42.02 Nafamostat mesilate enhances effect of gemcitabine + nab-paclitaxel therapy for pancreatic cancer.

T. Horiuchi1,2, H. Shiba1, N. Saito1,2, Y. Shirai1,2, R. Iwase1, K. Haruki1, Y. Fujiwara1, K. Furukawa1, T. Uwagawa1, T. Ohashi2, K. Yanaga1 1The Jikei University School Of Medicine,Department Of Surgery,,Tokyo, , Japan 2The Jikei University School Of Medicine,Department Of Gene Therapy, Research Center For Medical Science,Tokyo, , Japan

Introduction: Pancreatic cancer has a poor prognosis among visceral cancers, with an overall 5-year survival rate of around 5%. Recently, gemcitabine plus nab-paclitaxel therapy was reported to be superior to gemcitabine alone in patients with advanced pancreatic cancer. However, the tumor resistance to chemotherapy is common and activation of nuclear factor-kappa B (NF-κB) plays a key role in attenuation of anti-tumor effect of chemotherapy in pancreatic cancer. We previously reported that nafamostat mesilate, a synthetic serine protease inhibitor, inhibited NF-κB activation and induced antitumor effects for pancreatic cancer. We hypothesized that nafamostat mesilate downregulates the activity of NF-κB and improves therapeutic outcome of pancreatic cancer.

Methods: In vitro, we assessed NF-κB activity, cell viability, induction of caspase cascade, and quantification of apoptosis of human pancreatic cancer cell lines (PANC-1, MIA PaCa-2, ASPC-1) in the following five groups: 1) gemcitabine and nab-paclitaxel, 2) nafamostat mesilate alone, 3) triple combination (gemcitabine, nab-paclitaxel and nafamostat mesilate), or 4) vehicle as control. In vivo, we established orthotopic pancreatic cancer model in mice by injection of PANC-1 cells to the pancreas. At six weeks after injection, the animals were treated with nafamostat mesilate 3 times a week (Nafamostat mesilate group), with gemcitabine plus nab-paclitaxel once a week (Chemotherapy group), or with combination of nafamostat mesilate 3 times a week and gemcitabine plus nab-paclitaxel once a week (Triple combination group). In Control group, only vehicle of gemcitabine and nafamostat mesilate were injected at the same time course.

Results:In vitro, NF-κB activity in Chemotherapy group was higher than that in Control group, and NF-kB activity was significantly surpressed in Nafamostat mesilate group and Triple combination group as compared with Control group and Chemotherapy group, respectively (PANC-1: p<0.001, MIAPaCa-2: p<0.001, ASPC-1: p<0.001). Cell viability in Triple combination group was lower than that in Chemotherapy group. Cleaved caspase-3 and 8 level in Triple combination group was the greatest in the 4 groups. Apoptosis in Triple combination group was significantly more frequent than that in Chemotherapy group. In vivo, tumor size in Tripe combination group was smaller than those in other groups. NF-κB activation of the excised tumor in Triple combination group was significantly inhibited in comparison with that in Chemotherapy group (p<0.01). Cleaved caspase-3 level and the number of apoptotic cells in the Triple combination group were the greatest in the 4 groups.

Conclusion:Combination chemotherapy of gemcitabine plus nab-paclitaxel with nafamostat mesilate exerts enhanced anti-tumor effect against orthotopic pancreatic cancer model in mice.