General Session 6: Advancements in Treatment of Cholangiocarcinoma and Hepatocellular Carcinoma

Josep Llovet, MD, PhD—Chair, Icahn School of Medicine at Mount Sinai

Jinsil Seong, MD, PhD—Chair, Yonsei University College of Medicine

Rachna T. Shroff, MD
The University of Arizona Cancer Center
Novel Therapies for Cholangiocarcinoma: One Size Fits All?

Key points:

  • IDH1 mutations: Common alteration, awaiting results of ClarlDHy trial this year, other agents in early phase studies
  • FGFR2 fusions: Multiple agents in trials for 1st line and refractory populations, promising ORR, resistance mechanisms being understood
  • BRAF/HER2/DNA repair: lower frequency alterations, but still targetable, BATCH trial coming as well as others, combination strategies
  • lmmunotherapy: single agent pembro disappointing, but a role for 10 combinations? awaiting results


  • Tissue vs. cfDNA ➔availability of tissue
  • ABC-06 results due ➔new 2nd line SOC with FOLFOX?
  • Single-arm studies ➔truly need randomized studies
  • Are all CCA created equal? ➔how to stratify
  • Basket trials ➔allows for nimble evaluation of rare subsets with shared controls
  • Role of resistance ➔repeat biopsy vs. cfDNA

Conclusion - One size does NOT fit all!

  • it is a new era in the treatment of CCA!
  • Sequencing can and should be done on ALL patients
  • Actionable targets exist

➔  go after them with smart clinical trials

Melinda Bachini
Cholangiocarcinoma Foundation
The Patient Perspective on Cancer Treatment


• Patients' decision making is somewhat influenced by their social support system, but ultimately patients are making the  decision
• Patients are willing to take significant risks to receive surgical and non-surgical treatments
• Patient hesitancy to receive a placebo should inform and guide clinical trials design, but patients are willing to enroll

Bruno Sangro, MD, PhD
Clinica Universidad de Navarra and CIBEREHD
State of the Art Management of Advanced Hepatocellular Carcinoma

Take-Away Points

  • Sorafenib and Lenvatinib are the standard of care for first­line therapy patients with advanced HCC.
  • The choice of a second-line agent cannot be based on etiology, tumor burden or available biomarkers.
  • Combination therapies should prove increased efficacy before they are recommended

Open and Urgent Questions

  • Combinations should be based an a better understanding of the mechanisms of resistance

-           Tumor bio sies should be mandato in all trials

  • Sequencing should be explored as much as combinations

-           Risk models and scores

  • Early transition to systemic chemotherapy (alone or in combination with locoregional therapy) should be investigated prospectively

Josep Llovet, MD, PhD
Icahn School of Medicine at Mount Sinai
Current and Upcoming Clinical Trials