Molecular Approaches to Treating Pediatric Leukemias

Glucocorticoids and selumetinib are highly synergistic in RAS pathway mutated childhood acute lymphoblastic leukemia through upregulation of BIM.

(2019) Elizabeth C. Matheson et al.   HAEMATOLOGICA

Properties of FDA-approved small molecule protein kinase inhibitors

(2019) Robert Roskoski   PHARMACOLOGICAL RESEARCH

Distinct mutation spectrum, clinical outcome and therapeutic responses of typical complex/monosomy karyotype acute myeloid leukemia carrying TP53 mutations

(2019) Garret M.K. Leung et al.   AMERICAN JOURNAL OF HEMATOLOGY

Therapeutic targeting of mutant p53 in pediatric acute lymphoblastic leukemia

(2019) Salih Demir et al.   HAEMATOLOGICA

Neddylation: a novel modulator of the tumor microenvironment

(2019) Lisha Zhou et al.   Molecular Cancer

TAS4464, a highly potent and selective inhibitor of NEDD8 activating enzyme, suppresses neddylation and shows antitumor activity in diverse cancer models

(2019) Chihoko Yoshimura et al.   MOLECULAR CANCER THERAPEUTICS

TET2 Function in Hematopoietic Malignancies, Immune Regulation, and DNA Repair

(2019) Yimei Feng et al.   Frontiers in Oncology

Mechanisms of Progression of Myeloid Preleukemia to Transformed Myeloid Leukemia in Children with Down Syndrome

(2019) Maurice Labuhn et al.   CANCER CELL

Histone Deacetylase Inhibition with Panobinostat Combined with Intensive Induction Chemotherapy in Older Patients with Acute Myeloid Leukemia: Phase I Study Results

(2019) Matthew J. Wieduwilt et al.   CLINICAL CANCER RESEARCH

A high-throughput screen indicates gemcitabine and JAK inhibitors may be useful for treating pediatric AML

(2019) Christina D. Drenberg et al.   Nature Communications

Rewiring the Epigenetic Networks in MLL-Rearranged Leukemias: Epigenetic Dysregulation and Pharmacological Interventions

(2019) Anthony K. N. Chan et al.   Frontiers in Cell and Developmental Biology

Design of the First-in-Class, Highly Potent Irreversible Inhibitor Targeting the Menin-MLL Protein-Protein Interaction

(2018) Shilin Xu et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Ribociclib, a Cdk4/Cdk6 kinase inhibitor, enhances glucocorticoid sensitivity in B-acute lymphoblastic leukemia (B-All)

(2018) Roberta Bortolozzi et al.   BIOCHEMICAL PHARMACOLOGY

A phase 1 study of azacitidine combined with chemotherapy in childhood leukemia: a report from the TACL consortium

(2018) Weili Sun et al.   BLOOD

Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, combined with azacitidine in patients with AML

(2018) Ronan T. Swords et al.   BLOOD

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

(2018) Eytan M. Stein et al.   BLOOD

Tyrosine kinase inhibitor–induced defects in DNA repair sensitize FLT3(ITD)-positive leukemia cells to PARP1 inhibitors

(2018) Silvia Maifrede et al.   BLOOD

CBFβ-SMMHC Inhibition Triggers Apoptosis by Disrupting MYC Chromatin Dynamics in Acute Myeloid Leukemia

(2018) John Anto Pulikkan et al.   CELL

Complexity of Blocking Bivalent Protein–Protein Interactions: Development of a Highly Potent Inhibitor of the Menin–Mixed-Lineage Leukemia Interaction

(2018) Dmitry Borkin et al.   JOURNAL OF MEDICINAL CHEMISTRY

Successes and challenges in the treatment of pediatric acute myeloid leukemia: a retrospective analysis of the AML-BFM trials from 1987 to 2012

(2018) Mareike Rasche et al.   LEUKEMIA

Front-line imatinib treatment in children and adolescents with chronic myeloid leukemia: results from a phase III trial

(2018) Meinolf Suttorp et al.   LEUKEMIA

Kinase-targeted cancer therapies: progress, challenges and future directions

(2018) Khushwant S. Bhullar et al.   Molecular Cancer

The landscape of genomic alterations across childhood cancers

(2018) Susanne N. Gröbner et al.   NATURE

Kinase inhibitors: the road ahead

(2018) Fleur M. Ferguson et al.   NATURE REVIEWS DRUG DISCOVERY

Phase I trial of the mTOR inhibitor everolimus in combination with multi-agent chemotherapy in relapsed childhood acute lymphoblastic leukemia

