PDF(Pubmed)
Abstract：
Drug repurposing is considered a valid approach to accelerate therapeutic solutions for rare diseases. However, it is not as widely applied as it could be, due to several barriers that discourage both industry and academic institutions from pursuing this path. Herein we present the case of an academic multicentre study that considered the repurposing of the old drug guanabenz as a therapeutic strategy in amyotrophic lateral sclerosis. The difficulties encountered are discussed as an example of the barriers that academics involved in this type of study may face. Although further development of the drug for this target population was hampered for several reasons, the study was successful in many ways. Firstly, because the hypothesis tested was confirmed in a sub-population, leading to alternative innovative solutions that are now under clinical investigation. In addition, the study was informative and provided new insights into the disease, which are now giving new impetus to laboratory research. The message from this example is that even a repurposing study with an old product has the potential to generate innovation and interest from industry partners, provided it is based on a sound rationale, the study design is adequate to ensure meaningful results, and the investigators keep the full clinical development picture in mind.
摘要：
药物再利用被认为是加速罕见疾病治疗解决方案的有效方法。然而,它没有尽可能广泛地应用，由于阻碍行业和学术机构走这条路的几个障碍。在此，我们介绍了一项学术多中心研究的案例，该研究认为将旧药物guanabenz重新用作肌萎缩性侧索硬化症的治疗策略。讨论了遇到的困难，作为参与此类研究的学者可能面临的障碍的一个例子。尽管针对该目标人群的药物的进一步开发由于几个原因而受到阻碍，这项研究在许多方面都是成功的。首先,因为测试的假设在一个亚群体中得到了证实，导致目前正在临床研究中的替代创新解决方案。此外,这项研究提供了信息，并提供了对这种疾病的新见解，它们现在为实验室研究提供了新的动力。这个例子的信息是，即使是对旧产品的重新利用研究也有可能引起行业合作伙伴的创新和兴趣，只要它是基于一个合理的理由，研究设计足以确保有意义的结果，研究人员将完整的临床发展状况牢记在心。
