March 22, 2017



Natural Standard Bottom Line Monograph, Copyright © 2013 (www.naturalstandard.com). Commercial distribution prohibited. This monograph is intended for informational purposes only, and should not be interpreted as specific medical advice. You should consult with a qualified healthcare provider before making decisions about therapies and/or health conditions.

While some complementary and alternative techniques have been studied scientifically, high-quality data regarding safety, effectiveness, and mechanism of action are limited or controversial for most therapies. Whenever possible, it is recommended that practitioners be licensed by a recognized professional organization that adheres to clearly published standards. In addition, before starting a new technique or engaging a practitioner, it is recommended that patients speak with their primary healthcare provider(s). Potential benefits, risks (including financial costs), and alternatives should be carefully considered. The below monograph is designed to provide historical background and an overview of clinically-oriented research, and neither advocates for or against the use of a particular therapy.

Related Terms

  • A1, A2, A3, A4, A5, A10, A10-1, AS2-1, AS2-5, AS5, antineoplaston A, antineoplaston Ch, antineoplaston F, antineoplaston H, antineoplaston K, antineoplaston L, antineoplaston O, 3-N-phenylacetylaminopiperidine-2,6 dione, phenylacetylglutamine (PAG), phenylacetylisoglutamine (PAIG), phenylacetic acid (PAA), 3-phenylacetylamino-2,6-piperidinedione, sodium phenylacetate.


  • Antineoplastons are a group of naturally occurring peptide fractions, which were observed by Stanislaw Burzynski, MD, PhD in the late 1970s to be absent in the urine of cancer patients. It was hypothesized that these substances may have anti-tumor properties. In the 1980s, Burzynski identified chemical structures for several of these antineoplastons, and developed a process to prepare them synthetically. Antineoplaston A10, identified as 3-phenylacetylamino-2,6-piperidinedione, was the first to be synthesized.

  • The use of antineoplastons in the treatment of various cancer types has been studied in the laboratory and in animals, and in limited preliminary human research. In 1991, the Cancer Therapy Evaluation Program of the National Cancer Institute (NCI) examined records of seven patients with brain tumors treated at the Burzynski Clinic in Texas. Based on their findings, the NCI sponsored a brain tumor clinical trial. However, due to difficulty recruiting patients, and a disagreement over study design, this research was canceled. The results in nine patients included prior to cancellation were reported, but were not conclusive. In 1997, Dr. Burzynski had legal troubles for permitting antineoplastons to be shipped out of Texas.

  • There is a lack of sufficient evidence from randomized, controlled trials in support of antineoplastons as a cancer treatment, and antineoplastons are not FDA approved therapies. Antineoplastons are not widely available in the United States, and safety and efficacy are not proven. Multiple studies of antineoplastons in various cancers have been sponsored by the Burzynski Research Institute. In recent years, antineoplastons have also been suggested as treatment for other conditions such as Parkinson's disease, sickle cell anemia, and thalassemia.

Scientific Evidence


These uses have been tested in humans or animals. Safety and effectiveness have not always been proven. Some of these conditions are potentially serious, and should be evaluated by a qualified healthcare provider.



There is inconclusive scientific evidence regarding the effectiveness of antineoplastons in the treatment of cancer. Several preliminary human studies (case series, phase I/II trials) have examined antineoplaston types A2, A5, A10, AS2-1, and AS2-5 for a variety of cancer types. It remains unclear if antineoplastons are effective, or what doses may be safe. Until better research is available, no clear conclusion can be drawn.



A small preliminary study published by Dr. Burzynski and colleagues in 1992 reported increased energy and weight in patients with HIV, as well as a decreased number of opportunistic infections, and increased CD4+ counts overall. These patients were treated with antineoplaston AS2-1. However, this evidence cannot be considered conclusive. Currently, there are drug therapy regimens available for HIV with clearly demonstrated effects ("HAART" or "highly active anti-retroviral therapy), and patients with HIV are recommended to consult with a physician about treatment options.


Sickle cell anemia/thalassemia

A small preliminary study reported positive findings, but there is currently insufficient evidence to make a clear recommendation in this area.


