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Antineoplaston AS2-1 is a 4:1 mixture of phenylacetate and phenylacetylglutamine (as sodium salts), which are degradation products of the agent A10. It has been investigated primarily as an experimental anticancer therapy. The proposed mechanisms include inhibition of L-glutamine incorporation into tumor-cell proteins (leading to cell cycle arrest in G1 phase and inhibition of mitosis), possible inhibition of RAS oncogene expression, activation of the tumor suppressor gene p53 (resulting in cell differentiation and apoptosis), and normalization of hypermethylation at promoter regions in various genes including tumor suppressor genes[1][2][8]. Antineoplaston AS2-1 has been studied mainly for brain tumors such as gliomas, medulloblastoma, pineoblastoma, anaplastic astrocytoma, as well as other cancers[4][5][6][7]. Clinical trials have reported mixed results regarding efficacy; toxicity is generally mild to moderate but can include neurocortical effects at higher doses[5][6][9].
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