Kimberly Stegmaier | Education
Medical School
Harvard Medical School
Boston, MA
Residency
Pediatrics
Boston Combined Residency Program (BCRP)
Boston, MA
Residency
Pediatrics
Boston Children's Hospital
Boston, MA
Fellowship
Pediatric Hematology -Oncology
Boston Children's Hospital/Dana-Farber Cancer Institute
Boston, MA
Kimberly Stegmaier | Publications
Prospects for understanding and exploiting the consequences of hyperactivation lethality. Trends Cancer. 2025 May 19. View Prospects for understanding and exploiting the consequences of hyperactivation lethality. Abstract
Inflammatory signaling pathways play a role in SYK inhibitor resistant AML. Sci Rep. 2025 Apr 05; 15(1):11673. View Inflammatory signaling pathways play a role in SYK inhibitor resistant AML. Abstract
Rewiring the fusion oncoprotein EWS/FLI1 in Ewing sarcoma with bivalent small molecules. bioRxiv. 2025 Mar 17. View Rewiring the fusion oncoprotein EWS/FLI1 in Ewing sarcoma with bivalent small molecules. Abstract
A compendium of Amplification-Related Gain Of Sensitivity genes in human cancer. Nat Commun. 2025 Jan 27; 16(1):1077. View A compendium of Amplification-Related Gain Of Sensitivity genes in human cancer. Abstract
Lineage-dependence of the neuroblastoma surfaceome defines tumor cell state-dependent and independent immunotherapeutic targets. Neuro Oncol. 2025 Jan 18. View Lineage-dependence of the neuroblastoma surfaceome defines tumor cell state-dependent and independent immunotherapeutic targets. Abstract
CEBPA repression by MECOM blocks differentiation to drive aggressive leukemias. bioRxiv. 2024 Dec 30. View CEBPA repression by MECOM blocks differentiation to drive aggressive leukemias. Abstract
Targeting the Sodium-Potassium Pump as a Therapeutic Strategy in Acute Myeloid Leukemia. Cancer Res. 2024 Oct 15; 84(20):3354-3370. View Targeting the Sodium-Potassium Pump as a Therapeutic Strategy in Acute Myeloid Leukemia. Abstract
Lineage-dependence of the neuroblastoma surfaceome defines tumor cell state-dependent and independent immunotherapeutic targets. bioRxiv. 2024 Jul 02. View Lineage-dependence of the neuroblastoma surfaceome defines tumor cell state-dependent and independent immunotherapeutic targets. Abstract
Pharmacological targeting of the cancer epigenome. Nat Cancer. 2024 06; 5(6):844-865. View Pharmacological targeting of the cancer epigenome. Abstract
The KAT module of the SAGA complex maintains the oncogenic gene expression program in MYCN-amplified neuroblastoma. Sci Adv. 2024 May 31; 10(22):eadm9449. View The KAT module of the SAGA complex maintains the oncogenic gene expression program in MYCN-amplified neuroblastoma. Abstract
Nucleotide depletion promotes cell fate transitions by inducing DNA replication stress. Dev Cell. 2024 08 19; 59(16):2203-2221.e15. View Nucleotide depletion promotes cell fate transitions by inducing DNA replication stress. Abstract
Targeting TRIP13 in favorable histology Wilms tumor with nuclear export inhibitors synergizes with doxorubicin. Commun Biol. 2024 04 08; 7(1):426. View Targeting TRIP13 in favorable histology Wilms tumor with nuclear export inhibitors synergizes with doxorubicin. Abstract
STAT3 couples activated tyrosine kinase signaling to the oncogenic core transcriptional regulatory circuitry of anaplastic large cell lymphoma. Cell Rep Med. 2024 Mar 19; 5(3):101472. View STAT3 couples activated tyrosine kinase signaling to the oncogenic core transcriptional regulatory circuitry of anaplastic large cell lymphoma. Abstract
The UBE2J2/UBE2K-MARCH5 ubiquitination machinery regulates apoptosis in response to venetoclax in acute myeloid leukemia. Leukemia. 2024 03; 38(3):652-656. View The UBE2J2/UBE2K-MARCH5 ubiquitination machinery regulates apoptosis in response to venetoclax in acute myeloid leukemia. Abstract
PPM1D modulates hematopoietic cell fitness and response to DNA damage and is a therapeutic target in myeloid malignancy. Blood. 2023 12 14; 142(24):2079-2091. View PPM1D modulates hematopoietic cell fitness and response to DNA damage and is a therapeutic target in myeloid malignancy. Abstract
EZH2 Cooperates with BRD4-NUT to Drive NUT Carcinoma Growth by Silencing Key Tumor Suppressor Genes. Cancer Res. 2023 12 01; 83(23):3956-3973. View EZH2 Cooperates with BRD4-NUT to Drive NUT Carcinoma Growth by Silencing Key Tumor Suppressor Genes. Abstract
Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma. Clin Cancer Res. 2023 11 14; 29(22):4613-4626. View Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma. Abstract
DHODH: a promising target in the treatment of T-cell acute lymphoblastic leukemia. Blood Adv. 2023 11 14; 7(21):6685-6701. View DHODH: a promising target in the treatment of T-cell acute lymphoblastic leukemia. Abstract
EZH2 synergizes with BRD4-NUT to drive NUT carcinoma growth through silencing of key tumor suppressor genes. bioRxiv. 2023 Aug 16. View EZH2 synergizes with BRD4-NUT to drive NUT carcinoma growth through silencing of key tumor suppressor genes. Abstract
Phase 2 trial of palbociclib and ganitumab in patients with relapsed Ewing sarcoma. Cancer Med. 2023 07; 12(14):15207-15216. View Phase 2 trial of palbociclib and ganitumab in patients with relapsed Ewing sarcoma. Abstract
Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors. bioRxiv. 2023 Apr 07. View Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors. Abstract
Highly connected 3D chromatin networks established by an oncogenic fusion protein shape tumor cell identity. Sci Adv. 2023 03 31; 9(13):eabo3789. View Highly connected 3D chromatin networks established by an oncogenic fusion protein shape tumor cell identity. Abstract
Leukemia core transcriptional circuitry is a sparsely interconnected hierarchy stabilized by incoherent feed-forward loops. bioRxiv. 2023 Mar 15. View Leukemia core transcriptional circuitry is a sparsely interconnected hierarchy stabilized by incoherent feed-forward loops. Abstract
Depletion of creatine phosphagen energetics with a covalent creatine kinase inhibitor. Nat Chem Biol. 2023 07; 19(7):815-824. View Depletion of creatine phosphagen energetics with a covalent creatine kinase inhibitor. Abstract
Mitochondrial fusion is a therapeutic vulnerability of acute myeloid leukemia. Leukemia. 2023 04; 37(4):765-775. View Mitochondrial fusion is a therapeutic vulnerability of acute myeloid leukemia. Abstract
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 The ETS transcription factor ETV6 constrains the transcriptional activity of EWS-FLI to promote Ewing sarcoma. Abstract
Transcriptional Antagonism by CDK8 Inhibition Improves Therapeutic Efficacy of MEK Inhibitors. Cancer Res. 2023 01 18; 83(2):285-300. View Transcriptional Antagonism by CDK8 Inhibition Improves Therapeutic Efficacy of MEK Inhibitors. Abstract
BAF Complex Maintains Glioma Stem Cells in Pediatric H3K27M Glioma. Cancer Discov. 2022 12 02; 12(12):2880-2905. View BAF Complex Maintains Glioma Stem Cells in Pediatric H3K27M Glioma. Abstract
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 12; 127(12):2220-2226. View 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. Abstract
VRK1 as a synthetic lethal target in VRK2 promoter-methylated cancers of the nervous system. JCI Insight. 2022 10 10; 7(19). View VRK1 as a synthetic lethal target in VRK2 promoter-methylated cancers of the nervous system. Abstract
Transcriptional Plasticity Drives Leukemia Immune Escape. Blood Cancer Discov. 2022 09 06; 3(5):394-409. View Transcriptional Plasticity Drives Leukemia Immune Escape. Abstract
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 Transition to a mesenchymal state in neuroblastoma confers resistance to anti-GD2 antibody via reduced expression of ST8SIA1. Abstract
Unleashing Cell-Intrinsic Inflammation as a Strategy to Kill AML Blasts. Cancer Discov. 2022 07 06; 12(7):1760-1781. View Unleashing Cell-Intrinsic Inflammation as a Strategy to Kill AML Blasts. Abstract
Mitochondrial Dysfunction Is a Driver of SP-2509 Drug Resistance in Ewing Sarcoma. Mol Cancer Res. 2022 07 06; 20(7):1035-1046. View Mitochondrial Dysfunction Is a Driver of SP-2509 Drug Resistance in Ewing Sarcoma. Abstract
Identification of an Epi-metabolic dependency on EHMT2/G9a in T-cell acute lymphoblastic leukemia. Cell Death Dis. 2022 06 17; 13(6):551. View Identification of an Epi-metabolic dependency on EHMT2/G9a in T-cell acute lymphoblastic leukemia. Abstract
IKAROS and MENIN coordinate therapeutically actionable leukemogenic gene expression in MLL-r acute myeloid leukemia. Nat Cancer. 2022 05; 3(5):595-613. View IKAROS and MENIN coordinate therapeutically actionable leukemogenic gene expression in MLL-r acute myeloid leukemia. Abstract
