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开发热带寄生虫病新药的最新举措和策略。

Recent initiatives and strategies to developing new drugs for tropical parasitic diseases.

机构信息

University of California San Francisco, Sandler Center for Basic Research in Parasitic Diseases, California Institute for Quantitative Biosciences, Byers Hall 501E, 1700 4th Street, San Francisco, CA 94158, USA.

出版信息

Expert Opin Drug Discov. 2008 Feb;3(2):173-86. doi: 10.1517/17460441.3.2.173.

Abstract

BACKGROUND

Despite the toll of tropical parasitic diseases on human life in the developing world, present therapies still rely on drugs developed decades ago. In many cases, the clinical usefulness of these compounds is limited due to poor efficacy, toxicity and the constant attrition of drug resistance. The absence of a profit incentive regarding diseases afflicting the very poor has resulted in a lack of investment by the pharmaceutical industry in new chemotherapies.

OBJECTIVE

Given this background, this review addresses what alternative economic and scientific strategies have been implemented to procure novel drugs.

METHODS

The latest chemical, genetic and screening technologies to discover and develop drugs for tropical parasitic diseases are reviewed. In many cases these strategies are being implemented within the framework of public-private partnerships established to sustain dynamic drug development portfolios. Examples of public-private partnerships and their portfolios are discussed. Further, the contribution of dedicated academic screening centres to target discovery and preclinical prosecution of new small molecules is also highlighted. In every case, the latest scientific literature is cited, but also relevant press releases and website information to indicate the present vitality in the field.

CONCLUSION

The tools, institutions and consortia are now in place and evolving to deliver new pharmaceuticals. Short-term results have already been realised in the clinic, mainly through the provision of new formulations of existing drugs. Long-term and consistent investment will be required, however, to identify, develop and clinically validate new chemical entities.

摘要

背景

尽管热带寄生虫病对发展中国家的人类生命造成了严重影响,但目前的治疗方法仍然依赖于几十年前开发的药物。在许多情况下,由于疗效不佳、毒性和耐药性的不断削弱,这些化合物的临床实用性受到限制。由于利润激励不足,针对贫困人群的疾病,制药行业缺乏对新化疗药物的投资。

目的

鉴于这种背景,本文探讨了为获取新型药物而实施的替代经济和科学策略。

方法

本文回顾了用于发现和开发热带寄生虫病药物的最新化学、遗传和筛选技术。在许多情况下,这些策略是在为维持动态药物开发组合而建立的公私合作伙伴关系框架内实施的。讨论了公私合作伙伴关系及其投资组合的实例。此外,还强调了专门的学术筛选中心在发现目标和推进新小分子的临床前研究方面的贡献。在每种情况下,都引用了最新的科学文献,但也引用了相关的新闻稿和网站信息,以表明该领域目前的活力。

结论

现在已经有了用于提供新药物的工具、机构和联盟,并在不断发展。在临床上已经取得了短期成果,主要是通过提供现有药物的新制剂。然而,需要长期和持续的投资,以识别、开发和临床验证新的化学实体。

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