Antineoplastons (PDQ®): Complementary and alternative medicine - Patient Information [NCI]

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Antineoplastons (PDQ®): Complementary and alternative medicine - Patient Information [NCI]

This information is produced and provided by the National Cancer Institute (NCI). The information in this topic may have changed since it was written. For the most current information, contact the National Cancer Institute via the Internet web site at or call 1-800-4-CANCER.



  • Antineoplastons are chemical compounds that are found normally in urine and blood. For use in medical research, antineoplastons can be made from chemicals in a laboratory. (See Question 1.)
  • Antineoplaston therapy was developed by Dr. S. R. Burzynski, who proposed the use of antineoplastons as a possible cancer treatment in 1976. (See Question 2.)
  • No randomized, controlled trials showing the effectiveness of antineoplastons have been published in peer-reviewed scientific journals. (See Question 6.)
  • Nonrandomized clinical trials are ongoing at Dr. Burzynski's clinic to study the effect of antineoplastons on cancer. (See Question 6.)
  • Antineoplastons have caused mild side effects and some serious nervous system problems. (See Question 7.)
  • Antineoplastons are not approved by the U. S. Food and Drug Administration for the prevention or treatment of any disease. (See Question 8.)

Questions and Answers About Antineoplastons

What are antineoplastons?

Antineoplastons are a group of chemical compounds that are found normally in urine and blood. They are made up mostly of amino acids (the building blocks of protein) and peptides (compounds made of two or more amino acids). For use in medical research, antineoplastons were originally taken from human urine, but they are now made from chemicals in the laboratory.

What is the history of the discovery and use of antineoplastons as a complementary and alternative treatment for cancer?

Antineoplaston therapy was developed by Dr. S. R. Burzynski. He proposed that there must be a process in the body that controls how a cell develops, and that this process fails when a cell divides endlessly and develops into a tumor. He suggested that certain natural substances, which he named "antineoplastons," switch an abnormal cell back onto the path of normal development. Since peptides are considered to be the carriers of instructions in the body, he began looking for peptides that may be present in the blood of cancer patients. After comparing the blood of healthy people to the blood of people with cancer, Dr. Burzynski found that people with cancer have lower amounts of a certain group of chemicals. He found these same chemicals in urine and suggested that some of these chemicals can be used to stop certain cancer cells from dividing.

Dr. Burzynski separated and removed several different types of antineoplastons from the urine of healthy people. He tested these antineoplastons on normal and abnormal cells to see their effect and found that some types of antineoplastons were more effective on more types of abnormal cells than others. He called this type antineoplaston A. He later developed and tested antineoplastons A1, A2, A3, A4, and A5. He found that A2 had the most effect on tumor cells and named the active ingredient in it A10. Other antineoplastons followed. In 1976, Dr. Burzynski proposed the use of antineoplastons as a possible cancer treatment and began treating patients in clinical trials at his own clinic. (See Question 6.) Since 1980, Dr. Burzynski has made the antineoplastons from chemicals in his laboratory, instead of taking them from urine or blood.

What is the theory behind the claim that antineoplastons are useful in treating cancer?

According to Dr. Burzynski, when the body does not have enough antineoplastons, cells that begin to develop abnormally are not corrected, and tumors form and grow. He suggests that antineoplaston therapy supplies the body with the substances needed to correct the abnormal development of the cell and allow it to develop normally or to die a natural cell death, while healthy cells are not affected.

How are antineoplastons administered?

Antineoplastons have been given in different ways. Today, most antineoplastons are given by mouth or by injection (shot).

Have any preclinical (laboratory or animal) studies been conducted using antineoplastons?

Research in a laboratory or using animals is done to find out if a drug, procedure, or treatment is likely to be useful in humans. These preclinical studies are done before testing in humans is begun.

Dr. Burzynski did laboratory studies to see how antineoplastons affect human cancer cells. He reported that antineoplaston A killed human cancer cells but had no effect on animal tumor cells. Other types of antineoplastons have not been tested in animals.

Japanese scientists tested some types of antineoplastons on human liver cancer cells. High doses were needed to slow the growth of the cells or cause them to die.

Several laboratory-made antineoplastons have been tested on various types of cells and were reported to be more effective than the natural form taken from urine.

(See the PDQ health professional summary on Antineoplastons for more information on preclinical study results.)

Have any clinical trials (research studies with people) of antineoplastons been conducted?

To date, no phase IIIrandomized, controlled trials of antineoplastons as a treatment for cancer have been conducted.

Many cancer patients have been treated with antineoplastons at Dr. Burzynski's clinic and studied there. A few trials and case studies have been done outside of the clinic. Some of the cancers studied include breast, bladder, cervical, prostate, liver, and lung cancers, leukemia, lymphoma, and brain tumors.

Published information includes results from phase I clinical trials, phase II clinical trials, and case reports. The following antineoplastons were studied in clinical trials:

  • Antineoplaston A
  • Antineoplaston A10
  • Antineoplaston AS2-1
  • Antineoplaston AS2-5
  • Antineoplaston A2
  • Antineoplaston A3
  • Antineoplaston A5

Safety of Antineoplastons

Phase I trials are the first step in testing a new treatment in people. In these studies, researchers test to see what dose is safe, how the treatment should be given (such as by mouth or by injection), and how often it should be given.

