Kimberly Stegmaier, MD

Kimberly Stegmaier, MD

Pediatric Hematology/Oncology

Contact Information




888-733-4662 (New Pediatric Patients)
617-632-3270 (Established Pediatric Patients)


Kimberly Stegmaier, MD

Kimberly Stegmaier, M.D., is a Professor of Pediatrics at Harvard Medical School, a Principal Investigator in Pediatric Oncology at the Dana-Farber Cancer Institute (DFCI), and an Attending 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.



Co-Director of the Hematologic Malignancy Center (Basic and Translational Research)
Ted Williams Chair
Professor of Pediatrics, Harvard Medical School

Clinical Interests

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

Board Certification

  • Pediatric Hematology/Oncology, 2002


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


  • 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

  • Helen Mentor, Dana-Farber Cancer Institute's Helen Gurley Brown (HGB) Presidential Initiative, 2020-2022


    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.



      • DHODH: a promising target in the treatment of T-cell acute lymphoblastic leukemia. Blood Adv. 2023 Nov 14; 7(21):6685-6701. View in: Pubmed

      • EZH2 synergizes with BRD4-NUT to drive NUT carcinoma growth through silencing of key tumor suppressor genes. Cancer Res. 2023 Sep 25. View in: Pubmed

      • Integration of genomic sequencing drives therapeutic targeting of PDGFRA in T-cell acute lymphoblastic leukemia/lymphoblastic lymphoma. Clin Cancer Res. 2023 Sep 19. View in: Pubmed

      • PPM1D modulates hematopoietic cell fitness and response to DNA damage and is a therapeutic target in myeloid malignancy. Blood. 2023 Aug 18. View in: Pubmed

      • EZH2 synergizes with BRD4-NUT to drive NUT carcinoma growth through silencing of key tumor suppressor genes. bioRxiv. 2023 Aug 16. View in: Pubmed

      • Phase 2 trial of palbociclib and ganitumab in patients with relapsed Ewing sarcoma. Cancer Med. 2023 07; 12(14):15207-15216. View in: Pubmed

      • Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors. bioRxiv. 2023 Apr 07. View in: Pubmed

      • Highly connected 3D chromatin networks established by an oncogenic fusion protein shape tumor cell identity. Sci Adv. 2023 03 31; 9(13):eabo3789. View in: Pubmed

      • Leukemia core transcriptional circuitry is a sparsely interconnected hierarchy stabilized by incoherent feed-forward loops. bioRxiv. 2023 Mar 15. View in: Pubmed

      • Depletion of creatine phosphagen energetics with a covalent creatine kinase inhibitor. Nat Chem Biol. 2023 07; 19(7):815-824. View in: Pubmed

      • Mitochondrial fusion is a therapeutic vulnerability of acute myeloid leukemia. Leukemia. 2023 04; 37(4):765-775. View in: Pubmed

      • The ETS transcription factor ETV6 constrains the transcriptional activity of EWS-FLI to promote Ewing sarcoma. Nat Cell Biol. 2023 02; 25(2):285-297. View in: Pubmed

      • Transcriptional Antagonism by CDK8 Inhibition Improves Therapeutic Efficacy of MEK Inhibitors. Cancer Res. 2023 01 18; 83(2):285-300. View in: Pubmed

      • BAF Complex Maintains Glioma Stem Cells in Pediatric H3K27M Glioma. Cancer Discov. 2022 12 02; 12(12):2880-2905. View in: Pubmed

      • Adverse prognostic impact of the loss of STAG2 protein expression in patients with newly diagnosed localised Ewing sarcoma: A report from the Children's Oncology Group. Br J Cancer. 2022 Dec; 127(12):2220-2226. View in: Pubmed

      • VRK1 as a synthetic lethal target in VRK2 promoter-methylated cancers of the nervous system. JCI Insight. 2022 10 10; 7(19). View in: Pubmed

      • Transcriptional Plasticity Drives Leukemia Immune Escape. Blood Cancer Discov. 2022 09 06; 3(5):394-409. View in: Pubmed

      • Transition to a mesenchymal state in neuroblastoma confers resistance to anti-GD2 antibody via reduced expression of ST8SIA1. Nat Cancer. 2022 08; 3(8):976-993. View in: Pubmed

      • Unleashing Cell-Intrinsic Inflammation as a Strategy to Kill AML Blasts. Cancer Discov. 2022 07 06; 12(7):1760-1781. View in: Pubmed

