College of Veterinary, Medical and Life Sciences, University of Glasgow, Glasgow G12 8QQ, UK.
Cell Signal. 2022 Nov;99:110446. doi: 10.1016/j.cellsig.2022.110446. Epub 2022 Aug 19.
The use of small molecule drugs to inhibit active protein targets has revolutionised the treatment options for many diseases in the past 30 years. The greatly improved pharmacokinetic properties of modern drugs combined with enhanced cell permeability and oral bioavailability has made these molecules ideal for reaching protein targets of interest in cells and inhibiting disease-driven signalling pathways. However, these small molecule drugs have several limitations which have opened the doors for the development of a new class of compounds, known as proteolysis targeting chimeras (PROTACs). These next generation drugs actively and specifically degrade designated protein targets and hold the potential to greatly expand the druggable genome, including previously drug-resistant targets.
在过去的 30 年中,小分子药物抑制活性蛋白靶标在许多疾病的治疗选择上带来了革命性的变化。现代药物大大改善的药代动力学特性,加上增强的细胞通透性和口服生物利用度,使这些分子成为到达细胞中感兴趣的蛋白靶标并抑制疾病驱动的信号通路的理想选择。然而,这些小分子药物存在一些局限性,这为开发一类新的化合物开辟了道路,这类化合物被称为蛋白水解靶向嵌合体(PROTACs)。这些新一代药物可主动且特异性地降解指定的蛋白靶标,并有可能极大地扩展可药物治疗的基因组,包括以前耐药的靶标。