Antineoplastons

Health Care Professional Information

Common Name

Antineoplaston treatment

Clinical Summary

Antineoplastons refer to mixtures of peptides, amino acids, and other organic substances that were first isolated from human urine and blood by Stanislaw Burzynski. He claims to have used antineoplastons to treat a variety of cancers based on the belief that they promote the body's natural defenses against cancer. In 1993, the National Cancer Institute sponsored clinical trials to investigate the antitumor potential of antineoplastons in patients with brain tumors (4). The trials were closed two years later as poor patient accrual precluded conclusions about the efficacy of the treatment. A Mayo clinic study found no benefit (1).
Antineoplastons have also been tested in patients with glioma (6).
Status on clinical trials using antineoplastons as an investigational drug for various cancers remain unknown (5).
Adverse reactions observed include confusion, sleepiness, and exacerbation of underlying seizures, headache, vomiting, and fatigue.

Purported Uses
Constituents
  • A10 capsules: 50mg of 3-phenylacetylamino 2,6-piperidinedione
  • A10 injection: Mixture of sodium salts of phenylacetylglutamine and phenylacetylisoglutamine in 4:1 ratio
  • AS2-1 capsules: 500mg of a 4:1 phenylacetate and phenylacetylglutamine
  • AS2-1 injection: Mixture of phenylacetate and phenylacetylglutamine in a 4:1 ratio
    (2)
Mechanism of Action

The proposed mechanisms for the anticancer activity of antineoplastons include activation of the tumor suppressor gene p53 by phenylacetate (PN) and AS2-1, metabolites of A10 (3-phenyl-acetylamino-2, 6-peperidinedione) (2). Phenylacetylglutamine (PG), the main component of A10-I is thought to inhibit uptake of amino acids that are essential for cancer cell growth (2). A10 was shown in a study to inhibit neutrophil apoptosis in breast cancer cells. Since depletion of neutrophils is associated with development of cancer, researchers suggest a role for A10 as an adjuvant therapy for breast cancer (3).

Warnings

There is no conclusive evidence to support the antineoplaston theory.

Adverse Reactions
  • Seizures
  • Sleepiness
    (1)
  • Anemia
  • Hypernatremia
  • Fever
  • Slurred speech
  • Skin rash
  • Diuresis
    (2)
References
  1. Buckner JC, Malkin MG, Reed E et al. Phase II study of antineoplastons A10 (NSC 648539) and AS2-1 (NSC 620261) in patients with recurrent glioma. Mayo Clin Proc 1999; 74(2):137-145.
  2. Burzynski SR. The present state of antineoplaston research (1). Integr Cancer Ther 2004; 3(1):47-58.
  3. Badria F, Mabed M, El Awadi M, Abou-Zeid L, Al Nashar E, Hawas S. Immune modulatory potentials of antineoplaston A-10 in breast cancer patients. Cancer Lett 2000; 157(1):57-63.
  4. The National Cancer Institute web site. Antineoplastons.
    http://www.cancer.gov/cancertopics/pdq/cam/antineoplastons/HealthProfessional/page2. Accessed March 21, 2012.
  5. Antineoplastons. http://www.clinicaltrials.gov/ct2/results?term=antineoplastons. Accessed March 21, 2012.
  6. Burzynski SR, Janicki TJ, Weaver RA, Burzynski B. Targeted therapy with antineoplastons A10 and AS2-1 of high-grade, recurrent, and progressive brainstem glioma.  Integr Cancer Ther. 2006 Mar;5(1):40-7.

Consumer Information

How It Works

Bottom Line: There is no clear evidence to support the anticancer effects of antineoplastons in humans.

Antineoplastons are compounds that were first isolated from human urine and blood by Stanislaw Burzynski. He claims that they promote the body's natural defenses against cancer. However, there is insufficient evidence to support this theory. Adverse reactions from antineoplaston treatment include confusion, sleepiness, and exacerbation of seizures, headache, vomiting, and fatigue.

Purported Uses

Cancer Treatment
Although a few studies were performed to test the efficacy of antineoplastons, definitive data are lacking.

Patient Warnings

There is no conclusive evidence to support the antineoplaston theory.

Side Effects
  • Seizures
  • Sleepiness
  • Anemia
  • Hypernatremia
  • Fever
  • Slurred speech
  • Skin rash
  • Diuresis
E-mail your questions and comments to aboutherbs@mskcc.org.