Immunotherapy of liver cancer: open label phase II clinical study / Шинэ санаа Шинэ нээлт

ABSTRACT

Background: Hepatocellular carcinoma (HCC) is a global health problem and chronic hepatitis B and C infections account for majority of liver cancer cases. HCC is the most common malignancy in Mongolia with high fatality rate; highlighting the unmet need for better treatments. The increasing number of clinical studies is now devoted to development of various immunotherapies. Objective: To evaluate the clinical benefit of an oral immunomodulator derived from pooled blood of hepatitis B and C virus carriers - originally indicated for treatment of chronic hepatitis as a therapeutic vaccine. Patients Nine female and twelve male patients between ages 51-71 (median 59.2) presented with advanced, inoperable HCC characterized by elevated serum levels of alpha-fetoprotein (AFP) biomarker. As no other treatment options were available, patients consented to receive one daily tablet of vaccine. Results: After average 2.2 months of treatment 19 out of 21 (90.5%) patients experienced six-fold decrease of AFP from median 122.5 to 19.2 IU/ml (P<0.002 by Wilcoxon matched-pairs test); of these eight (42.1%) had exhibited levels (median 4.4 IU/ml) below upper limit 10 IU/ml of normal range. The decrease in AFP was correlated with tumor clearance or regression on CT scan. Two patients who did not experienced decline in AFP had died, the remaining are alive after median follow-up of 11 months (range 1-57), which is 3.7-times longer than 3 months survival in historical controls with advanced HCC (P<0.0001). None of patients experienced adverse effects. Conclusion: The results suggest that our immunotherapy holds promise as safe, effective and fast-acting intervention for HCC. Further studies are required to confirm this preliminary observation.