Antineoplaston Therapy in Treating Children With Primary Malignant Brain Tumors

Phase II Study of Antineoplastons A10 and AS2-1 in Children With Primary Malignant Brain Tumors

RATIONALE: Current therapies for children with primary malignant brain tumors provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with primary malignant brain tumors.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children with primary malignant brain tumors.

OBJECTIVES:

• To determine the efficacy of Antineoplaston therapy in children with primary malignant brain tumors 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 primary malignant brain tumors.

OVERVIEW: This is a single arm, open-label study in which children with primary malignant brain tumors 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

DISEASE CHARACTERISTICS:

• Histologically confirmed incurable primary malignant brain tumor that has progressed, recurred, or persisted after initial therapy (must have failed standard therapy).
• Evidence of residual tumor by MRI performed within two weeks prior to study entry

PATIENT CHARACTERISTICS:

Age:

• 6 months to 17 years

Performance status:

• Karnofsky 60-100%

Life expectancy:

• At least 2 months

Hematopoietic:

• WBC at least 1,500/mm^3
• Platelet count greater than 50,000/mm^3

Hepatic:

• No hepatic failure
• Bilirubin no greater than 2.5 mg/dL
• SGOT/SGPT no greater than 5 times upper limit of normal

Renal:

• Creatinine no greater than 2.5 mg/dL

Cardiovascular:

• No severe heart disease
• No uncontrolled hypertension

Pulmonary:

• No severe lung disease

Other:

• Not pregnant or nursing
• Fertile patients must use effective contraception during and for 4 weeks after study participation
• No serious active infections or fever
• No other serious concurrent disease

PRIOR CONCURRENT THERAPY:

Biologic therapy:

• At least 4 weeks since prior immunotherapy and recovered
• No concurrent immunomodulating agents

Chemotherapy:

• At least 4 weeks since prior chemotherapy (6 weeks for nitrosoureas) and recovered
• No concurrent antineoplastic agents

Endocrine therapy:

• Recovered from prior endocrine therapy
• Concurrent corticosteroids for cerebral edema allowed

Radiotherapy:

• At least 8 weeks since prior radiotherapy (unless clear radiological evidence of progression) and recovered

Surgery:

• At least 4 weeks since prior surgery (unless clear radiological evidence of progression) and recovered

Other:

• No prior antineoplaston therapy