Antineoplaston Therapy in Treating Patients With Brain Stem Glioma

OBJECTIVES:

- To determine the efficacy of Antineoplaston therapy in patients with a brain stem
glioma, 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 patients with a brain
stem glioma.

OVERVIEW: This is a single arm, open-label study in which patients 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 in measured utilizing MRI scans, which are
performed every 8 weeks for the first 2 years, every 3 months for the 3rd and 4th years,
every 6 months for the 5th and 6th years, and annually thereafter.

PROJECTED ACCRUAL: A total of 20-40 patients will be accrued for this study.

DISEASE CHARACTERISTICS:

- Histologically confirmed (except if medically contraindicated) brain stem glioma that
is unlikely to respond to existing therapy and for which no curative therapy exists.

- Tumor must be at least 5 mm in maximum diameter

PATIENT CHARACTERISTICS:

Age:

- 6 months or greater

Performance status:

- Karnofsky 60-100%

Life expectancy:

- At least 2 months

Hematopoietic:

- WBC at least 2,000/mm^3

- Platelet count greater than 50,000/mm^3

Hepatic:

- Bilirubin no greater than 2.5 mg/dL

- SGOT/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 severe heart disease

- No uncontrolled hypertension

- No history of congestive heart failure

- No 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

- No concurrent immunomodulating agents

Chemotherapy:

- At least 4 weeks since prior chemotherapy (6 weeks for nitrosoureas)

- No concurrent antineoplastic agents

Endocrine therapy:

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

Radiotherapy:

- At least 8 weeks since prior radiotherapy

Surgery:

- Not specified

Other:

- No prior Antineoplaston therapy