacy and safety of cabozantinib for patients with advanced hepatocellular carcinoma based on albumin-bilirubin grade. Br J Cancer. 2022 03; 126(4):569-575.. View in PubMed
• A Phase I, Open-Label, Dose-Escalation Study of the OX40 Agonist Ivuxolimab in Patients with Locally Advanced or Metastatic Cancers. Clin Cancer Res. 2022 01 01; 28(1):71-83.. View in PubMed
• Society for Immunotherapy of Cancer (SITC) clinical practice guideline on immunotherapy for the treatment of hepatocellular carcinoma. J Immunother Cancer. 2021 09; 9(9).. View in PubMed
• Infigratinib (BGJ398) in previously treated patients with advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements: mature results from a multicentre, open-label, single-arm, phase 2 study. Lancet Gastroenterol Hepatol. 2021 10; 6(10):803-815.. View in PubMed
• IMbrave 151: a randomized phase II trial of atezolizumab combined with bevacizumab and chemotherapy in patients with advanced biliary tract cancer. Ther Adv Med Oncol. 2021; 13:17588359211036544.. View in PubMed
• Pharmacokinetics and safety of niraparib in patients with moderate hepatic impairment. Cancer Chemother Pharmacol. 2021 11; 88(5):825-836.. View in PubMed
• Etiology and Outcomes of Hepatocellular Carcinoma in an Ethnically Diverse Population: The Multiethnic Cohort. Cancers (Basel). 2021 Jul 12; 13(14).. View in PubMed
• Phase I, First-in-Human Study of the Probody Therapeutic CX-2029 in Adults with Advanced Solid Tumor Malignancies. Clin Cancer Res. 2021 08 15; 27(16):4521-4530.. View in PubMed
• CheckMate 040 cohort 5: A phase I/II study of nivolumab in patients with advanced hepatocellular carcinoma and Child-Pugh B cirrhosis. J Hepatol. 2021 09; 75(3):600-609.. View in PubMed
• Cabozantinib: An evolving therapy for hepatocellular carcinoma. Cancer Treat Rev. 2021 Jul; 98:102221.. View in PubMed
• Comparative Efficacy of Atezolizumab plus Bevacizumab and Other Treatment Options for Patients with Unresectable Hepatocellular Carcinoma: A Network Meta-Analysis. Liver Cancer. 2021 Jun; 10(3):240-248.. View in PubMed
• A phase I study of intravenous fenretinide (4-HPR) for patients with malignant solid tumors. Cancer Chemother Pharmacol. 2021 04; 87(4):525-532.. View in PubMed
• Liver Cancer Immunity. Hepatology. 2021 01; 73.. View in PubMed
• Presentation, Management, and Outcomes Across the Rural-Urban Continuum for Hepatocellular Carcinoma. JNCI Cancer Spectr. 2021 02; 5(1).. View in PubMed
• Efficacy and Safety of Nivolumab Plus Ipilimumab in Patients With Advanced Hepatocellular Carcinoma Previously Treated With Sorafenib: The CheckMate 040 Randomized Clinical Trial. JAMA Oncol. 2020 Nov 01; 6(11):e204564.. View in PubMed
• Phase I and Biomarker Study of the Wnt Pathway Modulator DKN-01 in Combination with Gemcitabine/Cisplatin in Advanced Biliary Tract Cancer. Clin Cancer Res. 2020 12 01; 26(23):6158-6167.. View in PubMed
• Phase II Trial of Neoadjuvant Bevacizumab with Modified FOLFOX7 in Patients with Stage II and III Rectal Cancer. Oncologist. 2020 12; 25(12):e1879-e1885.. View in PubMed
• Second-line cabozantinib after sorafenib treatment for advanced hepatocellular carcinoma: a subgroup analysis of the phase 3 CELESTIAL trial. ESMO Open. 2020 08; 5(4).. View in PubMed
• Association of inflammatory biomarkers with clinical outcomes in nivolumab-treated patients with advanced hepatocellular carcinoma. J Hepatol. 2020 12; 73(6):1460-1469.. View in PubMed
