Division of Endocrinology, Diabetes, and Metabolism, Department of Medicine, University of Alabama at Birmingham, Birmingham, AL.
UAB Comprehensive Diabetes Center, University of Alabama at Birmingham, Birmingham, AL.
Diabetes. 2020 Sep;69(9):2003-2016. doi: 10.2337/db19-1001. Epub 2020 Jul 1.
Targeting retinoid X receptor (RXR) has been proposed as one of the therapeutic strategies to treat individuals with metabolic syndrome, as RXR heterodimerizes with multiple nuclear receptors that regulate genes involved in metabolism. Despite numerous efforts, RXR ligands (rexinoids) have not been approved for clinical trials to treat metabolic syndrome due to the serious side effects such as hypertriglyceridemia and altered thyroid hormone axis. In this study, we demonstrate a novel rexinoid-like small molecule, UAB126, which has positive effects on metabolic syndrome without the known side effects of potent rexinoids. Oral administration of UAB126 ameliorated obesity, insulin resistance, hepatic steatosis, and hyperlipidemia without changes in food intake, physical activity, and thyroid hormone levels. RNA-sequencing analysis revealed that UAB126 regulates the expression of genes in the liver that are modulated by several nuclear receptors, including peroxisome proliferator-activated receptor α and/or liver X receptor in conjunction with RXR. Furthermore, UAB126 not only prevented but also reversed obesity-associated metabolic disorders. The results suggest that optimized modulation of RXR may be a promising strategy to treat metabolic disorders without side effects. Thus, the current study reveals that UAB126 could be an attractive therapy to treat individuals with obesity and its comorbidities.
靶向视黄醇 X 受体 (RXR) 已被提议作为治疗代谢综合征患者的一种治疗策略,因为 RXR 与多种核受体形成异二聚体,这些核受体调节参与代谢的基因。尽管进行了大量努力,但由于严重的副作用,如高甘油三酯血症和甲状腺激素轴改变,RXR 配体(类维甲酸)尚未被批准用于治疗代谢综合征的临床试验。在这项研究中,我们证明了一种新型的类维甲酸小分子 UAB126,它对代谢综合征有积极作用,而没有已知的强效类维甲酸的副作用。UAB126 的口服给药改善了肥胖、胰岛素抵抗、肝脂肪变性和高血脂,而食物摄入、体力活动和甲状腺激素水平没有变化。RNA 测序分析显示,UAB126 调节了肝脏中受几种核受体(包括过氧化物酶体增殖物激活受体 α 和/或肝 X 受体)调节的基因的表达,与 RXR 结合。此外,UAB126 不仅预防而且逆转了与肥胖相关的代谢紊乱。结果表明,优化 RXR 的调节可能是一种治疗代谢紊乱而无副作用的有前途的策略。因此,本研究表明 UAB126 可能是治疗肥胖及其合并症患者的一种有吸引力的疗法。