As noted in the General Information section, Burzynski first proposed antineoplastons as a naturally occurring biochemical defense against cancer in 1976 as a result of his study of cybernetic systems and information theory. The search for information-bearing peptides in body fluids led him to separate peptides from human blood and subsequently from human urine. He called these substances antineoplastons and categorized them according to their general and specific anticarcinogenic potential. In 1980, the developer characterized the chemical structures of antineoplastons and began preparing them synthetically rather than isolating them from human urine. Preparations now used in clinical studies to treat cancer are antineoplastons A10, AS2-5, AS2-1, A2, A3, and A5.

From 1991 to 1995, the National Cancer Institute initiated phase II clinical trials of antineoplastons A10 and AS2-1. Protocols for two phase II clinical trials were originally developed by investigators from several cancer centers, with review and input from both the developer and NCI. The National Institutes of Health, Office of Alternative Medicine, now known as the National Center for Complementary and Alternative Medicine, provided funding for the trials. Three centers (Memorial Sloan-Kettering Cancer Center, the Mayo Clinic, and the Warren Grant Magnuson Clinical Center at NIH) began accruing participants for these NCI-sponsored studies in 1993. However, by August 1995 only nine patients had entered the trials; despite efforts by the developer, NCI staff, and investigators to reach agreement on proposed changes to increase patient accrual and dose, the studies were closed prematurely in August 1995.[1-3]

The developer and investigators in Japan have reported several case series showing varying results using antineoplastons as a clinical therapy against several different types of cancer, alone or in combination with standard chemotherapy.[4-15] These studies are described in more detail in the Human/Clinical Studies section of this document. Most of these studies were phase I trials or their equivalent; therefore, the only objective of these trials was safety.

Other uses of antineoplastons suggested by the developer include treatment of conditions such as Parkinson’s disease, sickle cell anemia, and thalassemia.[16]

References

1. The antineoplaston anomaly: how a drug was used for decades in thousands of patients, with no safety, efficacy data. The Cancer Letter 24 (36):1998 Also available online ¹. Last accessed October 9, 2007. 
   
   
2. Burzynski SR: Efficacy of antineoplastons A10 and AS2-1. Mayo Clin Proc 74 (6): 641-2, 1999.  [PUBMED Abstract]
   
   
3. Hammer MR, Jonas WB: Managing social conflict in complementary and alternative medicine research: the case of antineoplastons. Integr Cancer Ther 3 (1): 59-65, 2004.  [PUBMED Abstract]
   
   
4. Burzynski SR, Stolzmann Z, Szopa B, et al.: Antineoplaston A in cancer therapy. (I). Physiol Chem Phys 9 (6): 485-500, 1977.  [PUBMED Abstract]
   
   
5. Tsuda H, Hara H, Eriguchi N, et al.: Toxicological study on antineoplastons A-10 and AS2-1 in cancer patients. Kurume Med J 42 (4): 241-9, 1995.  [PUBMED Abstract]
   
   
6. Burzynski SR: Toxicology studies on antineoplaston AS2-5 injections in cancer patients. Drugs Exp Clin Res 12 (Suppl 1): 17-24, 1986.  [PUBMED Abstract]
   
   
7. Burzynski SR, Burzynski B, Mohabbat MO: Toxicology studies on antineoplaston AS2-1 injections in cancer patients. Drugs Exp Clin Res 12 (Suppl 1): 25-35, 1986.  [PUBMED Abstract]
   
   
8. Burzynski SR, Kubove E: Toxicology studies on antineoplaston A10 injections in cancer patients. Drugs Exp Clin Res 12 (Suppl 1): 47-55, 1986.  [PUBMED Abstract]
   
   
9. Burzynski SR, Kubove E, Burzynski B: Treatment of hormonally refractory cancer of the prostate with antineoplaston AS2-1. Drugs Exp Clin Res 16 (7): 361-9, 1990.  [PUBMED Abstract]
   
   
10. Burzynski SR, Kubove E, Burzynski B: Phase I clinical studies of antineoplaston A5 injections. Drugs Exp Clin Res 13 (Suppl 1): 37-43, 1987.  [PUBMED Abstract]
    
    
11. Burzynski SR, Kubove E: Phase I clinical studies of antineoplaston A3 injections. Drugs Exp Clin Res 13 (Suppl 1): 17-29, 1987.  [PUBMED Abstract]
    
    
12. Burzynski SR, Kubove E: Initial clinical study with antineoplaston A2 injections in cancer patients with five years' follow-up. Drugs Exp Clin Res 13 (Suppl 1): 1-11, 1987.  [PUBMED Abstract]
    
    
13. Sugita Y, Tsuda H, Maruiwa H, et al.: The effect of Antineoplaston, a new antitumor agent on malignant brain tumors. Kurume Med J 42 (3): 133-40, 1995.  [PUBMED Abstract]
    
    
14. Tsuda H, Sata M, Kumabe T, et al.: Quick response of advanced cancer to chemoradiation therapy with antineoplastons. Oncol Rep 5 (3): 597-600, 1998 May-Jun.  [PUBMED Abstract]
    
    
15. Kumabe T, Tsuda H, Uchida M, et al.: Antineoplaston treatment for advanced hepatocellular carcinoma. Oncol Rep 5 (6): 1363-7, 1998 Nov-Dec.  [PUBMED Abstract]
    
    
16. Burzynski SR: Potential of antineoplastons in diseases of old age. Drugs Aging 7 (3): 157-67, 1995.  [PUBMED Abstract]
    
    

Glossary Terms

Practices used instead of standard treatments. They generally are not recognized by the medical community as standard or conventional medical approaches. Examples of alternative medicine include dietary supplements, megadose vitamins, herbal preparations, special teas, acupuncture, massage therapy, magnet therapy, spiritual healing, and meditation.

