Stegmaier Lab Publications
Visit PubMed for a complete list of publications from the Stegmaier Lab.
Selected Publications
Combining menin and MEK inhibition to target poor prognosis KMT2A-rearranged RAS pathway-mutant acute myeloid leukemia
Scheidegger N.; Schneider C.; Alexe G.; ... Stegmaier, K.
Blood Advances. 2026. PDF
Identifying targeted therapies for CBFA2T3::GLIS2 acute myeloid leukemia
Gonzales, F.; Schneider, C.; Alexe G.; ... Stegmaier, K.
Leukemia. 2026. PDF
Rewiring the Fusion Oncoprotein EWSR1::FLI1 in Ewing Sarcoma with Bivalent Small Molecules
Bond, M. J.; Golden, R. P.; DiGiovanni, G.; … Stegmaier, K.
Journal of the American Chemical Society. 2025. PDF
Targeting the Sodium-Potassium Pump as a Therapeutic Strategy in Acute Myeloid Leukemia
Schneider, C.; Spaink, H.; Alexe, G.; … Stegmaier, K.
Cancer Research. 2024. PDF
The KAT Module of the SAGA Complex Maintains the Oncogenic Gene Expression Program in MYCN-Amplified Neuroblastoma
Malone, C. F.; Mabe, N. W.; Forman, A. B.; … Stegmaier, K.
Science Advances. 2024. PDF
The UBE2J2/UBE2K-MARCH5 Ubiquitination Machinery Regulates Apoptosis in Response to Venetoclax in Acute Myeloid Leukemia
Lin, S.; Schneider, C.; Su, A. H.; … Stegmaier, K.
Leukemia. 2024. PDF
The ETS Transcription Factor ETV6 Constrains the Transcriptional Activity of EWS-FLI to Promote Ewing Sarcoma
Lu, D. Y.; Ellegast, J. M.; Ross, K. N.; … Stegmaier, K.
Nature Cell Biology. 2023. PDF
Transcriptional Antagonism by CDK8 Inhibition Improves Therapeutic Efficacy of MEK Inhibitors
Malone, C. F.; Kim, M.; Alexe, G.; … Stegmaier, K.
Cancer Research. 2023. PDF
VRK1 as a Synthetic Lethal Target in VRK2 Promoter-Methylated Cancers of the Nervous System
So, J.; Mabe, N. W.; Englinger, B.; … Stegmaier, K.; Filbin, M. G.; Hahn, W. C.
JCI Insight. 2022. PDF
Transition to a Mesenchymal State in Neuroblastoma Confers Resistance to Anti-GD2 Antibody via Reduced Expression of ST8SIA1
Mabe, N. W.; Huang, M.; Dalton, G. N.; … Stegmaier, K.; Majzner, R. G.
Nature Cancer. 2022. PDF
Unleashing Cell-Intrinsic Inflammation as a Strategy to Kill AML Blasts
Ellegast, J. M.; Alexe, G.; Hamze, A.; … Stegmaier, K.
Cancer Discovery. 2022. PDF
Targeting Serine Hydroxymethyltransferases 1 and 2 for T-Cell Acute Lymphoblastic Leukemia Therapy
Pikman, Y.; Ocasio-Martinez, N.; Alexe, G.; … Stegmaier, K.
Leukemia. 2022. PDF
EP300 Selectively Controls the Enhancer Landscape of MYCN-Amplified Neuroblastoma
Durbin, A.; Wang, T.; Wimalasena, V.K.; ... Stegmaier, K.; Look, T.; & Qi, J.
Cancer Discovery. 2022. PDF
An In Vivo CRISPR Screening Platform for Prioritizing Therapeutic Targets in AML
Lin, S.; Larrue, C.; Scheidegger, N. K.; … Stegmaier, K.
Cancer Discovery. 2022. PDF
TRIM8 Modulates the EWS/FLI Oncoprotein to Promote Survival in Ewing Sarcoma
Seong, B. K. A.; Dharia, N. V.; Lin, S.; … Stegmaier, K.
Cancer Cell. 2021. PubMed
Selective Modulation of a Pan-Essential Protein as a Therapeutic Strategy in Cancer
Malone, C. F.; Dharia, N. V.; Kugener, G.; … Stegmaier, K.
Cancer Discovery. 2021. PubMed
STAG2 Loss Rewires Oncogenic and Developmental Programs to Promote Metastasis in Ewing Sarcoma
Adane, B.; Alexe, G.; Seong, B. K. A.; … Stegmaier, K.
Cancer Cell. 2021. PDF
Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory, or High-Risk Leukemias: A Report from the LEAP Consortium
Pikman, Y.; Tasian, S. K.; Sulis, M. L.; … Stegmaier, K.
Cancer Discovery. 2021. PubMed
A First-Generation Pediatric Cancer Dependency Map
Dharia, N. V.; Kugener, G.; Guenther, L. M.; … Vazquez, F.; Stegmaier, K.
