Antineoplaston Therapy in Treating Patients With Recurrent or Refractory Waldenstrom's Macroglobulinemia

Phase II Study of Antineoplastons A10 and AS2-1 in Patients With Macroglobulinemia of Waldenstrom

Current therapies for Recurrent or Refractory Waldenstrom's Macroglobulinemia provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of Recurrent or Refractory Waldenstrom's Macroglobulinemia.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on patients with Recurrent or Refractory Waldenstrom's Macroglobulinemia.

Recurrent or Refractory Waldenstrom's Macroglobulinemia patients receive gradually escalating doses of intravenous Antineoplaston therapy (Atengenal + Astugenal) until the maximum tolerated dose is reached. Treatment continues up to 12 months in the absence of disease progression or unacceptable toxicity.

OBJECTIVES:

• To determine the efficacy of Antineoplaston therapy in patients with Recurrent or Refractory Waldenstrom's Macroglobulinemia, 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 Recurrent or Refractory Waldenstrom's Macroglobulinemia.
• To determine objective response, tumor size is measured utilizing physical examination, radiologic studies, and bone marrow biopsies as necessary, 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.

DISEASE CHARACTERISTICS:

• Histologically confirmed recurrent or progressive Waldenstrom's macroglobulinemia that is unlikely to respond to existing therapy, including surgery, radiotherapy, and chemotherapy

  • Evidence of tumor by MRI, CT scan, chest x-ray, or radionuclide scan
  • Must have received and failed at least one standard first-line therapy (e.g., corticosteroid/alkylator combination)

• Biochemical evidence of Waldenstrom's macroglobulinemia

  • Abnormal proteins in serum and urine

PATIENT CHARACTERISTICS:

Age:

• 18 and over

Performance status:

• Karnofsky 60-100%

Life expectancy:

• At least 2 months

Hematopoietic:

• WBC at least 2000/mm3
• Platelet count at least 50,000/mm3

Hepatic:

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

Renal:

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

Cardiovascular:

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

Pulmonary:

• No serious lung disease, such as chronic obstructive pulmonary disease

Other:

• Not pregnant or nursing
• Fertile patients must use effective contraception during and for 4 weeks after study
• No active infection
• No non-malignant systemic disease
• Not a high medical or psychiatric risk

PRIOR CONCURRENT THERAPY:

Biologic therapy:

• At least 4 weeks since prior immunotherapy
• No concurrent immunomodulating agent

Chemotherapy:

• See Disease Characteristics
• At least 4 weeks (6 weeks since nitrosoureas) since prior chemotherapy
• No concurrent antineoplastic agents

Endocrine therapy:

• See Disease Characteristics
• Concurrent corticosteroids allowed

Radiotherapy:

• See Disease Characteristics
• At least 8 weeks since prior radiotherapy

Surgery:

• See Disease Characteristics
• Recovered from prior surgery

Other:

• No prior antineoplastons
• Prior cytodifferentiating agents allowed