Canavan Disease
A USU study indicates acetate supplementation therapy could be effective in treating the fatal disease.
Canavan disease (CD) is a relatively rare, but always fatal, inherited, degenerative brain disorder. Currently, there is no cure for the disease, nor is there a standard course of treatment.
Research by a USU team headed by Aryan Namboodiri, Ph.D., indicates that a dietary supplement could be used as an effective treatment for CD. An article on the findings was published in the online Proceedings of the National Academy of Sciences.
USU Study Points to Potential Therapy
A disorder of the central nervous system, CD is characterized by the destruction of myelin—the brain's white matter. The myelin degenerates into spongy tissue riddled with microscopic fluid-filled spaces. Large amounts of N-acetylaspartic acid (NAA) in the brain are associated with CD. However, the relation to accumulated NAA and the cause of the destruction of myelin are not known.
Studies conducted at USU provide the first direct evidence that defective myelin synthesis, resulting from a deficiency of NAA-derived acetate, is involved in the pathogenesis of CD. These results make a strong case for acetate supplementation as a potential therapy for this devastating disease.