Penn State Milton S. Hershey Medical Center and Cancer Institute, 500 University Drive CH046, Office T4419, Hershey, PA 17033, USA.
Mol Cancer Ther. 2012 Nov;11(11):2321-30. doi: 10.1158/1535-7163.MCT-12-0578. Epub 2012 Sep 10.
Despite significant treatment advances over the past decade, multiple myeloma (MM) remains largely incurable. In this study we found that MM cells were remarkably sensitive to the death-inducing effects of a new class of sangivamycin-like molecules (SLM). A panel of structurally related SLMs selectively induced apoptosis in MM cells but not other tumor or nonmalignant cell lines at submicromolar concentrations. SLM6 was the most active compound in vivo, where it was well tolerated and significantly inhibited growth and induced apoptosis of MM tumors. We determined that the anti-MM activity of SLM6 was mediated by direct inhibition of cyclin-dependent kinase 9 (CDK9), which resulted in transcriptional repression of oncogenes that are known to drive MM progression (MAF, CCND1, MYC, and others). Furthermore, SLM6 showed superior in vivo anti-MM activity more than the CDK inhibitor flavopiridol, which is currently in clinical trials for MM. These findings show that SLM6 is a novel CDK9 inhibitor with promising preclinical activity as an anti-MM agent.
尽管在过去十年中取得了重大的治疗进展,但多发性骨髓瘤(MM)仍然基本无法治愈。在这项研究中,我们发现 MM 细胞对一类新型桑吉瓦霉素样分子(SLM)的诱导细胞死亡效应非常敏感。一组结构相关的 SLM 可在亚微摩尔浓度下选择性地诱导 MM 细胞凋亡,但不诱导其他肿瘤或非恶性细胞系凋亡。SLM6 是体内最活跃的化合物,它具有良好的耐受性,可显著抑制 MM 肿瘤的生长并诱导其凋亡。我们确定 SLM6 的抗 MM 活性是通过直接抑制细胞周期蛋白依赖性激酶 9(CDK9)介导的,这导致已知驱动 MM 进展的癌基因转录抑制(MAF、CCND1、MYC 等)。此外,SLM6 在体内显示出优于 CDK 抑制剂 flavopiridol 的抗 MM 活性,后者目前正在临床试验中用于 MM。这些发现表明 SLM6 是一种新型 CDK9 抑制剂,具有作为抗 MM 药物的有前途的临床前活性。