Drug Development
Progress is reported toward acetate supplementation therapy for Canavan disease
Published in Medical Letter on the CDC and FDA, November 20th, 2005
"Canavan disease (CD) is a fatal genetic neurodegenerative disorder caused by mutations in the gene for aspartoacylase, an enzyme that hydrolyzes N-acetylaspartate (NAA) into L-aspartate and acetate. Because aspartoacylase is localized in oligodendrocytes, and NAA-derived acetate is incorporated into myelin lipids, we hypothesize that an acetate deficiency in oligodendrocytes is responsible for the pathology in CD, and we propose acetate supplementation as a possible therapy," scientists writing in the Journal of Pharmacology and Experimental Therapeutics report.
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