Cystine uptake inhibition potentiates front-line therapies in acute myeloid leukemia. Leukemia. 2022 06; 36(6):1585-1595. View Cystine uptake inhibition potentiates front-line therapies in acute myeloid leukemia. Abstract
The proteogenomic subtypes of acute myeloid leukemia. Cancer Cell. 2022 03 14; 40(3):301-317.e12. View The proteogenomic subtypes of acute myeloid leukemia. Abstract
EP300 Selectively Controls the Enhancer Landscape of MYCN-Amplified Neuroblastoma. Cancer Discov. 2022 03 01; 12(3):730-751. View EP300 Selectively Controls the Enhancer Landscape of MYCN-Amplified Neuroblastoma. Abstract
A distinct core regulatory module enforces oncogene expression in KMT2A-rearranged leukemia. Genes Dev. 2022 03 01; 36(5-6):368-389. View A distinct core regulatory module enforces oncogene expression in KMT2A-rearranged leukemia. Abstract
SHMT2 inhibition disrupts the TCF3 transcriptional survival program in Burkitt lymphoma. Blood. 2022 01 27; 139(4):538-553. View SHMT2 inhibition disrupts the TCF3 transcriptional survival program in Burkitt lymphoma. Abstract
An In Vivo CRISPR Screening Platform for Prioritizing Therapeutic Targets in AML. Cancer Discov. 2022 02; 12(2):432-449. View An In Vivo CRISPR Screening Platform for Prioritizing Therapeutic Targets in AML. Abstract
Targeting serine hydroxymethyltransferases 1 and 2 for T-cell acute lymphoblastic leukemia therapy. Leukemia. 2022 02; 36(2):348-360. View Targeting serine hydroxymethyltransferases 1 and 2 for T-cell acute lymphoblastic leukemia therapy. Abstract
TRIM8 modulates the EWS/FLI oncoprotein to promote survival in Ewing sarcoma. Cancer Cell. 2021 09 13; 39(9):1262-1278.e7. View TRIM8 modulates the EWS/FLI oncoprotein to promote survival in Ewing sarcoma. Abstract
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 Synthetic Lethal Interaction between the ESCRT Paralog Enzymes VPS4A and VPS4B in Cancers Harboring Loss of Chromosome 18q or 16q. Abstract
STAG2 loss rewires oncogenic and developmental programs to promote metastasis in Ewing sarcoma. Cancer Cell. 2021 06 14; 39(6):827-844.e10. View STAG2 loss rewires oncogenic and developmental programs to promote metastasis in Ewing sarcoma. Abstract
Gene Fusions Create Partner and Collateral Dependencies Essential to Cancer Cell Survival. Cancer Res. 2021 08 01; 81(15):3971-3984. View Gene Fusions Create Partner and Collateral Dependencies Essential to Cancer Cell Survival. Abstract
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 The synergy of BET inhibitors with aurora A kinase inhibitors in MYCN-amplified neuroblastoma is heightened with functional TP53. Abstract
Selective Modulation of a Pan-Essential Protein as a Therapeutic Strategy in Cancer. Cancer Discov. 2021 09; 11(9):2282-2299. View Selective Modulation of a Pan-Essential Protein as a Therapeutic Strategy in Cancer. Abstract
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 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. Abstract
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 Targeting acute myeloid leukemia dependency on VCP-mediated DNA repair through a selective second-generation small-molecule inhibitor. Abstract
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 RAD21 is a driver of chromosome 8 gain in Ewing sarcoma to mitigate replication stress. Abstract
A first-generation pediatric cancer dependency map. Nat Genet. 2021 Apr; 53(4):529-538. View A first-generation pediatric cancer dependency map. Abstract
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 Bromodomain and extra-terminal inhibitors-A consensus prioritisation after the Paediatric Strategy Forum for medicinal product development of epigenetic modifiers in children-ACCELERATE. Abstract
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 Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory, or High-Risk Leukemias: A Report from the LEAP Consortium. Abstract
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 Network-based systems pharmacology reveals heterogeneity in LCK and BCL2 signaling and therapeutic sensitivity of T-cell acute lymphoblastic leukemia. Abstract
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 Synthetic Lethal Interaction between the ESCRT Paralog Enzymes VPS4A and VPS4B in Cancers Harboring Loss of Chromosome 18q or 16q. Abstract
