Saito Satoshi, Ihara Masafumi
Department of Neurology, National Cerebral and Cardiovascular Center.
Brain Nerve. 2019 Sep;71(9):936-942. doi: 10.11477/mf.1416201385.
The aim of drug repositioning is to identify and develop new uses for existing drugs. Many drugs for neurological disorders have been established by drug repositioning. Conventional drug repositioning approaches are based on chance findings or close clinical observations. Nowadays, computational drug repositioning can complement these approaches. Drug repositioning has many advantages. For example, unexpected adverse events occur less frequently in clinical trials using repositioned drugs than in trials with new drugs because safety information for repositioned drugs has often already accumulated before the start of a clinical trial. Another advantage is that drug repositioning can shorten the time needed for drug development and thereby reduce costs related to drug innovation. If results of pharmacological and toxic tests are available, pre-clinical studies may also be omitted. Drug repositioning is especially promising in the development of treatments for neurodegenerative disorders because the development of new drugs for chronic disorders often takes more than 20 years and has a high financial burden. Strategic patenting is important to cooperate with pharmaceutical companies and to accomplish drug repositioning.
药物重新定位的目的是识别并开发现有药物的新用途。许多用于治疗神经系统疾病的药物都是通过药物重新定位确定的。传统的药物重新定位方法基于偶然发现或密切的临床观察。如今,计算药物重新定位可以补充这些方法。药物重新定位有许多优点。例如,在使用重新定位药物的临床试验中,意外不良事件的发生频率低于使用新药的试验,因为重新定位药物的安全信息通常在临床试验开始前就已经积累。另一个优点是,药物重新定位可以缩短药物开发所需的时间,从而降低与药物创新相关的成本。如果有药理学和毒理学测试结果,也可以省略临床前研究。药物重新定位在神经退行性疾病治疗的开发中特别有前景,因为针对慢性疾病的新药开发通常需要20多年,且经济负担沉重。战略专利对于与制药公司合作以及实现药物重新定位很重要。