阿尔茨海默病、帕金森病、脑缺血、脑胶质瘤等中枢神经系统（CNS）疾病发病率呈逐年上升趋势。此类疾病病理机制复杂，病灶位于CNS深处，且受到血脑屏障（BBB）的严格限制，传统药物脑部递送效率低、治疗效果有限。中药活性成分以其多靶点、多途径的神经保护潜力备受关注，但生物利用度低、BBB穿透能力弱的问题，严重制约了其临床推广与应用。基于中医药归经理论与现代BBB转运机制，系统梳理纳米载体递送、受体介导主动靶向、微环境响应释药、鼻腔-脑通路及仿生递送等策略，并总结各类策略在多种CNS疾病模型中的应用进展。同时分析中药脑靶向递送制剂临床转化面临的载体安全性、疾病异质性及多成分协同递送等瓶颈。未来，结合智能诊疗平台、多机制协同递送体系及人工智能辅助设计技术，有望推动中药脑靶向制剂的临床转化进程，为研发高效CNS靶向中药制剂提供可行路径。

Incidence of central nervous system (CNS) disorders such as Alzheimer’s disease, Parkinson’s disease, cerebral ischemia, and glioblastoma is rising annually. These diseases are characterized by complex pathogenic mechanisms, deep lesion locations within the CNS, and strict limitations imposed by the blood-brain barrier (BBB), resulting in low drug delivery efficiency and limited therapeutic efficacy with conventional medications. Active components from traditional Chinese medicine (TCM) have attracted significant attention due to their multi-target, multi-pathway neuroprotective potential. However, their clinical application remains hindered by low bioavailability and poor BBB penetration. Based on TCM meridian theory and modern understanding of BBB transport mechanisms, this review systematically summarizes various strategies for brain targeting, including nanocarrier-based delivery, receptor-mediated active targeting, microenvironment-responsive drug release, nasal-to-brain pathways, and biomimetic delivery systems, and evaluates their progress in diverse CNS disease models. It also analyzes key challenges in clinical translation, such as carrier safety, disease heterogeneity, and the coordinated delivery of multiple active ingredients. In the future, integrating intelligent diagnostic platforms, multi-mechanism synergistic delivery systems, and artificial intelligence-assisted design technologies holds promise for accelerating the clinical development of TCM brain-targeted formulations, providing a viable pathway toward the creation of highly effective CNS-targeted TCM therapeutics.

R283.6;R943

2025年乌鲁木齐市“红山英才”计划项目