Targeted therapy for fusion-driven high-risk acute leukemia

The DOT1L inhibitor pinometostat reduces H3K79 methylation and has modest clinical activity in adult acute leukemia

(2018) Eytan M. Stein et al.   BLOOD

A Non-catalytic Function of SETD1A Regulates Cyclin K and the DNA Damage Response

(2018) Takayuki Hoshii et al.   CELL

Larotrectinib for paediatric solid tumours harbouring NTRK gene fusions: phase 1 results from a multicentre, open-label, phase 1/2 study

(2018) Theodore W Laetsch et al.   LANCET ONCOLOGY

LKB1, Salt-Inducible Kinases, and MEF2C Are Linked Dependencies in Acute Myeloid Leukemia

(2018) Yusuke Tarumoto et al.   MOLECULAR CELL

Efficacy of Larotrectinib in TRK Fusion–Positive Cancers in Adults and Children

(2018) Alexander Drilon et al.   NEW ENGLAND JOURNAL OF MEDICINE

High Frequency and Poor Outcome of Philadelphia Chromosome–Like Acute Lymphoblastic Leukemia in Adults

(2017) Kathryn G. Roberts et al.   JOURNAL OF CLINICAL ONCOLOGY

Improved Outcomes With Retinoic Acid and Arsenic Trioxide Compared With Retinoic Acid and Chemotherapy in Non–High-Risk Acute Promyelocytic Leukemia: Final Results of the Randomized Italian-German APL0406 Trial

(2017) Uwe Platzbecker et al.   JOURNAL OF CLINICAL ONCOLOGY

Mechanisms of Pinometostat (EPZ-5676) Treatment–Emergent Resistance in MLL -Rearranged Leukemia

(2017) Carly T. Campbell et al.   MOLECULAR CANCER THERAPEUTICS

Pharmacological perturbation of CDK9 using selective CDK9 inhibition or degradation

(2017) Calla M Olson et al.   Nature Chemical Biology

Structural basis of PROTAC cooperative recognition for selective protein degradation

(2017) Morgan S Gadd et al.   Nature Chemical Biology

Safety and Antitumor Activity of the Multitargeted Pan-TRK, ROS1, and ALK Inhibitor Entrectinib: Combined Results from Two Phase I Trials (ALKA-372-001 and STARTRK-1)

(2017) Alexander Drilon et al.   Cancer Discovery

A Next-Generation TRK Kinase Inhibitor Overcomes Acquired Resistance to Prior TRK Kinase Inhibition in Patients with TRK Fusion–Positive Solid Tumors

(2017) Alexander Drilon et al.   Cancer Discovery

Precision Medicine in Children and Young Adults with Hematologic Malignancies and Blood Disorders: The Columbia University Experience

(2017) Lianna J. Marks et al.   Frontiers in Pediatrics

Integration of Next-Generation Sequencing to Treat Acute Lymphoblastic Leukemia with Targetable Lesions: The St. Jude Children’s Research Hospital Approach

(2017) Hiroto Inaba et al.   Frontiers in Pediatrics

Hyper-CVAD plus ponatinib versus hyper-CVAD plus dasatinib as frontline therapy for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia: A propensity score analysis

(2016) Koji Sasaki et al.   CANCER

NUP98 Fusion Proteins Interact with the NSL and MLL1 Complexes to Drive Leukemogenesis

(2016) Haiming Xu et al.   CANCER CELL

Breaking Cryo-EM Resolution Barriers to Facilitate Drug Discovery

(2016) Alan Merk et al.   CELL

Phase I Study of Selinexor, a Selective Inhibitor of Nuclear Export, in Combination With Fludarabine and Cytarabine, in Pediatric Relapsed or Refractory Acute Leukemia

(2016) Thomas B. Alexander et al.   JOURNAL OF CLINICAL ONCOLOGY

Complementary activities of DOT1L and Menin inhibitors in MLL-rearranged leukemia

(2016) C Dafflon et al.   LEUKEMIA

NUP98 is rearranged in 3.8% of pediatric AML forming a clinical and molecular homogenous group with a poor prognosis

