Department of Pharmaceutical Sciences, College of Pharmacy, University of Illinois at Chicago, Chicago, IL, USA.
Department of Microbial Pathogens and Immunity, Rush University Medical Center, Chicago, IL, USA.
Nat Commun. 2023 Jun 22;14(1):3737. doi: 10.1038/s41467-023-39444-y.
Only praziquantel is available for treating schistosomiasis, a disease affecting more than 200 million people. Praziquantel-resistant worms have been selected for in the lab and low cure rates from mass drug administration programs suggest that resistance is evolving in the field. Thioredoxin glutathione reductase (TGR) is essential for schistosome survival and a validated drug target. TGR inhibitors identified to date are irreversible and/or covalent inhibitors with unacceptable off-target effects. In this work, we identify noncovalent TGR inhibitors with efficacy against schistosome infections in mice, meeting the criteria for lead progression indicated by WHO. Comparisons with previous in vivo studies with praziquantel suggests that these inhibitors outperform the drug of choice for schistosomiasis against juvenile worms.
目前仅有吡喹酮可用于治疗血吸虫病,这种疾病影响着超过 2 亿人。在实验室中已经选择出了对吡喹酮具有抗药性的蠕虫,大规模药物管理项目中的低治愈率表明,这种抗药性正在野外进化。硫氧还蛋白-谷胱甘肽还原酶(TGR)对血吸虫的生存至关重要,是一个经过验证的药物靶点。迄今为止发现的 TGR 抑制剂是非共价的、不可逆的和/或具有不可接受的脱靶效应的共价抑制剂。在这项工作中,我们鉴定了对小鼠血吸虫感染具有疗效的非共价 TGR 抑制剂,符合世界卫生组织(WHO)所指出的先导化合物进展标准。与之前用吡喹酮进行的体内研究相比,这些抑制剂在治疗血吸虫病的效果上优于首选药物。