Rawat Mukul, Padalino Gilda, Adika Edem, Okombo John, Yeo Tomas, Brancale Andrea, Fidock David A, Hoffmann Karl F, Lee Marcus C S
Biological Chemistry and Drug Discovery, Wellcome Centre for Anti-Infectives Research, University of Dundee, Dundee, United Kingdom.
Wellcome Sanger Institute, Wellcome Genome Campus, Hinxton, United Kingdom.
PLoS Pathog. 2025 Feb 3;21(2):e1012216. doi: 10.1371/journal.ppat.1012216. eCollection 2025 Feb.
The human pathogens Plasmodium and Schistosoma are each responsible for over 200 million infections annually, especially in low- and middle-income countries. There is a pressing need for new drug targets for these diseases, driven by emergence of drug-resistance in Plasmodium and an overall dearth of drug targets against Schistosoma. Here, we explored the opportunity for pathogen-hopping by evaluating a series of quinoxaline-based anti-schistosomal compounds for their activity against P. falciparum. We identified compounds with low nanomolar potency against 3D7 and multidrug-resistant strains. In vitro resistance selections using wildtype and mutator P. falciparum lines revealed a low propensity for resistance. Only one of the series, compound 22, yielded resistance mutations, including point mutations in a non-essential putative hydrolase pfqrp1, as well as copy number amplification of a phospholipid-translocating ATPase, pfatp2, a potential target. Notably, independently generated CRISPR-edited mutants in pfqrp1 also showed resistance to compound 22 and a related analogue. Moreover, previous lines with pfatp2 copy number variations were similarly less susceptible to challenge with the new compounds. Finally, we examined whether the predicted hydrolase activity of PfQRP1 underlies its mechanism of resistance, showing that both mutation of the putative catalytic triad and a more severe loss of function mutation elicited resistance. Collectively, we describe a compound series with potent activity against two important pathogens and their potential target in P. falciparum.
人类病原体疟原虫和血吸虫每年各自导致超过2亿人感染,尤其是在低收入和中等收入国家。由于疟原虫中耐药性的出现以及针对血吸虫的药物靶点总体匮乏,迫切需要针对这些疾病的新药物靶点。在此,我们通过评估一系列基于喹喔啉的抗血吸虫化合物对恶性疟原虫的活性,探索了跨病原体应用的机会。我们鉴定出了对3D7和多重耐药菌株具有低纳摩尔效力的化合物。使用野生型和突变型恶性疟原虫株进行的体外耐药性筛选显示耐药性倾向较低。该系列中只有一种化合物22产生了耐药性突变,包括在一个非必需的假定水解酶pfqrp1中的点突变,以及一种潜在靶点磷脂转运ATP酶pfatp2的拷贝数扩增。值得注意的是,在pfqrp1中独立产生的CRISPR编辑突变体也对化合物22和一种相关类似物表现出耐药性。此外,先前具有pfatp2拷贝数变异的株系对新化合物的挑战同样不太敏感。最后,我们研究了PfQRP1预测的水解酶活性是否是其耐药机制的基础,结果表明假定催化三联体的突变和更严重的功能丧失突变均引发了耐药性。我们共同描述了一个对两种重要病原体及其在恶性疟原虫中的潜在靶点具有强效活性的化合物系列。