小分子化合物筛选技术在药物研发中的应用及进展

Application and progress of small molecule compound screening techniques in drug research and development

摘要:

近年来，随着疾病机制研究的深入及技术手段的革新，小分子药物研发不断推陈出新，在癌症、糖尿病和自身免疫性疾病等领域的应用日益广泛。成千上万的疾病相关蛋白曾经被认为是“不可成药”靶点，传统的新药研究方法无法得到药物候选分子。如今传统筛选技术的突破创新和先进辅助技术的交叉结合，为很多“不可成药”靶点筛选出了有效的小分子化合物候选分子，并不断地推进临床阶段。该文将围绕最新研究成果，对小分子药物的基本特性、筛选流程及其在疾病中的具体应用进行综述，并分析经典与新兴小分子化合物筛选技术方法特征及优化方向，探讨辅助筛选技术发展，旨在为小分子药物的筛选与开发提供全面的理论基础和实践指导，以促进高效、安全的新药研发。

Abstract:

In recent years, with the deepening of disease mechanism research and innovations in technological methods, the development of small molecule drugs has continuously introduced new advances. Their applications have become increasingly widespread in fields such as cancer, diabetes mellitus, and autoimmune diseases. In the past, thousands of disease-related proteins were considered "undruggable" targets, and traditional drug discovery methods were unable to yield viable drug candidates. However, breakthroughs in traditional screening techniques, combined with advanced auxiliary technologies, have now enabled the identification of effective small molecule candidates for many "undruggable" targets, further advancing them to clinical stages. This review will focus on the latest research findings, providing an overview of the basic characteristics, screening processes, and specific applications of small molecules in disease treatment. It will also analyze the features and optimization directions of classical and emerging small molecule screening techniques, as well as explore the development of auxiliary screening technologies. The aim is to provide a comprehensive theoretical foundation and practical guidance for the screening and development of small molecule drugs, thereby promoting the efficient and safe development of new drugs.