(2016) S Struski et al.   LEUKEMIA

Leukemia-Associated Nup214 Fusion Proteins Disturb the XPO1-Mediated Nuclear-Cytoplasmic Transport Pathway and Thereby the NF-κB Signaling Pathway

(2016) Shoko Saito et al.   MOLECULAR AND CELLULAR BIOLOGY

The genomic landscape of core-binding factor acute myeloid leukemias

(2016) Zachary J Faber et al.   NATURE GENETICS

Transformation of human CD34+ hematopoietic progenitor cells with DEK-NUP214 induces AML in an immunocompromised mouse model

(2016) H Qin et al.   ONCOGENE

Implementation of next generation sequencing into pediatric hematology-oncology practice: moving beyond actionable alterations

(2016) Jennifer A. Oberg et al.   Genome Medicine

Selective Small Molecule Induced Degradation of the BET Bromodomain Protein BRD4

(2015) Michael Zengerle et al.   ACS Chemical Biology

Modular PROTAC Design for the Degradation of Oncogenic BCR-ABL

(2015) Ashton C. Lai et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Pharmacologic Inhibition of the Menin-MLL Interaction Blocks Progression of MLL Leukemia In Vivo

(2015) Dmitry Borkin et al.   CANCER CELL

Hijacking the E3 Ubiquitin Ligase Cereblon to Efficiently Target BRD4

(2015) Jing Lu et al.   CHEMISTRY & BIOLOGY

Menin-MLL inhibitors block oncogenic transformation by MLL-fusion proteins in a fusion partner-independent manner

(2015) S He et al.   LEUKEMIA

Discovery of cancer drug targets by CRISPR-Cas9 screening of protein domains

(2015) Junwei Shi et al.   NATURE BIOTECHNOLOGY

Catalytic in vivo protein knockdown by small-molecule PROTACs

(2015) Daniel P Bondeson et al.   Nature Chemical Biology

SWI/SNF-mutant cancers depend on catalytic and non-catalytic activity of EZH2

(2015) Kimberly H Kim et al.   NATURE MEDICINE

A small-molecule inhibitor of the aberrant transcription factor CBF -SMMHC delays leukemia in mice

(2015) A. Illendula et al.   SCIENCE

Phthalimide conjugation as a strategy for in vivo target protein degradation

(2015) G. E. Winter et al.   SCIENCE

Targetable Kinase-Activating Lesions in Ph-like Acute Lymphoblastic Leukemia

(2015) Kathryn G. Roberts et al.   NEW ENGLAND JOURNAL OF MEDICINE

Long-term outcome with dasatinib after imatinib failure in chronic-phase chronic myeloid leukemia: follow-up of a phase 3 study

(2014) N. P. Shah et al.   BLOOD

Childhood and adolescent cancer statistics, 2014

(2014) Elizabeth Ward et al.   CA-A CANCER JOURNAL FOR CLINICIANS

SYK Is a Critical Regulator of FLT3 in Acute Myeloid Leukemia

(2014) Alexandre Puissant et al.   CANCER CELL

Long-term follow-up of imatinib in pediatric Philadelphia chromosome-positive acute lymphoblastic leukemia: Children's Oncology Group Study AALL0031

(2014) K R Schultz et al.   LEUKEMIA

Phase II trial of HyperCVAD and Dasatinib in patients with relapsed Philadelphia chromosome positive acute lymphoblastic leukemia or blast phase chronic myeloid leukemia

(2013) Ohad Benjamini et al.   AMERICAN JOURNAL OF HEMATOLOGY

CBFA2T3-GLIS2 fusion transcript is a novel common feature in pediatric, cytogenetically normal AML, not restricted to FAB M7 subtype

(2013) R. Masetti et al.   BLOOD

Potent inhibition of DOT1L as treatment of MLL-fusion leukemia

(2013) S. R. Daigle et al.   BLOOD

Synergy against PML-RARa: targeting transcription, proteolysis, differentiation, and self-renewal in acute promyelocytic leukemia