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 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. Abstract
Rapid and direct control of target protein levels with VHL-recruiting dTAG molecules. Nat Commun. 2020 09 18; 11(1):4687. View Rapid and direct control of target protein levels with VHL-recruiting dTAG molecules. Abstract
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 The Folate Cycle Enzyme MTHFR Is a Critical Regulator of Cell Response to MYC-Targeting Therapies. Abstract
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 Blockade of Oncogenic NOTCH1 with the SERCA Inhibitor CAD204520 in T Cell Acute Lymphoblastic Leukemia. Abstract
Targeting EZH2 for the treatment of hepatosplenic T-cell lymphoma. Blood Adv. 2020 04 14; 4(7):1265-1269. View Targeting EZH2 for the treatment of hepatosplenic T-cell lymphoma. Abstract
Selective USP7 inhibition elicits cancer cell killing through a p53-dependent mechanism. Sci Rep. 2020 03 24; 10(1):5324. View Selective USP7 inhibition elicits cancer cell killing through a p53-dependent mechanism. Abstract
Targeting DUBs to degrade oncogenic proteins. Br J Cancer. 2020 04; 122(8):1121-1123. View Targeting DUBs to degrade oncogenic proteins. Abstract
Preclinical efficacy for a novel tyrosine kinase inhibitor, ArQule 531 against acute myeloid leukemia. J Hematol Oncol. 2020 01 28; 13(1):8. View Preclinical efficacy for a novel tyrosine kinase inhibitor, ArQule 531 against acute myeloid leukemia. Abstract
STRIPAK directs PP2A activity toward MAP4K4 to promote oncogenic transformation of human cells. Elife. 2020 Jan 08; 9. View STRIPAK directs PP2A activity toward MAP4K4 to promote oncogenic transformation of human cells. Abstract
Salt-inducible kinase inhibition suppresses acute myeloid leukemia progression in vivo. Blood. 2020 01 02; 135(1):56-70. View Salt-inducible kinase inhibition suppresses acute myeloid leukemia progression in vivo. Abstract
Resistance Mechanisms to SYK Inhibition in Acute Myeloid Leukemia. Cancer Discov. 2020 02; 10(2):214-231. View Resistance Mechanisms to SYK Inhibition in Acute Myeloid Leukemia. Abstract
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 EWS-FLI1 modulated alternative splicing of ARID1A reveals novel oncogenic function through the BAF complex. Abstract
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 Small-Molecule and CRISPR Screening Converge to Reveal Receptor Tyrosine Kinase Dependencies in Pediatric Rhabdoid Tumors. Abstract
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 Single-cell cloning of human T-cell lines reveals clonal variation in cell death responses to chemotherapeutics. Abstract
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 Neuronal differentiation and cell-cycle programs mediate response to BET-bromodomain inhibition in MYC-driven medulloblastoma. Abstract
Targeting chromatin complexes in fusion protein-driven malignancies. Nat Rev Cancer. 2019 05; 19(5):255-269. View Targeting chromatin complexes in fusion protein-driven malignancies. Abstract
Therapeutic discovery for marrow failure with MDS predisposition using pluripotent stem cells. JCI Insight. 2019 04 30; 5. View Therapeutic discovery for marrow failure with MDS predisposition using pluripotent stem cells. Abstract
Synthetic Lethality of Wnt Pathway Activation and Asparaginase in Drug-Resistant Acute Leukemias. Cancer Cell. 2019 04 15; 35(4):664-676.e7. View Synthetic Lethality of Wnt Pathway Activation and Asparaginase in Drug-Resistant Acute Leukemias. Abstract
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 High-throughput Chemical Screening Identifies Focal Adhesion Kinase and Aurora Kinase B Inhibition as a Synergistic Treatment Combination in Ewing Sarcoma. Abstract
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 Correction: Detection of circulating tumour DNA is associated with inferior outcomes in Ewing sarcoma and osteosarcoma: a report from the Children's Oncology Group. Abstract
Ushering in the next generation of precision trials for pediatric cancer. Science. 2019 Mar 15; 363(6432):1175-1181. View Ushering in the next generation of precision trials for pediatric cancer. Abstract
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 Virtual Screening Identifies Irreversible FMS-like Tyrosine Kinase 3 Inhibitors with Activity toward Resistance-Conferring Mutations. Abstract
