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Kimberly Stegmaier, MD


Pediatric Hematology/Oncology

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Physician

  • Co-Director of the Pediatric Hematologic Malignancy Program
  • Physician
  • Ted Williams Chair
  • Associate Professor of Pediatrics, Harvard Medical School

Clinical Interests

  • Cancer genomics
  • Chemical screening
  • Ewing sarcoma
  • Hematologic malignancies
  • Neuroblastoma

Contact Information

  • Appointments888-733-4662 (new)
    617-632-3270 (established)
  • Office Phone Number617-632-4985
  • Fax617-632-4850

Bio

Kimberly Stegmaier, M.D., is an Associate Professor of Pediatrics at Harvard Medical School, a Principal Investigator in Pediatric Oncology at the Dana-Farber Cancer Institute (DFCI), and anAttending Physician at the Boston Children’s Hospital Boston (BCH) and DFCI. She is the Co-director of the Pediatric Hematologic Malignancies Program at DFCI and BCH and is also an Associate Member of the Broad Institute of Harvard and MIT. She received her B.S. from Duke University and her M.D. from Harvard Medical School. She completed her residency at BCH and a post-doctoral pediatric hematology-oncology fellowship at DFCI/BCH. In 2006, she launched her own laboratory effort at DFCI.

Dr. Stegmaier’s laboratory integrates chemical biology, genomic, and proteomic approaches to discover new lead compounds and protein targets for cancer therapy. She has focused her efforts on the acute leukemias and two pediatric solid tumors of childhood: Ewing sarcoma and neuroblastoma.

Board Certification:

  • Pediatric Hematology/Oncology, 2002

Fellowship:

  • Boston Children's Hospital/Dana-Farber Cancer Institute, Pediatric Hematology/Oncology

Residency:

  • Boston Combined Residency Program, Boston Children's Hospital/Boston Medical Center, Pediatrics

Medical School:

  • Harvard Medical School

Recent Awards:

  • Joanne Levy, MD, Memorial Award for Outstanding Achievement, American Society of  Hematology 2006
  • Genome Technology “Tomorrow’s Principal Investigator” Award 2007
  • American Society for Clinical Investigation, Elected Member 2009
  • SU2C Innovative Research Grant Recipient 2009
  • Society for Pediatric Research (SPR) Young Investigator Award 2012
  • Stephen E. Sallan Leadership Award 2012
  • Osler Young Investigator Award, Interurban Clinical Club 2013
  • Clifford Barger Excellence in Mentoring Award, Harvard Medical School 2014

Research

Principal Investigator

My research program focuses on the integration of “omic” approaches for the identification of new protein targets and small-molecule modulators of malignancy with an eye toward clinical translation. Cancer discovery efforts in my laboratory have focused on the alteration of the malignant state (e.g., AML and neuroblastoma differentiation) and the modulation of pharmacologically challenging oncoproteins (e.g., EWS/FLI in Ewing sarcoma, MYCN in neuroblastoma, and NOTCH1 in T-ALL.) Most recently, we are applying an integrated approach to discover new therapeutic opportunities in these malignancies with deep genomic characterization of primary tumors, kinase activity profiling for immediately druggable targets, functional genomic screening for new tumor dependencies and chemical screening for modulators of relevant oncogenic drivers. Clinical trials for patients with AML and Ewing sarcoma have resulted from our research and a trial testing BET bromodomain inhibitors in patients with MYCN amplified neuroblastoma is in development.

 

