B-cell lymphoma extra large (BCL-XL) is a well-validated cancer target. However, the on-target and dose-limiting thrombocytopenia limits the use of BCL-XL inhibitors, such as ABT263, as safe and effective anticancer agents. To reduce the toxicity of ABT263, we converted it into DT2216, a BCL-XL proteolysis-targeting chimera (PROTAC), that targets BCL-XL to the Von Hippel-Lindau (VHL) E3 ligase for degradation. We found that DT2216 was more potent against various BCL-XL-dependent leukemia and cancer cells but considerably less toxic to platelets than ABT263 in vitro because VHL is poorly expressed in platelets. In vivo, DT2216 effectively inhibits the growth of several xenograft tumors as a single agent or in combination with other chemotherapeutic agents, without causing appreciable thrombocytopenia. These findings demonstrate the potential to use PROTAC technology to reduce on-target drug toxicities and rescue the therapeutic potential of previously undruggable targets. Furthermore, DT2216 may be developed as a safe first-in-class anticancer agent targeting BCL-XL.

译文

b细胞淋巴瘤特大 (BCL-XL) 是一个经过充分验证的癌症靶标。然而,目标和剂量限制性血小板减少症限制了BCL-XL抑制剂 (如ABT263) 作为安全有效的抗癌药物的使用。为了降低ABT263的毒性,我们将其转化为DT2216,一种BCL-XL蛋白水解靶向嵌合体 (PROTAC),该嵌合体将BCL-XL靶向Von Hippel-Lindau (VHL) E3连接酶降解。我们发现DT2216在体外对各种BCL-XL依赖性白血病和癌细胞更有效,但对血小板的毒性比ABT263小得多,因为VHL在血小板中表达不佳。在体内,DT2216作为单一药物或与其他化学治疗剂组合有效抑制几种异种移植瘤的生长,而不会引起明显的血小板减少症。这些发现证明了使用PROTAC技术降低靶向药物毒性和挽救先前无法治疗的靶标的治疗潜力的潜力。此外,DT2216可以被开发为靶向BCL-XL的安全的一流抗癌剂。

+1
+2
100研值 100研值 ¥99课程
检索文献一次
下载文献一次

去下载>

成功解锁2个技能,为你点赞

《SCI写作十大必备语法》
解决你的SCI语法难题!

技能熟练度+1

视频课《玩转文献检索》
让你成为检索达人!

恭喜完成新手挑战

手机微信扫一扫,添加好友领取

免费领《Endnote文献管理工具+教程》

微信扫码, 免费领取

手机登录

获取验证码
登录