Antineoplaston Therapy in Treating Children With Rhabdoid Tumor of the Central Nervous System

Phase II Study of Antineoplastons A10 and AS2-1 in Children With Rhabdoid Tumor of the Central Nervous System

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

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (> 6 months of age) with Rhabdoid tumors.

OVERVIEW: This is a single arm, open-label study in which children with Rhabdoid 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.

OBJECTIVES:

• To determine the efficacy of Antineoplaston therapy in children with a Rhabdoid tumor, 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 Rhabdoid tumor.
• 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 Rhabdoid tumor of the central nervous system that is unlikely to respond to existing therapy and for which no curative therapy exists
• Measurable tumor by MRI scan performed within two weeks prior to study entry
• Tumor must be at least 5 mm

PATIENT CHARACTERISTICS:

Age:

• 6 months to 17 years

Performance status:

• Karnofsky 60-100%

Life expectancy:

• At least 2 months

Hematopoietic:

• WBC at least 2000/mm^3
• Platelet count greater than 50,000/mm^3

Hepatic:

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

Renal:

• Creatinine no greater than 2.5 mg/dL
• No history of renal conditions that contraindicate high dosages of sodium

Cardiovascular:

• No uncontrolled hypertension
• No severe heart disease
• No history of congestive heart failure
• No history of other cardiovascular conditions that contraindicate high dosages of sodium

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 concomitant 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:

• Concurrent corticosteroids for cerebral edema allowed (must be on a stable dose for at least 1 week before study entry)

Radiotherapy:

• At least 8 weeks since prior radiotherapy and recovered

Surgery:

• Not specified

Other:

• No prior antineoplaston treatment