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This investigational antioxidant combination therapy, developed by Dr. Aurora Pujol and colleagues at the Bellvitge Biomedical Research Institute, consists of alpha-lipoic acid, N-acetylcysteine (NAC), and alpha-tocopherol (vitamin E). It is specifically designed to treat adrenomyeloneuropathy (AMN), the adult-onset form of X-linked adrenoleukodystrophy (X-ALD). The therapy targets oxidative stress, which is recognized as an early and critical factor in the neurodegenerative cascade of X-ALD. By combining these three small-molecule antioxidants, the treatment aims to reduce oxidative damage, restore redox balance, and halt the axonal degeneration in the spinal cord that leads to spastic paraparesis. A Phase II open-label pilot study published in 2019 demonstrated that high doses of this combination were well-tolerated and could correct biomarkers of oxidative stress in patients with AMN, following successful preclinical trials in mouse models.
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