Bohnenberger H, Kaderali L, Ströbel P, Yepes D, Plessmann U, Dharia NV, Yao S, Heydt C, Merkelbach-Bruse S, Emmert A, Hoffmann J, Bodemeyer J, Reuter-Jessen K, Lois AM, Dröge LH, Baumeister P, Walz C, Biggemann L, Walter R, Häupl B, Comoglio F, Pan KT, Scheich S, Lenz C, Küffer S, Bremmer F, Kitz J, Sitte M, Beißbarth T, Hinterthaner M, Sebastian M, Lotz J, Schildhaus HU, Wolff H, Danner BC, Brandts C, Büttner R, Canis M, Stegmaier K, Serve H, Urlaub H, Oellerich T. Comparative proteomics reveals a diagnostic signature for pulmonary head-and-neck cancer metastasis. EMBO Mol Med. 2018 Aug 10.
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Pikman Y, Stegmaier K. Targeted therapy for fusion-driven high-risk acute leukemia. Blood. 2018 Jul 26.
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Stolte B, Iniguez AB, Dharia NV, Robichaud AL, Conway AS, Morgan AM, Alexe G, Schauer NJ, Liu X, Bird GH, Tsherniak A, Vazquez F, Buhrlage SJ, Walensky LD, Stegmaier K. Genome-scale CRISPR-Cas9 screen identifies druggable dependencies in TP53 wild-type Ewing sarcoma. J Exp Med. 2018 Aug 06; 215(8):2137-2155.
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Zhang C, D'Alessandro A, Wellendorf AM, Mohmoud F, Serrano-Lopez J, Perentesis JP, Komurov K, Alexe G, Stegmaier K, Whitsett JA, Grimes HL, Cancelas JA. KLF5 controls glutathione metabolism to suppress p190-BCR-ABL+ B-cell lymphoblastic leukemia. Oncotarget. 2018 Jul 03; 9(51):29665-29679.
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Gorthi A, Romero JC, Loranc E, Cao L, Lawrence LA, Goodale E, Iniguez AB, Bernard X, Masamsetti VP, Roston S, Lawlor ER, Toretsky JA, Stegmaier K, Lessnick SL, Chen Y, Bishop AJR. Author Correction: EWS-FLI1 increases transcription to cause R-loops and block BRCA1 repair in Ewing sarcoma. Nature. 2018 Jul; 559(7715):E11.
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Place AE, Pikman Y, Stevenson KE, Harris MH, Pauly M, Sulis ML, Hijiya N, Gore L, Cooper TM, Loh ML, Roti G, Neuberg DS, Hunt SK, Orloff-Parry S, Stegmaier K, Sallan SE, Silverman LB. Phase I trial of the mTOR inhibitor everolimus in combination with multi-agent chemotherapy in relapsed childhood acute lymphoblastic leukemia. Pediatr Blood Cancer. 2018 Jul; 65(7):e27062.
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Baldoni S, Del Papa B, Dorillo E, Aureli P, De Falco F, Rompietti C, Sorcini D, Varasano E, Cecchini D, Zei T, Di Tommaso A, Rosati E, Alexe G, Roti G, Stegmaier K, Di Ianni M, Falzetti F, Sportoletti P. Bepridil exhibits anti-leukemic activity associated with NOTCH1 pathway inhibition in chronic lymphocytic leukemia. Int J Cancer. 2018 Aug 15; 143(4):958-970.
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Gorthi A, Romero JC, Loranc E, Cao L, Lawrence LA, Goodale E, Iniguez AB, Bernard X, Masamsetti VP, Roston S, Lawlor ER, Toretsky JA, Stegmaier K, Lessnick SL, Chen Y, Bishop AJR. EWS-FLI1 increases transcription to cause R-loops and block BRCA1 repair in Ewing sarcoma. Nature. 2018 03 15; 555(7696):387-391.
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Wagner FF, Benajiba L, Campbell AJ, Weïwer M, Sacher JR, Gale JP, Ross L, Puissant A, Alexe G, Conway A, Back M, Pikman Y, Galinsky I, DeAngelo DJ, Stone RM, Kaya T, Shi X, Robers MB, Machleidt T, Wilkinson J, Hermine O, Kung A, Stein AJ, Lakshminarasimhan D, Hemann MT, Scolnick E, Zhang YL, Pan JQ, Stegmaier K, Holson EB. Exploiting an Asp-Glu "switch" in glycogen synthase kinase 3 to design paralog-selective inhibitors for use in acute myeloid leukemia. Sci Transl Med. 2018 Mar 07; 10(431).
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Iniguez AB, Stolte B, Wang EJ, Conway AS, Alexe G, Dharia NV, Kwiatkowski N, Zhang T, Abraham BJ, Mora J, Kalev P, Leggett A, Chowdhury D, Benes CH, Young RA, Gray NS, Stegmaier K. EWS/FLI Confers Tumor Cell Synthetic Lethality to CDK12 Inhibition in Ewing Sarcoma. Cancer Cell. 2018 Feb 12; 33(2):202-216.e6.
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Chen L, Alexe G, Dharia NV, Ross L, Iniguez AB, Conway AS, Wang EJ, Veschi V, Lam N, Qi J, Gustafson WC, Nasholm N, Vazquez F, Weir BA, Cowley GS, Ali LD, Pantel S, Jiang G, Harrington WF, Lee Y, Goodale A, Lubonja R, Krill-Burger JM, Meyers RM, Tsherniak A, Root DE, Bradner JE, Golub TR, Roberts CW, Hahn WC, Weiss WA, Thiele CJ, Stegmaier K. CRISPR-Cas9 screen reveals a MYCN-amplified neuroblastoma dependency on EZH2. J Clin Invest. 2018 Jan 02; 128(1):446-462.
