Dey Isha, Shah Kalpit, Bradbury Neil A
Department of Physiology and Biophysics, Chicago Medical School, North Chicago, Illinois, USA.
J Genet Syndr Gene Ther. 2016 Feb;7(1). doi: 10.4172/2157-7412.1000284. Epub 2016 Jan 30.
The recent FDA approval of two drugs to treat the basic defect in cystic fibrosis has given hope to patients and their families battling this devastating disease. Over many years, with heavy financial investment from Vertex Pharmaceuticals and the Cystic Fibrosis Foundation, pre-clinical evaluation of thousands of synthetic drugs resulted in the production of Kalydeco and Orkambi. Yet, despite the success of this endeavor, many other compounds have been proposed as therapeutic agents in the treatment of CF. Of note, several of these compounds are naturally occurring, and are present in spices from the grocery store and over the counter preparations in health food stores. In this short review, we look at three such compounds, genistein, curcumin, and resveratrol, and evaluate the scientific support for their use as therapeutic agents in the treatment of patients with CF.
美国食品药品监督管理局(FDA)近期批准了两种治疗囊性纤维化基本缺陷的药物,这给与这种毁灭性疾病作斗争的患者及其家人带来了希望。多年来,在Vertex制药公司和囊性纤维化基金会的大量资金投入下,对数千种合成药物进行的临床前评估催生了Kalydeco和Orkambi。然而,尽管这项努力取得了成功,但仍有许多其他化合物被提议作为治疗囊性纤维化的治疗药物。值得注意的是,其中几种化合物是天然存在的,存在于杂货店的香料和健康食品店的非处方制剂中。在这篇简短的综述中,我们研究了三种这样的化合物,即染料木黄酮、姜黄素和白藜芦醇,并评估了它们作为治疗囊性纤维化患者的治疗药物的科学依据。