Common Names

  • Antineoplaston treatment

For Patients & Caregivers

How It Works

There is a lack of 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, worsening 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
  • Dehydration
  • Fever
  • Slurred speech
  • Skin rash
  • Increased urination
  • Stomach fullness
  • Vein inflammation
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For Healthcare Professionals

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 2 years later as poor patient accrual precluded conclusions about the efficacy of the treatment. In addition, a Mayo clinic study found no benefit (1). Antineoplastons have also been tested in patients with gliomas (6) (7) (8) (10), but lack of a rigid scientific approach in these studies has been questioned (9) (11). A preliminary study of antineoplastons adjunctive to hepatic arterial infusion post-hepatectomy did not improve overall survival, although cancer-specific survival was higher (12). The status of other clinical trials using antineoplastons as investigational drugs for various cancers remains unknown (5).

Adverse reactions observed include confusion, sleepiness, and exacerbation of underlying seizures, headache, vomiting, fatigue, stomach fullness, and phlebitis.

Purported Uses
  • Cancer treatment
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).


There is no conclusive evidence to support the antineoplaston theory.

Adverse Reactions

Seizures, sleepiness, anemia, hypernatremia, fever, slurred speech, skin rash, diuresis, stomach fullness, and phlebitis (1) (2) (12).

  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. Accessed March 10, 2015.

  5. Antineoplastons. Accessed March 10, 2015.

  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.

  7. Burzynski SR, Janicki TJ, Burzynski GS, Marszalek A. Long-term survival (>13 years) in a child with recurrent diffuse pontine gliosarcoma: a case report. J Pediatr Hematol Oncol. 2014 Oct;36(7):e433-9.

  8. Burzynski SR, Burzynski GS, Janicki TJ, et al. Complete response and long-term survival (>20 years) of a child with tectal glioma: a case report. Pediatr Neurosurg. 2015;50(2):99-103.

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