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 reported to be under way there for many types of cancer. More than 40 years after antineoplastons were first discovered, published clinical trial results are available only for a relatively small number of patients, mostly preliminary reports, uncontrolled studies, or case reports. According to the National Cancer Institute, “No randomized controlled trials showing the effectiveness of antineoplastons have been published in the peer-reviewed scientific literature.” In other words, there is no convincing evidence showing that antineoplastons actually work.
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 by mouth, injection into a vein or muscle, and by other routes. The treatment usually lasts from 8 - 12 months. Treatment can cost from $7,000 to $9,500 per month or more, depending on the type of treatment, number of consultations, and the need for surgery to implant a catheter for drug delivery. Available information suggests that health insurance plans often do not reimburse costs linked to this treatment.
Antineoplaston therapy was developed by Stanislaw Burzynski, MD, PhD. Initial treatments are given over the course of 1 - 3 weeks at a clinic in Houston, founded by Dr. Burzynski. Further treatments might be given at home, but could require monthly visits to a doctor. In the past, many of the patients who received antineoplaston treatment were also treated with surgery, radiation, chemotherapy, or combinations of these standard treatments at other centers before visiting Dr. Burzynski’s clinic. Some patients also received chemotherapy or radiation 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. Patients cannot receive conventional anti-cancer treatment from other doctors while they are taking part in these antineoplaston studies.
What is the history behind it?
According to the Burzynski clinic Web site, in 1967 Dr. Burzynski identified naturally-occurring peptides in the human body that he concluded control the growth of cancer and called antineoplastons. 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 from human blood and urine, antineoplastons are now man-made.
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. He established the Burzynski Research Institute in 1984, but his methods for conducting and reporting clinical research have been criticized for not following 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. Of note, the FDA warned the Burzynski Research Clinic Review Board in 2009 about not following the rules for protecting human subjects in its clinical trials.
What is the evidence?
A number of patients claim to have been helped by antineoplaston therapy, but these kinds of 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 studies that were too small to demonstrate clear success. Most of these studies were directed by Dr. Burzynski himself. Results from a few small studies conducted by a 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 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, 1 patient died after slight improvement, while 1 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.
Dr. Burzynski is still conducting clinical trials of antineoplaston therapy at his clinic. Many of these clinical trials include certain standard cancer treatments such as radiation and chemotherapy treatments along with antineoplastons.
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 results of randomized controlled trials—the type of study that is required to prove that a new anti-cancer drug works – published in peer-reviewed medical journals. These types of clinical trials are required if a drug is to be approved by the FDA.
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."
Proponents claim that antineoplaston therapy is “nontoxic” However, reported side effects range from mild to serious, including fatigue (extreme tiredness), anemia (low red blood cell counts), headaches, numbness, skin rash, swelling of the hands and face, chills, fever, dizziness, changes in blood pressure, unpleasant body odor during treatment, and sleepiness. High levels of blood sodium and abnormal calcium levels can also cause significant problems with this therapy. Other serious side effects can involve the brain, and include confusion, seizures, and swelling in the brain. This has required that the treatment be stopped in some patients.
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.
To learn more
More information from your American Cancer Society
Here is more information you might find helpful. You also can order free copies of our documents from our toll-free number, 1-800-227-2345, or read them on our Web site, www.cancer.org.
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).
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 Clinic. Accessed at www.burzynskiclinic.com/ on March 27, 2012.
Burzynski SR. Biologically active peptides in human urine. I. Isolation of a group of medium-sized peptides. Physiol Chem Phys. 1973;5:437-447.
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.
Cassileth BR. The Alternative Medicine Handbook: The Complete Reference Guide to Alternative and Complementary Therapies. New York, NY: W.W. Norton & Co; 1998.
ClinicalTrials.gov. Antineoplastons. Accessed at http://clinicaltrials.gov/ct2/results?term=antineoplastons on March 28, 2012.
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.
National Cancer Institute. Antineoplastons PDQ (updated 8/16/12). Accessed at www.cancer.gov/cancertopics/pdq/cam/antineoplastons/healthprofessional on December 7, 2012.
University of California, San Diego, Moores Cancer Center. Antineoplastons. Accessed at http://moores.ucsd.edu/Outreach/PublicEducation/CAMs/antineoplastons.asp on March 28, 2012.
US Congress, Office of Technology Assessment. Pharmacologic and Biologic Treatments. In: Unconventional Cancer Treatments: OTA-H-405. Washington, DC: US Government Printing Office; 1990.
US Food and Drug Administration. Burzynski Research Institute Warning Letter, Ref: 10-HFD-45-09-01. October 5, 2009. Accessed at http://www.fda.gov/ICECI/EnforcementActions/WarningLetters/2009/ucm192711.htm on March 28, 2012.
Weil A. Antineoplastons: a bogus cancer treatment? Andrew Weil MD. Accessed at: http://www.drweil.com/drw/u/id/QAA400003 on March 28, 2012.
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: 12/07/2012