Biotech Research and Innovation Centre, Faculty of Health, University of Copenhagen, Copenhagen, Denmark.
Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK.
Nat Rev Drug Discov. 2024 Mar;23(3):218-231. doi: 10.1038/s41573-023-00847-7. Epub 2023 Dec 19.
In spite of major efforts and investment in development of psychiatric drugs, many clinical trials have failed in recent decades, and clinicians still prescribe drugs that were discovered many years ago. Although multiple reasons have been discussed for the drug development deadlock, we focus here on one of the major possible biological reasons: differences between the characteristics of drug targets in preclinical models and the corresponding targets in patients. Importantly, based on technological advances in single-cell analysis, we propose here a framework for the use of available and newly emerging knowledge from single-cell and spatial omics studies to evaluate and potentially improve the translational predictivity of preclinical models before commencing preclinical and, in particular, clinical studies. We believe that these recommendations will improve preclinical models and the ability to assess drugs in clinical trials, reducing failure rates in expensive late-stage trials and ultimately benefitting psychiatric drug discovery and development.
尽管在精神药物研发方面投入了大量的精力和资金,但近几十年来,许多临床试验都失败了,临床医生仍然开处方使用多年前发现的药物。尽管人们已经讨论了造成药物研发僵局的多种原因,但我们在这里关注的是其中一个主要的生物学原因:临床前模型中药物靶点的特征与患者体内相应靶点之间的差异。重要的是,基于单细胞分析技术的进步,我们在这里提出了一个框架,利用单细胞和空间组学研究中现有的和新兴的知识,在进行临床前研究,特别是临床研究之前,评估和潜在地提高临床前模型的转化预测能力。我们相信,这些建议将改进临床前模型,并提高在临床试验中评估药物的能力,降低昂贵的后期试验的失败率,最终使精神药物的发现和开发受益。