In the phase I trials of antineoplastons, side effects were usually mild and did not last long.

The most severe harmful side effects occurred in a phase II trial. Phase II cancer trials study how a treatment works against certain types of cancer and how it affects the body. A phase II trial of antineoplastons A10 and AS2-1 in brain tumor patients reported severe nervous system side effects including sleepiness, confusion, seizures, and swelling near the brain.

(See Question 7.)

Effect of Antineoplastons on Brain Tumors, Prostate Cancer, and Liver Cancer

Studies have reported on the effect of antineoplastons in certain types of cancer:

  • The effect of antineoplastons A10 and AS2-1 on brain tumors was studied at Dr. Burzynski's clinic and at the Mayo Clinic. A brain tumor study done in Japan did not report the type of antineoplaston used.
  • The effect of antineoplaston AS2-1 on prostate cancer was studied at Dr. Burzynski's clinic.
  • The effect of antineoplaston A10 on liver cancer is discussed in a case report from Japan.

These studies reported mixed results, including some cancer remissions (signs and symptoms of cancer decreased or went away). Other investigators have not been able to obtain the same results reported by Dr. Burzynski and his team. Some of the patients in the reported studies received standard treatments in addition to the antineoplastons. In those cases, it is not known if responses and side effects were caused by antineoplaston therapy, the other treatments, or both. One additional independent report (a study from Japan) was completed but does not have the same findings as the Burzynski report.

(See the PDQ health professional summary on Antineoplastons for detailed information on clinical trial results.)

Randomizedcontrolled trials give the highest level of evidence. In these trials, volunteers are put randomly (by chance) into one of 2 or more groups that compare different treatments. One group (called the control group) does not receive the new treatment being studied. The control group is compared to the groups that receive the new treatment, to see if the new treatment works. No randomized, controlled trials showing the effectiveness of antineoplastons have been published in peer-reviewed scientific journals.

In 1991, the National Cancer Institute (NCI) reviewed some of Dr. Burzynski's cases and decided to conduct clinical trials on antineoplastons at cancer centers. By August 1995, only 9 patients had enrolled and the clinical trials were closed before being completed. The U. S. Food and Drug Administration (FDA) gave Dr. Burzynski permission to conduct clinical trials of antineoplaston therapy at his own clinic. Ongoing non-randomized clinical trials at the Burzynski clinic continue to study the effect of antineoplastons on cancer.

The antineoplastons now used in clinical trials are A10, AS2-5, AS2-1, A2, A3, and A5. Information about ongoing clinical trials is available from the NCI Web site.

Have any side effects or risks been reported from antineoplastons?

Antineoplaston side effects included mild, short-term side effects as well as serious nervous system problems.

The following mild side effects have been noted:

  • Anemia (lower than normal number of red blood cells).
  • High blood pressure.
  • Dizziness.
  • Gas.
  • Fever and chills.
  • Feeling very tired.
  • Headaches.
  • Abnormal levels of calcium in the blood.
  • Dry or itchy skin rash.
  • Nausea and vomiting.
  • Numbness.
  • Irregular heartbeat.
  • Swelling caused by excess fluid in body tissues.
  • Swelling, pain, or stiffness in small joints.

Serious nervous system side effects included the following:

  • Extreme sleepiness.
  • Confusion.
  • Seizures.
  • Swelling near the brain.
Are antineoplastons approved by the U.S. Food and Drug Administration (FDA) for use as a cancer treatment in the United States?

Antineoplastons are not approved by the FDA for the prevention or treatment of any disease. In the United States, antineoplaston therapy can be obtained only in clinical trials at Dr. Burzynski's clinic.

Current Clinical Trials

Check NCI's list of cancer clinical trials for U.S. cancer CAM clinical trials on antineoplastons therapy that are actively enrolling patients.

General information about clinical trials is available from the NCI Web site.

Changes to This Summary (07 / 26 / 2011)

The PDQcancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.

Editorial changes were made to this summary.

General CAM Information

Complementary and alternative medicine (CAM)—also referred to as integrative medicine—includes a broad range of healing philosophies, approaches, and therapies. A therapy is generally called complementary when it is used in addition to conventional treatments; it is often called alternative when it is used instead of conventional treatment. (Conventional treatments are those that are widely accepted and practiced by the mainstream medical community.) Depending on how they are used, some therapies can be considered either complementary or alternative. Complementary and alternative therapies are used in an effort to prevent illness, reduce stress, prevent or reduce side effects and symptoms, or control or cure disease.

Unlike conventional treatments for cancer, complementary and alternative therapies are often not covered by insurance companies. Patients should check with their insurance provider to find out about coverage for complementary and alternative therapies.

Cancer patients considering complementary and alternative therapies should discuss this decision with their doctor, nurse, or pharmacist as they would any therapeutic approach, because some complementary and alternative therapies may interfere with their standard treatment or may be harmful when used with conventional treatment.