      • Mitochondrial Dysfunction Is a Driver of SP-2509 Drug Resistance in Ewing Sarcoma. Mol Cancer Res. 2022 07 06; 20(7):1035-1046. View in: Pubmed

      • Identification of an Epi-metabolic dependency on EHMT2/G9a in T-cell acute lymphoblastic leukemia. Cell Death Dis. 2022 Jun 17; 13(6):551. View in: Pubmed

      • IKAROS and MENIN coordinate therapeutically actionable leukemogenic gene expression in MLL-r acute myeloid leukemia. Nat Cancer. 2022 05; 3(5):595-613. View in: Pubmed

      • Cystine uptake inhibition potentiates front-line therapies in acute myeloid leukemia. Leukemia. 2022 06; 36(6):1585-1595. View in: Pubmed

      • The proteogenomic subtypes of acute myeloid leukemia. Cancer Cell. 2022 03 14; 40(3):301-317.e12. View in: Pubmed

      • A distinct core regulatory module enforces oncogene expression in KMT2A-rearranged leukemia. Genes Dev. 2022 03 01; 36(5-6):368-389. View in: Pubmed

      • EP300 Selectively Controls the Enhancer Landscape of MYCN-Amplified Neuroblastoma. Cancer Discov. 2022 03 01; 12(3):730-751. View in: Pubmed

      • SHMT2 inhibition disrupts the TCF3 transcriptional survival program in Burkitt lymphoma. Blood. 2022 01 27; 139(4):538-553. View in: Pubmed

      • An In Vivo CRISPR Screening Platform for Prioritizing Therapeutic Targets in AML. Cancer Discov. 2022 02; 12(2):432-449. View in: Pubmed

      • Targeting serine hydroxymethyltransferases 1 and 2 for T-cell acute lymphoblastic leukemia therapy. Leukemia. 2022 02; 36(2):348-360. View in: Pubmed

      • TRIM8 modulates the EWS/FLI oncoprotein to promote survival in Ewing sarcoma. Cancer Cell. 2021 09 13; 39(9):1262-1278.e7. View in: Pubmed

      • Synthetic Lethal Interaction between the ESCRT Paralog Enzymes VPS4A and VPS4B in Cancers Harboring Loss of Chromosome 18q or 16q. Cell Rep. 2021 Jul 13; 36(2):109367. View in: Pubmed

      • STAG2 loss rewires oncogenic and developmental programs to promote metastasis in Ewing sarcoma. Cancer Cell. 2021 06 14; 39(6):827-844.e10. View in: Pubmed

      • The synergy of BET inhibitors with aurora A kinase inhibitors in MYCN-amplified neuroblastoma is heightened with functional TP53. Neoplasia. 2021 06; 23(6):624-633. View in: Pubmed

      • Gene Fusions Create Partner and Collateral Dependencies Essential to Cancer Cell Survival. Cancer Res. 2021 08 01; 81(15):3971-3984. View in: Pubmed

      • Selective Modulation of a Pan-Essential Protein as a Therapeutic Strategy in Cancer. Cancer Discov. 2021 09; 11(9):2282-2299. View in: Pubmed

      • Structure-activity relationship study of THZ531 derivatives enables the discovery of BSJ-01-175 as a dual CDK12/13 covalent inhibitor with efficacy in Ewing sarcoma. Eur J Med Chem. 2021 Oct 05; 221:113481. View in: Pubmed

      • Targeting acute myeloid leukemia dependency on VCP-mediated DNA repair through a selective second-generation small-molecule inhibitor. Sci Transl Med. 2021 03 31; 13(587). View in: Pubmed

      • RAD21 is a driver of chromosome 8 gain in Ewing sarcoma to mitigate replication stress. Genes Dev. 2021 04 01; 35(7-8):556-572. View in: Pubmed

      • A first-generation pediatric cancer dependency map. Nat Genet. 2021 04; 53(4):529-538. View in: Pubmed

      • Bromodomain and extra-terminal inhibitors-A consensus prioritisation after the Paediatric Strategy Forum for medicinal product development of epigenetic modifiers in children-ACCELERATE. Eur J Cancer. 2021 03; 146:115-124. View in: Pubmed

      • Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory, or High-Risk Leukemias: A Report from the LEAP Consortium. Cancer Discov. 2021 06; 11(6):1424-1439. View in: Pubmed

