Compounds for the prevention or treatment of X linked adrenoleukodystrophy
Aurora Pujol
Institut d'Investigació Biomèdica de Bellvitge
Compounds for the prevention or treatment of X linked adrenoleukodystrophy: 2-Propylpentanoic acid (valproic acid) and its monosodium salt are useful orally for the prevention and/or treatment of X linked adrenoleukodystrophy (X-ALD) in human, for both phenotypes: adrenomyeloneuropathy and cerebral childhood adrenoleukodystrophy, alone or in combination with other substances. Valproic acid increases ABCD2 gene expression levels, not only in human cell lines, but also in rat and human neural tissue, and in peripheral mononuclear cells of X-ALD patients. As valproic acid is an excellently examined active pharmaceutical ingredient in terms of toxicology, side effects and dose, and it has already been in clinical use for more than three decades, the present invention provides a new agent for the prevention or treatment of a so-far fatal disease.
Currently, we are considering the possibility of reformulate the patent with a cocktail containing VPA and antioxidant compounds, as derived from experimental work on progress.
Filing date: 11 May, 2006
Licenced date: 31 Dec, 2012
P200601202