Antineoplaston A10 has been investigated primarily for its potential use in cancer treatment. It is part of a group of compounds derived from amino acids and peptides, studied for their ability to influence the growth and differentiation of cancer cells. Research has focused on its role in modulating gene expression and inhibiting tumor cell proliferation, particularly in brain tumors and other aggressive cancers. It has been used in experimental therapies, often in combination with other antineoplastons, aiming to restore normal cellular function without damaging healthy tissue. Despite ongoing interest, its clinical application remains limited and controversial due to insufficient evidence from large-scale, controlled trials. Use has been largely restricted to investigational settings.