To date, no phase III randomized, controlled trials of antineoplastons as a treatment for cancer have been conducted. Publications have taken the form of case reports, phase I clinical trials, toxicity studies, and phase II clinical trials. Phase I toxicity studies are the first group discussed below. The studies are categorized by the antineoplaston investigated. The second group of studies involves patients with various malignancies. Table 1 is a summary of dose regimens for all human studies. Table 2 summarizes the following clinical trials and appears at the end of this section.
Phase I Toxicity Studies for Specific Antineoplastons
The studies discussed below are phase I toxicity studies in patients with various types of malignancies, including bladder cancer, breast cancer, and leukemias. The studies are listed by the antineoplastons administered. The effect of a specific antineoplaston under investigation is difficult to ascertain because of the confounding effect of previous therapies. Unless specifically noted, all studies were conducted by the developer and his associates at his research institute.
A 1977 article reported on 21 patients with advanced cancer or leukemia who were treated with antineoplaston A and followed for up to 9 months. Patients ranged in age from 14 to 75 years and had cancers of various types. Eight patients received no previous therapies, and 13 patients had been previously treated with chemotherapy and radiation therapy. Antineoplaston A was administered intravenously (IV), intramuscularly (IM), rectally, by bladder instillation, intrapleurally, and by application to the skin. Tolerance to antineoplaston A depended on the method of administration and the type of neoplasm.
Fever and chills, the main side effects, occurred only after IV or IM administration at the beginning of treatment. Fever lasted for a few hours, followed by subnormal temperatures and lowered blood pressure. Premedication with salicylates, adrenocorticotrophic hormone, or corticosteroids were used to treat the fever or suppress it. Only patients with chronic lymphocytic leukemia, transitional cell carcinoma of the bladder, metastatic adenocarcinoma of the rectum, squamous cell carcinoma of the cervix, and synovial sarcoma reacted with fever to low doses of antineoplaston A. No severe adverse reactions were reported, even when patients were treated with very high doses of the formulation (refer to Table 1). No toxicities were reported in any patient. Platelet and white blood cell counts were elevated after a month of treatment but gradually returned to normal.
Four patients obtained complete tumor response (two cases of bladder cancer, one case of breast cancer, and one case of acute lymphocytic leukemia); four patients obtained partial tumor response (two cases of chronic lymphocytic leukemia, one case of rectosigmoid adenocarcinoma, and one case of synovial sarcoma); six patients had stable disease; and two patients discontinued treatment. There were five deaths during the study that were not attributed to antineoplaston A toxicity.