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Author Spotlight: Investigating Liver Cancer Pathogenesis Using Patient-Derived Organoids
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Author Spotlight: Investigating Liver Cancer Pathogenesis Using Patient-Derived Organoids

Published on: August 18, 2023

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Novel targeted therapy strategies for biliary tract cancers and hepatocellular carcinoma

Thomas T DeLeon1, Daniel H Ahn1, James M Bogenberger1

  • 1Division of Hematology Oncology, Department of Medicine, Mayo Clinic, Scottsdale, AZ 85259, USA.

Future Oncology (London, England)
|February 21, 2018
PubMed

Insights

Hepatobiliary cancers cause many deaths globally, with limited treatments. Emerging biomarkers and therapies, like nivolumab for hepatocellular carcinoma and FGFR2/IDH1 targets for cholangiocarcinoma, offer new hope.

Area of Science:

  • Oncology
  • Gastroenterology
  • Cancer Research

Background:

  • Hepatobiliary cancers represent a significant global health burden, ranking as the second leading cause of cancer-related mortality worldwide.
  • There is a critical unmet need for effective systemic therapies in advanced and metastatic stages of these malignancies.
  • Current treatment options for hepatobiliary cancers are limited, highlighting the necessity for novel therapeutic strategies.

Purpose of the Study:

  • To review emerging therapeutic biomarkers and innovative treatment concepts for hepatobiliary cancers.
  • To highlight promising advancements in hepatocellular carcinoma and intrahepatic cholangiocarcinoma.
  • To identify additional biomarkers requiring further investigation in the broader category of hepatobiliary cancers.

Main Methods:

  • Literature review of recent advancements in systemic therapy for hepatobiliary cancers.
  • Analysis of approved and investigational therapies targeting specific molecular pathways and immune checkpoints.
  • Identification and categorization of potential biomarkers for therapeutic targeting.

Main Results:

  • Nivolumab has shown promise and received US FDA approval for hepatocellular carcinoma.
  • Intrahepatic cholangiocarcinoma is seeing advancements with therapies targeting FGFR2, IDH1, and immune checkpoint inhibitors.
  • Several biomarkers including FGF19, ERRFI1, TERT, BAP1, BRAF, CDKN2A, tumor mutational burden, and ERBB2 (HER2/neu) warrant further exploration.

Conclusions:

  • Emerging biomarkers and novel therapeutic strategies hold significant promise for improving outcomes in hepatobiliary cancers.
  • Targeted therapies and immunotherapies represent key areas of development for these challenging diseases.
  • Continued research into novel agents and biomarkers is crucial to address the unmet needs in advanced hepatobiliary cancers.
Keywords:
biomarkerscholangiocarcinomagallbladder cancerhepatocellular cancerimmunotherapytargeted therapy

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