Lovastatin for X-Linked Adrenoleukodystrophy

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Abstract
X-linked adrenoleukodystrophy is an inherited recessive disorder characterized by a defect in peroxisomal β-oxidation of very-long-chain fatty acids (those with more than 22 carbon atoms) and secondary neuroinflammatory damage.1,2 Even though the accumulation of very-long-chain fatty acids in plasma and tissues occurs early, the neurologic manifestations are not observed until the age of 4 to 8 years for childhood adrenoleukodystrophy or 20 to 30 years for adrenomyeloneuropathy. The neurologic damage in X-linked adrenoleukodystrophy may be mediated by the activation of astrocytes and the induction of proinflammatory cytokines. At present, dietary restrictions and therapy with Lorenzo's oil are used to lower plasma levels of very-long-chain fatty acids, but these measures do not address the neuroinflammatory aspects of the disorder.1,2