      • Network-based systems pharmacology reveals heterogeneity in LCK and BCL2 signaling and therapeutic sensitivity of T-cell acute lymphoblastic leukemia. Nat Cancer. 2021 03; 2(3):284-299. View in: Pubmed

      • Synthetic Lethal Interaction between the ESCRT Paralog Enzymes VPS4A and VPS4B in Cancers Harboring Loss of Chromosome 18q or 16q. Cell Rep. 2020 12 15; 33(11):108493. View in: Pubmed

      • Paediatric Strategy Forum for medicinal product development of epigenetic modifiers for children: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration. Eur J Cancer. 2020 11; 139:135-148. View in: Pubmed

      • Rapid and direct control of target protein levels with VHL-recruiting dTAG molecules. Nat Commun. 2020 09 18; 11(1):4687. View in: Pubmed

      • The Folate Cycle Enzyme MTHFR Is a Critical Regulator of Cell Response to MYC-Targeting Therapies. Cancer Discov. 2020 12; 10(12):1894-1911. View in: Pubmed

      • Blockade of Oncogenic NOTCH1 with the SERCA Inhibitor CAD204520 in T Cell Acute Lymphoblastic Leukemia. Cell Chem Biol. 2020 06 18; 27(6):678-697.e13. View in: Pubmed

      • Targeting EZH2 for the treatment of hepatosplenic T-cell lymphoma. Blood Adv. 2020 04 14; 4(7):1265-1269. View in: Pubmed

      • Selective USP7 inhibition elicits cancer cell killing through a p53-dependent mechanism. Sci Rep. 2020 03 24; 10(1):5324. View in: Pubmed

      • Targeting DUBs to degrade oncogenic proteins. Br J Cancer. 2020 04; 122(8):1121-1123. View in: Pubmed

      • Preclinical efficacy for a novel tyrosine kinase inhibitor, ArQule 531 against acute myeloid leukemia. J Hematol Oncol. 2020 01 28; 13(1):8. View in: Pubmed

      • STRIPAK directs PP2A activity toward MAP4K4 to promote oncogenic transformation of human cells. Elife. 2020 Jan 08; 9. View in: Pubmed

      • Salt-inducible kinase inhibition suppresses acute myeloid leukemia progression in vivo. Blood. 2020 01 02; 135(1):56-70. View in: Pubmed

      • Resistance Mechanisms to SYK Inhibition in Acute Myeloid Leukemia. Cancer Discov. 2020 02; 10(2):214-231. View in: Pubmed

      • EWS-FLI1 modulated alternative splicing of ARID1A reveals novel oncogenic function through the BAF complex. Nucleic Acids Res. 2019 10 10; 47(18):9619-9636. View in: Pubmed

      • Small-Molecule and CRISPR Screening Converge to Reveal Receptor Tyrosine Kinase Dependencies in Pediatric Rhabdoid Tumors. Cell Rep. 2019 08 27; 28(9):2331-2344.e8. View in: Pubmed

      • Single-cell cloning of human T-cell lines reveals clonal variation in cell death responses to chemotherapeutics. Cancer Genet. 2019 09; 237:69-77. View in: Pubmed

      • Neuronal differentiation and cell-cycle programs mediate response to BET-bromodomain inhibition in MYC-driven medulloblastoma. Nat Commun. 2019 06 03; 10(1):2400. View in: Pubmed

      • Targeting chromatin complexes in fusion protein-driven malignancies. Nat Rev Cancer. 2019 05; 19(5):255-269. View in: Pubmed

      • Therapeutic discovery for marrow failure with MDS predisposition using pluripotent stem cells. JCI Insight. 2019 04 30; 5. View in: Pubmed

      • Synthetic Lethality of Wnt Pathway Activation and Asparaginase in Drug-Resistant Acute Leukemias. Cancer Cell. 2019 04 15; 35(4):664-676.e7. View in: Pubmed

      • High-throughput Chemical Screening Identifies Focal Adhesion Kinase and Aurora Kinase B Inhibition as a Synergistic Treatment Combination in Ewing Sarcoma. Clin Cancer Res. 2019 07 15; 25(14):4552-4566. View in: Pubmed

      • Correction: Detection of circulating tumour DNA is associated with inferior outcomes in Ewing sarcoma and osteosarcoma: a report from the Children's Oncology Group. Br J Cancer. 2019 Apr; 120(8):869. View in: Pubmed

