Abstract
Protocol BT-23, a Phase II study, was performed to determine the efficacy and safety of combination Antineoplaston A10 and Antineoplaston AS2-1 (ANP) in children with an optic pathway glioma (OPG). Sixteen patients (12 males, 4 females) ages 1 to 16 years (median age 4.5 years) were treated. The patients received ANP, by intravenous infusion, six times daily (i.e., every four hours). The length of treatment extended from a minimum of 4.1 weeks to a maximum of 210.0 weeks. The median dosage of A10 was 8.07 g/kg/d while for AS2-1 itwas 0.39 g/kg/d.Patients’ best response to ANP was determined: 12.5% of this patient population achieved a complete response, 18.8% achieved a partial response, and 31.3% maintained stable disease. The median overall survival was 78.5 months (95% CI: 37.6-92.3) while median progression-free survival was 45.4 months (95% CI: 7.1-71.4). ANPwas associated with a12% incidence of Grade 3 and a12% incidence of Grade 4 adverse drug experiences (ADEs). There were no Grade 5 ADEs. Grade 3 ADEs included somnolence and elevated SGOT; Grade 4 ADEsincluded hypernatremia and hypokalemia. Long-term quality of life was excellent with no chronic toxicity identified. Children with an OPG respond very well to ANP with good efficacy and toxicity profiles.
Highlights
Optic pathway gliomas (OPG) constitute approximately 6% of pediatric brain tumors (Jahraus & Tarbell, 2006; Thiagalingam, Flaherty, Billson & North, 2004)
Optic pathway gliomas are the most common type of brain tumor associated with neurofibromatosis 1 (NF-1) and growth of the glioma in some NF-1 patients is so slow that treatment is usually not recommended (Pollack & Mulvihill, 1996; Tow, Chandela, Miller & Avellino, 2003)
In a Phase II study of ANP in patients with an astrocytoma, which was opened for enrollment in 1988, a complete responses (CR) with more than 28 years of progression-free survival (PFS) was documented in a 7-year-old child diagnosed with a large, inoperable optic pathway glioma (OPG) (S Burzynski, Kubove & B Burzynski, 1992; Hawkins & Friedman, 1992)
Summary
Optic pathway gliomas (OPG) constitute approximately 6% of pediatric brain tumors (Jahraus & Tarbell, 2006; Thiagalingam, Flaherty, Billson & North, 2004). Cranial neuropathy, and endocrine dysfunction, including growth hormone deficiency, thyroid dysregulation, and gonadotropin deficiency, are delayed toxicities that can develop in children and adolescents following radiation therapy for OPGs (Burzynski, 2006). Such therapy predisposes patients to the development of a second primary cancer. In a Phase II study of ANP in patients with an astrocytoma, which was opened for enrollment in 1988, a CR with more than 28 years of progression-free survival (PFS) was documented in a 7-year-old child diagnosed with a large, inoperable OPG (S Burzynski, Kubove & B Burzynski, 1992; Hawkins & Friedman, 1992) This patient, who had a short life expectancy and was never treated with RT or chemotherapy, is tumor-free and continues to enjoy good health and a productive life.
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