(2018) Andrew E. Place et al.   PEDIATRIC BLOOD & CANCER

Monitoring and Management of Toxicities of Novel B Cell Signaling Agents

(2018) Joanna Rhodes et al.   Current Oncology Reports

A phase 1/2, open-label, dose-escalation study of midostaurin in children with relapsed or refractory acute leukaemia

(2018) C. Michel Zwaan et al.   BRITISH JOURNAL OF HAEMATOLOGY

Mechanisms of PARP inhibitor sensitivity and resistance

(2018) Alan D. D’Andrea   DNA REPAIR

PARP inhibitor re‑sensitizes Adriamycin resistant leukemia cells through DNA damage and apoptosis

(2018) Jie Wu et al.   Molecular Medicine Reports

Epigenetic drug combination induces remission in mouse xenograft models of pediatric acute myeloid leukemia

(2017) Anilkumar Gopalakrishnapillai et al.   LEUKEMIA RESEARCH

Jak Stat signaling and cancer: Opportunities, benefits and side effects of targeted inhibition

(2017) Bernd Groner et al.   MOLECULAR AND CELLULAR ENDOCRINOLOGY

From basic apoptosis discoveries to advanced selective BCL-2 family inhibitors

(2017) Avi Ashkenazi et al.   NATURE REVIEWS DRUG DISCOVERY

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

Assessment of Overall Survival, Quality of Life, and Safety Benefits Associated With New Cancer Medicines

(2017) Sebastian Salas-Vega et al.   JAMA Oncology

Dexamethasone vs prednisone in induction treatment of pediatric ALL: results of the randomized trial AIEOP-BFM ALL 2000

(2016) A. Moricke et al.   BLOOD

Therapy with low-dose azacitidine for MDS in children and young adults: a retrospective analysis of the EWOG-MDS study group

(2016) Annamaria M. Cseh et al.   BRITISH JOURNAL OF HAEMATOLOGY

Drugging the undruggables: exploring the ubiquitin system for drug development

(2016) Xiaodong Huang et al.   CELL RESEARCH

A Phase 1 Study of the PARP Inhibitor Veliparib in Combination with Temozolomide in Acute Myeloid Leukemia

(2016) Ivana Gojo et al.   CLINICAL CANCER RESEARCH

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

(2016) C Dafflon et al.   LEUKEMIA

Decitabine enhances chemosensitivity of early T-cell precursor-acute lymphoblastic leukemia cell lines and patient-derived samples

(2016) Benjamin Y. Lu et al.   LEUKEMIA & LYMPHOMA

Pediatric low-grade gliomas: implications of the biologic era

(2016) Roger J. Packer et al.   NEURO-ONCOLOGY

Mutational landscape, clonal evolution patterns, and role of RAS mutations in relapsed acute lymphoblastic leukemia

(2016) Koichi Oshima et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Targeting BCL-2 and ABL/LYN in Philadelphia chromosome–positive acute lymphoblastic leukemia

(2016) Jessica T. Leonard et al.   Science Translational Medicine

The role of the proteasome in AML

(2016) C M Csizmar et al.   Blood Cancer Journal

Everolimus induces Met inactivation by disrupting the FKBP12/Met complex

(2016) Lucia Raimondo et al.   Oncotarget

Long-Term Side Effects of Tyrosine Kinase Inhibitors in Chronic Myeloid Leukemia

(2016) Lauren Caldemeyer et al.   Current Hematologic Malignancy Reports

P53-MDM2 Pathway: Evidences for A New Targeted Therapeutic Approach in B-Acute Lymphoblastic Leukemia

(2016) Stefania Trino et al.   Frontiers in Pharmacology

The JAK-STAT Pathway: Impact on Human Disease and Therapeutic Intervention

(2015) John J. O'Shea et al.   Annual Review of Medicine

Pevonedistat (MLN4924), a First-in-Class NEDD8-activating enzyme inhibitor, in patients with acute myeloid leukaemia and myelodysplastic syndromes: a phase 1 study