Evaluation of CAM Approaches

It is important that the same rigorous scientific evaluation used to assess conventional approaches be used to evaluate CAM therapies. The National Cancer Institute (NCI) and the National Center for Complementary and Alternative Medicine (NCCAM) are sponsoring a number of clinical trials (research studies) at medical centers to evaluate CAM therapies for cancer.

Conventional approaches to cancer treatment have generally been studied for safety and effectiveness through a rigorous scientific process that includes clinical trials with large numbers of patients. Less is known about the safety and effectiveness of complementary and alternative methods. Few CAM therapies have undergone rigorous evaluation. A small number of CAM therapies originally considered to be purely alternative approaches are finding a place in cancer treatment—not as cures, but as complementary therapies that may help patients feel better and recover faster. One example is acupuncture. According to a panel of experts at a National Institutes of Health (NIH) Consensus Conference in November 1997, acupuncture has been found to be effective in the management of chemotherapy-associated nausea and vomiting and in controlling pain associated with surgery. In contrast, some approaches, such as the use of laetrile, have been studied and found ineffective or potentially harmful.

The NCI Best Case Series Program, which was started in 1991, is one way CAM approaches that are being used in practice are being investigated. The program is overseen by the NCI's Office of Cancer Complementary and Alternative Medicine (OCCAM). Health care professionals who offer alternative cancer therapies submit their patients' medical records and related materials to OCCAM. OCCAM conducts a critical review of the materials and develops follow-up research strategies for approaches deemed to warrant NCI-initiated research.

Questions to Ask Your Health Care Provider About CAM

When considering complementary and alternative therapies, patients should ask their health care provider the following questions:

  • What side effects can be expected?
  • What are the risks associated with this therapy?
  • Do the known benefits outweigh the risks?
  • What benefits can be expected from this therapy?
  • Will the therapy interfere with conventional treatment?
  • Is this therapy part of a clinical trial?
  • If so, who is sponsoring the trial?
  • Will the therapy be covered by health insurance?

To Learn More About CAM

National Center for Complementary and Alternative Medicine (NCCAM)

The National Center for Complementary and Alternative Medicine (NCCAM) at the National Institutes of Health (NIH) facilitates research and evaluation of complementary and alternative practices, and provides information about a variety of approaches to health professionals and the public.

NCCAM Clearinghouse
Post Office Box 7923 Gaithersburg, MD 20898–7923
Telephone: 1–888–644–6226 (toll free) 301–519–3153 (for International callers)
TTY (for deaf and hard of hearing callers): 1–866–464–3615
Fax: 1–866–464–3616
Web site:

CAM on PubMed

NCCAM and the NIH National Library of Medicine (NLM) jointly developed CAM on PubMed, a free and easy-to-use search tool for finding CAM-related journal citations. As a subset of the NLM's PubMed bibliographic database, CAM on PubMed features more than 230,000 references and abstracts for CAM-related articles from scientific journals. This database also provides links to the Web sites of over 1,800 journals, allowing users to view full-text articles. (A subscription or other fee may be required to access full-text articles.) CAM on PubMed is available through the NCCAM Web site. It can also be accessed through NLM PubMed bibliographic database by selecting the "Limits" tab and choosing "Complementary Medicine" as a subset.

Office of Cancer Complementary and Alternative Medicine

The NCI Office of Cancer Complementary and Alternative Medicine (OCCAM) coordinates the activities of the NCI in the area of complementary and alternative medicine (CAM). OCCAM supports CAM cancer research and provides information about cancer-related CAM to health providers and the general public via the NCI Web site.

National Cancer Institute (NCI) Cancer Information Service

U.S. residents may call the NCI Cancer Information Service toll free at 1-800-4-CANCER (1-800-422-6237) Monday through Friday from 8:00 am to 8:00 pm. A trained Cancer Information Specialist is available to answer your questions.

Food and Drug Administration

The Food and Drug Administration (FDA) regulates drugs and medical devices to ensure that they are safe and effective.

Food and Drug Administration
5600 Fishers Lane
Rockville, MD 20857
Telephone: 1–888–463–6332 (toll free)
Web site:

Federal Trade Commission

The Federal Trade Commission (FTC) enforces consumer protection laws. Publications available from the FTC include:

  • Who Cares: Sources of Information About Health Care Products and Services
  • Fraudulent Health Claims: Don't Be Fooled
Consumer Response Center
Federal Trade Commission
Washington, DC 20580
Telephone: 1-877-FTC-HELP (1-877-382-4357) (toll free)
TTY (for deaf and hearing impaired callers): 202-326-2502
Web site:

Last Revised: 2011-07-26

If you want to know more about cancer and how it is treated, or if you wish to know about clinical trials for your type of cancer, you can call the NCI's Cancer Information Service at 1-800-422-6237, toll free. A trained information specialist can talk with you and answer your questions.

This information does not replace the advice of a doctor. Healthwise, Incorporated disclaims any warranty or liability for your use of this information.