Pilot study of albendazole in patients with advanced malignancy - Effect on serum tumor markers/high incidence of neutropenia

Our preclinical studies have shown that the widely used antiparasitic drug albendazole has potent antiproliferative activity against colorectal cancer (CRC) and hepatocellular carcinoma (HCC). This trial was designed to evalu ate albendazole in a small number of patients (n = 7) with either HCC or CR C and hepatic metastases refractory to other forms of therapy. Albendazole was given at 10 mg/kg/day orally in two divided doses for a period of 28 da ys. To follow the effect of treatment, tumor markers, carcinoembryonic anti gen (CEA) or alpha -feto protein (AFP), were measured routinely in these pa tients. A range of hematological and biochemical indices were also serially measured to monitor bone marrow, kidney or liver toxicity. Albendazole the rapy resulted in a decrease in CEA in 2 patients. In the remaining 5 with m easurable tumor markers, serum CEA or AFP was stabilized in 3 patients, whi le in the other 2, after an initial stabilization (5-10 days), the markers began to increase. In the 7 patients completing the trial, albendazole was well tolerated and there were no significant changes in any hematological, kidney or liver function tests, but 3 patients were withdrawn for severe ne utropenia which was probably contributory to the death of 1 patient. These data support our previous experimental results demonstrating that albendazo le has antitumor effects. Copyright (C) 2001 S. Karger AG, Basel.