000131777 001__ 131777 000131777 005__ 20240229105012.0 000131777 0247_ $$2doi$$a10.1172/JCI93801 000131777 0247_ $$2pmid$$apmid:29227286 000131777 0247_ $$2ISSN$$a0021-9738 000131777 0247_ $$2ISSN$$a1558-8238 000131777 0247_ $$2altmetric$$aaltmetric:30321455 000131777 037__ $$aDKFZ-2018-00074 000131777 041__ $$aeng 000131777 082__ $$a610 000131777 1001_ $$0P:(DE-He78)6333b389d0abc96ef7f2f6e049a8f8c4$$aDietrich, Sascha$$b0$$eFirst author 000131777 245__ $$aDrug-perturbation-based stratification of blood cancer. 000131777 260__ $$aAnn Arbor, Mich.$$bASCJ$$c2018 000131777 3367_ $$2DRIVER$$aarticle 000131777 3367_ $$2DataCite$$aOutput Types/Journal article 000131777 3367_ $$0PUB:(DE-HGF)16$$2PUB:(DE-HGF)$$aJournal Article$$bjournal$$mjournal$$s1680786356_21947 000131777 3367_ $$2BibTeX$$aARTICLE 000131777 3367_ $$2ORCID$$aJOURNAL_ARTICLE 000131777 3367_ $$00$$2EndNote$$aJournal Article 000131777 520__ $$aAs new generations of targeted therapies emerge and tumor genome sequencing discovers increasingly comprehensive mutation repertoires, the functional relationships of mutations to tumor phenotypes remain largely unknown. Here, we measured ex vivo sensitivity of 246 blood cancers to 63 drugs alongside genome, transcriptome, and DNA methylome analysis to understand determinants of drug response. We assembled a primary blood cancer cell encyclopedia data set that revealed disease-specific sensitivities for each cancer. Within chronic lymphocytic leukemia (CLL), responses to 62% of drugs were associated with 2 or more mutations, and linked the B cell receptor (BCR) pathway to trisomy 12, an important driver of CLL. Based on drug responses, the disease could be organized into phenotypic subgroups characterized by exploitable dependencies on BCR, mTOR, or MEK signaling and associated with mutations, gene expression, and DNA methylation. Fourteen percent of CLLs were driven by mTOR signaling in a non-BCR-dependent manner. Multivariate modeling revealed immunoglobulin heavy chain variable gene (IGHV) mutation status and trisomy 12 as the most important modulators of response to kinase inhibitors in CLL. Ex vivo drug responses were associated with outcome. This study overcomes the perception that most mutations do not influence drug response of cancer, and points to an updated approach to understanding tumor biology, with implications for biomarker discovery and cancer care. 000131777 536__ $$0G:(DE-HGF)POF3-317$$a317 - Translational cancer research (POF3-317)$$cPOF3-317$$fPOF III$$x0 000131777 588__ $$aDataset connected to CrossRef, PubMed, 000131777 7001_ $$aOleś, Małgorzata$$b1 000131777 7001_ $$aLu, Junyan$$b2 000131777 7001_ $$0P:(DE-He78)b07a76205b49e86733668b7201520d19$$aSellner, Leopold$$b3 000131777 7001_ $$aAnders, Simon$$b4 000131777 