Institute for Genetic Medicine, University of Southern California, Los Angeles, California 90033, United States.
J Med Chem. 2011 Mar 24;54(6):1640-54. doi: 10.1021/jm101202j. Epub 2011 Feb 22.
The lymphoid tyrosine phosphatase LYP, encoded by the PTPN22 gene, is a critical regulator of signaling in T cells and recently emerged as a candidate target for therapy of autoimmune diseases. Here, by library screening, we identified a series of noncompetitive inhibitors of LYP that showed activity in primary T cells. Kinetic analysis confirmed that binding of the compounds to the phosphatase is nonmutually exclusive with respect to a known bidentate competitive inhibitor. The mechanism of action of the lead inhibitor compound 4e was studied by a combination of hydrogen/deuterium-exchange mass spectrometry and molecular modeling. The results suggest that the inhibitor interacts critically with a hydrophobic patch located outside the active site of the phosphatase. Targeting of secondary allosteric sites is viewed as a promising yet unexplored approach to develop pharmacological inhibitors of protein tyrosine phosphatases. Our novel scaffold could be a starting point to attempt development of "nonactive site" anti-LYP pharmacological agents.
淋巴特异酪氨酸磷酸酶 LYP 由 PTPN22 基因编码,是 T 细胞信号转导的关键调节因子,最近被认为是治疗自身免疫性疾病的候选靶点。在这里,我们通过文库筛选,鉴定出一系列非竞争性 LYP 抑制剂,它们在原代 T 细胞中具有活性。动力学分析证实,化合物与磷酸酶的结合与已知的双齿竞争性抑制剂不相互排斥。通过氢/氘交换质谱和分子建模的组合,研究了先导抑制剂化合物 4e 的作用机制。结果表明,抑制剂与位于磷酸酶活性位点之外的疏水区关键相互作用。靶向二级变构位点被认为是开发蛋白酪氨酸磷酸酶药理学抑制剂的一种有前途但尚未探索的方法。我们的新型支架可以作为尝试开发“非活性位点”抗 LYP 药理制剂的起点。