Target and Agent Prioritization for the Children’s Oncology Group—National Cancer Institute Pediatric MATCH Trial

Abstract LB-178: Genomic profiling of 1239 diverse pediatric cancers identifies novel discoveries across tumors

(2016) Juliann Chmielecki et al.   CANCER RESEARCH

Identification of Oncogenic and Drug-Sensitizing Mutations in the Extracellular Domain of FGFR2

(2015) J. Tanizaki et al.   CANCER RESEARCH

The BET Bromodomain Inhibitor OTX015 Affects Pathogenetic Pathways in Preclinical B-cell Tumor Models and Synergizes with Targeted Drugs

(2015) M. Boi et al.   CLINICAL CANCER RESEARCH

Molecular Pathways: Targeting the Cyclin D-CDK4/6 Axis for Cancer Treatment

(2015) T. VanArsdale et al.   CLINICAL CANCER RESEARCH

CDK4 Amplification Reduces Sensitivity to CDK4/6 Inhibition in Fusion-Positive Rhabdomyosarcoma

(2015) M. E. Olanich et al.   CLINICAL CANCER RESEARCH

Detection, Characterization, and Inhibition of FGFR-TACC Fusions in IDH Wild-type Glioma

(2015) A. L. Di Stefano et al.   CLINICAL CANCER RESEARCH

Phase 2 study of dovitinib in patients with relapsed or refractory multiple myeloma with or without t(4;14) translocation

(2015) Christof Scheid et al.   EUROPEAN JOURNAL OF HAEMATOLOGY

Integrative Clinical Sequencing in the Management of Refractory or Relapsed Cancer in Youth

(2015) Rajen J. Mody et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Reproducible and Sustained Efficacy of Targeted Therapy With Vemurafenib in Patients With BRAFV600E-Mutated Erdheim-Chester Disease

(2015) Julien Haroche et al.   JOURNAL OF CLINICAL ONCOLOGY

The Genomic Landscape of Childhood and Adolescent Melanoma

(2015) Charles Lu et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

BRCA1 Circos: a visualisation resource for functional analysis of missense variants

(2015) Ankita Jhuraney et al.   JOURNAL OF MEDICAL GENETICS

Molecularly targeted therapy based on tumour molecular profiling versus conventional therapy for advanced cancer (SHIVA): a multicentre, open-label, proof-of-concept, randomised, controlled phase 2 trial

(2015) Christophe Le Tourneau et al.   LANCET ONCOLOGY

Relapsed neuroblastomas show frequent RAS-MAPK pathway mutations

(2015) Thomas F Eleveld et al.   NATURE GENETICS

Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations

(2015) David M. Hyman et al.   NEW ENGLAND JOURNAL OF MEDICINE

The ALK/ROS1 Inhibitor PF-06463922 Overcomes Primary Resistance to Crizotinib in ALK-Driven Neuroblastoma

(2015) N. R. Infarinato et al.   Cancer Discovery

An Oncogenic NTRK Fusion in a Patient with Soft-Tissue Sarcoma with Response to the Tropomyosin-Related Kinase Inhibitor LOXO-101

(2015) R. C. Doebele et al.   Cancer Discovery

Targeting CDK4 and CDK6: From Discovery to Therapy

(2015) C. J. Sherr et al.   Cancer Discovery

Vemurafenib Use in an Infant for High-Risk Langerhans Cell Histiocytosis

(2015) Sébastien Héritier et al.   JAMA Oncology

BET bromodomain protein inhibition is a therapeutic option for medulloblastoma

(2015) Anton George Henssen et al.   Oncotarget

Mutually exclusive recurrent somatic mutations in MAP2K1 and BRAF support a central role for ERK activation in LCH pathogenesis

(2014) R. Chakraborty et al.   BLOOD

ALK Mutations Confer Differential Oncogenic Activation and Sensitivity to ALK Inhibition Therapy in Neuroblastoma

(2014) Scott C. Bresler et al.   CANCER CELL

ERK Mutations Confer Resistance to Mitogen-Activated Protein Kinase Pathway Inhibitors

(2014) E. M. Goetz et al.   CANCER RESEARCH

Loss of NF1 in Cutaneous Melanoma Is Associated with RAS Activation and MEK Dependence