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Roti G, Qi J, Kitara S, Sanchez-Martin M, Saur Conway A, Varca AC, Su A, Wu L, Kung AL, Ferrando AA, Bradner JE, Stegmaier K. Leukemia-specific delivery of mutant NOTCH1 targeted therapy. J Exp Med. 2018 Jan 02; 215(1):197-216.
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Meyers RM, Bryan JG, McFarland JM, Weir BA, Sizemore AE, Xu H, Dharia NV, Montgomery PG, Cowley GS, Pantel S, Goodale A, Lee Y, Ali LD, Jiang G, Lubonja R, Harrington WF, Strickland M, Wu T, Hawes DC, Zhivich VA, Wyatt MR, Kalani Z, Chang JJ, Okamoto M, Stegmaier K, Golub TR, Boehm JS, Vazquez F, Root DE, Hahn WC, Tsherniak A. Computational correction of copy number effect improves specificity of CRISPR-Cas9 essentiality screens in cancer cells. Nat Genet. 2017 Dec; 49(12):1779-1784.
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LaRochelle JR, Fodor M, Ellegast JM, Liu X, Vemulapalli V, Mohseni M, Stams T, Buhrlage SJ, Stegmaier K, LaMarche MJ, Acker MG, Blacklow SC. Identification of an allosteric benzothiazolopyrimidone inhibitor of the oncogenic protein tyrosine phosphatase SHP2. Bioorg Med Chem. 2017 12 15; 25(24):6479-6485.
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Lobbardi R, Pinder J, Martinez-Pastor B, Theodorou M, Blackburn JS, Abraham BJ, Namiki Y, Mansour M, Abdelfattah NS, Molodtsov A, Alexe G, Toiber D, de Waard M, Jain E, Boukhali M, Lion M, Bhere D, Shah K, Gutierrez A, Stegmaier K, Silverman LB, Sadreyev RI, Asara JM, Oettinger MA, Haas W, Look AT, Young RA, Mostoslavsky R, Dellaire G, Langenau DM. TOX Regulates Growth, DNA Repair, and Genomic Instability in T-cell Acute Lymphoblastic Leukemia. Cancer Discov. 2017 11; 7(11):1336-1353.
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Malone CF, Stegmaier K. Scratching the Surface of Immunotherapeutic Targets in Neuroblastoma. Cancer Cell. 2017 09 11; 32(3):271-273.
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Weisberg EL, Puissant A, Stone R, Sattler M, Buhrlage SJ, Yang J, Manley PW, Meng C, Buonopane M, Daley JF, Lazo S, Wright R, Weinstock DM, Christie AL, Stegmaier K, Griffin JD. Characterization of midostaurin as a dual inhibitor of FLT3 and SYK and potentiation of FLT3 inhibition against FLT3-ITD-driven leukemia harboring activated SYK kinase. Oncotarget. 2017 Jul 06.
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Weisberg EL, Puissant A, Stone R, Sattler M, Buhrlage SJ, Yang J, Manley PW, Meng C, Buonopane M, Daley JF, Lazo S, Wright R, Weinstock DM, Christie AL, Stegmaier K, Griffin JD. Characterization of midostaurin as a dual inhibitor of FLT3 and SYK and potentiation of FLT3 inhibition against FLT3-ITD-driven leukemia harboring activated SYK kinase. Oncotarget. 2017 Aug 08; 8(32):52026-52044.
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Mohr S, Doebele C, Comoglio F, Berg T, Beck J, Bohnenberger H, Alexe G, Corso J, Ströbel P, Wachter A, Beissbarth T, Schnütgen F, Cremer A, Haetscher N, Göllner S, Rouhi A, Palmqvist L, Rieger MA, Schroeder T, Bönig H, Müller-Tidow C, Kuchenbauer F, Schütz E, Green AR, Urlaub H, Stegmaier K, Humphries RK, Serve H, Oellerich T. Hoxa9 and Meis1 Cooperatively Induce Addiction to Syk Signaling by Suppressing miR-146a in Acute Myeloid Leukemia. Cancer Cell. 2017 04 10; 31(4):549-562.e11.
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Fenouille N, Bassil CF, Ben-Sahra I, Benajiba L, Alexe G, Ramos A, Pikman Y, Conway AS, Burgess MR, Li Q, Luciano F, Auberger P, Galinsky I, DeAngelo DJ, Stone RM, Zhang Y, Perkins AS, Shannon K, Hemann MT, Puissant A, Stegmaier K. The creatine kinase pathway is a metabolic vulnerability in EVI1-positive acute myeloid leukemia. Nat Med. 2017 Mar; 23(3):301-313.
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Pikman Y, Alexe G, Roti G, Conway AS, Furman A, Lee ES, Place AE, Kim S, Saran C, Modiste R, Weinstock DM, Harris M, Kung AL, Silverman LB, Stegmaier K. Synergistic Drug Combinations with a CDK4/6 Inhibitor in T-cell Acute Lymphoblastic Leukemia. Clin Cancer Res. 2017 Feb 15; 23(4):1012-1024.
