RATIONALE: Antineoplastons are naturally-occurring substances that may also be made in the
laboratory. Antineoplastons may inhibit the growth of cancer cells.
PURPOSE: This phase II trial is studying how well antineoplaston therapy works in treating
children with primitive neuroectodermal tumors that has not responded to standard therapy.
- Demonstrate the antitumor activity of antineoplastons A10 and AS2-1 in children with
primitive neuroectodermal tumors by determining the proportion of patients who
experience an objective tumor response.
- Evaluate the adverse effects of and tolerance to this regimen in these patients.
OUTLINE: This is an open-label study.
Patients receive gradually escalating doses of intravenous antineoplaston A10 and
antineoplaston AS2-1 6 times a day until the maximum tolerated dose is reached. Treatment
continues for at least 2 months in the absence of disease progression or unacceptable
toxicity. After 2 months, patients with responding or stable disease may continue treatment.
Tumors are measured every 8 weeks for 2 years, every 3 months for the third and fourth
years, every 6 months for the fifth and sixth years, and then annually thereafter.
PROJECTED ACCRUAL: A total of 20-40 patients will be accrued for this study.
- Histologically confirmed (except if medically contraindicated) incurable primitive
- Evidence of progressive or recurrent tumor by MRI or CT scan performed within 2
weeks prior to study entry
- Must have received and failed prior standard therapy
- Tumor must be at least 5 mm
- 6 months to 17 years
- Karnofsky 60-100%
- At least 2 months
- WBC at least 2000/mm^3
- Platelet count greater than 50,000/mm^3
- Bilirubin no greater than 2.5 mg/dL
- SGOT and SGPT no greater than 5 times upper limit of normal
- No hepatic insufficiency
- Creatinine no greater than 2.5 mg/dL
- No history of renal conditions that contraindicate high dosages of sodium
- No uncontrolled hypertension
- No severe heart disease
- No history of congestive heart failure
- No other cardiovascular conditions that contraindicate high dosages of sodium
- No severe lung disease
- Not pregnant or nursing
- Fertile patients must use effective contraception during and for 4 weeks after study
- No serious active infections or fever
- No other serious concomitant disease
PRIOR CONCURRENT THERAPY:
- At least 4 weeks since prior immunotherapy and recovered
- No concurrent immunomodulating agents
- At least 4 weeks since prior chemotherapy (6 weeks for nitrosoureas) and recovered
- No concurrent antineoplastic agents
- Concurrent corticosteroids for cerebral edema allowed (must be on a stable dose for
at least 1 week prior to study entry)
- At least 8 weeks since prior radiotherapy and recovered
- Not specified
- No prior antineoplaston treatment