(2015) Ronan T. Swords et al.   BRITISH JOURNAL OF HAEMATOLOGY

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

(2015) Dmitry Borkin et al.   CANCER CELL

CDK4/6 Inhibitor PD 0332991 Sensitizes Acute Myeloid Leukemia to Cytarabine-Mediated Cytotoxicity

(2015) C. Yang et al.   CANCER RESEARCH

Effective Targeting of the P53-MDM2 Axis in Preclinical Models of Infant MLL-Rearranged Acute Lymphoblastic Leukemia

(2015) J. Richmond et al.   CLINICAL CANCER RESEARCH

A Phase I/II Study of the Janus Kinase (JAK)1 and 2 Inhibitor Ruxolitinib in Patients With Relapsed or Refractory Acute Myeloid Leukemia

(2015) Naveen Pemmaraju et al.   Clinical Lymphoma Myeloma & Leukemia

Targeting DOT1L and HOX gene expression in MLL-rearranged leukemia and beyond

(2015) Chun-Wei Chen et al.   EXPERIMENTAL HEMATOLOGY

Collaborative Efforts Driving Progress in Pediatric Acute Myeloid Leukemia

(2015) C. Michel Zwaan et al.   JOURNAL OF CLINICAL ONCOLOGY

Childhood Acute Lymphoblastic Leukemia: Progress Through Collaboration

(2015) Ching-Hon Pui et al.   JOURNAL OF CLINICAL ONCOLOGY

Small PARP inhibitor PJ-34 induces cell cycle arrest and apoptosis of adult T-cell leukemia cells

(2015) Xue Tao Bai et al.   Journal of Hematology & Oncology

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

(2015) S He et al.   LEUKEMIA

Specific Antileukemic Activity of PD0332991, a CDK4/6 Inhibitor, against Philadelphia Chromosome-Positive Lymphoid Leukemia

(2015) A. Nemoto et al.   MOLECULAR CANCER THERAPEUTICS

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

(2015) A. Illendula et al.   SCIENCE

Molecular mechanism for USP7-mediated DNMT1 stabilization by acetylation

(2015) Jingdong Cheng et al.   Nature Communications

MLL-Rearranged Acute Lymphoblastic Leukemias Activate BCL-2 through H3K79 Methylation and Are Sensitive to the BCL-2-Specific Antagonist ABT-199

(2015) Juliana M. Benito et al.   Cell Reports

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

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

A therapeutic trial of decitabine and vorinostat in combination with chemotherapy for relapsed/refractory acute lymphoblastic leukemia

(2014) Michael J. Burke et al.   AMERICAN JOURNAL OF HEMATOLOGY

Ras pathway mutations are prevalent in relapsed childhood acute lymphoblastic leukemia and confer sensitivity to MEK inhibition

(2014) J. Irving et al.   BLOOD

Clinical relevance of molecular aberrations in paediatric acute myeloid leukaemia at first relapse

(2014) Costa Bachas et al.   BRITISH JOURNAL OF HAEMATOLOGY

A phase 1 clinical trial of vorinostat in combination with decitabine in patients with acute myeloid leukaemia or myelodysplastic syndrome

(2014) Mark Kirschbaum et al.   BRITISH JOURNAL OF HAEMATOLOGY

MLL fusion-driven activation ofCDK6potentiates proliferation inMLL-rearranged infant ALL

(2014) Marieke Van der Linden et al.   CELL CYCLE

Milestones in the Curability of Pediatric Cancers

(2014) Melissa M. Hudson et al.   JOURNAL OF CLINICAL ONCOLOGY

Evaluation of the In Vitro and In Vivo Efficacy of the JAK Inhibitor AZD1480 against JAK-Mutated Acute Lymphoblastic Leukemia

(2014) S. Suryani et al.   MOLECULAR CANCER THERAPEUTICS

Targeting the PI3K/mTOR Pathway in Pediatric Hematologic Malignancies

(2014) Sarah K. Tasian et al.   Frontiers in Oncology

Recent Advancements of Bortezomib in Acute Lymphocytic Leukemia Treatment

(2013) Xiao-Li Du et al.   ACTA HAEMATOLOGICA

Leukemic transformation by the MLL-AF6 fusion oncogene requires the H3K79 methyltransferase Dot1l

(2013) A. J. Deshpande et al.   BLOOD

Mutant IDH1 promotes leukemogenesis in vivo and can be specifically targeted in human AML

(2013) A. Chaturvedi et al.   BLOOD

Treat cancers by targeting survivin: Just a dream or future reality?

