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.
In a 2014 study done in Japan, antineoplaston AS2-1 was tested in human colon cancer cells. High concentrations of AS2-1 were needed to be effective in the cancer cells. Since the concentrations of AS2-1 seen in this cell study are 4 times higher than concentrations seen in cancer patients who have received it, these findings may not be useful in clinical studies.
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 III randomized, 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.)
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.)
Randomized controlled 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.
- Fever and chills.
- Feeling very tired.
- Abnormal levels of calcium in the blood.
- Dry or itchy skin rash.
- Nausea and vomiting.
- 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.
- Swelling near the brain.
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.