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Townsend EC, Murakami MA, Christodoulou A, Christie AL, Köster J, DeSouza TA, Morgan EA, Kallgren SP, Liu H, Wu SC, Plana O, Montero J, Stevenson KE, Rao P, Vadhi R, Andreeff M, Armand P, Ballen KK, Barzaghi-Rinaudo P, Cahill S, Clark RA, Cooke VG, Davids MS, DeAngelo DJ, Dorfman DM, Eaton H, Ebert BL, Etchin J, Firestone B, Fisher DC, Freedman AS, Galinsky IA, Gao H, Garcia JS, Garnache-Ottou F, Graubert TA, Gutierrez A, Halilovic E, Harris MH, Herbert ZT, Horwitz SM, Inghirami G, Intlekofer AM, Ito M, Izraeli S, Jacobsen ED, Jacobson CA, Jeay S, Jeremias I, Kelliher MA, Koch R, Konopleva M, Kopp N, Kornblau SM, Kung AL, Kupper TS, LeBoeuf NR, LaCasce AS, Lees E, Li LS, Look AT, Murakami M, Muschen M, Neuberg D, Ng SY, Odejide OO, Orkin SH, Paquette RR, Place AE, Roderick JE, Ryan JA, Sallan SE, Shoji B, Silverman LB, Soiffer RJ, Steensma DP, Stegmaier K, Stone RM, Tamburini J, Thorner AR, van Hummelen P, Wadleigh M, Wiesmann M, Weng AP, Wuerthner JU, Williams DA, Wollison BM, Lane AA, Letai A, Bertagnolli MM, Ritz J, Brown M, Long H, Aster JC, Shipp MA, Griffin JD, Weinstock DM. The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 07 11; 30(1):183.
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Hong AL, Tseng YY, Cowley GS, Jonas O, Cheah JH, Kynnap BD, Doshi MB, Oh C, Meyer SC, Church AJ, Gill S, Bielski CM, Keskula P, Imamovic A, Howell S, Kryukov GV, Clemons PA, Tsherniak A, Vazquez F, Crompton BD, Shamji AF, Rodriguez-Galindo C, Janeway KA, Roberts CW, Stegmaier K, van Hummelen P, Cima MJ, Langer RS, Garraway LA, Schreiber SL, Root DE, Hahn WC, Boehm JS. Integrated genetic and pharmacologic interrogation of rare cancers. Nat Commun. 2016 06 22; 7:11987.
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Pikman Y, Puissant A, Alexe G, Furman A, Chen LM, Frumm SM, Ross L, Fenouille N, Bassil CF, Lewis CA, Ramos A, Gould J, Stone RM, DeAngelo DJ, Galinsky I, Clish CB, Kung AL, Hemann MT, Vander Heiden MG, Banerji V, Stegmaier K. Targeting MTHFD2 in acute myeloid leukemia. J Exp Med. 2016 06 27; 213(7):1285-306.
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Aguirre AJ, Meyers RM, Weir BA, Vazquez F, Zhang CZ, Ben-David U, Cook A, Ha G, Harrington WF, Doshi MB, Kost-Alimova M, Gill S, Xu H, Ali LD, Jiang G, Pantel S, Lee Y, Goodale A, Cherniack AD, Oh C, Kryukov G, Cowley GS, Garraway LA, Stegmaier K, Roberts CW, Golub TR, Meyerson M, Root DE, Tsherniak A, Hahn WC. Genomic Copy Number Dictates a Gene-Independent Cell Response to CRISPR/Cas9 Targeting. Cancer Discov. 2016 08; 6(8):914-29.
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Wagner FF, Bishop JA, Gale JP, Shi X, Walk M, Ketterman J, Patnaik D, Barker D, Walpita D, Campbell AJ, Nguyen S, Lewis M, Ross L, Weïwer M, An WF, Germain AR, Nag PP, Metkar S, Kaya T, Dandapani S, Olson DE, Barbe AL, Lazzaro F, Sacher JR, Cheah JH, Fei D, Perez J, Munoz B, Palmer M, Stegmaier K, Schreiber SL, Scolnick E, Zhang YL, Haggarty SJ, Holson EB, Pan JQ. Inhibitors of Glycogen Synthase Kinase 3 with Exquisite Kinome-Wide Selectivity and Their Functional Effects. ACS Chem Biol. 2016 07 15; 11(7):1952-63.
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Townsend EC, Murakami MA, Christodoulou A, Christie AL, Köster J, DeSouza TA, Morgan EA, Kallgren SP, Liu H, Wu SC, Plana O, Montero J, Stevenson KE, Rao P, Vadhi R, Andreeff M, Armand P, Ballen KK, Barzaghi-Rinaudo P, Cahill S, Clark RA, Cooke VG, Davids MS, DeAngelo DJ, Dorfman DM, Eaton H, Ebert BL, Etchin J, Firestone B, Fisher DC, Freedman AS, Galinsky IA, Gao H, Garcia JS, Garnache-Ottou F, Graubert TA, Gutierrez A, Halilovic E, Harris MH, Herbert ZT, Horwitz SM, Inghirami G, Intlekofer AM, Ito M, Izraeli S, Jacobsen ED, Jacobson CA, Jeay S, Jeremias I, Kelliher MA, Koch R, Konopleva M, Kopp N, Kornblau SM, Kung AL, Kupper TS, LeBoeuf NR, LaCasce AS, Lees E, Li LS, Look AT, Murakami M, Muschen M, Neuberg D, Ng SY, Odejide OO, Orkin SH, Paquette RR, Place AE, Roderick JE, Ryan JA, Sallan SE, Shoji B, Silverman LB, Soiffer RJ, Steensma DP, Stegmaier K, Stone RM, Tamburini J, Thorner AR, van Hummelen P, Wadleigh M, Wiesmann M, Weng AP, Wuerthner JU, Williams DA, Wollison BM, Lane AA, Letai A, Bertagnolli MM, Ritz J, Brown M, Long H, Aster JC, Shipp MA, Griffin JD, Weinstock DM. The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 04 11; 29(4):574-586.
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Lindblad O, Cordero E, Puissant A, Macaulay L, Ramos A, Kabir NN, Sun J, Vallon-Christersson J, Haraldsson K, Hemann MT, Borg Å, Levander F, Stegmaier K, Pietras K, Rönnstrand L, Kazi JU. Aberrant activation of the PI3K/mTOR pathway promotes resistance to sorafenib in AML. Oncogene. 2016 09 29; 35(39):5119-31.