(2013) Mohane Selvaraj Coumar et al.   CANCER TREATMENT REVIEWS

Safety and pharmacokinetics of the antisense oligonucleotide (ASO) LY2181308 as a single-agent or in combination with idarubicin and cytarabine in patients with refractory or relapsed acute myeloid leukemia (AML)

(2013) Harry P. Erba et al.   INVESTIGATIONAL NEW DRUGS

Dasatinib in Children and Adolescents With Relapsed or Refractory Leukemia: Results of the CA180-018 Phase I Dose-Escalation Study of the Innovative Therapies for Children With Cancer Consortium

(2013) C. Michel Zwaan et al.   JOURNAL OF CLINICAL ONCOLOGY

A Phase I Study of EZN-3042, a Novel Survivin Messenger Ribonucleic Acid (mRNA) Antagonist, Administered in Combination With Chemotherapy in Children With Relapsed Acute Lymphoblastic Leukemia (ALL)

(2013) Elizabeth A. Raetz et al.   JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY

Targeting the PI3K/Akt/mTOR signaling pathway in B-precursor acute lymphoblastic leukemia and its therapeutic potential

(2013) L M Neri et al.   LEUKEMIA

Histone deacetylase inhibitors induce apoptosis in myeloid leukemia by suppressing autophagy

(2013) M V Stankov et al.   LEUKEMIA

mTOR kinase structure, mechanism and regulation

(2013) Haijuan Yang et al.   NATURE

Targeted Inhibition of Mutant IDH2 in Leukemia Cells Induces Cellular Differentiation

(2013) F. Wang et al.   SCIENCE

Bortezomib with chemotherapy is highly active in advanced B-precursor acute lymphoblastic leukemia: Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL) Study

(2012) Y. H. Messinger et al.   BLOOD

Structural insights into inhibition of the bivalent menin-MLL interaction by small molecules in leukemia

(2012) A. Shi et al.   BLOOD

Pediatric acute lymphoblastic leukemia: where are we going and how do we get there?

(2012) C.-H. Pui et al.   BLOOD

Epigenetic reprogramming reverses the relapse-specific gene expression signature and restores chemosensitivity in childhood B-lymphoblastic leukemia

(2012) T. Bhatla et al.   BLOOD

Cancer Epigenetics: From Mechanism to Therapy

(2012) Mark A. Dawson et al.   CELL

Targeting Mutant p53 in Human Tumors

(2012) Brian D. Lehmann et al.   JOURNAL OF CLINICAL ONCOLOGY

Targeting p53 in Vivo: A First-in-Human Study With p53-Targeting Compound APR-246 in Refractory Hematologic Malignancies and Prostate Cancer

(2012) Sören Lehmann et al.   JOURNAL OF CLINICAL ONCOLOGY

Improved Survival for Children and Adolescents With Acute Lymphoblastic Leukemia Between 1990 and 2005: A Report From the Children's Oncology Group

(2012) Stephen P. Hunger et al.   JOURNAL OF CLINICAL ONCOLOGY

Targeting survivin overcomes drug resistance in acute lymphoblastic leukemia

(2011) E. Park et al.   BLOOD

NEDD8 Pathways in Cancer, Sine Quibus Non

(2011) Ian R. Watson et al.   CANCER CELL

APR-246 exhibits anti-leukemic activity and synergism with conventional chemotherapeutic drugs in acute myeloid leukemia cells

(2010) Dina Ali et al.   EUROPEAN JOURNAL OF HAEMATOLOGY

The (un)targeted cancer kinome

(2010) Oleg Fedorov et al.   Nature Chemical Biology

DNMT3AMutations in Acute Myeloid Leukemia

(2010) Timothy J. Ley et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Comparison of Azacitidine and Decitabine Activities in Acute Myeloid Leukemia Cell Lines

(2010) Paul W. Hollenbach et al.   PLoS One

Long-term results of five consecutive trials in childhood acute lymphoblastic leukemia performed by the ALL-BFM study group from 1981 to 2000

(2009) A Möricke et al.   LEUKEMIA

DNA Methyltransferase and Histone Deacetylase Inhibitors in the Treatment of Myelodysplastic Syndromes

(2008) Elizabeth A. Griffiths et al.   SEMINARS IN HEMATOLOGY