Unusually Long Survival (More Than Five Years to More Than 31 Years) in Twelve Patients with Relapsed Medulloblastoma Treated with Antineoplastons in Phase II Studies

Background: Medulloblastoma (MB), a grade 4 embryonal neoplasm, is the most common malignant brain tumor in children and adolescents. Relapsed MB (RMB) carries a poor prognosis with less than 10% surviving more than 5 years post-relapse. Objective: Twelve patients with RMB were treated with Antineoplastons A-10 and AS-2-1 (ANP) according to phase II clinical studies. They are presented here, emphasizing unusually long survival and a lack of long-term negative sequelae. Methods: For clinical studies, the eligibility criteria had to include a Karnofsky/Lansky (KPS/LPS) score of at least sixty and at least a 2- month life expectancy. IV ANP was given to all patients, while some also received maintenance treatment with oral ANP. IV ANP infusions utilized a subclavian catheter and an automated pump. The maximum tolerated doses of A10 and AS2-1 were achieved. Outcome criteria were objective response, survival, and toxicity after ANP therapy. Results: The Kaplan-Meier Survival analysis for the 46 RMB patients gave a median survival of 2.42 years. Twelve of 46 RMB patients survived from more than 5 years to more than 31 years (26.1%). Five of these patients survived more than 12 years, a definition of cure. The patients were between the ages of 1.5 to 29.7 years old with a 9.2-year median age. LPS/KPS ranged from 50 to 100 with a median score of 60. One patient experienced a serious adverse event (somnolence), possibly attributed to ANP, from which he quickly recovered fully. Conclusions: ANP therapy shows promise for the long-term survival of RMB patients with no long-term toxicity.