      • Ushering in the next generation of precision trials for pediatric cancer. Science. 2019 Mar 15; 363(6432):1175-1181. View in: Pubmed

      • Virtual Screening Identifies Irreversible FMS-like Tyrosine Kinase 3 Inhibitors with Activity toward Resistance-Conferring Mutations. J Med Chem. 2019 03 14; 62(5):2428-2446. View in: Pubmed

      • MDM2 and MDM4 Are Therapeutic Vulnerabilities in Malignant Rhabdoid Tumors. Cancer Res. 2019 05 01; 79(9):2404-2414. View in: Pubmed

      • The kinases IKBKE and TBK1 regulate MYC-dependent survival pathways through YB-1 in AML and are targets for therapy. Blood Adv. 2018 12 11; 2(23):3428-3442. View in: Pubmed

      • Resistance to Epigenetic-Targeted Therapy Engenders Tumor Cell Vulnerabilities Associated with Enhancer Remodeling. Cancer Cell. 2018 12 10; 34(6):922-938.e7. View in: Pubmed

      • A Combination CDK4/6 and IGF1R Inhibitor Strategy for Ewing Sarcoma. Clin Cancer Res. 2019 02 15; 25(4):1343-1357. View in: Pubmed

      • Creatine kinase pathway inhibition alters GSK3 and WNT signaling in EVI1-positive AML. Leukemia. 2019 03; 33(3):800-804. View in: Pubmed

      • TRPS1 Is a Lineage-Specific Transcriptional Dependency in Breast Cancer. Cell Rep. 2018 10 30; 25(5):1255-1267.e5. View in: Pubmed

      • Precision Targeting of BFL-1/A1 and an ATM Co-dependency in Human Cancer. Cell Rep. 2018 09 25; 24(13):3393-3403.e5. View in: Pubmed

      • Comparative proteomics reveals a diagnostic signature for pulmonary head-and-neck cancer metastasis. EMBO Mol Med. 2018 09; 10(9). View in: Pubmed

      • Detection of circulating tumour DNA is associated with inferior outcomes in Ewing sarcoma and osteosarcoma: a report from the Children's Oncology Group. Br J Cancer. 2018 08; 119(5):615-621. View in: Pubmed

      • Selective gene dependencies in MYCN-amplified neuroblastoma include the core transcriptional regulatory circuitry. Nat Genet. 2018 09; 50(9):1240-1246. View in: Pubmed

      • Targeted therapy for fusion-driven high-risk acute leukemia. Blood. 2018 09 20; 132(12):1241-1247. View in: Pubmed

      • Genome-scale CRISPR-Cas9 screen identifies druggable dependencies in TP53 wild-type Ewing sarcoma. J Exp Med. 2018 08 06; 215(8):2137-2155. View in: Pubmed

      • KLF5 controls glutathione metabolism to suppress p190-BCR-ABL+ B-cell lymphoblastic leukemia. Oncotarget. 2018 Jul 03; 9(51):29665-29679. View in: Pubmed

      • Author Correction: EWS-FLI1 increases transcription to cause R-loops and block BRCA1 repair in Ewing sarcoma. Nature. 2018 07; 559(7715):E11. View in: Pubmed

      • Phase I trial of the mTOR inhibitor everolimus in combination with multi-agent chemotherapy in relapsed childhood acute lymphoblastic leukemia. Pediatr Blood Cancer. 2018 07; 65(7):e27062. View in: Pubmed

      • Bepridil exhibits anti-leukemic activity associated with NOTCH1 pathway inhibition in chronic lymphocytic leukemia. Int J Cancer. 2018 08 15; 143(4):958-970. View in: Pubmed

      • EWS-FLI1 increases transcription to cause R-loops and block BRCA1 repair in Ewing sarcoma. Nature. 2018 03 15; 555(7696):387-391. View in: Pubmed

      • 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 03 07; 10(431). View in: Pubmed

      • EWS/FLI Confers Tumor Cell Synthetic Lethality to CDK12 Inhibition in Ewing Sarcoma. Cancer Cell. 2018 02 12; 33(2):202-216.e6. View in: Pubmed

      • CRISPR-Cas9 screen reveals a MYCN-amplified neuroblastoma dependency on EZH2. J Clin Invest. 2018 01 02; 128(1):446-462. View in: Pubmed

      • Leukemia-specific delivery of mutant NOTCH1 targeted therapy. J Exp Med. 2018 01 02; 215(1):197-216. View in: Pubmed