(2013) Guilherme Augusto dos Santos et al.   JOURNAL OF EXPERIMENTAL MEDICINE

A Phase 2 Trial of Ponatinib in Philadelphia Chromosome–Positive Leukemias

(2013) J.E. Cortes et al.   NEW ENGLAND JOURNAL OF MEDICINE

Retinoic Acid and Arsenic Trioxide for Acute Promyelocytic Leukemia

(2013) Francesco Lo-Coco et al.   NEW ENGLAND JOURNAL OF MEDICINE

Lenalidomide Causes Selective Degradation of IKZF1 and IKZF3 in Multiple Myeloma Cells

(2013) J. Kronke et al.   SCIENCE

The Myeloma Drug Lenalidomide Promotes the Cereblon-Dependent Destruction of Ikaros Proteins

(2013) G. Lu et al.   SCIENCE

Targeting JAK1/2 and mTOR in murine xenograft models of Ph-like acute lymphoblastic leukemia

(2012) S. L. Maude et al.   BLOOD

Genetic Alterations Activating Kinase and Cytokine Receptor Signaling in High-Risk Acute Lymphoblastic Leukemia

(2012) Kathryn G. Roberts et al.   CANCER CELL

Imatinib after induction for treatment of children and adolescents with Philadelphia-chromosome-positive acute lymphoblastic leukaemia (EsPhALL): a randomised, open-label, intergroup study

(2012) Andrea Biondi et al.   LANCET ONCOLOGY

The same pocket in menin binds both MLL and JUND but has opposite effects on transcription

(2012) Jing Huang et al.   NATURE

Menin-MLL inhibitors reverse oncogenic activity of MLL fusion proteins in leukemia

(2012) Jolanta Grembecka et al.   Nature Chemical Biology

NUP98 gene fusions and hematopoietic malignancies: common themes and new biologic insights

(2011) S. M. Gough et al.   BLOOD

Selective Killing of Mixed Lineage Leukemia Cells by a Potent Small-Molecule DOT1L Inhibitor

(2011) Scott R. Daigle et al.   CANCER CELL

MLL-Rearranged Leukemia Is Dependent on Aberrant H3K79 Methylation by DOT1L

(2011) Kathrin M. Bernt et al.   CANCER CELL

Crystal Structure of Menin Reveals Binding Site for Mixed Lineage Leukemia (MLL) Protein

(2011) Marcelo J. Murai et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Improved outcome with hematopoietic stem cell transplantation in a poor prognostic subgroup of infants with mixed-lineage-leukemia (MLL)-rearranged acute lymphoblastic leukemia: results from the Interfant-99 Study

(2010) G. Mann et al.   BLOOD

Molecular Basis of the Mixed Lineage Leukemia-Menin Interaction

(2010) Jolanta Grembecka et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Inhibition of CRM1-mediated Nuclear Export of Transcription Factors by Leukemogenic NUP98 Fusion Proteins

(2010) Akiko Takeda et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Arsenic Trioxide Controls the Fate of the PML-RAR  Oncoprotein by Directly Binding PML

(2010) X. W. Zhang et al.   SCIENCE

Proteomic and Genetic Approaches Identify Syk as an AML Target

(2009) Cynthia K. Hahn et al.   CANCER CELL

Improved Early Event-Free Survival With Imatinib in Philadelphia Chromosome–Positive Acute Lymphoblastic Leukemia: A Children's Oncology Group Study

(2009) Kirk R. Schultz et al.   JOURNAL OF CLINICAL ONCOLOGY

A subtype of childhood acute lymphoblastic leukaemia with poor treatment outcome: a genome-wide classification study

(2009) Monique L Den Boer et al.   LANCET ONCOLOGY

Deletion ofIKZF1and Prognosis in Acute Lymphoblastic Leukemia

(2009) Charles G. Mullighan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Acute promyelocytic leukemia: from highly fatal to highly curable

(2008) Z.-Y. Wang et al.   BLOOD