Other common name(s): antineoplastons, A10 (Atengenal, Cengenal), AS2-1 (Astugenal, Fengenal)Scientific/medical name(s): 3-phenylacetylamino-2,6-piperidinedione, phenylacetic acid, phenylacetylglutamine, phenylacetylisoglutamine
Antineoplaston therapy is a complementary/alternative cancer treatment that involves using a group of synthetic chemicals called antineoplastons intended to protect the body from disease. Antineoplastons are made up mostly of peptides and amino acids originally taken from human blood and urine.
Thousands of patients have been treated with antineoplastons, mostly at a single clinic, and clinical trials are under way there for many types of cancer. Published clinical trial results are available for a relatively small number of patients, and the effectiveness of antineoplastons as a cancer therapy remains uncertain. Most cancer specialists believe there is insufficient evidence to recommend use of antineoplastons except perhaps in the context of clinical trials that will provide reliable information on the safety and effectiveness of this treatment.
How is it promoted for use?
Supporters have claimed antineoplastons are a part of something called the body’s natural biochemical defense system. This system is said to act independently of the body’s immune system and to protect against diseases like cancer, which involve a breakdown in the information processing of the body’s cells.
Proponents claim antineoplaston therapy has been successful in treating many forms of cancer. They claim people with cancer don’t have enough naturally occurring antineoplastons and that this therapy replenishes the body’s supply, allowing the biochemical defense system of the body to induce cancer cells to stop growing and to develop features that resemble normal cells (cell differentiation).
What does it involve?
Antineoplastons are given orally or by injection into a vein. The duration of treatment usually ranges from eight to twelve months. A year of treatment can cost from $30,000 to $60,000, depending on the type of treatment, number of consultations, and the need for surgery to implant a catheter for drug delivery.
Antineoplaston therapy was developed by Stanislaw Burzynski, MD, PhD. Initial treatments are given over the course of one to three weeks at a clinic in Houston, founded by Dr. Burzynski. (Other U.S. centers are participating in studies to evaluate this treatment, as well as some centers in other countries.) Further treatments may be given “at home,” but require monthly visits to a doctor, either at the Houston clinic or elsewhere with one of Dr. Burzynski’s research colleagues. In the past, many of the patients who received antineoplaston treatment also were treated with surgery, radiation, chemotherapy, or combinations of these standard treatments at other centers, and some received chemotherapy prescribed by Dr. Burzynski. Currently, antineoplaston treatment is available in the United States only through participation in clinical trials led by Dr. Burzynski and his colleagues. To be eligible for these clinical studies, patients must have cancer that is growing despite conventional treatments. Patients cannot receive conventional anticancer treatments while they are participating in these antineoplaston studies.
What is the history behind it?
According to the Burzynski Patient Group Web site, “In 1967, Dr. Burzynski identified naturally occurring peptides in the human body that he concluded control the growth of cancer.” That same year, he graduated from the Medical Academy in Lublin, Poland. His first article published in the medical literature, which discussed an effect of urinary peptides on growth of cancer cells in laboratory dishes (tissue culture), was published in 1973 while he was working as a researcher at Baylor College of Medicine in Houston. Although Burzynski initially isolated the chemicals that he named antineoplastons from human blood and urine, he later produced these naturally occurring substances in his laboratory.
In 1977, Dr. Burzynski opened his own clinic, where he has used antineoplaston therapy to treat patients for a variety of cancers. He claims that the therapy has cured many patients of their illnesses. However, his methods for conducting and reporting clinical research have been criticized for not following appropriate scientific standards.
In the United States today, antineoplaston therapy can only be given to patients who go to Dr. Burzynski’s clinic and enroll in his clinical trials approved by the U.S. Food and Drug Administration (FDA).
What is the evidence?
Some patients claim to have been helped by antineoplaston therapy, but these anecdotal reports are not considered evidence of effectiveness by the medical community, either for this or for any other type of therapy. Some promising results for the use of antineoplaston therapy have been reported in small studies. Most of these studies were directed by Dr. Burzynski himself. Results from a few small studies conducted by one group of researchers in Japan have also been published. However, the available clinical evidence consists of early-phase clinical trials and best case series.
During the 1980s, the United States Congressional Office of Technology Assessment (OTA) reviewed medical journal articles describing cases of cancer patients whom Dr. Burzynski had treated with antineoplaston therapy. Its report, published in 1990, concludes that, “Despite a substantial number of preliminary clinical studies published by Burzynski and his associates describing outcomes among the patients he treated with Antineoplastons and an attempt at a ‘best case’ review, there is still a lack of valid information to judge whether this treatment is likely to be beneficial to cancer patients.” The OTA report criticized Burzynski’s research process and noted that his definitions of advanced cancer and of complete and partial cancer remission were not used in accordance with generally accepted definitions. One example they pointed to was a patient said to have had a complete remission after treatment with antineoplastons. The report concluded, however, that this claim was inappropriate because the cancer had been removed by surgery before the antineoplaston treatment was started.
In 1982, consultants to the Ontario (Canada) Ministry of Health visited Burzynski’s clinic and reviewed records of twelve patients selected by Burzynski from among the thousands he had treated. According to the OTA report, the Canadian doctors “found no examples of objective response to Antineoplastons.” In 1985, the Canadian Bureau of Prescription Drugs examined the records of Canadian doctors who had treated patients at Dr. Burzynski’s clinic in Houston. Of 36 patients, 32 had died without showing signs of improvement. Of the remaining 4, one patient died after slight improvement, while one patient died after stabilizing for a year. The 2 remaining patients had widespread cancer.