MDM2 and MDM4 Are Therapeutic Vulnerabilities in Malignant Rhabdoid Tumors. Cancer Res. 2019 05 01; 79(9):2404-2414. View MDM2 and MDM4 Are Therapeutic Vulnerabilities in Malignant Rhabdoid Tumors. Abstract
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 The kinases IKBKE and TBK1 regulate MYC-dependent survival pathways through YB-1 in AML and are targets for therapy. Abstract
Resistance to Epigenetic-Targeted Therapy Engenders Tumor Cell Vulnerabilities Associated with Enhancer Remodeling. Cancer Cell. 2018 12 10; 34(6):922-938.e7. View Resistance to Epigenetic-Targeted Therapy Engenders Tumor Cell Vulnerabilities Associated with Enhancer Remodeling. Abstract
A Combination CDK4/6 and IGF1R Inhibitor Strategy for Ewing Sarcoma. Clin Cancer Res. 2019 02 15; 25(4):1343-1357. View A Combination CDK4/6 and IGF1R Inhibitor Strategy for Ewing Sarcoma. Abstract
Creatine kinase pathway inhibition alters GSK3 and WNT signaling in EVI1-positive AML. Leukemia. 2019 03; 33(3):800-804. View Creatine kinase pathway inhibition alters GSK3 and WNT signaling in EVI1-positive AML. Abstract
TRPS1 Is a Lineage-Specific Transcriptional Dependency in Breast Cancer. Cell Rep. 2018 10 30; 25(5):1255-1267.e5. View TRPS1 Is a Lineage-Specific Transcriptional Dependency in Breast Cancer. Abstract
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 Precision Targeting of BFL-1/A1 and an ATM Co-dependency in Human Cancer. Abstract
Comparative proteomics reveals a diagnostic signature for pulmonary head-and-neck cancer metastasis. EMBO Mol Med. 2018 09; 10(9). View Comparative proteomics reveals a diagnostic signature for pulmonary head-and-neck cancer metastasis. Abstract
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 Detection of circulating tumour DNA is associated with inferior outcomes in Ewing sarcoma and osteosarcoma: a report from the Children's Oncology Group. Abstract
Selective gene dependencies in MYCN-amplified neuroblastoma include the core transcriptional regulatory circuitry. Nat Genet. 2018 09; 50(9):1240-1246. View Selective gene dependencies in MYCN-amplified neuroblastoma include the core transcriptional regulatory circuitry. Abstract
Targeted therapy for fusion-driven high-risk acute leukemia. Blood. 2018 09 20; 132(12):1241-1247. View Targeted therapy for fusion-driven high-risk acute leukemia. Abstract
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 Genome-scale CRISPR-Cas9 screen identifies druggable dependencies in TP53 wild-type Ewing sarcoma. Abstract
KLF5 controls glutathione metabolism to suppress p190-BCR-ABL+ B-cell lymphoblastic leukemia. Oncotarget. 2018 Jul 03; 9(51):29665-29679. View KLF5 controls glutathione metabolism to suppress p190-BCR-ABL+ B-cell lymphoblastic leukemia. Abstract
Author Correction: EWS-FLI1 increases transcription to cause R-loops and block BRCA1 repair in Ewing sarcoma. Nature. 2018 07; 559(7715):E11. View Author Correction: EWS-FLI1 increases transcription to cause R-loops and block BRCA1 repair in Ewing sarcoma. Abstract
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 Phase I trial of the mTOR inhibitor everolimus in combination with multi-agent chemotherapy in relapsed childhood acute lymphoblastic leukemia. Abstract
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 Bepridil exhibits anti-leukemic activity associated with NOTCH1 pathway inhibition in chronic lymphocytic leukemia. Abstract
EWS-FLI1 increases transcription to cause R-loops and block BRCA1 repair in Ewing sarcoma. Nature. 2018 03 15; 555(7696):387-391. View EWS-FLI1 increases transcription to cause R-loops and block BRCA1 repair in Ewing sarcoma. Abstract
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 Exploiting an Asp-Glu "switch" in glycogen synthase kinase 3 to design paralog-selective inhibitors for use in acute myeloid leukemia. Abstract
EWS/FLI Confers Tumor Cell Synthetic Lethality to CDK12 Inhibition in Ewing Sarcoma. Cancer Cell. 2018 02 12; 33(2):202-216.e6. View EWS/FLI Confers Tumor Cell Synthetic Lethality to CDK12 Inhibition in Ewing Sarcoma. Abstract
CRISPR-Cas9 screen reveals a MYCN-amplified neuroblastoma dependency on EZH2. J Clin Invest. 2018 01 02; 128(1):446-462. View CRISPR-Cas9 screen reveals a MYCN-amplified neuroblastoma dependency on EZH2. Abstract