(2014) Moriah H. Nissan et al.   CANCER RESEARCH

RecurrentMYOD1mutations in pediatric and adult sclerosing and spindle cell rhabdomyosarcomas: Evidence for a common pathogenesis

(2014) Narasimhan P. Agaram et al.   GENES CHROMOSOMES & CANCER

Effect of Selumetinib vs Chemotherapy on Progression-Free Survival in Uveal Melanoma

(2014) Richard D. Carvajal et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

The BET Bromodomain Inhibitor I-BET151 Acts Downstream of Smoothened Protein to Abrogate the Growth of Hedgehog Protein-driven Cancers

(2014) Jun Long et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

BRAF-V600Eexpression in precursor versus differentiated dendritic cells defines clinically distinct LCH risk groups

(2014) Marie-Luise Berres et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Discovery and saturation analysis of cancer genes across 21 tumour types

(2014) Michael S. Lawrence et al.   NATURE

Epigenetic targeting of Hedgehog pathway transcriptional output through BET bromodomain inhibition

(2014) Yujie Tang et al.   NATURE MEDICINE

The clinical development of MEK inhibitors

(2014) Yujie Zhao et al.   Nature Reviews Clinical Oncology

Targeting RAS–ERK signalling in cancer: promises and challenges

(2014) Ahmed A. Samatar et al.   NATURE REVIEWS DRUG DISCOVERY

The effect of everolimus on renal angiomyolipoma in patients with tuberous sclerosis complex being treated for subependymal giant cell astrocytoma: subgroup results from the randomized, placebo-controlled, Phase 3 trial EXIST-1

(2014) J. C. Kingswood et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

Combined BRAF and MEK Inhibition versus BRAF Inhibition Alone in Melanoma

(2014) Georgina V. Long et al.   NEW ENGLAND JOURNAL OF MEDICINE

Initial testing (stage 1) of the PARP inhibitor BMN 673 by the pediatric preclinical testing program:PALB2mutation predicts exceptionalin vivoresponse to BMN 673

(2014) Malcolm A. Smith et al.   PEDIATRIC BLOOD & CANCER

FGFR2 Is Amplified in the NCI-H716 Colorectal Cancer Cell Line and Is Required for Growth and Survival

(2014) Anjili Mathur et al.   PLoS One

Complementary genomic approaches highlight the PI3K/mTOR pathway as a common vulnerability in osteosarcoma

(2014) Jennifer A. Perry et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

TRKing Down an Old Oncogene in a New Era of Targeted Therapy

(2014) Aria Vaishnavi et al.   Cancer Discovery

NSD3-NUT Fusion Oncoprotein in NUT Midline Carcinoma: Implications for a Novel Oncogenic Mechanism

(2014) C. A. French et al.   Cancer Discovery

Inflammatory Myofibroblastic Tumors Harbor Multiple Potentially Actionable Kinase Fusions

(2014) C. M. Lovly et al.   Cancer Discovery

Activating mTOR Mutations in a Patient with an Extraordinary Response on a Phase I Trial of Everolimus and Pazopanib

(2014) N. Wagle et al.   Cancer Discovery

Synthetic lethal interaction between PI3K/Akt/mTOR and Ras/MEK/ERK pathway inhibition in rhabdomyosarcoma

(2013) Monika Katharina Guenther et al.   CANCER LETTERS

Novel Oncogenic PDGFRA Mutations in Pediatric High-Grade Gliomas

(2013) B. S. Paugh et al.   CANCER RESEARCH

Neoadjuvant Imatinib in Advanced Primary or Locally Recurrent Dermatofibrosarcoma Protuberans: A Multicenter Phase II DeCOG Trial with Long-term Follow-up

(2013) S. Ugurel et al.   CLINICAL CANCER RESEARCH

Dual CDK4/CDK6 Inhibition Induces Cell-Cycle Arrest and Senescence in Neuroblastoma

(2013) J. Rader et al.   CLINICAL CANCER RESEARCH

Inhibition of BET Bromodomain Targets Genetically Diverse Glioblastoma

(2013) Z. Cheng et al.   CLINICAL CANCER RESEARCH

Dual Blockade of the PI3K/AKT/mTOR (AZD8055) and RAS/MEK/ERK (AZD6244) Pathways Synergistically Inhibits Rhabdomyosarcoma Cell Growth In Vitro and In Vivo