*Key to grades:A: Strong scientific evidence for this use; B: Good scientific evidence for this use; C: Unclear scientific evidence for this use; D: Fair scientific evidence against this use (it may not work); F: Strong scientific evidence against this use (it likely does not work).


The below uses are based on tradition or scientific theories. They often have not been thoroughly tested in humans, and safety and effectiveness have not always been proven. Some of these conditions are potentially serious and should be evaluated by a qualified health care professional.

  • Acute lymphocytic leukemia, adenocarcinoma, aging, astrocytoma, basal cell epithelioma, brain/central nervous system tumors, cholesterol/triglyceride abnormalities, chronic lymphocytic leukemia, encephalitis, glioblastoma, hepatocellular carcinoma, leukocytosis, malignant melanoma, medulloblastoma, metastatic synovial sarcoma, Parkinson's disease, promyelocytic leukemia, recurrent glioma, thrombocytosis.


The below doses are based on scientific research, publications, traditional use, or expert opinion. Many herbs and supplements have not been thoroughly tested, and safety and effectiveness may not be proven. Brands may be made differently, with variable ingredients, even within the same brand. The below doses may not apply to all products. You should read product labels, and discuss doses with a qualified healthcare provider before starting therapy.

Adults (18 years and older)

  • Various doses of antineoplastons have been used in preliminary studies. Safety and efficacy are not proven for any specific dose or use. Doses of antineoplaston A10 used by mouth in studies range from 10 to 40 grams daily or 100 to 288 milligrams per kilogram of body weight per day. Duration of use has varied. Antineoplaston AS2-1 has been studied at doses from 12 to 30 grams daily or 97 to 130 milligrams per kilogram of body weight per day. Antineoplastons have also been studied when applied to the skin, injected through the veins (intravenous) and injected into muscles (intramuscular).

Children (younger than 18 years)

  • There is insufficient available data to safely recommend the use of antineoplastons in children.


The U.S. Food and Drug Administration does not strictly regulate herbs and supplements. There is no guarantee of strength, purity or safety of products, and effects may vary. You should always read product labels. If you have a medical condition, or are taking other drugs, herbs, or supplements, you should speak with a qualified healthcare provider before starting a new therapy. Consult a healthcare provider immediately if you experience side effects.


  • Allergic skin rash has been reported after injection of antineoplaston AS2-1. Individuals who have reacted to antineoplastons in the past should avoid this therapy.

Side Effects and Warnings

  • Adverse effects are reported in several preliminary studies. It is not clear how common these reactions are, or if they occur more frequently than with placebo. Since many patients taking antineoplastons have been diagnosed with serious illnesses such as advanced cancers, it is not clear if these effects may be from the illnesses themselves, or caused by antineoplastons.

  • Antineoplaston therapy has been associated with drowsiness, headache, fatigue, mild dizziness/vertigo, and confusion. Antineoplaston A10 is retained in the brain tissue of animals, although the importance of this in humans is not known. Weakness, nausea, vomiting, upset stomach, abdominal pain, and increased flatulence (gas) have been reported.

  • Various types of antineoplastons administered from weeks to years have been associated with sore throat, fever, chills, reduced blood albumin levels, liver function test abnormalities, low blood sugar levels (hypoglycemia), low potassium, and a strong body odor similar to urine.

  • Palpitations, high blood pressure (hypertension), and mild peripheral edema (water retention) have been noted. Chest pressure and irregular or fast heartbeat have also been observed. Joint swelling, muscle/joint pain, muscle contractions in the throat, weakness, and finger rigidity have been reported in clinical trials.

  • Decreases in blood platelets, red blood cells, and white blood cells have been observed. Other serious reported effects include slow or abnormal breathing, metabolic/electrolyte abnormalities, cerebral edema (brain swelling), dangerously low blood pressure (hypotension), and death.

Pregnancy and Breastfeeding

  • The safety of antineoplastons during pregnancy or breastfeeding is not known, and therefore cannot be recommended.


Most herbs and supplements have not been thoroughly tested for interactions with other herbs, supplements, drugs, or foods. The interactions listed below are based on reports in scientific publications, laboratory experiments, or traditional use. You should always read product labels. If you have a medical condition, or are taking other drugs, herbs, or supplements, you should speak with a qualified healthcare provider before starting a new therapy.

