作者
Johannes Popow,William Farnaby,Andreas Gollner,Christiane Kofink,Gerhard W. Fischer,Melanie Wurm,David Zollman,Andre Wijaya,Nikolai Mischerikow,Carina Hasenoehrl,Polina Prokofeva,Heribert Arnhof,Silvia Arce-Solano,Sammy Bell,Georg Boeck,Emelyne Diers,Aileen B. Frost,Jake Goodwin‐Tindall,Jale Karolyi‐Oezguer,Shakil Khan,Theresa Klawatsch,Manfred Koegl,Roland Kousek,Barbara Kratochvil,Katrin Kropatsch,Arnel A. Lauber,Ross McLennan,Sabine Olt,Daniel Peter,Oliver Petermann,Vanessa Roessler,Peggy Stolt-Bergner,Patrick Strack,Eva Strauss,Nicole Trainor,Vesna Vetma,Claire Whitworth,Siying Zhong,Jens Quant,Harald Weinstabl,Bernhard Küster,Peter Ettmayer,Alessio Ciulli
摘要
Despite the high prevalence of cancers driven by KRAS mutations, to date only the G12C mutation has been clinically proven to be druggable via covalent targeting of the mutated cysteine amino acid residue (1). However, in many cancer indications other KRAS mutations, such as G12D and -V, are far more prevalent and small molecule concepts that can address a wider variety of oncogenic KRAS alleles are in high clinical demand (2). Here we show that a single small molecule can be used to simultaneously and potently degrade 13 out of 17 of the most prevalent oncogenic KRAS alleles, including those not yet tractable by inhibitors. Compared with inhibition, degradation of oncogenic KRAS results in more profound and sustained pathway modulation across a broad range of KRAS mutant cell lines. As a result, KRAS degraders inhibit growth of the majority of cancer cell lines driven by KRAS mutations while sparing models without genetic KRAS aberrations. Finally, we demonstrate that pharmacological degradation of oncogenic KRAS leads to tumour regression in vivo. Together, these findings unveil a new path towards addressing KRAS driven cancers with small molecule degraders.