Antineoplastons (generic name)

treats Sickle cell anemia/thalassemia, HIV, and Cancer
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Phenylacetate, Sodium phenylacetate


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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.


DISCLAIMER: 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.

Cancer: 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.
Grade: C

HIV: 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.
Grade: C

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.
Grade: C


WARNING: DISCLAIMER: The below uses are based on tradition, scientific theories, or limited research. 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 healthcare provider. There may be other proposed uses that are not listed below.
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.


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.


DISCLAIMER: Many complementary techniques are practiced by healthcare professionals with formal training, in accordance with the standards of national organizations. However, this is not universally the case, and adverse effects are possible. Due to limited research, in some cases only limited safety information is available.


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.

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