Abstract

The paper reports the evaluation of efficacy and safety of Antineoplastons A10 and AS2-1 (ANP) in a phase II study of newly diagnosed patients with anaplastic astrocytoma (AA). The study was designed as a single-arm, two-stage, phase II trial of ANP as monotherapy. The primary endpoint, was to determine the overall response rate (confirmed complete response (CR) or partial response (PR)) to ANP, based on disappearance or more than 50% reduction of tumor size by contrast-enhanced magnetic resonance imaging (MRI). The goal of the study was a response rate to ANP of not less than 10% or 4 patients. 19 patients (12 men, 7 women) ages 22-60 years (median age, 44) were treated. The patients received ANP daily every four hours (median dose of A10 5.3 g/kg/d and AS2-1 0.3 g/kg/d). The duration of ANP ranged from 3 to 175 weeks. A complete response was documented in 21%, and stable disease in 26% of patients. The study was closed for enrollment after 19 of its subjects had been admitted, because the goal of trial had been accomplished. Progression-free survival and overall survival at 2 years was 16% and 37% and at 10 years was 5% and 14% respectively. Only 11% of patients reported grade 3 toxicities consisting of hypokalemia and fatigue, and 11% reported grade 4 toxicities consisting of hypernatremia and hypokalemia. There was no chronic toxicity and there was a high quality of life. ANP shows efficacy with very good toxicity profile in patients with newly diagnosed AA.