The translational challenge in Chagas disease drug development

Chagas disease is a neglected tropical disease caused by the protozoan parasite Trypanosoma cruzi. There is an urgent need for safe, effective, and accessible new treatments since the currently approved drugs have serious limitations. Drug development for Chagas disease has historically been hampered by the complexity of the disease, critical knowledge gaps, and lack of coordinated R&D efforts. This review covers some of the translational challenges associated with the progression of new chemical entities from preclinical to clinical phases of development, and discusses how recent technological advances might allow the research community to answer key questions relevant to the disease and to overcome hurdles in R&D for Chagas disease.

恰加斯病（Chagas disease）是由原生动物寄生虫克氏锥虫（Trypanosoma cruzi）引发的一种被忽视的热带病。当前获批的治疗药物存在严重局限性，因此亟需开发安全、有效且可及的新型治疗方案。长期以来，恰加斯病的药物研发受限于该疾病的复杂性、关键知识空白以及缺乏协调一致的研发（R&D）工作。本综述探讨了新化学实体从临床前研发阶段推进至临床研发阶段所面临的转化挑战，并讨论了近年来的技术进步如何助力科研界解答与该疾病相关的核心问题，同时攻克恰加斯病研发过程中的各类障碍。