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Kovar H, Amatruda J, Brunet E, Burdach S, Cidre-Aranaz F, de Alava E, Dirksen U, van der Ent W, Grohar P, Grünewald TG, Helman L, Houghton P, Iljin K, Korsching E, Ladanyi M, Lawlor E, Lessnick S, Ludwig J, Meltzer P, Metzler M, Mora J, Moriggl R, Nakamura T, Papamarkou T, Radic Sarikas B, Rédini F, Richter GH, Rossig C, Schadler K, Schäfer BW, Scotlandi K, Sheffield NC, Shelat A, Snaar-Jagalska E, Sorensen P, Stegmaier K, Stewart E, Sweet-Cordero A, Szuhai K, Tirado OM, Tirode F, Toretsky J, Tsafou K, Üren A, Zinovyev A, Delattre O. The second European interdisciplinary Ewing sarcoma research summit--A joint effort to deconstructing the multiple layers of a complex disease. Oncotarget. 2016 Feb 23; 7(8):8613-24.
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Harris MH, DuBois SG, Glade Bender JL, Kim A, Crompton BD, Parker E, Dumont IP, Hong AL, Guo D, Church A, Stegmaier K, Roberts CW, Shusterman S, London WB, MacConaill LE, Lindeman NI, Diller L, Rodriguez-Galindo C, Janeway KA. Multicenter Feasibility Study of Tumor Molecular Profiling to Inform Therapeutic Decisions in Advanced Pediatric Solid Tumors: The Individualized Cancer Therapy (iCat) Study. JAMA Oncol. 2016 Jan 28.
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Stieglitz E, Taylor-Weiner AN, Chang TY, Gelston LC, Wang YD, Mazor T, Esquivel E, Yu A, Seepo S, Olsen SR, Rosenberg M, Archambeault SL, Abusin G, Beckman K, Brown PA, Briones M, Carcamo B, Cooper T, Dahl GV, Emanuel PD, Fluchel MN, Goyal RK, Hayashi RJ, Hitzler J, Hugge C, Liu YL, Messinger YH, Mahoney DH, Monteleone P, Nemecek ER, Roehrs PA, Schore RJ, Stine KC, Takemoto CM, Toretsky JA, Costello JF, Olshen AB, Stewart C, Li Y, Ma J, Gerbing RB, Alonzo TA, Getz G, Gruber TA, Golub TR, Stegmaier K, Loh ML. Corrigendum: The genomic landscape of juvenile myelomonocytic leukemia. Nat Genet. 2016 Jan; 48(1):101.
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Neumann T, Benajiba L, Göring S, Stegmaier K, Schmidt B. Evaluation of Improved Glycogen Synthase Kinase-3a Inhibitors in Models of Acute Myeloid Leukemia. J Med Chem. 2015 Nov 25; 58(22):8907-19.
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Stieglitz E, Taylor-Weiner AN, Chang TY, Gelston LC, Wang YD, Mazor T, Esquivel E, Yu A, Seepo S, Olsen S, Rosenberg M, Archambeault SL, Abusin G, Beckman K, Brown PA, Briones M, Carcamo B, Cooper T, Dahl GV, Emanuel PD, Fluchel MN, Goyal RK, Hayashi RJ, Hitzler J, Hugge C, Liu YL, Messinger YH, Mahoney DH, Monteleone P, Nemecek ER, Roehrs PA, Schore RJ, Stine KC, Takemoto CM, Toretsky JA, Costello JF, Olshen AB, Stewart C, Li Y, Ma J, Gerbing RB, Alonzo TA, Getz G, Gruber T, Golub T, Stegmaier K, Loh ML. The genomic landscape of juvenile myelomonocytic leukemia. Nat Genet. 2015 Nov; 47(11):1326-1333.
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Kennedy AL, Vallurupalli M, Chen L, Crompton B, Cowley G, Vazquez F, Weir BA, Tsherniak A, Parasuraman S, Kim S, Alexe G, Stegmaier K. Functional, chemical genomic, and super-enhancer screening identify sensitivity to cyclin D1/CDK4 pathway inhibition in Ewing sarcoma. Oncotarget. 2015 Oct 06; 6(30):30178-93.
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Boros K, Puissant A, Back M, Alexe G, Bassil CF, Sinha P, Tholouli E, Stegmaier K, Byers RJ, Rodig SJ. Increased SYK activity is associated with unfavorable outcome among patients with acute myeloid leukemia. Oncotarget. 2015 Sep 22; 6(28):25575-87.
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Marques Howarth M, Simpson D, Ngok SP, Nieves B, Chen R, Siprashvili Z, Vaka D, Breese MR, Crompton BD, Alexe G, Hawkins DS, Jacobson D, Brunner AL, West R, Mora J, Stegmaier K, Khavari P, Sweet-Cordero EA. Long noncoding RNA EWSAT1-mediated gene repression facilitates Ewing sarcoma oncogenesis. J Clin Invest. 2014 Dec; 124(12):5275-90.
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Cowley GS, Weir BA, Vazquez F, Tamayo P, Scott JA, Rusin S, East-Seletsky A, Ali LD, Gerath WF, Pantel SE, Lizotte PH, Jiang G, Hsiao J, Tsherniak A, Dwinell E, Aoyama S, Okamoto M, Harrington W, Gelfand E, Green TM, Tomko MJ, Gopal S, Wong TC, Li H, Howell S, Stransky N, Liefeld T, Jang D, Bistline J, Meyers BH, Armstrong SA, Anderson KC, Stegmaier K, Reich M, Pellman D, Boehm JS, Mesirov JP, Golub TR, Root DE, Hahn WC. Erratum: Parallel genome-scale loss of function screens in 216 cancer cell lines for the identification of context-specific genetic dependencies. Sci Data. 2014; 1:140044.
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Moore NF, Azarova AM, Bhatnagar N, Ross KN, Drake LE, Frumm S, Liu QS, Christie AL, Sanda T, Chesler L, Kung AL, Gray NS, Stegmaier K, George RE. Molecular rationale for the use of PI3K/AKT/mTOR pathway inhibitors in combination with crizotinib in ALK-mutated neuroblastoma. Oncotarget. 2014 Sep 30; 5(18):8737-49.