Leukemia-specific delivery of mutant NOTCH1 targeted therapy. J Exp Med. 2018 01 02; 215(1):197-216. View Leukemia-specific delivery of mutant NOTCH1 targeted therapy. Abstract
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 Computational correction of copy number effect improves specificity of CRISPR-Cas9 essentiality screens in cancer cells. Abstract
Identification of an allosteric benzothiazolopyrimidone inhibitor of the oncogenic protein tyrosine phosphatase SHP2. Bioorg Med Chem. 2017 12 15; 25(24):6479-6485. View Identification of an allosteric benzothiazolopyrimidone inhibitor of the oncogenic protein tyrosine phosphatase SHP2. Abstract
TOX Regulates Growth, DNA Repair, and Genomic Instability in T-cell Acute Lymphoblastic Leukemia. Cancer Discov. 2017 11; 7(11):1336-1353. View TOX Regulates Growth, DNA Repair, and Genomic Instability in T-cell Acute Lymphoblastic Leukemia. Abstract
Scratching the Surface of Immunotherapeutic Targets in Neuroblastoma. Cancer Cell. 2017 09 11; 32(3):271-273. View Scratching the Surface of Immunotherapeutic Targets in Neuroblastoma. Abstract
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 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. Abstract
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 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. Abstract
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 Hoxa9 and Meis1 Cooperatively Induce Addiction to Syk Signaling by Suppressing miR-146a in Acute Myeloid Leukemia. Abstract
The creatine kinase pathway is a metabolic vulnerability in EVI1-positive acute myeloid leukemia. Nat Med. 2017 Mar; 23(3):301-313. View The creatine kinase pathway is a metabolic vulnerability in EVI1-positive acute myeloid leukemia. Abstract
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 Synergistic Drug Combinations with a CDK4/6 Inhibitor in T-cell Acute Lymphoblastic Leukemia. Abstract
The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 07 11; 30(1):183. View The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Abstract
Integrated genetic and pharmacologic interrogation of rare cancers. Nat Commun. 2016 06 22; 7:11987. View Integrated genetic and pharmacologic interrogation of rare cancers. Abstract
Targeting MTHFD2 in acute myeloid leukemia. J Exp Med. 2016 06 27; 213(7):1285-306. View Targeting MTHFD2 in acute myeloid leukemia. Abstract
Genomic Copy Number Dictates a Gene-Independent Cell Response to CRISPR/Cas9 Targeting. Cancer Discov. 2016 Aug; 6(8):914-29. View Genomic Copy Number Dictates a Gene-Independent Cell Response to CRISPR/Cas9 Targeting. Abstract
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 Inhibitors of Glycogen Synthase Kinase 3 with Exquisite Kinome-Wide Selectivity and Their Functional Effects. Abstract
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 Multicenter Feasibility Study of Tumor Molecular Profiling to Inform Therapeutic Decisions in Advanced Pediatric Solid Tumors: The Individualized Cancer Therapy (iCat) Study. Abstract
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 The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Abstract
Aberrant activation of the PI3K/mTOR pathway promotes resistance to sorafenib in AML. Oncogene. 2016 09 29; 35(39):5119-31. View Aberrant activation of the PI3K/mTOR pathway promotes resistance to sorafenib in AML. Abstract
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 The second European interdisciplinary Ewing sarcoma research summit--A joint effort to deconstructing the multiple layers of a complex disease. Abstract
Corrigendum: The genomic landscape of juvenile myelomonocytic leukemia. Nat Genet. 2016 Jan; 48(1):101. View Corrigendum: The genomic landscape of juvenile myelomonocytic leukemia. Abstract
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 Evaluation of Improved Glycogen Synthase Kinase-3a Inhibitors in Models of Acute Myeloid Leukemia. Abstract
The genomic landscape of juvenile myelomonocytic leukemia. Nat Genet. 2015 Nov; 47(11):1326-1333. View The genomic landscape of juvenile myelomonocytic leukemia. Abstract
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 Functional, chemical genomic, and super-enhancer screening identify sensitivity to cyclin D1/CDK4 pathway inhibition in Ewing sarcoma. Abstract
