Kimberly Stegmaier, MD
Attending Physician, Dana-Farber/Boston Children's Cancer and Blood Disorders Center; Co-Director, Hematologic Malignancy Center (Basic & Translational Research); Ted Williams Chair, Dana-Farber Cancer Institute
Professor of Pediatrics, Harvard Medical School
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Kimberly Stegmaier, MD
Attending Physician, Dana-Farber/Boston Children's Cancer and Blood Disorders Center; Co-Director, Hematologic Malignancy Center (Basic & Translational Research); Ted Williams Chair, Dana-Farber Cancer Institute
Professor of Pediatrics, Harvard Medical School
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
Publications
Prospects for understanding and exploiting the consequences of hyperactivation lethality. View Abstract
Inflammatory signaling pathways play a role in SYK inhibitor resistant AML. View Abstract
Rewiring the fusion oncoprotein EWS/FLI1 in Ewing sarcoma with bivalent small molecules. View Abstract
A compendium of Amplification-Related Gain Of Sensitivity genes in human cancer. View Abstract
Lineage-dependence of the neuroblastoma surfaceome defines tumor cell state-dependent and independent immunotherapeutic targets. View Abstract
CEBPA repression by MECOM blocks differentiation to drive aggressive leukemias. View Abstract
Targeting the Sodium-Potassium Pump as a Therapeutic Strategy in Acute Myeloid Leukemia. View Abstract
Lineage-dependence of the neuroblastoma surfaceome defines tumor cell state-dependent and independent immunotherapeutic targets. View Abstract
Pharmacological targeting of the cancer epigenome. View Abstract
The KAT module of the SAGA complex maintains the oncogenic gene expression program in MYCN-amplified neuroblastoma. View Abstract
Nucleotide depletion promotes cell fate transitions by inducing DNA replication stress. View Abstract
Targeting TRIP13 in favorable histology Wilms tumor with nuclear export inhibitors synergizes with doxorubicin. View Abstract
STAT3 couples activated tyrosine kinase signaling to the oncogenic core transcriptional regulatory circuitry of anaplastic large cell lymphoma. View Abstract
The UBE2J2/UBE2K-MARCH5 ubiquitination machinery regulates apoptosis in response to venetoclax in acute myeloid leukemia. View Abstract
PPM1D modulates hematopoietic cell fitness and response to DNA damage and is a therapeutic target in myeloid malignancy. View Abstract
EZH2 Cooperates with BRD4-NUT to Drive NUT Carcinoma Growth by Silencing Key Tumor Suppressor Genes. View Abstract
Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma. View Abstract
DHODH: a promising target in the treatment of T-cell acute lymphoblastic leukemia. View Abstract
EZH2 synergizes with BRD4-NUT to drive NUT carcinoma growth through silencing of key tumor suppressor genes. View Abstract
Phase 2 trial of palbociclib and ganitumab in patients with relapsed Ewing sarcoma. View Abstract
Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors. View Abstract
Highly connected 3D chromatin networks established by an oncogenic fusion protein shape tumor cell identity. View Abstract
Leukemia core transcriptional circuitry is a sparsely interconnected hierarchy stabilized by incoherent feed-forward loops. View Abstract
Depletion of creatine phosphagen energetics with a covalent creatine kinase inhibitor. View Abstract
Mitochondrial fusion is a therapeutic vulnerability of acute myeloid leukemia. View Abstract
The ETS transcription factor ETV6 constrains the transcriptional activity of EWS-FLI to promote Ewing sarcoma. View Abstract
Transcriptional Antagonism by CDK8 Inhibition Improves Therapeutic Efficacy of MEK Inhibitors. View Abstract
BAF Complex Maintains Glioma Stem Cells in Pediatric H3K27M Glioma. View 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. View Abstract
VRK1 as a synthetic lethal target in VRK2 promoter-methylated cancers of the nervous system. View Abstract
Transcriptional Plasticity Drives Leukemia Immune Escape. View Abstract
Transition to a mesenchymal state in neuroblastoma confers resistance to anti-GD2 antibody via reduced expression of ST8SIA1. View Abstract
Unleashing Cell-Intrinsic Inflammation as a Strategy to Kill AML Blasts. View Abstract
Mitochondrial Dysfunction Is a Driver of SP-2509 Drug Resistance in Ewing Sarcoma. View Abstract
Identification of an Epi-metabolic dependency on EHMT2/G9a in T-cell acute lymphoblastic leukemia. View Abstract
IKAROS and MENIN coordinate therapeutically actionable leukemogenic gene expression in MLL-r acute myeloid leukemia. View Abstract
Cystine uptake inhibition potentiates front-line therapies in acute myeloid leukemia. View Abstract
