Canavan Disease - Treatment

Treatment

There is no cure for Canavan disease, nor is there a standard course of treatment. Treatment is symptomatic and supportive, but there is an experimental treatment using lithium citrate. When a person has Canavan Disease, his or her levels of N-acetyl aspartate are chronically elevated. The lithium citrate has proven that, in a rat genetic model of Canavan Disease, the lithium citrate significantly decreased the levels of N-acetyl aspartate. When tested on a human, the subject reversed during a two week wash-out period after withdrawal of lithium. The investigation revealed both decreased N-acetyl aspartate levels in regions of the brain tested and magnetic resonance spectroscopic values that are more characteristic of normal development and myelination. This evidence suggests that a larger controlled trial of lithium may be warranted as supportive therapy for children with Canavan disease.

In addition, there are experimental trials of gene therapy. A healthy gene is cloned to take over for the defective one that causes Canavan disease.

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