Increased SYK activity is associated with unfavorable outcome among patients with acute myeloid leukemia. Oncotarget. 2015 Sep 22; 6(28):25575-87. View Increased SYK activity is associated with unfavorable outcome among patients with acute myeloid leukemia. Abstract
Long noncoding RNA EWSAT1-mediated gene repression facilitates Ewing sarcoma oncogenesis. J Clin Invest. 2014 Dec; 124(12):5275-90. View Long noncoding RNA EWSAT1-mediated gene repression facilitates Ewing sarcoma oncogenesis. Abstract
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 Erratum: Parallel genome-scale loss of function screens in 216 cancer cell lines for the identification of context-specific genetic dependencies. Abstract
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 Parallel genome-scale loss of function screens in 216 cancer cell lines for the identification of context-specific genetic dependencies. Abstract
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 Molecular rationale for the use of PI3K/AKT/mTOR pathway inhibitors in combination with crizotinib in ALK-mutated neuroblastoma. Abstract
The genomic landscape of pediatric Ewing sarcoma. Cancer Discov. 2014 Nov; 4(11):1326-41. View The genomic landscape of pediatric Ewing sarcoma. Abstract
New Approaches to Target T-ALL. Front Oncol. 2014; 4:170. View New Approaches to Target T-ALL. Abstract
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 Triplication of a 21q22 region contributes to B cell transformation through HMGN1 overexpression and loss of histone H3 Lys27 trimethylation. Abstract
Targeting Csnk1a1 in leukemia. J Exp Med. 2014 Apr 07; 211(4):594. View Targeting Csnk1a1 in leukemia. Abstract
An epigenetic mechanism of resistance to targeted therapy in T cell acute lymphoblastic leukemia. Nat Genet. 2014 Apr; 46(4):364-70. View An epigenetic mechanism of resistance to targeted therapy in T cell acute lymphoblastic leukemia. Abstract
SYK is a critical regulator of FLT3 in acute myeloid leukemia. Cancer Cell. 2014 Feb 10; 25(2):226-42. View SYK is a critical regulator of FLT3 in acute myeloid leukemia. Abstract
MUC1-C oncoprotein promotes FLT3 receptor activation in acute myeloid leukemia cells. Blood. 2014 Jan 30; 123(5):734-42. View MUC1-C oncoprotein promotes FLT3 receptor activation in acute myeloid leukemia cells. Abstract
A phase II study of the EGFR inhibitor gefitinib in patients with acute myeloid leukemia. Leuk Res. 2014 Apr; 38(4):430-4. View A phase II study of the EGFR inhibitor gefitinib in patients with acute myeloid leukemia. Abstract
Mutational heterogeneity in cancer and the search for new cancer-associated genes. Nature. 2013 Jul 11; 499(7457):214-218. View Mutational heterogeneity in cancer and the search for new cancer-associated genes. Abstract
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 Vivo RNAi screening identifies a leukemia-specific dependence on integrin beta 3 signaling. Abstract
Selective HDAC1/HDAC2 inhibitors induce neuroblastoma differentiation. Chem Biol. 2013 May 23; 20(5):713-25. View Selective HDAC1/HDAC2 inhibitors induce neuroblastoma differentiation. Abstract
SYK regulates mTOR signaling in AML. Leukemia. 2013 Nov; 27(11):2118-28. View SYK regulates mTOR signaling in AML. Abstract
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 High-throughput tyrosine kinase activity profiling identifies FAK as a candidate therapeutic target in Ewing sarcoma. Abstract
Targeting MYCN in neuroblastoma by BET bromodomain inhibition. Cancer Discov. 2013 Mar; 3(3):308-23. View Targeting MYCN in neuroblastoma by BET bromodomain inhibition. Abstract
Complementary genomic screens identify SERCA as a therapeutic target in NOTCH1 mutated cancer. Cancer Cell. 2013 Mar 18; 23(3):390-405. View Complementary genomic screens identify SERCA as a therapeutic target in NOTCH1 mutated cancer. Abstract
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 The intersection of genetic and chemical genomic screens identifies GSK-3a as a target in human acute myeloid leukemia. Abstract
Genetic and proteomic approaches to identify cancer drug targets. Br J Cancer. 2012 Jan 17; 106(2):254-61. View Genetic and proteomic approaches to identify cancer drug targets. Abstract
Targeting NOTCH1 in hematopoietic malignancy. Crit Rev Oncog. 2011; 16(1-2):103-15. View Targeting NOTCH1 in hematopoietic malignancy. Abstract