The proteogenomic subtypes of acute myeloid leukemia. View Abstract
EP300 Selectively Controls the Enhancer Landscape of MYCN-Amplified Neuroblastoma. View Abstract
A distinct core regulatory module enforces oncogene expression in KMT2A-rearranged leukemia. View Abstract
SHMT2 inhibition disrupts the TCF3 transcriptional survival program in Burkitt lymphoma. View Abstract
An In Vivo CRISPR Screening Platform for Prioritizing Therapeutic Targets in AML. View Abstract
Targeting serine hydroxymethyltransferases 1 and 2 for T-cell acute lymphoblastic leukemia therapy. View Abstract
TRIM8 modulates the EWS/FLI oncoprotein to promote survival in Ewing sarcoma. View Abstract
Synthetic Lethal Interaction between the ESCRT Paralog Enzymes VPS4A and VPS4B in Cancers Harboring Loss of Chromosome 18q or 16q. View Abstract
STAG2 loss rewires oncogenic and developmental programs to promote metastasis in Ewing sarcoma. View Abstract
Gene Fusions Create Partner and Collateral Dependencies Essential to Cancer Cell Survival. View Abstract
The synergy of BET inhibitors with aurora A kinase inhibitors in MYCN-amplified neuroblastoma is heightened with functional TP53. View Abstract
Selective Modulation of a Pan-Essential Protein as a Therapeutic Strategy in Cancer. View 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. View Abstract
Targeting acute myeloid leukemia dependency on VCP-mediated DNA repair through a selective second-generation small-molecule inhibitor. View Abstract
RAD21 is a driver of chromosome 8 gain in Ewing sarcoma to mitigate replication stress. View Abstract
A first-generation pediatric cancer dependency map. View Abstract
Bromodomain and extra-terminal inhibitors-A consensus prioritisation after the Paediatric Strategy Forum for medicinal product development of epigenetic modifiers in children-ACCELERATE. View Abstract
Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory, or High-Risk Leukemias: A Report from the LEAP Consortium. View Abstract
Network-based systems pharmacology reveals heterogeneity in LCK and BCL2 signaling and therapeutic sensitivity of T-cell acute lymphoblastic leukemia. View Abstract
Synthetic Lethal Interaction between the ESCRT Paralog Enzymes VPS4A and VPS4B in Cancers Harboring Loss of Chromosome 18q or 16q. View 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. View Abstract
Rapid and direct control of target protein levels with VHL-recruiting dTAG molecules. View Abstract
The Folate Cycle Enzyme MTHFR Is a Critical Regulator of Cell Response to MYC-Targeting Therapies. View Abstract
Blockade of Oncogenic NOTCH1 with the SERCA Inhibitor CAD204520 in T Cell Acute Lymphoblastic Leukemia. View Abstract
Targeting EZH2 for the treatment of hepatosplenic T-cell lymphoma. View Abstract
Selective USP7 inhibition elicits cancer cell killing through a p53-dependent mechanism. View Abstract
Targeting DUBs to degrade oncogenic proteins. View Abstract
Preclinical efficacy for a novel tyrosine kinase inhibitor, ArQule 531 against acute myeloid leukemia. View Abstract
STRIPAK directs PP2A activity toward MAP4K4 to promote oncogenic transformation of human cells. View Abstract
Salt-inducible kinase inhibition suppresses acute myeloid leukemia progression in vivo. View Abstract
Resistance Mechanisms to SYK Inhibition in Acute Myeloid Leukemia. View Abstract
EWS-FLI1 modulated alternative splicing of ARID1A reveals novel oncogenic function through the BAF complex. View Abstract
Small-Molecule and CRISPR Screening Converge to Reveal Receptor Tyrosine Kinase Dependencies in Pediatric Rhabdoid Tumors. View Abstract
Single-cell cloning of human T-cell lines reveals clonal variation in cell death responses to chemotherapeutics. View Abstract
Neuronal differentiation and cell-cycle programs mediate response to BET-bromodomain inhibition in MYC-driven medulloblastoma. View Abstract
Targeting chromatin complexes in fusion protein-driven malignancies. View Abstract
Therapeutic discovery for marrow failure with MDS predisposition using pluripotent stem cells. View Abstract
Synthetic Lethality of Wnt Pathway Activation and Asparaginase in Drug-Resistant Acute Leukemias. View Abstract
High-throughput Chemical Screening Identifies Focal Adhesion Kinase and Aurora Kinase B Inhibition as a Synergistic Treatment Combination in Ewing Sarcoma. View 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. View Abstract
Ushering in the next generation of precision trials for pediatric cancer. View Abstract
Virtual Screening Identifies Irreversible FMS-like Tyrosine Kinase 3 Inhibitors with Activity toward Resistance-Conferring Mutations. View Abstract