      • Computational correction of copy number effect improves specificity of CRISPR-Cas9 essentiality screens in cancer cells. Nat Genet. 2017 Dec; 49(12):1779-1784. View in: Pubmed

      • Identification of an allosteric benzothiazolopyrimidone inhibitor of the oncogenic protein tyrosine phosphatase SHP2. Bioorg Med Chem. 2017 12 15; 25(24):6479-6485. View in: Pubmed

      • TOX Regulates Growth, DNA Repair, and Genomic Instability in T-cell Acute Lymphoblastic Leukemia. Cancer Discov. 2017 11; 7(11):1336-1353. View in: Pubmed

      • Scratching the Surface of Immunotherapeutic Targets in Neuroblastoma. Cancer Cell. 2017 09 11; 32(3):271-273. View in: Pubmed

      • 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. View in: Pubmed

      • 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. View in: Pubmed

      • 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. View in: Pubmed

      • The creatine kinase pathway is a metabolic vulnerability in EVI1-positive acute myeloid leukemia. Nat Med. 2017 Mar; 23(3):301-313. View in: Pubmed

      • Synergistic Drug Combinations with a CDK4/6 Inhibitor in T-cell Acute Lymphoblastic Leukemia. Clin Cancer Res. 2017 Feb 15; 23(4):1012-1024. View in: Pubmed

      • The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 07 11; 30(1):183. View in: Pubmed

      • Integrated genetic and pharmacologic interrogation of rare cancers. Nat Commun. 2016 06 22; 7:11987. View in: Pubmed

      • Targeting MTHFD2 in acute myeloid leukemia. J Exp Med. 2016 06 27; 213(7):1285-306. View in: Pubmed

      • Genomic Copy Number Dictates a Gene-Independent Cell Response to CRISPR/Cas9 Targeting. Cancer Discov. 2016 08; 6(8):914-29. View in: Pubmed

      • 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. View in: Pubmed

      • 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 May 01; 2(5):608-615. View in: Pubmed

      • The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 04 11; 29(4):574-586. View in: Pubmed

      • Aberrant activation of the PI3K/mTOR pathway promotes resistance to sorafenib in AML. Oncogene. 2016 09 29; 35(39):5119-31. View in: Pubmed

      • 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. View in: Pubmed

      • Corrigendum: The genomic landscape of juvenile myelomonocytic leukemia. Nat Genet. 2016 Jan; 48(1):101. View in: Pubmed

      • Evaluation of Improved Glycogen Synthase Kinase-3a Inhibitors in Models of Acute Myeloid Leukemia. J Med Chem. 2015 Nov 25; 58(22):8907-19. View in: Pubmed

      • The genomic landscape of juvenile myelomonocytic leukemia. Nat Genet. 2015 Nov; 47(11):1326-1333. View in: Pubmed

      • 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. View in: Pubmed

      • Increased SYK activity is associated with unfavorable outcome among patients with acute myeloid leukemia. Oncotarget. 2015 Sep 22; 6(28):25575-87. View in: Pubmed

      • Long noncoding RNA EWSAT1-mediated gene repression facilitates Ewing sarcoma oncogenesis. J Clin Invest. 2014 Dec; 124(12):5275-90. View in: Pubmed

      • 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. View in: Pubmed

      • 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. View in: Pubmed

      • 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. View in: Pubmed

      • The genomic landscape of pediatric Ewing sarcoma. Cancer Discov. 2014 Nov; 4(11):1326-41. View in: Pubmed

      • New Approaches to Target T-ALL. Front Oncol. 2014; 4:170. View in: Pubmed

      • 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. View in: Pubmed

      • Targeting Csnk1a1 in leukemia. J Exp Med. 2014 Apr 07; 211(4):594. View in: Pubmed

      • An epigenetic mechanism of resistance to targeted therapy in T cell acute lymphoblastic leukemia. Nat Genet. 2014 Apr; 46(4):364-70. View in: Pubmed

      • SYK is a critical regulator of FLT3 in acute myeloid leukemia. Cancer Cell. 2014 Feb 10; 25(2):226-42. View in: Pubmed

      • MUC1-C oncoprotein promotes FLT3 receptor activation in acute myeloid leukemia cells. Blood. 2014 Jan 30; 123(5):734-42. View in: Pubmed