Nature Genetics. 2021. PDF
Targeting Acute Myeloid Leukemia Dependency on VCP-Mediated DNA Repair through a Selective Second-Generation Small-Molecule Inhibitor
Roux, B.; Vaganay, C.; Vargas, J. D.; … Puissant, A.; Stegmaier, K.; Benajiba, L.
Science Translational Medicine. 2021. PubMed
Targeting EZH2 for the Treatment of Hepatosplenic T-Cell Lymphoma
Pikman, Y.; Conway, A. S.; Robichaud, A. L.; … Stegmaier, K.
Blood Advances. 2020. PDF
Resistance Mechanisms to SYK Inhibition in Acute Myeloid Leukemia
Cremer, A.; Ellegast, J. M.; Alexe, G.; … Stegmaier, K.
Cancer Discovery. 2020. PubMed
High-Throughput Chemical Screening Identifies Focal Adhesion Kinase and Aurora Kinase B Inhibition as a Synergistic Treatment Combination in Ewing Sarcoma
Wang, S.; Hwang, E. E.; Guha, R.; … Stegmaier, K.; Crompton, B. D.
Clinical Cancer Research. 2019. PDF
Ushering in the next Generation of Precision Trials for Pediatric Cancer
DuBois, S. G.; Corson, L. B.; Stegmaier, K.; Janeway, K. A.
Science. 2019.
Creatine Kinase Pathway Inhibition Alters GSK3 and WNT Signaling in EVI1-Positive AML
Benajiba, L.; Alexe, G.; Su, A.; … Stegmaier, K.; Puissant, A.
Leukemia. 2019.
A Combination CDK4/6 and IGF1R Inhibitor Strategy for Ewing Sarcoma
Guenther, L. M.; Dharia, N. V.; Ross, L.; … Stegmaier, K.
Clinical Cancer Research. 2019. PDF
Resistance to Epigenetic-Targeted Therapy Engenders Tumor Cell Vulnerabilities Associated with Enhancer Remodeling
Iniguez, A. B.; Alexe, G.; Wang, E. J.; … Stegmaier, K.
Cancer Cell. 2018. PDF
Targeted Therapy for Fusion-Driven High-Risk Acute Leukemia
Pikman, Y.; Stegmaier, K.
Blood. 2018. PDF
Selective Gene Dependencies in MYCN-Amplified Neuroblastoma Include the Core Transcriptional Regulatory Circuitry
Durbin, A. D.; Zimmerman, M. W.; Dharia, N. V.; … Young, R. A.; Look, A. T.; Stegmaier, K.
Nature Genetics. 2018.
Genome-Scale CRISPR-Cas9 Screen Identifies Druggable Dependencies in TP53 Wild-Type Ewing Sarcoma
Stolte, B.; Iniguez, A. B.; Dharia, N. V.; … Walensky, L. D.; Stegmaier, K.
Journal of Experimental Medicine. 2018. PDF
Exploiting an Asp-Glu “Switch” in Glycogen Synthase Kinase 3 to Design Paralog-Selective Inhibitors for Use in Acute Myeloid Leukemia
Wagner, F. F.; Benajiba, L.; Campbell, A. J.; Weïwer, M.; … Stegmaier, K.; Holson, E. B.
Science Translational Medicine. 2018.
EWS/FLI Confers Tumor Cell Synthetic Lethality to CDK12 Inhibition in Ewing Sarcoma
Iniguez, A. B.; Stolte, B.; Wang, E. J.; … Stegmaier, K.
Cancer Cell. 2018. PDF
Leukemia-Specific Delivery of Mutant NOTCH1 Targeted Therapy
Roti, G.; Qi, J.; Kitara, S.; Sanchez-Martin, M.; … Stegmaier, K. J
Journal of Experimental Medicine. 2018. PDF
CRISPR-Cas9 Screen Reveals a MYCN-Amplified Neuroblastoma Dependency on EZH2
Chen, L.; Alexe, G.; Dharia, N. V.; … Stegmaier, K.
Journal of Clinical Investigation. 2018.
Scratching the Surface of Immunotherapeutic Targets in Neuroblastoma
Malone, C. F.; Stegmaier, K.
Cancer Cell. 2017.
The Creatine Kinase Pathway Is a Metabolic Vulnerability in EVI1-Positive Acute Myeloid Leukemia
Fenouille, N.; Bassil, C. F.; Ben-Sahra, I.; … Stegmaier, K.
Nature Medicine. 2017.
Synergistic Drug Combinations with a CDK4/6 Inhibitor in T-Cell Acute Lymphoblastic Leukemia
Pikman, Y.; Alexe, G.; Roti, G.; Conway, A. S.; … Stegmaier, K.
Clinical Cancer Research. 2017.
Targeting MTHFD2 in Acute Myeloid Leukemia
Pikman, Y.; Puissant, A.; Alexe, G.; … Stegmaier, K.