In 1991, the National Cancer Institute (NCI) reviewed several ‘best cases’ (involving patients with brain tumors) chosen by Burzynski. According to a 1992 article in the Journal of the National Cancer Institute, “two NCI extramural investigators independently reviewed the case histories of some patients treated with antineoplastons. At the investigators’ recommendation, the NCI examined the case histories, pathology slides, and imaging studies from seven patients with primary brain tumors ….[T]he site visit team and, subsequently, the [NCI] Division of Cancer Treatment’s Decision Network Committee believed that evidence of possible antitumor effect was demonstrated.” NCI concluded that these results warranted further investigation through clinical trials at other medical centers. But because of disagreement between NCI researchers and Burzynski, the clinical trials were terminated in 1995. By 1999, the researchers concluded that only 6 of the 9 patients treated in that study could be evaluated according to the study’s initial requirements. None of the 6 showed evidence of tumor shrinkage. The researchers noted, however, that the small number of patients participating limited their ability to say with confidence that antineoplaston treatment had no benefit. Side effects of antineoplaston treatment included temporary sleepiness and confusion, and worsening of epilepsy (seizures) in patients who already had that problem (as a result of the tumors).
Dr. Burzynski currently has permission from the FDA to conduct clinical trials of antineoplaston therapy at his clinic. The NCI and researchers at several cancer centers are also conducting laboratory experiments on the peptides involved in antineoplaston therapy.
While many articles have been published and dozens of clinical trials against many types of cancer have been ongoing at Dr. Burzynski’s clinic for several years, there have not been any randomized controlled trials—the type of study that is required for new anticancer drugs to be approved by the FDA and recommended by conventional oncologists.
Although some proponents of antineoplaston therapy have suggested that the reviews of this treatment by conventional cancer specialists are biased by mistrust of alternative therapies, even some prominent figures in the field of alternative medicine have reservations about antineoplastons. According to Dr. Andrew Weil, “Over the years, Dr. Burzynski claims to have treated more than 8,000 patients, but his success rates are unknown. His Web site states only that he has helped ‘many’ people. If antineoplaston therapy works, we should have scientific studies showing what percentage of patients treated have survived and for how long, as well as evidence showing how Dr. Burzynski’s method stacks up against conventional cancer treatment…. Until we have credible scientific evidence showing what antineoplastons are, how they act in the body, and what realistic expectations of treatment with them might be, I see no reason for any cancer patient to take this route.”
Are there any possible problems or complications?
Proponents claim that antineoplaston therapy is ‘nontoxic.’ However, reported side effects include stomach gas, slight rashes, chills, fever, change in blood pressure, unpleasant body odor during treatment, sleepiness, confusion, and seizures. High levels of blood sodium can also be a significant problem with this therapy.
It is not known whether antineoplastons would cause any problems due to interactions with other medications.
Relying on this type of treatment alone and avoiding or delaying conventional medical care for cancer may have serious health consequences.
More information from your American Cancer Society The following information on complementary and alternative therapies may also be helpful to you. These materials may be found on our Web site (www.cancer.org) or ordered from our toll-free number (1-800-ACS-2345).
The ACS Operational Statement on Complementary and Alternative Methods of Cancer Management
The antineoplaston anomaly: how a drug was used for decades in thousands of patients, with no safety, efficacy data. Cancer Lett. 1998;24(36).
Antineoplastons. National Cancer Institute. Accessed at: http://cis.nci.nih.gov/fact/7_43.htm. on June 11, 2008.
Antineoplaston therapy. Burzynski Patient Group. Accessed at: http://burzynskipatientgroup.org/antitherapy.html on June 11, 2008.
Buckner JC, Malkin MG, Reed E, Cascino TL, Reid JM, Ames MM, Tong WP, Lim S, Figg WD. Phase II study of antineoplastons A10 (NSC 648539) and AS2-1 (NSC 620261) in patients with recurrent glioma. Mayo Clin Proc. 1999;74:137-145.
Burzynski SR. Biologically active peptides in human urine. I. Isolation of a group of medium-sized peptides. Physiol Chem Phys. 1973;5:437-447.
Cassileth BR. The Alternative Medicine Handbook: The Complete Reference Guide to Alternative and Complementary Therapies. New York, NY: W.W. Norton & Co; 1998.
Green S. ‘Antineoplastons’. An unproved cancer therapy. JAMA. 1992;267:2924-2928.
Hawkins MJ, Friedman MA. National Cancer Institute’s evaluation of unconventional cancer treatments. J Natl Cancer Inst. 1992;84:1699-1702.
US Congress, Office of Technology Assessment. Pharmacologic and Biologic Treatments. In: Unconventional Cancer Treatments: OTA-H-405. Washington, DC: US Government Printing Office; 1990.
Weil A. Antineoplastons: a bogus cancer treatment? Andrew Weil MD. Accessed at: http://www.drweil.com/drw/u/id/QAA400003 on June 11, 2008.
Note: This information may not cover all possible claims, uses, actions, precautions, side effects or interactions. It is not intended as medical advice, and should not be relied upon as a substitute for consultation with your doctor, who is familiar with your medical situation.
Last Revised: 11/01/2008