Department of Pathology, Medicine and Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA 02215, USA.
Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School, Boston, MA 02215, USA.
Sci Signal. 2024 Feb 27;17(825):eadf2670. doi: 10.1126/scisignal.adf2670.
More than 50% of human tumors display hyperactivation of the serine/threonine kinase AKT. Despite evidence of clinical efficacy, the therapeutic window of the current generation of AKT inhibitors could be improved. Here, we report the development of a second-generation AKT degrader, INY-05-040, which outperformed catalytic AKT inhibition with respect to cellular suppression of AKT-dependent phenotypes in breast cancer cell lines. A growth inhibition screen with 288 cancer cell lines confirmed that INY-05-040 had a substantially higher potency than our first-generation AKT degrader (INY-03-041), with both compounds outperforming catalytic AKT inhibition by GDC-0068. Using multiomic profiling and causal network integration in breast cancer cells, we demonstrated that the enhanced efficacy of INY-05-040 was associated with sustained suppression of AKT signaling, which was followed by induction of the stress mitogen-activated protein kinase (MAPK) c-Jun N-terminal kinase (JNK). Further integration of growth inhibition assays with publicly available transcriptomic, proteomic, and reverse phase protein array (RPPA) measurements established low basal JNK signaling as a biomarker for breast cancer sensitivity to AKT degradation. Together, our study presents a framework for mapping the network-wide signaling effects of therapeutically relevant compounds and identifies INY-05-040 as a potent pharmacological suppressor of AKT signaling.
超过 50%的人类肿瘤显示丝氨酸/苏氨酸激酶 AKT 的过度激活。尽管有临床疗效的证据,但当前一代 AKT 抑制剂的治疗窗口可以进一步改善。在这里,我们报告了第二代 AKT 降解剂 INY-05-040 的开发,该降解剂在乳腺癌细胞系中对 AKT 依赖性表型的细胞抑制方面优于催化 AKT 抑制。用 288 种癌细胞系进行的生长抑制筛选证实,与我们的第一代 AKT 降解剂(INY-03-041)相比,INY-05-040 的效力明显更高,这两种化合物的效力都超过了 GDC-0068 的催化 AKT 抑制。通过多组学分析和乳腺癌细胞中的因果网络整合,我们证明了 INY-05-040 增强疗效与 AKT 信号的持续抑制有关,随后诱导应激丝裂原激活蛋白激酶(MAPK)c-Jun N-末端激酶(JNK)。将生长抑制测定与公开的转录组、蛋白质组和反向相蛋白阵列(RPPA)测量进一步整合,确立了低基础 JNK 信号作为 AKT 降解对乳腺癌敏感性的生物标志物。总之,我们的研究提出了一个框架,用于绘制治疗相关化合物的网络范围信号效应图谱,并将 INY-05-040 鉴定为 AKT 信号的有效药理学抑制剂。