Journal of Experimental Medicine. 2016.
Evaluation of Improved Glycogen Synthase Kinase-3α Inhibitors in Models of Acute Myeloid Leukemia
Neumann, T.; Benajiba, L.; Göring, S.; Stegmaier, K.; Schmidt, B.
Journal of Medicinal Chemistry. 2015.
Functional, Chemical Genomic, and Super-Enhancer Screening Identify Sensitivity to Cyclin D1/CDK4 Pathway Inhibition in Ewing Sarcoma
Kennedy, A. L.; Vallurupalli, M.; Chen, L.; … Stegmaier, K.
Oncotarget. 2015.
Increased SYK Activity Is Associated with Unfavorable Outcome among Patients with Acute Myeloid Leukemia
Boros, K.; Puissant, A.; … Stegmaier, K.; Byers, R. J.; Rodig, S. J.
Oncotarget 2015.
The Genomic Landscape of Pediatric Ewing Sarcoma
Crompton, B. D.; Stewart, C.; … Getz, G.; Mora, J.; Stegmaier, K.
Cancer Discovery. 2014.
SYK Is a Critical Regulator of FLT3 in Acute Myeloid Leukemia
Puissant, A.; Fenouille, N.; Alexe, G.; … Stegmaier, K.
Cancer Cell. 2014.
SYK Regulates mTOR Signaling in AML
Carnevale, J.; Ross, L.; Puissant, A.; … Stegmaier, K.
Leukemia. 2013.
Selective HDAC1/HDAC2 Inhibitors Induce Neuroblastoma Differentiation
Frumm, S. M.; Fan, Z. P.; Ross, K. N.; … Stegmaier, K.
Chemistry & Biology. 2013.
High-Throughput Tyrosine Kinase Activity Profiling Identifies FAK as a Candidate Therapeutic Target in Ewing Sarcoma
Crompton, B. D.; Carlton, A. L.; Thorner, A. R.; … Stegmaier, K.
Cancer Research. 2013.
Complementary Genomic Screens Identify SERCA as a Therapeutic Target in NOTCH1 Mutated Cancer
Roti, G.; Carlton, A.; Ross, K. N.; … Stegmaier, K.
Cancer Cell. 2013.
Targeting MYCN in Neuroblastoma by BET Bromodomain Inhibition
Puissant, A.; Frumm, S. M.; Alexe, G.; … Stegmaier, K.
Cancer Discovery. 2013.
The Intersection of Genetic and Chemical Genomic Screens Identifies GSK-3α as a Target in Human Acute Myeloid Leukemia
Banerji, V.; Frumm, S. M.; Ross, K. N.; … Stegmaier, K.
Journal of Clinical Investigation. 2012.
Genetic and Proteomic Approaches to Identify Cancer Drug Targets
Roti, G.; Stegmaier, K.
British Journal of Cancer. 2012.
Chemical Genomics and Combo Therapy
Stegmaier, K.
Blood. 2010.
Proteomic and Genetic Approaches Identify Syk as an AML Target
Hahn, C. K.; Berchuck, J. E.; Ross, K. N.; … Stegmaier, K.
Cancer Cell. 2009.
Identification of AML1-ETO Modulators by Chemical Genomics
Corsello, S. M.; Roti, G.; Ross, K. N.; … Stegmaier, K.
Blood. 2009.
Phase II Study of Intermediate-Dose Cytarabine in Patients with Relapsed or Refractory Ewing Sarcoma: A Report from the Children’s Oncology Group
DuBois, S. G.; Krailo, M. D.; Lessnick, S. L.; … Stegmaier, K.
Pediatric Blood & Cancer. 2009.
High-Throughput Gene Expression Profiling of Memory Differentiation in Primary Human T Cells
Haining, W. N.; Angelosanto, J.; Brosnahan, K.; … Stegmaier, K.
BMC Immunology. 2008.
Expression-Based Screening Identifies the Combination of Histone Deacetylase Inhibitors and Retinoids for Neuroblastoma Differentiation
Hahn, C. K.; Ross, K. N.; Warrington, I. M.; … Stegmaier, K.
PNAS. 2008.
Signature-Based Small Molecule Screening Identifies Cytosine Arabinoside as an EWS/FLI Modulator in Ewing Sarcoma
Stegmaier, K.; Wong, J. S.; Ross, K. N.; … Golub, T. R.
PLoS Medicine. 2007.
Gefitinib Induces Myeloid Differentiation of Acute Myeloid Leukemia
Stegmaier, K.; Corsello, S. M.; Ross, K. N.; … Golub, T. R.
Blood. 2005.
Gene Expression-Based High-Throughput Screening (GE-HTS) and Application to Leukemia Differentiation
Stegmaier, K.; Ross, K. N.; Colavito, S. A.; … Golub, T. R.
Nature Genetics. 2004.