• Serum Alpha-fetoprotein Levels and Clinical Outcomes in the Phase III CELESTIAL Study of Cabozantinib versus Placebo in Patients with Advanced Hepatocellular Carcinoma. Clin Cancer Res. 2020 09 15; 26(18):4795-4804.. View in PubMed
• Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study. Lancet Oncol. 2020 06; 21(6):796-807.. View in PubMed
• Randomised phase II trial (SWOG S1310) of single agent MEK inhibitor trametinib Versus 5-fluorouracil or capecitabine in refractory advanced biliary cancer. Eur J Cancer. 2020 05; 130:219-227.. View in PubMed
• Atezolizumab and Bevacizumab Combination Therapy for Hepatocellular Carcinoma. Gastroenterol Hepatol (N Y). 2020 Mar; 16(3):145-148.. View in PubMed
• Diagnosis and management of toxicities of immune checkpoint inhibitors in hepatocellular carcinoma. J Hepatol. 2020 02; 72(2):320-341.. View in PubMed
• Assessment of Treatment With Sorafenib Plus Doxorubicin vs Sorafenib Alone in Patients With Advanced Hepatocellular Carcinoma: Phase 3 CALGB 80802 Randomized Clinical Trial. JAMA Oncol. 2019 Nov 01; 5(11):1582-1588.. View in PubMed
• Mandatory Research Biopsy Requirements Delay Initiation of Clinical Trials. Front Oncol. 2019; 9:968.. View in PubMed
• Adjuvant Therapy for Resected Biliary Tract Cancer: ASCO Clinical Practice Guideline. J Clin Oncol. 2019 04 20; 37(12):1015-1027.. View in PubMed
• Integrative Epigenetic Analysis Reveals Therapeutic Targets to the DNA Methyltransferase Inhibitor Guadecitabine (SGI-110) in Hepatocellular Carcinoma. Hepatology. 2018 10; 68(4):1412-1428.. View in PubMed
• Monitoring Twitter Conversations for Targeted Recruitment in Cancer Trials in Los Angeles County: Protocol for a Mixed-Methods Pilot Study. JMIR Res Protoc. 2018 Sep 25; 7(9):e177.. View in PubMed
• Cabozantinib in Patients with Advanced and Progressing Hepatocellular Carcinoma. N Engl J Med. 2018 Jul 05; 379(1):54-63.. View in PubMed
• A Randomized Phase II Open-Label Multi-Institution Study of the Combination of Bevacizumab and Erlotinib Compared to Sorafenib in the First-Line Treatment of Patients with Advanced Hepatocellular Carcinoma. Oncology. 2018; 94(6):329-339.. View in PubMed
• A multi-arm phase I dose escalating study of an oral NOTCH inhibitor BMS-986115 in patients with advanced solid tumours. Invest New Drugs. 2018 12; 36(6):1026-1036.. View in PubMed
• A phase I trial of escalating doses of cixutumumab (IMC-A12) and sorafenib in the treatment of advanced hepatocellular carcinoma. Cancer Chemother Pharmacol. 2018 05; 81(5):957-963.. View in PubMed
• Evaluation of hepatic impairment on pharmacokinetics and safety of crizotinib in patients with advanced cancer. Cancer Chemother Pharmacol. 2018 04; 81(4):659-670.. View in PubMed
• A phase 1, dose-escalation study of PF-06664178, an anti-Trop-2/Aur0101 antibody-drug conjugate in patients with advanced or metastatic solid tumors. Invest New Drugs. 2018 10; 36(5):836-847.. View in PubMed
• Phase II Study of BGJ398 in Patients With FGFR-Altered Advanced Cholangiocarcinoma. J Clin Oncol. 2018 01 20; 36(3):276-282.. View in PubMed
• Effect of First-Line Chemotherapy Combined With Cetuximab or Bevacizumab on Overall Survival in Patients With KRAS Wild-Type Advanced or Metastatic Colorectal Cancer: A Randomized Clinical Trial. JAMA. 2017 06 20; 317(23):2392-2401.. View in PubMed
