Selenium treatment in neuronal ceroid-lipofuscinosis

Neuronal ceroid-lipofuscinosis (NCL) refers to a group of disorders with devastating effects on the central nervous system. The accumulation of autofluorescent lipopigments containing lipid peroxides is considered a pathogenetic mechanism of the cell damage seen in NCL. Therapy aimed at preventing further lipid peroxidation, such as the Zeman regimen, did not slow progression of the disease. Therefore, Santavuori and Westermarck [Santavuori and Westermarck 1984] introduced treatment with a combination of selenium and vitamin E and reported favorable results with few side effects. We present information on the rationale for the use of selenium, recommendations on the daily intake, and reported side effects. However, our limited experience with selenium in this disorder does not permit conclusions. Additionally, careful studies are indicated before this treatment is dispensed routinely.