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Cowley GS, Weir BA, Vazquez F, Tamayo P, Scott JA, Rusin S, East-Seletsky A, Ali LD, Gerath WF, Pantel SE, Lizotte PH, Jiang G, Hsiao J, Tsherniak A, Dwinell E, Aoyama S, Okamoto M, Harrington W, Gelfand E, Green TM, Tomko MJ, Gopal S, Wong TC, Wong TC, Li H, Howell S, Stransky N, Liefeld T, Jang D, Bistline J, Hill Meyers B, Armstrong SA, Anderson KC, Stegmaier K, Reich M, Pellman D, Boehm JS, Mesirov JP, Golub TR, Root DE, Hahn WC. Parallel genome-scale loss of function screens in 216 cancer cell lines for the identification of context-specific genetic dependencies. Sci Data. 2014; 1:140035.
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Crompton BD, Stewart C, Taylor-Weiner A, Alexe G, Kurek KC, Calicchio ML, Kiezun A, Carter SL, Shukla SA, Mehta SS, Thorner AR, de Torres C, Lavarino C, Suñol M, McKenna A, Sivachenko A, Cibulskis K, Lawrence MS, Stojanov P, Rosenberg M, Ambrogio L, Auclair D, Seepo S, Blumenstiel B, DeFelice M, Imaz-Rosshandler I, Schwarz-Cruz Y Celis A, Rivera MN, Rodriguez-Galindo C, Fleming MD, Golub TR, Getz G, Mora J, Stegmaier K. The genomic landscape of pediatric Ewing sarcoma. Cancer Discov. 2014 Nov; 4(11):1326-41.
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Roti G, Stegmaier K. New Approaches to Target T-ALL. Front Oncol. 2014; 4:170.
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Lane AA, Chapuy B, Lin CY, Tivey T, Li H, Townsend EC, van Bodegom D, Day TA, Wu SC, Liu H, Yoda A, Alexe G, Schinzel AC, Sullivan TJ, Malinge S, Taylor JE, Stegmaier K, Jaffe JD, Bustin M, te Kronnie G, Izraeli S, Harris MH, Stevenson KE, Neuberg D, Silverman LB, Sallan SE, Bradner JE, Hahn WC, Crispino JD, Pellman D, Weinstock DM. Triplication of a 21q22 region contributes to B cell transformation through HMGN1 overexpression and loss of histone H3 Lys27 trimethylation. Nat Genet. 2014 Jun; 46(6):618-23.
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Stegmaier K. Targeting Csnk1a1 in leukemia. J Exp Med. 2014 Apr 07; 211(4):594.
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Knoechel B, Roderick JE, Williamson KE, Zhu J, Lohr JG, Cotton MJ, Gillespie SM, Fernandez D, Ku M, Wang H, Piccioni F, Silver SJ, Jain M, Pearson D, Kluk MJ, Ott CJ, Shultz LD, Brehm MA, Greiner DL, Gutierrez A, Stegmaier K, Kung AL, Root DE, Bradner JE, Aster JC, Kelliher MA, Bernstein BE. An epigenetic mechanism of resistance to targeted therapy in T cell acute lymphoblastic leukemia. Nat Genet. 2014 Apr; 46(4):364-70.
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Puissant A, Fenouille N, Alexe G, Pikman Y, Bassil CF, Mehta S, Du J, Kazi JU, Luciano F, Rönnstrand L, Kung AL, Aster JC, Galinsky I, Stone RM, DeAngelo DJ, Hemann MT, Stegmaier K. SYK is a critical regulator of FLT3 in acute myeloid leukemia. Cancer Cell. 2014 Feb 10; 25(2):226-42.
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Liu S, Yin L, Stroopinsky D, Rajabi H, Puissant A, Stegmaier K, Avigan D, Kharbanda S, Kufe D, Stone R. MUC1-C oncoprotein promotes FLT3 receptor activation in acute myeloid leukemia cells. Blood. 2014 Jan 30; 123(5):734-42.
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Deangelo DJ, Neuberg D, Amrein PC, Berchuck J, Wadleigh M, Sirulnik LA, Galinsky I, Golub T, Stegmaier K, Stone RM. A phase II study of the EGFR inhibitor gefitinib in patients with acute myeloid leukemia. Leuk Res. 2014 Apr; 38(4):430-4.
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Lawrence MS, Stojanov P, Polak P, Kryukov GV, Cibulskis K, Sivachenko A, Carter SL, Stewart C, Mermel CH, Roberts SA, Kiezun A, Hammerman PS, McKenna A, Drier Y, Zou L, Ramos AH, Pugh TJ, Stransky N, Helman E, Kim J, Sougnez C, Ambrogio L, Nickerson E, Shefler E, Cortés ML, Auclair D, Saksena G, Voet D, Noble M, DiCara D, Lin P, Lichtenstein L, Heiman DI, Fennell T, Imielinski M, Hernandez B, Hodis E, Baca S, Dulak AM, Lohr J, Landau DA, Wu CJ, Melendez-Zajgla J, Hidalgo-Miranda A, Koren A, McCarroll SA, Mora J, Crompton B, Onofrio R, Parkin M, Winckler W, Ardlie K, Gabriel SB, Roberts CWM, Biegel JA, Stegmaier K, Bass AJ, Garraway LA, Meyerson M, Golub TR, Gordenin DA, Sunyaev S, Lander ES, Getz G. Mutational heterogeneity in cancer and the search for new cancer-associated genes. Nature. 2013 Jul 11; 499(7457):214-218.
