RATIONALE: Current therapies for children with primitive neuroectodermal tumors that have not responded to standard therapy provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with primitive neuroectodermal tumors that have not responded to standard therapy. PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (\> 6 months of age) with primitive neuroectodermal tumors that has not responded to standard therapy.
OBJECTIVES: * To determine the efficacy of Antineoplaston therapy in children with primitive neuroectodermal tumors that has not responded to standard therapy, as measured by an objective response to therapy (complete response, partial response or stable disease). * To determine the safety and tolerance of Antineoplaston therapy in children with a brain tumor. OVERVIEW: This is a single arm, open-label study in which children with primitive neuroectodermal tumors that have not responded to standard therapy receive gradually escalating doses of intravenous Antineoplaston therapy (Atengenal + Astugenal) until the maximum tolerated dose is reached. Treatment continues for at least 12 months in the absence of disease progression or unacceptable toxicity. After 12 months, patients with a complete or partial response or with stable disease may continue treatment. To determine objective response, tumor size is measured utilizing MRI scans, which are performed every 8 weeks for the first two years, every 3 months for the third and fourth years, every 6 months for the 5th and sixth years, and annually thereafter. PROJECTED ACCRUAL: A total of 20-40 patients will be accrued to this study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
13
Children with a primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
Burzynski Clinic
Houston, Texas, United States
Number of Participants With Objective Response
Objective response rate per Response Assessment in Neuro-Oncology (RANO) for target lesions and assessed by MRI: Complete Response (CR), disappearance of all disease sustained for at least four weeks; Partial Response (PR), \>=50% decrease in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least four weeks.
Time frame: 12 months
Percentage of Participants Who Survived
6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival
Time frame: 6 months, 12 months, 24 months, 36 months, 48 months, 60 months
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