Deciphera Pharmaceuticals, Inc., Waltham, MA 02451, USA.
Emerald Biostructures, Bainbridge Island, WA 98110, USA.
Cancer Cell. 2019 May 13;35(5):738-751.e9. doi: 10.1016/j.ccell.2019.04.006.
Ripretinib (DCC-2618) was designed to inhibit the full spectrum of mutant KIT and PDGFRA kinases found in cancers and myeloproliferative neoplasms, particularly in gastrointestinal stromal tumors (GISTs), in which the heterogeneity of drug-resistant KIT mutations is a major challenge. Ripretinib is a "switch-control" kinase inhibitor that forces the activation loop (or activation "switch") into an inactive conformation. Ripretinib inhibits all tested KIT and PDGFRA mutants, and notably is a type II kinase inhibitor demonstrated to broadly inhibit activation loop mutations in KIT and PDGFRA, previously thought only achievable with type I inhibitors. Ripretinib shows efficacy in preclinical cancer models, and preliminary clinical data provide proof-of-concept that ripretinib inhibits a wide range of KIT mutants in patients with drug-resistant GISTs.
瑞普替尼(DCC-2618)旨在抑制癌症和骨髓增生性肿瘤中发现的突变型 KIT 和 PDGFRA 激酶的全谱,特别是在胃肠道间质瘤(GIST)中,药物耐药性 KIT 突变的异质性是一个主要挑战。瑞普替尼是一种“开关控制”激酶抑制剂,它迫使激活环(或激活“开关”)进入非活性构象。瑞普替尼抑制所有测试的 KIT 和 PDGFRA 突变体,特别是作为一种 II 型激酶抑制剂,广泛抑制 KIT 和 PDGFRA 的激活环突变,以前认为只有 I 型抑制剂才能实现。瑞普替尼在临床前癌症模型中显示出疗效,初步临床数据提供了概念验证,表明瑞普替尼抑制耐药性 GIST 患者广泛的 KIT 突变体。