      • A phase II study of the EGFR inhibitor gefitinib in patients with acute myeloid leukemia. Leuk Res. 2014 Apr; 38(4):430-4. View in: Pubmed

      • Mutational heterogeneity in cancer and the search for new cancer-associated genes. Nature. 2013 Jul 11; 499(7457):214-218. View in: Pubmed

      • In Vivo RNAi screening identifies a leukemia-specific dependence on integrin beta 3 signaling. Cancer Cell. 2013 Jul 08; 24(1):45-58. View in: Pubmed

      • Selective HDAC1/HDAC2 inhibitors induce neuroblastoma differentiation. Chem Biol. 2013 May 23; 20(5):713-25. View in: Pubmed

      • SYK regulates mTOR signaling in AML. Leukemia. 2013 Nov; 27(11):2118-28. View in: Pubmed

      • 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. View in: Pubmed

      • Complementary genomic screens identify SERCA as a therapeutic target in NOTCH1 mutated cancer. Cancer Cell. 2013 Mar 18; 23(3):390-405. View in: Pubmed

      • Targeting MYCN in neuroblastoma by BET bromodomain inhibition. Cancer Discov. 2013 Mar; 3(3):308-23. View in: Pubmed

      • 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. View in: Pubmed

      • Genetic and proteomic approaches to identify cancer drug targets. Br J Cancer. 2012 Jan 17; 106(2):254-61. View in: Pubmed

      • Targeting NOTCH1 in hematopoietic malignancy. Crit Rev Oncog. 2011; 16(1-2):103-15. View in: Pubmed

      • Chemical genomics and combo therapy. Blood. 2010 Dec 23; 116(26):5786-8. View in: Pubmed

      • Characterization of Notch1 antibodies that inhibit signaling of both normal and mutated Notch1 receptors. PLoS One. 2010 Feb 08; 5(2):e9094. View in: Pubmed

      • Proteomic and genetic approaches identify Syk as an AML target. Cancer Cell. 2009 Oct 06; 16(4):281-94. View in: Pubmed

      • Identification of AML1-ETO modulators by chemical genomics. Blood. 2009 Jun 11; 113(24):6193-205. View in: Pubmed

      • Genomic approaches to small molecule discovery. Leukemia. 2009 Jul; 23(7):1226-35. View in: Pubmed

      • 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. View in: Pubmed

      • High-throughput gene expression profiling of memory differentiation in primary human T cells. BMC Immunol. 2008 Aug 01; 9:44. View in: Pubmed

      • 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. View in: Pubmed

      • Literature Lab: a method of automated literature interrogation to infer biology from microarray analysis. BMC Genomics. 2007 Dec 18; 8:461. View in: Pubmed

      • Signature-based small molecule screening identifies cytosine arabinoside as an EWS/FLI modulator in Ewing sarcoma. PLoS Med. 2007 Apr; 4(4):e122. View in: Pubmed

      • Gene expression signature-based chemical genomic prediction identifies a novel class of HSP90 pathway modulators. Cancer Cell. 2006 Oct; 10(4):321-30. View in: Pubmed

      • A method for high-throughput gene expression signature analysis. Genome Biol. 2006; 7(7):R61. View in: Pubmed

      • Genomic approaches in acute leukemia. Best Pract Res Clin Haematol. 2006; 19(2):263-8. View in: Pubmed

      • 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. View in: Pubmed

      • Gefitinib induces myeloid differentiation of acute myeloid leukemia. Blood. 2005 Oct 15; 106(8):2841-8. View in: Pubmed

      • Gene expression-based high-throughput screening(GE-HTS) and application to leukemia differentiation. Nat Genet. 2004 Mar; 36(3):257-63. View in: Pubmed

      • 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. View in: Pubmed

      • TEL/AML-1 dimerizes and is associated with a favorable outcome in childhood acute lymphoblastic leukemia. Blood. 1996 Dec 01; 88(11):4252-8. View in: Pubmed

      • The TEL gene and human leukemia. Biochim Biophys Acta. 1996 Aug 08; 1288(1):M7-10. View in: Pubmed

      • 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. View in: Pubmed

      • Involvement of the TEL gene in hematologic malignancy by diverse molecular genetic mechanisms. Curr Top Microbiol Immunol. 1996; 211:279-88. View in: Pubmed

      • Frequent loss of heterozygosity at the TEL gene locus in acute lymphoblastic leukemia of childhood. Blood. 1995 Jul 01; 86(1):38-44. View in: Pubmed


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