Genomics Vault: A framework for precision medicine data management

Bhupinder Bhullar, Guy Gross, Hakan Akozek
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Abstract

A mixture of fumaric acid esters (FAEs) is approved for the oral therapy of psoriasis. However, for a long time the active ingredient of this mixture was unknown. We reviewed the in vitro data available for the different FAEs present in the multi compound drug and elaborate how they may contribute to possible clinical effects. Although helpful overall, many in vitro data must be viewed critically because the concentrations used in the experiments exceed the plasma levels reached in patients. The data suggest that dimethylfumarate (DMF) is the most active compound, mediating the major therapeutic effect after metabolization into monomethylfumarate (MMF) via an according receptor expressed on target cells. Identifying the active pharmaceutical ingredient within a mixture of compounds helps to subsequently eliminate unnecessary, potentially harmful compounds. This provides a promising example for an alternative precision medicine approach in clinical practice.
基因组库:精准医疗数据管理的框架
富马酸酯(FAEs)的混合物被批准用于口服治疗牛皮癣。然而,很长一段时间,这种混合物的有效成分是未知的。我们回顾了多种复合药物中存在的不同FAEs的体外数据,并详细说明了它们如何有助于可能的临床效果。虽然总体上是有帮助的,但许多体外数据必须谨慎看待,因为实验中使用的浓度超过了患者的血浆水平。这些数据表明富马酸二甲酯(DMF)是最活跃的化合物,通过靶细胞上表达的相应受体代谢为富马酸一甲酯(MMF)后介导主要的治疗作用。确定化合物混合物中的活性药物成分有助于随后消除不必要的、潜在有害的化合物。这为临床实践中的替代精准医学方法提供了一个有希望的例子。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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