Interactions with Drugs

  • Limited information is available about interactions with antineoplastons. Agents with adverse effects similar to antineoplastons may have additive effects, such as lowering blood potassium or glucose levels, or causing liver abnormalities. It is not known if antineoplastons add to the effects of chemotherapeutic drugs.

Interactions with Herbs and Supplements

  • Limited information is available about interactions with antineoplastons. Agents with adverse effects similar to antineoplastons may have additive effects, such as lowering blood potassium or glucose levels, or causing liver abnormalities.

Author Information

  • This information is based on a systematic review of scientific literature edited and peer-reviewed by contributors to the Natural Standard Research Collaboration (www.naturalstandard.com).


Natural Standard developed the above evidence-based information based on a thorough systematic review of the available scientific articles. For comprehensive information about alternative and complementary therapies on the professional level, go to www.naturalstandard.com. Selected references are listed below.

  1. Badria F, Mabed M, Khafagy W, et al. Potential utility of antineoplaston A-10 levels in breast cancer. Cancer Letters 2000;157(1):67-70. View Abstract

  2. Badria F, Mabed M, El Awadi M, et al. Immune modulatory potentials of antineoplaston A-10 in breast cancer patients. Cancer Lett. 8-31-2000;157(1):57-63. View Abstract

  3. 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. View Abstract

  4. Burzynski SR, Kubove E. Initial clinical study with antineoplaston A2 injections in cancer patients with five years' follow-up. Drugs Exp Clin Res 1987;13 Suppl 1:1-11. View Abstract

  5. Burzynski SR, Kubove E, Burzynski B. Treatment of hormonally refractory cancer of the prostate with antineoplaston AS2-1. Drugs Exp Clin Res 1990;16(7):361-369. View Abstract

  6. Burzynski SR. Potential of antineoplastons in diseases of old age. Drugs Aging 1995;7(3):157-167. View Abstract

  7. Green S. 'Antineoplastons'. An unproved cancer therapy. JAMA 6-3-1992;267(21):2924-2928. View Abstract

  8. Juszkiewicz M, Chodkowska A, Burzynski SR, et al. The influence of antineoplaston A5 on particular subtypes of central dopaminergic receptors. Drugs Exp Clin Res 1995;21(4):153-156. View Abstract

  9. Kumabe T, Tsuda H, Uchida M, Ogoh Y, et al. Antineoplaston treatment for advanced hepatocellular carcinoma. Oncol Rep 1998;5(6):1363-1367. View Abstract

  10. Liau MC, Szopa M, Burzynski B, et al. Quantitative assay of plasma and urinary peptides as an aid for the evaluation of cancer patients undergoing antineoplaston therapy. Drugs Exp Clin Res 1987;13 Suppl 1:61-70. View Abstract

  11. Soltysiak-Pawluczuk D, Burzynski SR. Cellular accumulation of antineoplaston AS21 in human hepatoma cells. Cancer Lett. 1-6-1995;88(1):107-112. View Abstract

  12. Sugita Y, Tsuda H, Maruiwa H, et al. The effect of antineoplaston, a new antitumor agent on malignant brain tumors. Kurume Med J 1995;42(3):133-140. View Abstract

  13. Tsuda H, Sata M, Saitsu H, et al. Antineoplaston AS2-1 for maintenance therapy in liver cancer. Oncology Reports 1997;4:1213-1216.

  14. Tsuda H, Iemura A, Sata M, et al. Inhibitory effect of antineoplaston A10 and AS2-1 on human hepatocellular carcinoma. Kurume Med J 1996;43(2):137-147. View Abstract

  15. Tsuda H, Sata M, Kumabe T, et al. Quick response of advanced cancer to chemoradiation therapy with antineoplastons. Oncol Rep. 1998;5(3):597-600. View Abstract

Copyright © 2013 Natural Standard (www.naturalstandard.com)

The information in this monograph is intended for informational purposes only, and is meant to help users better understand health concerns. Information is based on review of scientific research data, historical practice patterns, and clinical experience. This information should not be interpreted as specific medical advice. Users should consult with a qualified healthcare provider for specific questions regarding therapies, diagnosis and/or health conditions, prior to making therapeutic decisions.


March 22, 2017