The Walter and Eliza Hall Institute of Medical Research, Parkville, VIC 3052, Australia; University of Melbourne, Melbourne, VIC 3010, Australia.
Merck & Co., Inc., 770 Sumneytown Pike, West Point, PA 19486, USA.
Cell Host Microbe. 2020 Apr 8;27(4):642-658.e12. doi: 10.1016/j.chom.2020.02.005. Epub 2020 Feb 27.
Artemisin combination therapy (ACT) is the main treatment option for malaria, which is caused by the intracellular parasite Plasmodium. However, increased resistance to ACT highlights the importance of finding new drugs. Recently, the aspartic proteases Plasmepsin IX and X (PMIX and PMX) were identified as promising drug targets. In this study, we describe dual inhibitors of PMIX and PMX, including WM382, that block multiple stages of the Plasmodium life cycle. We demonstrate that PMX is a master modulator of merozoite invasion and direct maturation of proteins required for invasion, parasite development, and egress. Oral administration of WM382 cured mice of P. berghei and prevented blood infection from the liver. In addition, WM382 was efficacious against P. falciparum asexual infection in humanized mice and prevented transmission to mosquitoes. Selection of resistant P. falciparum in vitro was not achievable. Together, these show that dual PMIX and PMX inhibitors are promising candidates for malaria treatment and prevention.
青蒿素联合疗法(ACT)是治疗疟疾的主要方法,疟疾是由细胞内寄生虫疟原虫引起的。然而,ACT 耐药性的增加凸显了寻找新药的重要性。最近,天冬氨酸蛋白酶 Plasmepsin IX 和 X(PMIX 和 PMX)被确定为有前途的药物靶点。在这项研究中,我们描述了 PMIX 和 PMX 的双重抑制剂,包括 WM382,它可以阻断疟原虫生命周期的多个阶段。我们证明 PMX 是裂殖子入侵的主要调节剂,并直接调节入侵、寄生虫发育和逸出所需的蛋白质成熟。WM382 的口服给药可治愈 P.berghei 感染的小鼠,并防止肝脏的血液感染。此外,WM382 对人源化小鼠的恶性疟原虫无性感染有效,并防止向蚊子传播。在体外选择耐药性疟原虫是不可行的。综上所述,这些结果表明,双重 PMIX 和 PMX 抑制剂是治疗和预防疟疾的有前途的候选药物。