Chatelain Eric, Ioset Jean-Robert
Drugs for Neglected Diseases Initiative (DNDi), Geneva, Switzerland.
Drug Des Devel Ther. 2011 Mar 16;5:175-81. doi: 10.2147/DDDT.S16381.
New models of drug discovery have been developed to overcome the lack of modern and effective drugs for neglected diseases such as human African trypanosomiasis (HAT; sleeping sickness), leishmaniasis, and Chagas disease, which have no financial viability for the pharmaceutical industry. With the purpose of combining the skills and research capacity in academia, pharmaceutical industry, and contract researchers, public-private partnerships or product development partnerships aim to create focused research consortia that address all aspects of drug discovery and development. These consortia not only emulate the projects within pharmaceutical and biotechnology industries, eg, identification and screening of libraries, medicinal chemistry, pharmacology and pharmacodynamics, formulation development, and manufacturing, but also use and strengthen existing capacity in disease-endemic countries, particularly for the conduct of clinical trials. The Drugs for Neglected Diseases initiative (DNDi) has adopted a model closely related to that of a virtual biotechnology company for the identification and optimization of drug leads. The application of this model to the development of drug candidates for the kinetoplastid infections of HAT, Chagas disease, and leishmaniasis has already led to the identification of new candidates issued from DNDi's own discovery pipeline. This demonstrates that the model DNDi has been implementing is working but its DNDi, neglected diseases sustainability remains to be proven.
为克服针对被忽视疾病(如人类非洲锥虫病(昏睡病)、利什曼病和恰加斯病)缺乏现代有效药物的问题,已开发出新型药物研发模式,这些疾病对制药行业而言缺乏经济可行性。公私合营或产品开发合作旨在整合学术界、制药行业和合同研究机构的技能与研究能力,组建专注于药物研发各个方面的研究联盟。这些联盟不仅效仿制药和生物技术行业内的项目,如文库的识别与筛选、药物化学、药理学和药效学、制剂开发及生产,还利用并加强疾病流行国家的现有能力,尤其是开展临床试验的能力。被忽视疾病药物研发倡议组织(DNDi)采用了一种与虚拟生物技术公司密切相关的模式来识别和优化药物先导物。将该模式应用于治疗昏睡病、恰加斯病和利什曼病等动基体感染的候选药物开发,已促使从DNDi自身的发现流程中识别出新的候选药物。这表明DNDi一直在实施的模式是有效的,但其可持续性仍有待证明。