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Miller PG, Al-Shahrour F, Hartwell KA, Chu LP, Järås M, Puram RV, Puissant A, Callahan KP, Ashton J, McConkey ME, Poveromo LP, Cowley GS, Kharas MG, Labelle M, Shterental S, Fujisaki J, Silberstein L, Alexe G, Al-Hajj MA, Shelton CA, Armstrong SA, Root DE, Scadden DT, Hynes RO, Mukherjee S, Stegmaier K, Jordan CT, Ebert BL. In Vivo RNAi screening identifies a leukemia-specific dependence on integrin beta 3 signaling. Cancer Cell. 2013 Jul 08; 24(1):45-58.
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Frumm SM, Fan ZP, Ross KN, Duvall JR, Gupta S, VerPlank L, Suh BC, Holson E, Wagner FF, Smith WB, Paranal RM, Bassil CF, Qi J, Roti G, Kung AL, Bradner JE, Tolliday N, Stegmaier K. Selective HDAC1/HDAC2 inhibitors induce neuroblastoma differentiation. Chem Biol. 2013 May 23; 20(5):713-25.
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Carnevale J, Ross L, Puissant A, Banerji V, Stone RM, DeAngelo DJ, Ross KN, Stegmaier K. SYK regulates mTOR signaling in AML. Leukemia. 2013 Nov; 27(11):2118-28.
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Crompton BD, Carlton AL, Thorner AR, Christie AL, Du J, Calicchio ML, Rivera MN, Fleming MD, Kohl NE, Kung AL, Stegmaier K. High-throughput tyrosine kinase activity profiling identifies FAK as a candidate therapeutic target in Ewing sarcoma. Cancer Res. 2013 May 01; 73(9):2873-83.
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Puissant A, Frumm SM, Alexe G, Bassil CF, Qi J, Chanthery YH, Nekritz EA, Zeid R, Gustafson WC, Greninger P, Garnett MJ, McDermott U, Benes CH, Kung AL, Weiss WA, Bradner JE, Stegmaier K. Targeting MYCN in neuroblastoma by BET bromodomain inhibition. Cancer Discov. 2013 Mar; 3(3):308-23.
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Roti G, Carlton A, Ross KN, Markstein M, Pajcini K, Su AH, Perrimon N, Pear WS, Kung AL, Blacklow SC, Aster JC, Stegmaier K. Complementary genomic screens identify SERCA as a therapeutic target in NOTCH1 mutated cancer. Cancer Cell. 2013 Mar 18; 23(3):390-405.
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Banerji V, Frumm SM, Ross KN, Li LS, Schinzel AC, Hahn CK, Kakoza RM, Chow KT, Ross L, Alexe G, Tolliday N, Inguilizian H, Galinsky I, Stone RM, DeAngelo DJ, Roti G, Aster JC, Hahn WC, Kung AL, Stegmaier K. The intersection of genetic and chemical genomic screens identifies GSK-3a as a target in human acute myeloid leukemia. J Clin Invest. 2012 Mar; 122(3):935-47.
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Roti G, Stegmaier K. Genetic and proteomic approaches to identify cancer drug targets. Br J Cancer. 2012 Jan 17; 106(2):254-61.
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Roti G, Stegmaier K. Targeting NOTCH1 in hematopoietic malignancy. Crit Rev Oncog. 2011; 16(1-2):103-15.
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Stegmaier K. Chemical genomics and combo therapy. Blood. 2010 Dec 23; 116(26):5786-8.
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Aste-Amézaga M, Zhang N, Lineberger JE, Arnold BA, Toner TJ, Gu M, Huang L, Vitelli S, Vo KT, Haytko P, Zhao JZ, Baleydier F, L'Heureux S, Wang H, Gordon WR, Thoryk E, Andrawes MB, Tiyanont K, Stegmaier K, Roti G, Ross KN, Franlin LL, Wang H, Wang F, Chastain M, Bett AJ, Audoly LP, Aster JC, Blacklow SC, Huber HE. Characterization of Notch1 antibodies that inhibit signaling of both normal and mutated Notch1 receptors. PLoS One. 2010 Feb 08; 5(2):e9094.
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Hahn CK, Berchuck JE, Ross KN, Kakoza RM, Clauser K, Schinzel AC, Ross L, Galinsky I, Davis TN, Silver SJ, Root DE, Stone RM, DeAngelo DJ, Carroll M, Hahn WC, Carr SA, Golub TR, Kung AL, Stegmaier K. Proteomic and genetic approaches identify Syk as an AML target. Cancer Cell. 2009 Oct 06; 16(4):281-94.
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Corsello SM, Roti G, Ross KN, Chow KT, Galinsky I, DeAngelo DJ, Stone RM, Kung AL, Golub TR, Stegmaier K. Identification of AML1-ETO modulators by chemical genomics. Blood. 2009 Jun 11; 113(24):6193-205.
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Stegmaier K. Genomic approaches to small molecule discovery. Leukemia. 2009 Jul; 23(7):1226-35.
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DuBois SG, Krailo MD, Lessnick SL, Smith R, Chen Z, Marina N, Grier HE, Stegmaier K. Phase II study of intermediate-dose cytarabine in patients with relapsed or refractory Ewing sarcoma: a report from the Children's Oncology Group. Pediatr Blood Cancer. 2009 Mar; 52(3):324-7.
