Open Label Trial of Therapeutic Vaccine in Patients With Cholangiocarcinoma

Cholangiocarcinoma (CCA) is a malignant neoplasm originating from the epithelial cells lining the intra- or extrahepatic biliary ducts. It is the second-most common liver cancer, after hepatocellular carcinoma (HCC). About 6,000 people in the United States develop bile duct cancer each year. One-year survival is less than 25% and no effective and safe systemic treatments are currently available. Last year the completion of open-label phase 2 trial (NCT02256514) of hepcortespenlisimut-L (V5) has been reported, which has shown that two-third of Mongolian patients with advanced HCC had a favorable clinical response, including complete remissions and with overall survival over 90% after 1 year. So far a few patients with CCA were treated with V5, but it appeared that their response rate was somewhat inferior to patients with HCC since two (both with hemochromatosis) out six patients died within 6 months. In one patient who had improved clinically, the improvement was correlated with decrease in CA19-9 tumor marker, but no marker profile information is available in regard to other CCA patients. As V5 tablets are made from pooled blood of patients with HCC, in theory, they will be not very useful to patients with CCA. The goal of this project is to manufacture an immunotherapeutic formulation made from pooled heat- and chemically-inactivated blood from donors with CCA and initiate pilot open-label trial in 20 cholangiocarcinoma patients. This clinical trial will be conducted in collaboration with the National Cancer Center.