A Phase II study of antineoplastons A10 and AS2-1 in adult patients with recurrent glioblastoma multiforme based on Protocol BT-21

Gregory Burzynski 1 ,2, Stanislaw Burzynski1 ,2, Tomek Janicki1 ,2, Ania Marszalek1
1Burzynski Clinic, Houston, TX, USA, 2Burzynski Research Institute, Houston, TX, USA

Treatment of recurrent glioblastoma multiforme (GBM) creates one of the most difficult challanges in neuro-oncology. The aim of this presentation is to evaluate the responses and survival of 24 recurrent GBM patients and toxicity in all 33 eligible patients. The study accrued patients who developed disease progression during standard treatment within eight weeks from completion of radiation therapy (RT) and six weeks from chemotherapy. Forty candidates were registered but only 33 patients were eligible. The seven noneligible patients received less than 28 days of treatment with Antineoplastons A10 and AS2-1. Among the eligible patients there were 24 cases of recurrent GBM that progressed during prior treatment, four patients with anaplastic astrocytoma and five persistent GBM. Previous treatment included surgery in all patients (18 had tumor resection, and 6 underwent biopsy only), chemotherapy in 75% of patients and radiation therapy in 88% of patients. Antineoplastons were administered intravenously every four hours (median dose of A10 10.7 g/kg/d and AS2-1 0.43 g/kg/d) until objective response was documented or until progression. The median duration of ANP treatment was 13.2 weeks ranging (4.6 - 80.3). Responses were assessed by MRI repeated every eight weeks, and or PET scan. Objective responses were determined in 16.7% of cases (complete response, and partial response in 8.3% each). Progression-free survival at six months was 25%. Overall survival is 39.3% at one year, 4.4% at two years, five and ten years. The treatment was well tolerated with reversible grades 3 and 4 toxicity including four cases of hypernatremia, two of fatigue, two of hypokalemia, and a single case of somnolence.  There were no chronic toxicities. In conclusion, ANP is well tolerated and compares favorably to the current treatment for recurrent GBM.