Chemical genomics and combo therapy. Blood. 2010 Dec 23; 116(26):5786-8. View Chemical genomics and combo therapy. Abstract
Characterization of Notch1 antibodies that inhibit signaling of both normal and mutated Notch1 receptors. PLoS One. 2010 Feb 08; 5(2):e9094. View Characterization of Notch1 antibodies that inhibit signaling of both normal and mutated Notch1 receptors. Abstract
Proteomic and genetic approaches identify Syk as an AML target. Cancer Cell. 2009 Oct 06; 16(4):281-94. View Proteomic and genetic approaches identify Syk as an AML target. Abstract
Identification of AML1-ETO modulators by chemical genomics. Blood. 2009 Jun 11; 113(24):6193-205. View Identification of AML1-ETO modulators by chemical genomics. Abstract
Genomic approaches to small molecule discovery. Leukemia. 2009 Jul; 23(7):1226-35. View Genomic approaches to small molecule discovery. Abstract
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 Phase II study of intermediate-dose cytarabine in patients with relapsed or refractory Ewing sarcoma: a report from the Children's Oncology Group. Abstract
High-throughput gene expression profiling of memory differentiation in primary human T cells. BMC Immunol. 2008 Aug 01; 9:44. View High-throughput gene expression profiling of memory differentiation in primary human T cells. Abstract
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 Expression-based screening identifies the combination of histone deacetylase inhibitors and retinoids for neuroblastoma differentiation. Abstract
Literature Lab: a method of automated literature interrogation to infer biology from microarray analysis. BMC Genomics. 2007 Dec 18; 8:461. View Literature Lab: a method of automated literature interrogation to infer biology from microarray analysis. Abstract
Signature-based small molecule screening identifies cytosine arabinoside as an EWS/FLI modulator in Ewing sarcoma. PLoS Med. 2007 Apr; 4(4):e122. View Signature-based small molecule screening identifies cytosine arabinoside as an EWS/FLI modulator in Ewing sarcoma. Abstract
Gene expression signature-based chemical genomic prediction identifies a novel class of HSP90 pathway modulators. Cancer Cell. 2006 Oct; 10(4):321-30. View Gene expression signature-based chemical genomic prediction identifies a novel class of HSP90 pathway modulators. Abstract
Genomic approaches in acute leukemia. Best Pract Res Clin Haematol. 2006; 19(2):263-8. View Genomic approaches in acute leukemia. Abstract
A method for high-throughput gene expression signature analysis. Genome Biol. 2006; 7(7):R61. View A method for high-throughput gene expression signature analysis. Abstract
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 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. Abstract
Gefitinib induces myeloid differentiation of acute myeloid leukemia. Blood. 2005 Oct 15; 106(8):2841-8. View Gefitinib induces myeloid differentiation of acute myeloid leukemia. Abstract
Gene expression-based high-throughput screening(GE-HTS) and application to leukemia differentiation. Nat Genet. 2004 Mar; 36(3):257-63. View Gene expression-based high-throughput screening(GE-HTS) and application to leukemia differentiation. Abstract
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 The TEL gene contributes to the pathogenesis of myeloid and lymphoid leukemias by diverse molecular genetic mechanisms. Abstract
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 TEL/AML-1 dimerizes and is associated with a favorable outcome in childhood acute lymphoblastic leukemia. Abstract
The TEL gene and human leukemia. Biochim Biophys Acta. 1996 Aug 08; 1288(1):M7-10. View The TEL gene and human leukemia. Abstract
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 Mutational analysis of the candidate tumor suppressor genes TEL and KIP1 in childhood acute lymphoblastic leukemia. Abstract
Involvement of the TEL gene in hematologic malignancy by diverse molecular genetic mechanisms. Curr Top Microbiol Immunol. 1996; 211:279-88. View Involvement of the TEL gene in hematologic malignancy by diverse molecular genetic mechanisms. Abstract
Frequent loss of heterozygosity at the TEL gene locus in acute lymphoblastic leukemia of childhood. Blood. 1995 Jul 01; 86(1):38-44. View Frequent loss of heterozygosity at the TEL gene locus in acute lymphoblastic leukemia of childhood. Abstract