Drug intelligence / Profile preview

alpha-lipoic acid + N-acetylcysteine + alpha-tocopherol

Development stage
Unknown
Lead developer
Neurometabolic Diseases Laboratory
Modality
Small Molecules
Administration
Oral
01

Overview

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.

Other names
Antioxidant cocktailAntioxidant combination therapy (NAC, Lipoic Acid, Vitamin E)NAC + Lipoic Acid + Vitamin E
02

Targets

GSH (Glutathione)

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