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Antineoplaston AS2-5 is a synthetic antineoplastic agent developed by the Burzynski Clinic, consisting of a mixture of the amino acid derivatives phenylacetylglutamine (PAG) and phenylacetate (PN). Originally identified in human urine and later synthesized, antineoplastons are categorized by the developer as "biochemical correctors" that normalize the behavior of cancer cells by regulating gene expression. Specifically, AS2-5 is proposed to induce cell differentiation and apoptosis by upregulating tumor suppressor genes such as p53 and p21 while downregulating oncogenes like MYC and RAS. Despite decades of investigation in Phase 1 and Phase 2 clinical trials for various malignancies, including brain tumors and advanced solid cancers, the therapeutic efficacy of AS2-5 remains a subject of significant controversy within the mainstream medical community, and it has not received FDA approval for any indication.
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