Tempero Pharmaceuticals, Cambridge, Massachusetts, USA.
Nat Chem Biol. 2013 May;9(5):319-25. doi: 10.1038/nchembio.1223. Epub 2013 Mar 24.
In contrast to studies on class I histone deacetylase (HDAC) inhibitors, the elucidation of the molecular mechanisms and therapeutic potential of class IIa HDACs (HDAC4, HDAC5, HDAC7 and HDAC9) is impaired by the lack of potent and selective chemical probes. Here we report the discovery of inhibitors that fill this void with an unprecedented metal-binding group, trifluoromethyloxadiazole (TFMO), which circumvents the selectivity and pharmacologic liabilities of hydroxamates. We confirm direct metal binding of the TFMO through crystallographic approaches and use chemoproteomics to demonstrate the superior selectivity of the TFMO series relative to a hydroxamate-substituted analog. We further apply these tool compounds to reveal gene regulation dependent on the catalytic active site of class IIa HDACs. The discovery of these inhibitors challenges the design process for targeting metalloenzymes through a chelating metal-binding group and suggests therapeutic potential for class IIa HDAC enzyme blockers distinct in mechanism and application compared to current HDAC inhibitors.
与 I 类组蛋白去乙酰化酶(HDAC)抑制剂的研究相反,由于缺乏强效和选择性的化学探针,阐明 IIa 类 HDAC(HDAC4、HDAC5、HDAC7 和 HDAC9)的分子机制和治疗潜力受到阻碍。在这里,我们报告了一种抑制剂的发现,该抑制剂使用前所未有的三氟甲氧基噁二唑(TFMO)作为金属结合基团,填补了这一空白,克服了羟肟酸类化合物的选择性和药理学缺陷。我们通过晶体学方法证实了 TFMO 的直接金属结合,并利用化学蛋白质组学证明了 TFMO 系列相对于取代羟肟酸的类似物具有更高的选择性。我们进一步应用这些工具化合物来揭示依赖于 IIa 类 HDAC 催化活性位点的基因调控。这些抑制剂的发现挑战了通过螯合金属结合基团靶向金属酶的设计过程,并表明与当前的 HDAC 抑制剂相比,IIa 类 HDAC 酶抑制剂在机制和应用方面具有治疗潜力。