Isoflavones are plant-derived, biologically active compounds that are commonly used as natural drugs or diet supplements in the treatment of menopausal symptoms and as
antioxidants. Recently, it was proposed that
genistein (4',5,7-trihydroxyisoflavone) may be used in the treatment of patients suffering from Sanfilippo disease (
mucopolysaccharidosis type III), a severe
genetic disorder for which no
therapy is available. A pilot clinical study with this novel
therapy, called 'gene expression-targeted
isoflavone therapy' (GET IT), indicated that a standardized,
genistin-rich soy
isoflavone extract is effective in the treatment of such patients. Since various
isoflavone-containing products are commercially available, the content of the main
isoflavones were measured in such products. Extremely different amounts of
isoflavones were determined in various products, from 0.13 to 39 mg per
tablet. Only some of these products were found to be effective in inhibition of the synthesis of
glycosaminoglycans (compounds whose degradation is severely impaired in
mucopolysaccharidoses, including Sanfilippo disease) in cultured fibroblasts. Since in GET IT the dose of
genistein is calculated per patient's
body weight, the amount of this
isoflavone in a
tablet is crucial for this
therapy. Therefore, the results presented in this report indicate that a careful choice of a proper
isoflavone extract is necessary for GET IT.