Jiuming He, PhD | Chinese Academy of Medical Sciences; Yan Li, PhD; Kaijing Guo, MS; Jianpeng Huang, MS; Dongting Chen, MS
Competition Sponsor: Chinese Academy of Medical Sciences
Awardee Year: 2025
A novel small-molecule drug conjugate (SMDC) born from an original target and a unique mechanism, was designed to tackle refractory solid tumors like pancreatic cancer and glioblastoma. Its core advantages include: 1) Superior Efficacy: It demonstrates significantly higher tumor inhibition and survival extension than nab-paclitaxel in various challenging animal models. 2) Revolutionary Safety: Its tumor-specific activation mechanism drastically reduces systemic, immunogenic, and neurotoxicities, promising improved patient quality of life. 3) Dual-Targeting Delivery: It combines active targeting via a metabolism-associated transporter with passive targeting through nanomicelles, ensuring precise tumor accumulation. 4) Overcoming Clinical Barriers: It effectively penetrates the blood-brain barrier and dense tumor stroma, addressing the key limitation of poor solid tumor penetration seen with larger entities like ADCs. This SMDC aims to meet the urgent, high-unmet medical need in aggressive cancers with poor prognoses and limited therapeutic options, such as pancreatic cancer (median survival <1 year), glioblastoma, and chemotherapy-resistant lung cancer.