Department of Molecular Biology, University of Gdańsk, Kładki 24, 80-822 Gdańsk, Poland.
Phytother Res. 2010 Jan;24 Suppl 1:S109-13. doi: 10.1002/ptr.2944.
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.
异黄酮是一类植物来源的、具有生物活性的化合物,通常被用作治疗更年期症状的天然药物或饮食补充剂,也被用作抗氧化剂。最近,有人提出染料木黄酮(4',5,7-三羟基异黄酮)可能可用于治疗黏多糖贮积症 III 型(Sanfilippo 病)患者,这种严重的遗传疾病目前尚无有效的治疗方法。一项使用这种新型疗法(称为“基因表达靶向异黄酮疗法”)的初步临床试验表明,标准化的富含染料木苷的大豆异黄酮提取物对这类患者的治疗是有效的。由于市面上有各种含有异黄酮的产品,因此对这些产品中的主要异黄酮含量进行了测量。结果发现,不同产品中的异黄酮含量差异极大,每片从 0.13 毫克到 39 毫克不等。只有部分产品在培养的成纤维细胞中被发现可有效抑制黏多糖贮积症(包括 Sanfilippo 病)患者中糖胺聚糖的合成(这类化合物的降解在黏多糖贮积症中受到严重损害)。由于在 GET IT 中,染料木黄酮的剂量是根据每个患者的体重计算的,因此每片这种异黄酮的含量对这种疗法至关重要。因此,本报告中的结果表明,为了 GET IT 的顺利实施,必须仔细选择合适的异黄酮提取物。