KRAS / RAS 靶向 — 从平台化到 Ph3 验证
daraxonrasib ± GnP · 单药 ORR 47%;联合 ORR 55%,DCR 90%
自噬与胰腺导管腺癌(PDAC)耐药的关联是转化研究的长期热点,也是一个悬而未决的老问题。自噬在肿瘤中兼具促瘤与抑瘤双重角色,mTOR是其关键负调控检查点,氯喹/羟氯喹在临床前模型中与多种抗肿瘤药联用可增强细胞毒性,但自噬究竟贡献药效还是介导耐药一度“很大程度上未知”,其诱导或抑制的临床角色亦有待界定[1]。在PDAC中,遗传或药物抑制自噬在自发肿瘤模型和PDX中均显示疗效,且独立于p53状态[2];机制上MEK抑制经LKB1→AMPK→ULK1轴诱发保护性自噬,曲美替尼联合氯喹可使PDX退缩[3]。然而临床转化并不顺畅:转移性PDAC的羟氯喹II期研究中20例患者仅2例在2个月时无进展,中位PFS与OS分别只有46.5天和69.0天[5];术前随机II期研究虽见组织病理学反应显著改善(P=0.00016)[4]。加之耐药机制远不止自噬一条,如肿瘤相关成纤维细胞经外泌体miRNA靶向ACSL4、抑制铁死亡从而诱导吉西他滨耐药[6],卡点便在于:自噬介导的耐药是否有更可干预的代谢环节。
在AACR26海报场次当天,Soo-Youl Kim及同事关于自噬影响PDAC耐药的研究同步在线发表于Cancer Research(DOI: 10.1158/0008-5472.CAN-25-4517)。期刊页面显示,论文题为"Inhibiting Fatty Acid Oxidation Reverses Autophagy-Mediated…",2025年10月5日投稿、2026年4月10日接收,正式刊出于2026年7月2日的86卷13期3194–3212页[7][8]。
从标题可见,这项工作的推进在于干预点的前移与换轨:既往策略以羟氯喹直接封堵自噬通量[1][5],或以MEK抑制联合氯喹打断保护性自噬[3],本研究则把自噬介导的耐药与脂肪酸氧化这条代谢通路挂钩,提示不动自噬本身、抑制脂肪酸氧化即可逆转耐药,等于为这一耐药表型找到了新的可药切入点。
当然,目前可核实的仅是题录与发表信息,具体的细胞、动物证据与联合方案尚待全文披露;而转移背景下羟氯喹的有限疗效[5]与多重耐药通路并存的事实[6]也提醒,即便脂肪酸氧化抑制在临床前成立,能否转化为PDAC患者的生存获益、与化疗或自噬抑制如何排序组合,仍是需要前瞻研究回答的开放问题[1]。
- 1.Autophagy as a target for anticancer therapy · Nat Rev Clin Oncol 2011
- 2.Autophagy is critical for pancreatic tumor growth and progression in tumors with p53 alterations · Cancer Discov 2014
- 3.Protective autophagy elicited by RAF→MEK→ERK inhibition suggests a treatment strategy for RAS-driven cancers · Nat Med 2019
- 4.A Randomized Phase II Preoperative Study of Autophagy Inhibition with High-Dose Hydroxychloroquine and Gemcitabine/Nab-Paclitaxel in Pancreatic Cancer Patients · Clin Cancer Res 2020
- 5.Phase II and pharmacodynamic study of autophagy inhibition using hydroxychloroquine in patients with metastatic pancreatic adenocarcinoma · Oncologist 2014
- 6.Cancer-associated fibroblasts suppress ferroptosis and induce gemcitabine resistance in pancreatic cancer cells by secreting exosome-derived ACSL4-targeting miRNAs · Drug Resist Updat 2023
- 7.Inhibiting Fatty Acid Oxidation Reverses Autophagy-Mediated ... · aacrjournals.org 2026
- 8.Inhibiting Fatty Acid Oxidation Reverses Autophagy-Mediated ... · aacrjournals.org 2026
Presidential session #2 #AACR26
Dr. Mischel
@StanfordMed
Advancing cancer science to save lives
@carlhjune
@Penn
CAR-T applications solid tumors
Promising delivered locally #GBM
How to tune with #CAE for #PDAC
Promising #CART #solidtumors
@AACR
@OncoAlert
热度 14 · 8 赞 · 1 回复 · 5 转发
Kanwal Pratap Raghav, Jun Zhang, Myung-Ah Lee, Jingdong Zhang, John Park, Yanqiao Zhang, Susana Rosello Keranen, Byoung Chul Cho, Herbert Ho Fung Loong, Cheng Chen, Huan Li, Shaonan Wang, Yandong Shen, Zifei Fan, Qian Chen, Hui Wang, Jing Zhang, George Zhi Jian Chen, Jayesh Desai, Antoine Hollebecque
读摘要原文 →Eileen M. O'Reilly, Brian Wolpin, Shubham Pant, Joel Randolph Hecht, Jennifer Valerin, Dae Won Kim, Nilofer Azad, Kyaw Aung, Lin Tao, Sumit Kar, Hina Patel, Rashmi Vora, Aparna Hegde, Alexander Spira, Alexander Starodub, Benjamin Herzberg, Ignacio Garrido-Laguna
读摘要原文 →Brian M. Wolpin, Benjamin L. Musher, Gulam A. Manji, Wungki Park, Alexander Spira, Nilofer Azad, Vaia Florou, Tiago Biachi De Castria, Mark H. O'Hara, Erkut Borazanci, Aparna Parikh, John Strickler, Joel Randolph Hecht, Shubham Pant, Shiraj Sen, E. Gabriela Chiorean, W. Clay Gustafson, Hina Patel, Vidya Seshadri, Yan Zheng, Davendra Sohal
读摘要原文 →来源:Oriol Mirallas MD · x.com
