Laboratório de Síntese Orgânica Medicinal - LaSOM, Faculdade de Farmácia, Universidade Federal do Rio Grande do Sul, Av. Ipiranga 2752, Porto Alegre, RS, 90610-000, Brazil.
Faculdade de Ciências Farmacêuticas de Ribeirão Preto, Universidade de São Paulo, Av. do Café s/n, Ribeirão Preto, SP, 14040-903, Brazil.
Mol Divers. 2017 Aug;21(3):677-695. doi: 10.1007/s11030-017-9745-8. Epub 2017 May 18.
Malaria, a tropical parasitic disease caused by Plasmodium spp., continues to place a heavy social burden, with almost 200 million cases and more than 580,000 deaths per year. Plasmodium falciparum purine nucleoside phosphorylase (PfPNP) can be targeted for antimalarial drug design since its inhibition kills malaria parasites both in vitro and in vivo. Although the currently known inhibitors of PfPNP, immucillins, are orally available and of low toxicity to animals and humans, to the best of our knowledge, none of these compounds has entered clinical trials for the treatment of malaria. Using a pharmacophore-based virtual screening coupled to a consensual molecular docking approach, we identified 59 potential PfPNP inhibitors that are predicted to be orally absorbed in a Caco-2 cell model. Although most of these compounds are predicted to have high plasma protein binding levels, poor water solubility (except for compound 25) and CYP3A4 metabolic stability (except for 4, 7 and 8), four structures (4, 7, 8 and 25) remain as potential leads because of their plausible interaction with a specific hydrophobic pocket of PfPNP, which would confer them higher selectivity for PfPNP over human PNP. Additionally, both predicted Gibbs free energies for binding and molecular dynamics suggest that compound 4 may form a more stable complex with PfPNP than 5[Formula: see text]-methylthio-immucillin-H, a potent and selective inhibitor of PfPNP.
疟疾是一种由疟原虫属引起的热带寄生虫病,每年仍有近 2 亿例病例和超过 58 万人死亡,给社会带来沉重负担。疟原虫嘌呤核苷磷酸化酶(PfPNP)可以作为抗疟药物设计的靶点,因为其抑制剂在体外和体内都能杀死疟原虫。虽然目前已知的 PfPNP 抑制剂——免疫霉素类化合物具有口服可用性,且对动物和人类的毒性较低,但据我们所知,这些化合物都没有进入治疗疟疾的临床试验。本研究采用基于药效团的虚拟筛选与共识分子对接方法,鉴定出 59 种潜在的 PfPNP 抑制剂,这些抑制剂在 Caco-2 细胞模型中预测具有口服吸收性。尽管这些化合物中的大多数预测具有高血浆蛋白结合水平、较差的水溶性(除了化合物 25 外)和 CYP3A4 代谢稳定性(除了 4、7 和 8 外),但由于它们与 PfPNP 特定的疏水性口袋具有合理的相互作用,因此它们可能对 PfPNP 具有更高的选择性,而对人 PNP 的选择性较低,因此仍有 4、7、8 和 25 这四种结构作为潜在的先导化合物。此外,预测的结合吉布斯自由能和分子动力学都表明,与 5[Formula: see text]-甲基硫代-免疫霉素-H(一种强效和选择性的 PfPNP 抑制剂)相比,化合物 4 可能与 PfPNP 形成更稳定的复合物。