Department of Pediatric Nephrology, University Hospitals Leuven, Leuven, Belgium.
Drug Discov Today. 2013 Aug;18(15-16):785-92. doi: 10.1016/j.drudis.2013.02.003. Epub 2013 Feb 14.
Cysteamine is an amino thiol with the chemical formula HSCH2CH2NH2. Endogenously, cysteamine is derived from coenzyme A degradation, although its plasma concentrations are low. Most experience with cysteamine as a drug originates from the field of the orphan disease cystinosis, in which cysteamine is prescribed to decrease intralysosomal cystine accumulation. However, over the years, the drug has been used for several other applications both in vitro and in vivo. In this article, we review the different applications of cysteamine, ending with an overview of ongoing clinical trials for new indications, such as neurodegenerative disorders and nonalcoholic fatty liver disease (NAFLD). The recent development of an enteric-coated cysteamine formulation makes cysteamine more patient friendly and will extend its applicability for both old and new indications.
半胱胺是一种化学公式为 HSCH2CH2NH2 的含硫氨基酸。内源性的半胱胺来源于辅酶 A 的降解，尽管其在血浆中的浓度较低。半胱胺作为一种药物的大多数经验都来自于孤儿病胱氨酸症的领域，在该领域中，半胱胺被开处方以减少溶酶体腔内胱氨酸的积累。然而，多年来，该药物已在体外和体内用于其他几种应用。在本文中，我们综述了半胱胺的不同应用，最后概述了正在进行的临床试验，以确定新的适应症，如神经退行性疾病和非酒精性脂肪性肝病（NAFLD）。最近开发的一种肠包衣半胱胺制剂使半胱胺对患者更友好，并将扩大其在新旧适应症中的适用性。
