Hess Jeannine, Panic Gordana, Patra Malay, Mastrobuoni Luciano, Spingler Bernhard, Roy Saonli, Keiser Jennifer, Gasser Gilles
Department of Chemistry, University of Zurich , Winterthurerstrasse 190, CH-8057 Zurich, Switzerland.
Department of Medical Parasitology and Infection Biology, Swiss Tropical and Public Health Institute , Basel, Switzerland.
ACS Infect Dis. 2017 Sep 8;3(9):645-652. doi: 10.1021/acsinfecdis.7b00054. Epub 2017 Jul 18.
Schistosomiasis is a parasitic disease that affects more than 250 million people annually, mostly children in poor, tropical, rural areas. Only one treatment (praziquantel) is available, putting control efforts at risk should resistance occur. In pursuit of treatment alternatives, we derivatized an old antischistosomal agent, oxamniquine (OXA). Four organometallic derivatives of OXA were synthesized and tested against Schistosoma mansoni in vitro and in vivo. Of these, a ferrocenyl derivative, 1, killed larval and adult worms 24 h postexposure in vitro, in contrast to OXA, which lacks in vitro activity against adult worms. A dose of 200 mg/kg of 1 completely eliminated the worm burden in mice. Subsequently, a ruthenocenyl (5) and a benzyl derivative (6) of OXA were synthesized to probe the importance of the ferrocenyl group in 1. Compounds 1, 5, and 6 were lethal to both S. mansoni and S. haematobium adults in vitro. In vivo, at 100 mg/kg, all three compounds revealed S. mansoni worm burden reductions of 76 to 93%, commensurate with OXA. Our findings present three compounds with activity against S. mansoni in vitro, comparable activity in vivo, and high activity against S. haematobium in vitro. These compounds may possess a different binding mode or mode of action compared to OXA and present excellent starting points for further SAR studies.
血吸虫病是一种寄生虫病,每年影响超过2.5亿人,主要是贫困热带农村地区的儿童。目前仅有一种治疗药物(吡喹酮),一旦出现耐药性,控制工作将面临风险。为了寻找替代治疗方法,我们对一种古老的抗血吸虫药物奥沙尼喹(OXA)进行了衍生化。合成了四种OXA的有机金属衍生物,并在体外和体内对曼氏血吸虫进行了测试。其中,一种二茂铁基衍生物1在体外暴露24小时后可杀死幼虫和成虫,而OXA对成虫缺乏体外活性。200mg/kg的1剂量可完全消除小鼠体内的虫负荷。随后,合成了OXA的钌茂基衍生物(5)和苄基衍生物(6),以探究1中二茂铁基团的重要性。化合物1、5和6在体外对曼氏血吸虫和埃及血吸虫成虫均具有致死性。在体内,100mg/kg时,这三种化合物均使曼氏血吸虫的虫负荷降低了76%至93%,与OXA相当。我们的研究结果表明,有三种化合物在体外对曼氏血吸虫具有活性,在体内具有相当的活性,在体外对埃及血吸虫具有高活性。与OXA相比,这些化合物可能具有不同的结合模式或作用方式,为进一步的构效关系研究提供了极佳的起点。