• Combination epigenetic therapy in metastatic colorectal cancer (mCRC) with subcutaneous 5-azacitidine and entinostat: a phase 2 consortium/stand up 2 cancer study. Oncotarget. 2017 May 23; 8(21):35326-35338.. View in PubMed
• AKT Inhibition in Solid Tumors With AKT1 Mutations. J Clin Oncol. 2017 Jul 10; 35(20):2251-2259.. View in PubMed
• Nivolumab in patients with advanced hepatocellular carcinoma (CheckMate 040): an open-label, non-comparative, phase 1/2 dose escalation and expansion trial. Lancet. 2017 Jun 24; 389(10088):2492-2502.. View in PubMed
• The Promise of Immunotherapy in the Treatment of Hepatocellular Carcinoma. Am Soc Clin Oncol Educ Book. 2017; 37:311-317.. View in PubMed
• Clinical relevance of EMT and stem-like gene expression in circulating tumor cells of metastatic colorectal cancer patients. Pharmacogenomics J. 2018 01; 18(1):29-34.. View in PubMed
• A Phase I Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Combination Therapy with Refametinib plus Sorafenib in Patients with Advanced Cancer. Clin Cancer Res. 2016 05 15; 22(10):2368-76.. View in PubMed
• Guadecitabine (SGI-110) priming sensitizes hepatocellular carcinoma cells to oxaliplatin. Mol Oncol. 2015 Nov; 9(9):1799-814.. View in PubMed
• SWOG S0809: A Phase II Intergroup Trial of Adjuvant Capecitabine and Gemcitabine Followed by Radiotherapy and Concurrent Capecitabine in Extrahepatic Cholangiocarcinoma and Gallbladder Carcinoma. J Clin Oncol. 2015 Aug 20; 33(24):2617-22.. View in PubMed
• Phase I trial of sorafenib following liver transplantation in patients with high-risk hepatocellular carcinoma. Liver Cancer. 2015 Mar; 4(2):115-25.. View in PubMed
• A phase I, pharmacokinetic, and pharmacodynamic evaluation of the DNA methyltransferase inhibitor 5-fluoro-2′-deoxycytidine, administered with tetrahydrouridine. Cancer Chemother Pharmacol. 2015 Mar; 75(3):537-46.. View in PubMed
• Integrin genetic variants and stage-specific tumor recurrence in patients with stage II and III colon cancer. Pharmacogenomics J. 2015 Jun; 15(3):226-34.. View in PubMed
• A phase I study of PF-04449913, an oral hedgehog inhibitor, in patients with advanced solid tumors. Clin Cancer Res. 2015 03 01; 21(5):1044-51.. View in PubMed
• Phase I clinical trial of temsirolimus and vinorelbine in advanced solid tumors. Cancer Chemother Pharmacol. 2014 Dec; 74(6):1227-34.. View in PubMed
• Phase I study of the halichondrin B analogue eribulin mesylate in combination with cisplatin in advanced solid tumors. Br J Cancer. 2014 Dec 09; 111(12):2268-74.. View in PubMed
• A phase II trial of bevacizumab plus temsirolimus in patients with advanced hepatocellular carcinoma. Invest New Drugs. 2015 Feb; 33(1):241-6.. View in PubMed
• Impact of sex on the survival of patients with hepatocellular carcinoma: a Surveillance, Epidemiology, and End Results analysis. Cancer. 2014 Dec 01; 120(23):3707-16.. View in PubMed
• S0941: a phase 2 SWOG study of sorafenib and erlotinib in patients with advanced gallbladder carcinoma or cholangiocarcinoma. Br J Cancer. 2014 Feb 18; 110(4):882-7.. View in PubMed
• The effects of liver impairment on the pharmacokinetics of brivanib, a dual inhibitor of fibroblast growth factor receptor and vascular endothelial growth factor receptor tyrosine kinases. Cancer Chemother Pharmacol. 2013 Jul; 72(1):53-64.. View in PubMed

• Lenz, Heinz-Josef
• Terrault, Norah
• Quinn, David
• Matsuo, Koji
• Agulnik, Mark
• Zhang, Lin