Janin Yves L
Structure et Instabilité des Génomes (StrInG), Muséum National d'Histoire Naturelle, INSERM, CNRS, Alliance Sorbonne Université, 75005 Paris, France.
Beilstein J Org Chem. 2022 Sep 29;18:1355-1378. doi: 10.3762/bjoc.18.141. eCollection 2022.
This perspective is an attempt to document the problems that medicinal chemists are facing in drug discovery. It is also trying to identify relevant/possible, research areas in which academics can have an impact and should thus be the subject of grant calls. Accordingly, it describes how hit discovery happens, how compounds to be screened are selected from available chemicals and the possible reasons for the recurrent paucity of useful/exploitable results reported. This is followed by the successful hit to lead stories leading to recent and original antibacterials which are, or about to be, used in human medicine. Then, illustrated considerations and suggestions are made on the possible inputs of academic medicinal chemists. This starts with the observation that discovering a "good" hit in the course of a screening campaign still rely on a lot of luck - which is within the reach of academics -, that the hit to lead process requires a lot of chemistry and that if public-private partnerships can be important throughout these stages, they are absolute requirements for clinical trials. Concerning suggestions to improve the current hit success rate, one academic input in organic chemistry would be to identify new and pertinent chemical space, design synthetic accesses to reach these and prepare the corresponding chemical libraries. Concerning hit to lead programs on a given target, if no new hits are available, previously reported leads along with new structural data can be pertinent starting points to design, prepare and assay original analogues. In conclusion, this text is an actual plea illustrating that, in many countries, academic research in medicinal chemistry should be more funded, especially in the therapeutic area neglected by the industry. At the least, such funds would provide the intensive to secure series of hopefully relevant chemical entities which appears to often lack when considering the results of academic as well as industrial screening campaigns.
这篇综述旨在记录药物化学家在药物发现过程中面临的问题。它还试图确定学术界能够产生影响且应成为资助申请主题的相关/可能的研究领域。因此,它描述了苗头化合物发现的过程、如何从现有化学品中选择待筛选的化合物,以及所报道的有用/可开发结果反复匮乏的可能原因。接下来是成功的苗头到先导物的案例,这些案例产生了近期的原创抗菌药物,这些药物正在或即将用于人类医学。然后,针对学术药物化学家可能的贡献给出了具体的思考和建议。首先观察到,在筛选过程中发现一个“好的”苗头仍然很大程度上依赖运气——这是学术界能够做到的,从苗头到先导物的过程需要大量的化学工作,并且如果公私合作在这些阶段都很重要,那么它们对于临床试验来说是绝对必要的。关于提高当前苗头化合物成功率的建议,有机化学领域的一项学术贡献是确定新的相关化学空间,设计合成路线以进入这些空间并制备相应的化学文库。对于针对特定靶点的苗头到先导物项目,如果没有新的苗头,先前报道的先导物以及新的结构数据可以作为设计、制备和测试原创类似物的相关起点。总之,本文实际上是在呼吁,在许多国家,药物化学领域的学术研究应该获得更多资助,尤其是在被制药行业忽视的治疗领域。至少,这样的资金将有助于确保获得一系列有望相关的化学实体,而从学术和工业筛选活动的结果来看,这些实体似乎常常缺失。