MDM2 and MDM4 Are Therapeutic Vulnerabilities in Malignant Rhabdoid Tumors. View Abstract
The kinases IKBKE and TBK1 regulate MYC-dependent survival pathways through YB-1 in AML and are targets for therapy. View Abstract
Resistance to Epigenetic-Targeted Therapy Engenders Tumor Cell Vulnerabilities Associated with Enhancer Remodeling. View Abstract
A Combination CDK4/6 and IGF1R Inhibitor Strategy for Ewing Sarcoma. View Abstract
Creatine kinase pathway inhibition alters GSK3 and WNT signaling in EVI1-positive AML. View Abstract
TRPS1 Is a Lineage-Specific Transcriptional Dependency in Breast Cancer. View Abstract
Precision Targeting of BFL-1/A1 and an ATM Co-dependency in Human Cancer. View Abstract
Comparative proteomics reveals a diagnostic signature for pulmonary head-and-neck cancer metastasis. View Abstract
Detection of circulating tumour DNA is associated with inferior outcomes in Ewing sarcoma and osteosarcoma: a report from the Children's Oncology Group. View Abstract
Selective gene dependencies in MYCN-amplified neuroblastoma include the core transcriptional regulatory circuitry. View Abstract
Targeted therapy for fusion-driven high-risk acute leukemia. View Abstract
Genome-scale CRISPR-Cas9 screen identifies druggable dependencies in TP53 wild-type Ewing sarcoma. View Abstract
KLF5 controls glutathione metabolism to suppress p190-BCR-ABL+ B-cell lymphoblastic leukemia. View Abstract
Author Correction: EWS-FLI1 increases transcription to cause R-loops and block BRCA1 repair in Ewing sarcoma. View Abstract
Phase I trial of the mTOR inhibitor everolimus in combination with multi-agent chemotherapy in relapsed childhood acute lymphoblastic leukemia. View Abstract
Bepridil exhibits anti-leukemic activity associated with NOTCH1 pathway inhibition in chronic lymphocytic leukemia. View Abstract
EWS-FLI1 increases transcription to cause R-loops and block BRCA1 repair in Ewing sarcoma. View Abstract
Exploiting an Asp-Glu "switch" in glycogen synthase kinase 3 to design paralog-selective inhibitors for use in acute myeloid leukemia. View Abstract
EWS/FLI Confers Tumor Cell Synthetic Lethality to CDK12 Inhibition in Ewing Sarcoma. View Abstract
CRISPR-Cas9 screen reveals a MYCN-amplified neuroblastoma dependency on EZH2. View Abstract
Leukemia-specific delivery of mutant NOTCH1 targeted therapy. View Abstract
Computational correction of copy number effect improves specificity of CRISPR-Cas9 essentiality screens in cancer cells. View Abstract
Identification of an allosteric benzothiazolopyrimidone inhibitor of the oncogenic protein tyrosine phosphatase SHP2. View Abstract
TOX Regulates Growth, DNA Repair, and Genomic Instability in T-cell Acute Lymphoblastic Leukemia. View Abstract
Scratching the Surface of Immunotherapeutic Targets in Neuroblastoma. View 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. View 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. View Abstract
Hoxa9 and Meis1 Cooperatively Induce Addiction to Syk Signaling by Suppressing miR-146a in Acute Myeloid Leukemia. View Abstract
The creatine kinase pathway is a metabolic vulnerability in EVI1-positive acute myeloid leukemia. View Abstract
Synergistic Drug Combinations with a CDK4/6 Inhibitor in T-cell Acute Lymphoblastic Leukemia. View Abstract
The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. View Abstract
Integrated genetic and pharmacologic interrogation of rare cancers. View Abstract
Targeting MTHFD2 in acute myeloid leukemia. View Abstract
Genomic Copy Number Dictates a Gene-Independent Cell Response to CRISPR/Cas9 Targeting. View Abstract
Inhibitors of Glycogen Synthase Kinase 3 with Exquisite Kinome-Wide Selectivity and Their Functional Effects. View Abstract
Multicenter Feasibility Study of Tumor Molecular Profiling to Inform Therapeutic Decisions in Advanced Pediatric Solid Tumors: The Individualized Cancer Therapy (iCat) Study. View Abstract
The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. View Abstract
Aberrant activation of the PI3K/mTOR pathway promotes resistance to sorafenib in AML. View Abstract
The second European interdisciplinary Ewing sarcoma research summit--A joint effort to deconstructing the multiple layers of a complex disease. View Abstract
Corrigendum: The genomic landscape of juvenile myelomonocytic leukemia. View Abstract
Evaluation of Improved Glycogen Synthase Kinase-3a Inhibitors in Models of Acute Myeloid Leukemia. View Abstract
The genomic landscape of juvenile myelomonocytic leukemia. View Abstract
