Outcomes in Four Children with Persistent, Recurrent, and Progressive Gangliogliomas Treated in Phase II Studies with Antineoplastons A10 and AS2-1

Rationale: Gangliogliomas are generally benign tumors and are classified by the World Health Organization (WHO) as grade I or II tumors. However, in 1-5% of cases, gangliogliomas can behave more aggressively (WHO grade III) and have a worse prognosis. Four children with a ganglioglioma are presented to detail and discuss the efficacy of Antineoplastons A10 (Atengenal) and AS2-1 (Astugenal) in the treatment of gangliogliomas. Objectives: The children were treated with Antineoplastons A10 and AS2-1 (ANP therapy) at the Burzynski Clinic (BC) according to the phase II Protocols, BT-10 and BT-11. ANP therapy was delivered via subclavian catheter and infusion pump. During ANP therapy, tumor response was determined by comparison of sequential magnetic resonance imaging (MRI) of the brain with a baseline brain MRI. Findings: Of the four children treated for gangliogliomas, all had prior surgery but none had radiation therapy (RT) or chemotherapy. Two had recurrent, and progressive tumors with possible high-grade transformation (thalamo-mesencephalic region; temporal lobe with leptomeningeal spread) while two had tumors of the brain stem (persistent multicentric ganglioglioma; persistent and progressive ganglioglioma of the inferior medulla and superior cervical spinal cord), which are more difficult to treat and have a worse prognosis. Physical findings corresponded with the location of each child’s tumor. IV ANP therapy continued for 6.4 to 24.8 months. The two children with possible high-grade transformation achieved a partial response (PR) while the two children with brain stem tumors maintained stable disease (SD). Overall survival for these four children ranged from 10.3 to 22.4 years. Conclusions: The utilization of ANP therapy in children with gangliogliomas is presented. We conclude that ANP therapy is an attractive therapeutic option for children with gangliogliomas who are ineligible for or refuse surgical resection and/or demonstrate persistent, recurrent, or progressive disease with or without high-grade transformation following surgical resection..