7001_ $$aVelten, Britta$$b5 000131777 7001_ $$0P:(DE-He78)f385b8b832d53e921e7af2af3c625418$$aWu, Bian$$b6 000131777 7001_ $$0P:(DE-He78)c9be4ab60d1090c3d315a2ca6905e9ea$$aHüllein, Jennifer$$b7 000131777 7001_ $$0P:(DE-HGF)0$$ada Silva Liberio, Michelle$$b8 000131777 7001_ $$0P:(DE-He78)e8feda17d03b95bda3e8717e79dc07b8$$aWalther, Tatjana$$b9 000131777 7001_ $$0P:(DE-He78)dafca537433ee26708b96d89aba38d29$$aWagner, Lena$$b10 000131777 7001_ $$0P:(DE-He78)022daadde115569e96596dfcd21190a3$$aRabe, Sophie$$b11 000131777 7001_ $$aGhidelli-Disse, Sonja$$b12 000131777 7001_ $$aBantscheff, Marcus$$b13 000131777 7001_ $$aOleś, Andrzej K$$b14 000131777 7001_ $$0P:(DE-HGF)0$$aSłabicki, Mikołaj$$b15 000131777 7001_ $$aMock, Andreas$$b16 000131777 7001_ $$aOakes, Christopher C$$b17 000131777 7001_ $$aWang, Shihui$$b18 000131777 7001_ $$0P:(DE-He78)55dfd5b374ab9bccdb49e06e91345571$$aOppermann, Sina$$b19 000131777 7001_ $$0P:(DE-He78)1d028378e12ca6bdafa3b8b21bc5a9ea$$aLukas, Marina$$b20 000131777 7001_ $$aKim, Vladislav$$b21 000131777 7001_ $$0P:(DE-He78)45440b44791309bd4b7dbb4f73333f9b$$aSill, Martin$$b22 000131777 7001_ $$0P:(DE-He78)e15dfa1260625c69d6690a197392a994$$aBenner, Axel$$b23 000131777 7001_ $$aJauch, Anna$$b24 000131777 7001_ $$aSutton, Lesley Ann$$b25 000131777 7001_ $$aYoung, Emma$$b26 000131777 7001_ $$aRosenquist, Richard$$b27 000131777 7001_ $$0P:(DE-He78)ca03e7517472097f49f822f5d6b0056f$$aLiu, Xiyang$$b28 000131777 7001_ $$0P:(DE-He78)7f506158c27b5827cd9021cd3dad37c8$$aJethwa, Alexander$$b29 000131777 7001_ $$0P:(DE-He78)1121dc6777ecb3a3e48075bbd16246f2$$aLee, Kwang$$b30 000131777 7001_ $$aLewis, Joe$$b31 000131777 7001_ $$aPutzker, Kerstin$$b32 000131777 7001_ $$aLutz, Christoph$$b33 000131777 7001_ $$aRossi, Davide$$b34 000131777 7001_ $$aMokhir, Andriy$$b35 000131777 7001_ $$0P:(DE-He78)6fe098b268bbad1a939e14ed018b295f$$aOellerich, Thomas$$b36 000131777 7001_ $$aZirlik, Katja$$b37 000131777 7001_ $$aHerling, Marco$$b38 000131777 7001_ $$aNguyen-Khac, Florence$$b39 000131777 7001_ $$0P:(DE-He78)4301875630bc997edf491c694ae1f8a9$$aPlass, Christoph$$b40 000131777 7001_ $$aAndersson, Emma$$b41 000131777 7001_ $$aMustjoki, Satu$$b42 000131777 7001_ $$0P:(DE-He78)5bacb661d5d7c0220d8f996d980ad8de$$avon Kalle, Christof$$b43 000131777 7001_ $$aHo, Anthony D$$b44 000131777 7001_ $$aHensel, Manfred$$b45 000131777 7001_ $$aDürig, Jan$$b46 000131777 7001_ $$aRingshausen, Ingo$$b47 000131777 7001_ $$0P:(DE-He78)1beba8f953e7ae7e96e8d3e9a48f10f7$$aZapatka, Marc$$b48 000131777 7001_ $$0P:(DE-HGF)0$$aHuber, Wolfgang$$b49 000131777 7001_ $$0P:(DE-He78)f3d5f16b49eb47520def635be98d5576$$aZenz, Thorsten$$b50 000131777 773__ $$0PERI:(DE-600)2018375-6$$a10.1172/JCI93801$$gVol. 128, no. 1, p. 427 - 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