Precision Medicine and Immunotherapy Have Arrived for Cholangiocarcinoma: An Overview of Recent Approvals and Ongoing Clinical Trials

Article Tools REVIEW ARTICLES Precision Medicine Article Tools OPTIONS & TOOLS Export Citation Track Citation Add To Favorites Rights & Permissions COMPANION ARTICLES No companion articles ARTICLE CITATION DOI: 10.1200/PO.22.00573 JCO Precision Oncology no. 7 (2023) e2200573. Published online April 13, 2023. PMID: 37053534 Precision Medicine and Immunotherapy Have Arrived for Cholangiocarcinoma: An Overview of Recent Approvals and Ongoing Clinical Trials Thomas B. Karasic , MD1xThomas B. KarasicSearch for articles by this author; Jennifer R. Eads , MD1xJennifer R. EadsSearch for articles by this author; and Lipika Goyal , MD, MPhil2xLipika GoyalSearch for articles by this author Show More 1Division of Hematology/Oncology, University of Pennsylvania, Philadelphia, PA2Department of Medicine, Division of Hematology and Oncology, Stanford Cancer Center, Palo Alto, CA https://doi.org/10.1200/PO.22.00573 First Page Full Text PDF Figures and Tables © 2023 by American Society of Clinical OncologyCONTEXTKey ObjectiveTo summarize recent advances in precision medicine and immunotherapy for patients with cholangiocarcinoma.Knowledge GeneratedTargetable alterations are found in up to 40%-50% of patients with intrahepatic cholangiocarcinoma and up to 15%-20% of patients with extrahepatic cholangiocarcinoma. Effective therapies exist for FGFR2 fusions, IDH1 mutations, BRAF V600E mutations, NTRK and RET fusions, HER2 amplification/overexpression, MSI-high tumors, and TMB-high tumors, and data continue to emerge for other potential targets such as KRAS G12C mutations, MDM2 amplifications, and DNA repair deficiencies.RelevanceThe emerging therapies outlined in this review are likely to reshape the treatment landscape for cholangiocarcinoma in the coming years.SUPPORTL.G. receives funding from the American Cancer Society Clinical Scientist Development Grant 134,013‐CSDG‐19‐163‐01‐T.B.G., the NIH/NCI Gastrointestinal Cancer SPORE P50 CA127003, V Foundation for Cancer Research Translational Grant, and the Cholangiocarcinoma Foundation Andrea Marie Fuquay Research Fellowship.AUTHOR CONTRIBUTIONSConception and design: All authorsCollection and assembly of data: Thomas B. Karasic, Lipika GoyalData analysis and interpretation: All authorsManuscript writing: All authorsFinal approval of manuscript: All authorsAccountable for all aspects of the work: All authorsAUTHORS' DISCLOSURES OF POTENTIAL CONFLICTS OF INTERESTThe following represents disclosure information provided by authors of this manuscript. All relationships are considered compensated unless otherwise noted. Relationships are self-held unless noted. I = Immediate Family Member, Inst = My Institution. Relationships may not relate to the subject matter of this manuscript. For more information about ASCO's conflict of interest policy, please refer to www.asco.org/rwc or ascopubs.org/po/author-center.Open Payments is a public database containing information reported by companies about payments made to US-licensed physicians (Open Payments).Thomas B. KarasicHonoraria: Incyte, Pfizer, Ipsen, AstraZeneca/MedImmune, Taiho OncologyConsulting or Advisory Role: Nucorion, IncyteResearch Funding: Taiho Pharmaceutical (Inst), H3 Biomedicine (Inst), Bristol Myers Squibb (Inst), Lilly (Inst), Tempest Therapeutics (Inst), Xencor (Inst), Genentech (Inst)Jennifer R. EadsEmployment: Bristol Myers Squibb, Janssen OncologyStock and Other Ownership Interests: Bristol Myers Squibb/CelgeneConsulting or Advisory Role: Advanced Accelerator ApplicationsResearch Funding: Xencor (Inst), Tarveda Therapeutics (Inst), MedImmune (Inst), Incyte, Oncolys BioPharma (Inst), Seattle Genetics (Inst), Genentech (Inst), Hutchison MediPharma (Inst), AstraZeneca/MedImmune (Inst)Travel, Accommodations, Expenses: Bristol Myers Squibb, Janssen OncologyLipika GoyalConsulting or Advisory Role: Alentis Therapeutics, QED Therapeutics, AstraZeneca, Taiho Pharmaceutical, Incyte, Sirtex Medical, Genentech, Exelixis, TransThera Biosciences, Merck, Black Diamond Therapeutics, Synthekine, Eisai/H3 Biomedicine, Tyra Biosciences, Kinnate Biopharma, Compass Therapeutics, Blueprint Medicines, SERVIERUncompensated Relationships: Boehringer IngelheimNo other potential conflicts of interest were reported.