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核酸适配体(aptamer)
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谭蔚泓院士团队开发核酸适配体偶联药物ApDC,抗癌效果令人鼓舞
生物世界· 2025-09-25 10:30
Core Insights - The article discusses the rising incidence of cancer globally, with nearly 20 million new cases and 9.7 million cancer-related deaths reported in 2022, highlighting cancer as a leading cause of disease-related mortality [3] - Traditional cancer treatments like chemotherapy and radiotherapy have limitations due to their lack of tumor specificity, leading to significant side effects [3] - Targeted drug delivery strategies, particularly Aptamer-Drug Conjugates (ApDC), are emerging as promising alternatives for cancer treatment, allowing for selective delivery of cytotoxic drugs to tumor cells [3][6] Group 1: Research Development - A recent study published in "Signal Transduction and Targeted Therapy" introduced Sgc8c-M, an ApDC developed by linking a potent anti-mitotic agent MMAE with the PTK7 aptamer Sgc8c, aimed at treating cancers that overexpress PTK7 [4][6] - The study demonstrated the potential of Sgc8c-M through comprehensive evaluations from rodents to non-human primates, indicating its promise as a cancer therapy [4][9] Group 2: Mechanism and Efficacy - Sgc8c-M showed effective tumor regression in various PTK7-overexpressing cancer types, outperforming unlinked MMAE, the chemotherapy drug paclitaxel, and a PTK7-targeted antibody-drug conjugate [6][9] - Pharmacokinetic studies in mice revealed rapid accumulation of the drug in tumors while being quickly cleared from plasma and normal tissues, with over 75% of MMAE excreted within 24 hours [7] Group 3: Safety and Tolerability - Toxicokinetic assessments indicated that Sgc8c-M maintained consistent systemic drug exposure without accumulation after repeated dosing, and high therapeutic doses were found to be safe [7] - Further evaluations in non-human primates showed similar pharmacokinetic characteristics and good tolerability, with no significant accumulation observed after multiple doses [7]