This complementary and alternative medicine (CAM) information summary provides an overview of the use of antineoplastons as treatments for patients with cancer. The summary includes a brief history of the development of antineoplastons; a review of laboratory, animal, and human studies; and possible side effects associated with antineoplaston use.

This summary contains the following key information:

• Antineoplastons are drugs composed of chemical compounds that are naturally present in the urine and blood. They are an experimental cancer therapy that is purported to provide a natural biochemical substance that is excreted and therefore lacking in people with cancer.
• Antineoplastons were first proposed as a possible cancer treatment in 1976.
• Antineoplastons were originally isolated from human urine but are now synthesized from readily available chemicals in the developer’s laboratory.
• Antineoplastons are not approved by the United States Food and Drug Administration for the prevention or treatment of any disease.
• No randomized controlled trials showing the effectiveness of antineoplastons have been published in the peer-reviewed scientific literature.
• Antineoplaston side effects can include serious neurologic toxicity.
• Nonrandomized clinical trials investigating the anticancer efficacy of antineoplastons are underway at the developer’s institute.

Many of the medical and scientific terms used in this summary are hypertext linked (at first use in each section) to the NCI Dictionary, which is oriented toward nonexperts. When a linked term is clicked, a definition will appear in a separate window. All linked terms and their corresponding definitions will appear in a glossary in the printable version of the summary.

Reference citations in some PDQ CAM information summaries may include links to external Web sites that are operated by individuals or organizations for the purpose of marketing or advocating the use of specific treatments or products. These reference citations are included for informational purposes only. Their inclusion should not be viewed as an endorsement of the content of the Web sites, or of any treatment or product, by the PDQ Cancer CAM Editorial Board or the National Cancer Institute.

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