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Haining WN, Angelosanto J, Brosnahan K, Ross K, Hahn C, Russell K, Drury L, Norton S, Nadler L, Stegmaier K. High-throughput gene expression profiling of memory differentiation in primary human T cells. BMC Immunol. 2008 Aug 01; 9:44.
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Hahn CK, Ross KN, Warrington IM, Mazitschek R, Kanegai CM, Wright RD, Kung AL, Golub TR, Stegmaier K. Expression-based screening identifies the combination of histone deacetylase inhibitors and retinoids for neuroblastoma differentiation. Proc Natl Acad Sci U S A. 2008 Jul 15; 105(28):9751-6.
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Febbo PG, Mulligan MG, Slonina DA, Stegmaier K, Di Vizio D, Martinez PR, Loda M, Taylor SC. Literature Lab: a method of automated literature interrogation to infer biology from microarray analysis. BMC Genomics. 2007 Dec 18; 8:461.
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Stegmaier K, Wong JS, Ross KN, Chow KT, Peck D, Wright RD, Lessnick SL, Kung AL, Golub TR. Signature-based small molecule screening identifies cytosine arabinoside as an EWS/FLI modulator in Ewing sarcoma. PLoS Med. 2007 Apr; 4(4):e122.
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Hieronymus H, Lamb J, Ross KN, Peng XP, Clement C, Rodina A, Nieto M, Du J, Stegmaier K, Raj SM, Maloney KN, Clardy J, Hahn WC, Chiosis G, Golub TR. Gene expression signature-based chemical genomic prediction identifies a novel class of HSP90 pathway modulators. Cancer Cell. 2006 Oct; 10(4):321-30.
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Peck D, Crawford ED, Ross KN, Stegmaier K, Golub TR, Lamb J. A method for high-throughput gene expression signature analysis. Genome Biol. 2006; 7(7):R61.
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Stegmaier K. Genomic approaches in acute leukemia. Best Pract Res Clin Haematol. 2006; 19(2):263-8.
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Xu K, Guidez F, Glasow A, Chung D, Petrie K, Stegmaier K, Wang KK, Zhang J, Jing Y, Zelent A, Waxman S. Benzodithiophenes potentiate differentiation of acute promyelocytic leukemia cells by lowering the threshold for ligand-mediated corepressor/coactivator exchange with retinoic acid receptor alpha and enhancing changes in all-trans-retinoic acid-regulated gene expression. Cancer Res. 2005 Sep 01; 65(17):7856-65.
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Stegmaier K, Corsello SM, Ross KN, Wong JS, Deangelo DJ, Golub TR. Gefitinib induces myeloid differentiation of acute myeloid leukemia. Blood. 2005 Oct 15; 106(8):2841-8.
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Stegmaier K, Ross KN, Colavito SA, O'Malley S, Stockwell BR, Golub TR. Gene expression-based high-throughput screening(GE-HTS) and application to leukemia differentiation. Nat Genet. 2004 Mar; 36(3):257-63.
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Golub TR, Barker GF, Stegmaier K, Gilliland DG. The TEL gene contributes to the pathogenesis of myeloid and lymphoid leukemias by diverse molecular genetic mechanisms. Curr Top Microbiol Immunol. 1997; 220:67-79.
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McLean TW, Ringold S, Neuberg D, Stegmaier K, Tantravahi R, Ritz J, Koeffler HP, Takeuchi S, Janssen JW, Seriu T, Bartram CR, Sallan SE, Gilliland DG, Golub TR. TEL/AML-1 dimerizes and is associated with a favorable outcome in childhood acute lymphoblastic leukemia. Blood. 1996 Dec 01; 88(11):4252-8.
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Golub TR, McLean T, Stegmaier K, Carroll M, Tomasson M, Gilliland DG. The TEL gene and human leukemia. Biochim Biophys Acta. 1996 Aug 08; 1288(1):M7-10.
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Stegmaier K, Takeuchi S, Golub TR, Bohlander SK, Bartram CR, Koeffler HP. Mutational analysis of the candidate tumor suppressor genes TEL and KIP1 in childhood acute lymphoblastic leukemia. Cancer Res. 1996 Mar 15; 56(6):1413-7.
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Golub TR, Barker GF, Stegmaier K, Gilliland DG. Involvement of the TEL gene in hematologic malignancy by diverse molecular genetic mechanisms. Curr Top Microbiol Immunol. 1996; 211:279-88.
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Stegmaier K, Pendse S, Barker GF, Bray-Ward P, Ward DC, Montgomery KT, Krauter KS, Reynolds C, Sklar J, Donnelly M, et al. Frequent loss of heterozygosity at the TEL gene locus in acute lymphoblastic leukemia of childhood. Blood. 1995 Jul 01; 86(1):38-44.
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