Department of Microbiology, Tumor and Cell Biology and ‡Division of Translational Medicine and Chemical Biology, Department of Medical Biochemistry and Biophysics, Karolinska Institutet , 171 77 Stockholm, Sweden.
J Med Chem. 2013 Dec 27;56(24):9861-73. doi: 10.1021/jm401530a. Epub 2013 Dec 10.
Novel methods for treatment of African trypanosomiasis, caused by infection with Trypanosoma brucei are needed. Cordycepin (3'-deoxyadenosine, 1a) is a powerful trypanocidal compound in vitro but is ineffective in vivo because of rapid metabolic degradation by adenosine deaminase (ADA). We elucidated the structural moieties of cordycepin required for trypanocidal activity and designed analogues that retained trypanotoxicity while gaining resistance to ADA-mediated metabolism. 2-Fluorocordycepin (2-fluoro-3'-deoxyadenosine, 1b) was identified as a selective, potent, and ADA-resistant trypanocidal compound that cured T. brucei infection in mice. Compound 1b is transported through the high affinity TbAT1/P2 adenosine transporter and is a substrate of T. b. brucei adenosine kinase. 1b has good preclinical properties suitable for an oral drug, albeit a relatively short plasma half-life. We present a rapid and efficient synthesis of 2-halogenated cordycepins, also useful synthons for the development of additional novel C2-substituted 3'-deoxyadenosine analogues to be evaluated in development of experimental therapeutics.
需要开发新型方法来治疗由感染布氏锥虫引起的非洲锥虫病。在体外,虫草素(3'-脱氧腺苷,1a)是一种强大的杀锥虫化合物,但由于腺苷脱氨酶(ADA)的快速代谢降解,在体内无效。我们阐明了虫草素发挥杀锥虫活性所需的结构部分,并设计了保留对 ADA 介导的代谢具有毒性的类似物。2-氟虫草素(2-氟-3'-脱氧腺苷,1b)被鉴定为一种选择性、有效且对 ADA 具有抗性的杀锥虫化合物,可治愈感染小鼠的布氏锥虫。化合物 1b 通过高亲和力 TbAT1/P2 腺苷转运蛋白转运,是 T. b. brucei 腺苷激酶的底物。1b 具有良好的临床前特性,适合口服药物,尽管其血浆半衰期相对较短。我们提出了一种快速有效的 2-卤代虫草素合成方法,该方法也可用于开发其他新型 C2-取代 3'-脱氧腺苷类似物,以评估在实验治疗学中的开发。