Having to do with preventing or delaying the development of cancer.

A substance isolated from normal human blood and urine that is being tested as a type of treatment for some tumors and AIDS.

A tissue with red blood cells, white blood cells, platelets, and other substances suspended in fluid called plasma. Blood takes oxygen and nutrients to the tissues, and carries away wastes.

A term for diseases in which abnormal cells divide without control. Cancer cells can invade nearby tissues and can spread to other parts of the body through the blood and lymph systems. There are several main types of cancer. Carcinoma is cancer that begins in the skin or in tissues that line or cover internal organs. Sarcoma is cancer that begins in bone, cartilage, fat, muscle, blood vessels, or other connective or supportive tissue. Leukemia is cancer that starts in blood-forming tissue such as the bone marrow, and causes large numbers of abnormal blood cells to be produced and enter the blood. Lymphoma and multiple myeloma are cancers that begin in the cells of the immune system. Central nervous system cancers are cancers that begin in the tissues of the brain and spinal cord.

A group or series of case reports involving patients who were given similar treatment. Reports of case series usually contain detailed information about the individual patients. This includes demographic information (for example, age, gender, ethnic origin) and information on diagnosis, treatment, response to treatment, and follow-up after treatment.

Treatment with drugs that kill cancer cells.

A type of research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. Also called clinical trial.

The amount of medicine taken, or radiation given, at one time.

Liquid.

The National Cancer Institute, part of the National Institutes of Health of the United States Department of Health and Human Services, is the Federal Government's principal agency for cancer research. The National Cancer Institute conducts, coordinates, and funds cancer research, training, health information dissemination, and other programs with respect to the cause, diagnosis, prevention, and treatment of cancer. Access the National Cancer Institute Web site at http://www.cancer.gov. Also called NCI.

A federal agency that uses science to explore complementary and alternative medicine (CAM) practices, trains CAM researchers, and provides authoritative information about CAM to professionals and the public. NCCAM awards grants for research projects, training, and career development in CAM; sponsors conferences, educational programs, and exhibits; studies ways to use proven CAM practices along with conventional medical practice; and supports adding CAM to medical, dental, and nursing school programs. NCCAM is part of the National Institutes of Health. Also called NCCAM.

A federal agency in the U.S. that conducts biomedical research in its own laboratories; supports the research of non-Federal scientists in universities, medical schools, hospitals, and research institutions throughout the country and abroad; helps in the training of research investigators; and fosters communication of medical information. Access the National Institutes of Health Web site at http://www.nih.gov. Also called NIH.

NCI, part of the National Institutes of Health of the United States Department of Health and Human Services, is the Federal Government's principal agency for cancer research. It conducts, coordinates, and funds cancer research, training, health information dissemination, and other programs with respect to the cause, diagnosis, prevention, and treatment of cancer. Access the NCI Web site at http://www.cancer.gov. Also called National Cancer Institute.

A federal agency in the U.S. that conducts biomedical research in its own laboratories; supports the research of non-Federal scientists in universities, medical schools, hospitals, and research institutions throughout the country and abroad; helps in the training of research investigators; and fosters communication of medical information. Access the NIH Web site at http://www.nih.gov. Also called National Institutes of Health.

A molecule that contains two or more amino acids (the molecules that join together to form proteins). Peptides that contain many amino acids are called polypeptides or proteins.

The first step in testing a new treatment in humans. These studies test the best way to give a new treatment (for example, by mouth, intravenous infusion, or injection) and the best dose. The dose is usually increased a little at a time in order to find the highest dose that does not cause harmful side effects. Because little is known about the possible risks and benefits of the treatments being tested, phase I trials usually include only a small number of patients who have not been helped by other treatments.

A study to test whether a new treatment has an anticancer effect (for example, whether it shrinks a tumor or improves blood test results) and whether it works against a certain type of cancer.

An inherited disease in which the red blood cells have an abnormal crescent shape, block small blood vessels, and do not last as long as normal red blood cells. Sickle cell anemia is caused by a mutation (change) in one of the genes for hemoglobin (the substance inside red blood cells that binds to oxygen and carries it from the lungs to the tissues). It is most common in people of West and Central African descent. Also called sickle cell disease.

Treatment.

Fluid containing water and waste products. Urine is made by the kidneys, stored in the bladder, and leaves the body through the urethra.