临床阶段抗癌小分子 ONC201 的类似物 imipridone 家族的临床前评估显示 ONC212 具有强大的抗癌作用。
Preclinical evaluation of the imipridone family, analogs of clinical stage anti-cancer small molecule ONC201, reveals potent anti-cancer effects of ONC212.
机构信息
a Laboratory of Translational Oncology and Experimental Cancer Therapeutics, Molecular Therapeutics Program, Department of Hematology/Oncology, Fox Chase Cancer Center , Philadelphia , PA , USA.
b Provid Pharmaceuticals, Monmouth Junction , NJ , USA.
出版信息
Cell Cycle. 2017 Oct 2;16(19):1790-1799. doi: 10.1080/15384101.2017.1325046. Epub 2017 May 10.
Anti-cancer small molecule ONC201 upregulates the integrated stress response (ISR) and acts as a dual inactivator of Akt/ERK, leading to TRAIL gene activation. ONC201 is under investigation in multiple clinical trials to treat patients with cancer. Given the unique imipridone core chemical structure of ONC201, we synthesized a series of analogs to identify additional compounds with distinct therapeutic properties. Several imipridones with a broad range of in vitro potencies were identified in an exploration of chemical derivatives. Based on in vitro potency in human cancer cell lines and lack of toxicity to normal human fibroblasts, imipridones ONC206 and ONC212 were prioritized for further study. Both analogs inhibited colony formation, and induced apoptosis and downstream signaling that involves the integrated stress response and Akt/ERK, similar to ONC201. Compared to ONC201, ONC206 demonstrated improved inhibition of cell migration while ONC212 exhibited rapid kinetics of activity. ONC212 was further tested in >1000 human cancer cell lines in vitro and evaluated for safety and anti-tumor efficacy in vivo. ONC212 exhibited broad-spectrum efficacy at nanomolar concentrations across solid tumors and hematological malignancies. Skin cancer emerged as a tumor type with improved efficacy relative to ONC201. Orally administered ONC212 displayed potent anti-tumor effects in vivo, a broad therapeutic window and a favorable PK profile. ONC212 was efficacious in vivo in BRAF V600E melanoma models that are less sensitive to ONC201. Based on these findings, ONC212 warrants further development as a drug candidate. It is clear that therapeutic utility extends beyond ONC201 to include additional imipridones.
抗癌小分子 ONC201 上调整合应激反应(ISR),并充当 Akt/ERK 的双重失活剂,导致 TRAIL 基因激活。ONC201 正在多项临床试验中被用于治疗癌症患者。鉴于 ONC201 独特的 imipridone 核心化学结构,我们合成了一系列类似物,以鉴定具有独特治疗特性的其他化合物。在对化学衍生物的探索中,确定了一系列具有广泛体外效力的 imipridones。基于在人类癌细胞系中的体外效力和对正常人类成纤维细胞的毒性缺失,imipridones ONC206 和 ONC212 被优先用于进一步研究。这两种类似物均抑制集落形成,并诱导涉及整合应激反应和 Akt/ERK 的细胞凋亡和下游信号传导,与 ONC201 相似。与 ONC201 相比,ONC206 显示出对细胞迁移的抑制作用得到改善,而 ONC212 则表现出快速的活性动力学。ONC212 进一步在 >1000 种人类癌细胞系中进行了测试,并在体内评估了其安全性和抗肿瘤功效。ONC212 在纳米摩尔浓度下对实体瘤和血液恶性肿瘤表现出广谱疗效。与 ONC201 相比,皮肤癌的疗效有所提高。口服给予 ONC212 在体内显示出强大的抗肿瘤作用、广泛的治疗窗口和良好的 PK 特征。ONC212 在对 ONC201 敏感性较低的 BRAF V600E 黑色素瘤模型中具有体内疗效。基于这些发现,ONC212 值得进一步开发为候选药物。很明显,治疗效用不仅限于 ONC201,还包括其他 imipridones。