Machine-Learning-Accelerated Design of Ternary Carrier-Free Nanomedicine for Intranasal Therapy of Brain Metastatic Non-small-cell Lung Cancer

利用机器学习加速设计用于鼻内治疗脑转移性非小细胞肺癌的三元无载体纳米药物

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Abstract

Non-small-cell lung cancer (NSCLC) with brain metastases poses formidable therapeutic challenges due to acquired resistance and the inherent pharmacokinetic defects of traditional delivery. We developed an innovative lipoic acid-based self-assembled nanodrug (dabrafenib, trametinib, and lipoic acid self-assembly [DTL]) system, whose rational design was guided by a novel machine learning platform to overcome high-cost, empirical screening bottlenecks. Multifunctional lipoic acid, serving as a universal self-assembling molecule, enabled DTL's robust assembly and enhanced penetration across mucosal and solid tumor barriers via its unique thiol-mediated exchange mechanism while simultaneously exerting distinct antitumor efficacy. Intranasal administration of DTL achieved efficient dual-targeted delivery to both primary NSCLC and established intracranial metastases. Furthermore, compared to conventional targeted combination therapies, DTL induced diverse, multimodal tumor cell death (apoptosis, pyroptosis, and ferroptosis) and profoundly remodeled the immune microenvironment. In vivo, DTL markedly inhibited tumor growth with reduced toxicity, offering a clinically translatable strategy for advanced NSCLC.

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