Verteporfin showed antitumor effects in intrahepatic and extrahepatic cholangiocarcinoma cell lines, a new study published in Hepatobiliary & Pancreatic Diseases International found. Moreover, when combined with anti-PD-1, it inhibited tumor growth.

Blocking immune checkpoints with immunotherapy has shown little benefit in cholangiocarcinoma. However, research has shown that inhibiting YAP1 signaling using verteporfin could inhibit cell proliferation and induce apoptosis.

Here, a team of researchers led by Changqing Xie, MD, PhD, from the National Cancer Institute, National Institutes of Health, in Bethesda, Maryland evaluated the potential benefit of verteporfin plus anti-PD-1 in a mouse model of cholangiocarcinoma.

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The team first evaluated the cytotoxicity of verteporfin in human intrahepatic and extrahepatic cholangiocarcinoma cell lines. They then analyzed the effect of verteporfin on cell proliferation, apoptosis, and stemness. Finally, they assessed the efficacy of verteporfin and anti-PD-1 on their own and in combination in the mouse model.

The results showed that verteporfin reduced tumor cell growth and enhanced apoptosis in human cholangiocarcinoma tumor cells in a dose-dependent fashion and impaired stemness. Moreover, the combination of the 2 agents reduced tumor burden in the mouse model compared to verteporfin and anti-PD-1 alone.

The combination of the 2 agents could be a promising therapeutic strategy for cholangiocarcinoma, a disease with no effective immunotherapy, the authors said. They said that future clinical studies are necessary to determine whether the combination treatment could also be effective in delaying or halting the progression of the disease.

Cholangiocarcinoma is a group of rare malignant tumors originating from the cells of the biliary tree and one of the primary hepatobiliary malignant neoplasms. The prognosis of the disease is poor with the 5-year survival rate being only 10%.


Fu J. McGrath NA, Lee J, Wang X, Brar G, Xie G. Verteporfin synergizes the efficacy of anti-PD-1 in cholangiocarcinoma. Hepatobiliary Pancreat Dis Int. Published online March 10, 2022. doi:10.1016/j.hbpd.2022.03.006