Functional, chemical genomic, and super-enhancer screening identify sensitivity to cyclin D1/CDK4 pathway inhibition in Ewing sarcoma. View Abstract
Increased SYK activity is associated with unfavorable outcome among patients with acute myeloid leukemia. View Abstract
Long noncoding RNA EWSAT1-mediated gene repression facilitates Ewing sarcoma oncogenesis. View Abstract
Erratum: Parallel genome-scale loss of function screens in 216 cancer cell lines for the identification of context-specific genetic dependencies. View Abstract
Parallel genome-scale loss of function screens in 216 cancer cell lines for the identification of context-specific genetic dependencies. View Abstract
Molecular rationale for the use of PI3K/AKT/mTOR pathway inhibitors in combination with crizotinib in ALK-mutated neuroblastoma. View Abstract
The genomic landscape of pediatric Ewing sarcoma. View Abstract
New Approaches to Target T-ALL. View Abstract
Triplication of a 21q22 region contributes to B cell transformation through HMGN1 overexpression and loss of histone H3 Lys27 trimethylation. View Abstract
Targeting Csnk1a1 in leukemia. View Abstract
An epigenetic mechanism of resistance to targeted therapy in T cell acute lymphoblastic leukemia. View Abstract
SYK is a critical regulator of FLT3 in acute myeloid leukemia. View Abstract
MUC1-C oncoprotein promotes FLT3 receptor activation in acute myeloid leukemia cells. View Abstract
A phase II study of the EGFR inhibitor gefitinib in patients with acute myeloid leukemia. View Abstract
Mutational heterogeneity in cancer and the search for new cancer-associated genes. View Abstract
In Vivo RNAi screening identifies a leukemia-specific dependence on integrin beta 3 signaling. View Abstract
Selective HDAC1/HDAC2 inhibitors induce neuroblastoma differentiation. View Abstract
SYK regulates mTOR signaling in AML. View Abstract
High-throughput tyrosine kinase activity profiling identifies FAK as a candidate therapeutic target in Ewing sarcoma. View Abstract
Targeting MYCN in neuroblastoma by BET bromodomain inhibition. View Abstract
Complementary genomic screens identify SERCA as a therapeutic target in NOTCH1 mutated cancer. View Abstract
The intersection of genetic and chemical genomic screens identifies GSK-3a as a target in human acute myeloid leukemia. View Abstract
Genetic and proteomic approaches to identify cancer drug targets. View Abstract
Targeting NOTCH1 in hematopoietic malignancy. View Abstract
Chemical genomics and combo therapy. View Abstract
Characterization of Notch1 antibodies that inhibit signaling of both normal and mutated Notch1 receptors. View Abstract
Proteomic and genetic approaches identify Syk as an AML target. View Abstract
Identification of AML1-ETO modulators by chemical genomics. View Abstract
Genomic approaches to small molecule discovery. View Abstract
Phase II study of intermediate-dose cytarabine in patients with relapsed or refractory Ewing sarcoma: a report from the Children's Oncology Group. View Abstract
High-throughput gene expression profiling of memory differentiation in primary human T cells. View Abstract
Expression-based screening identifies the combination of histone deacetylase inhibitors and retinoids for neuroblastoma differentiation. View Abstract
Literature Lab: a method of automated literature interrogation to infer biology from microarray analysis. View Abstract
Signature-based small molecule screening identifies cytosine arabinoside as an EWS/FLI modulator in Ewing sarcoma. View Abstract
Gene expression signature-based chemical genomic prediction identifies a novel class of HSP90 pathway modulators. View Abstract
Genomic approaches in acute leukemia. View Abstract
A method for high-throughput gene expression signature analysis. View 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. View Abstract
Gefitinib induces myeloid differentiation of acute myeloid leukemia. View Abstract
Gene expression-based high-throughput screening(GE-HTS) and application to leukemia differentiation. View Abstract
The TEL gene contributes to the pathogenesis of myeloid and lymphoid leukemias by diverse molecular genetic mechanisms. View Abstract
TEL/AML-1 dimerizes and is associated with a favorable outcome in childhood acute lymphoblastic leukemia. View Abstract
The TEL gene and human leukemia. View Abstract
Mutational analysis of the candidate tumor suppressor genes TEL and KIP1 in childhood acute lymphoblastic leukemia. View Abstract
Involvement of the TEL gene in hematologic malignancy by diverse molecular genetic mechanisms. View Abstract
Frequent loss of heterozygosity at the TEL gene locus in acute lymphoblastic leukemia of childhood. View Abstract