(2013) J. Renshaw et al.   CLINICAL CANCER RESEARCH

Phase I Study of Vismodegib in Children with Recurrent or Refractory Medulloblastoma: A Pediatric Brain Tumor Consortium Study

(2013) A. Gajjar et al.   CLINICAL CANCER RESEARCH

BET Bromodomain Inhibition of MYC-Amplified Medulloblastoma

(2013) P. Bandopadhayay et al.   CLINICAL CANCER RESEARCH

Targeting FGFR with Dovitinib (TKI258): Preclinical and Clinical Data in Breast Cancer

(2013) F. Andre et al.   CLINICAL CANCER RESEARCH

Designing Transformative Clinical Trials in the Cancer Genome Era

(2013) Stefan Sleijfer et al.   JOURNAL OF CLINICAL ONCOLOGY

Tyrosine Kinase Inhibitor Therapy Induces Remission in a Patient With Refractory EBF1-PDGFRB–Positive Acute Lymphoblastic Leukemia

(2013) Brent W. Weston et al.   JOURNAL OF CLINICAL ONCOLOGY

MEK162 for patients with advanced melanoma harbouring NRAS or Val600 BRAF mutations: a non-randomised, open-label phase 2 study

(2013) Paolo A Ascierto et al.   LANCET ONCOLOGY

Safety and activity of crizotinib for paediatric patients with refractory solid tumours or anaplastic large-cell lymphoma: a Children's Oncology Group phase 1 consortium study

(2013) Yael P Mossé et al.   LANCET ONCOLOGY

Oncogenic and drug-sensitive NTRK1 rearrangements in lung cancer

(2013) Aria Vaishnavi et al.   NATURE MEDICINE

Implementing personalized cancer genomics in clinical trials

(2013) Richard Simon et al.   NATURE REVIEWS DRUG DISCOVERY

Crizotinib versus Chemotherapy in Advanced ALK-Positive Lung Cancer

(2013) Alice T. Shaw et al.   NEW ENGLAND JOURNAL OF MEDICINE

BET Inhibition Silences Expression of MYCN and BCL2 and Induces Cytotoxicity in Neuroblastoma Tumor Models

(2013) Anastasia Wyce et al.   PLoS One

Targeting MYCN in Neuroblastoma by BET Bromodomain Inhibition

(2013) Alexandre Puissant et al.   Cancer Discovery

PIK3CA Mutation H1047R Is Associated with Response to PI3K/AKT/mTOR Signaling Pathway Inhibitors in Early-Phase Clinical Trials

(2012) F. Janku et al.   CANCER RESEARCH

Sensitivity of Glioblastomas to Clinically Available MEK Inhibitors Is Defined by Neurofibromin 1 Deficiency

(2012) W. L. See et al.   CANCER RESEARCH

Cyclin-dependent kinase 4/6 inhibition in cancer therapy

(2012) Neil Johnson et al.   CELL CYCLE

MEK inhibition exhibits efficacy in human and mouse neurofibromatosis tumors

(2012) Walter J. Jessen et al.   JOURNAL OF CLINICAL INVESTIGATION

PI3K/AKT/mTOR Inhibitors in Patients With Breast and Gynecologic Malignancies HarboringPIK3CAMutations

(2012) Filip Janku et al.   JOURNAL OF CLINICAL ONCOLOGY

Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial

(2012) David Neal Franz et al.   LANCET

Selumetinib plus docetaxel for KRAS-mutant advanced non-small-cell lung cancer: a randomised, multicentre, placebo-controlled, phase 2 study

(2012) Pasi A Jänne et al.   LANCET ONCOLOGY

Medulloblastoma exome sequencing uncovers subtype-specific somatic mutations

(2012) Trevor J. Pugh et al.   NATURE

p16-Cdk4-Rb axis controls sensitivity to a cyclin-dependent kinase inhibitor PD0332991 in glioblastoma xenograft cells

(2012) L. Cen et al.   NEURO-ONCOLOGY

Inhibiting the Hedgehog Pathway in Patients with the Basal-Cell Nevus Syndrome

(2012) Jean Y. Tang et al.   NEW ENGLAND JOURNAL OF MEDICINE

Combined BRAF and MEK Inhibition in Melanoma with BRAF V600 Mutations

(2012) Keith T. Flaherty et al.   NEW ENGLAND JOURNAL OF MEDICINE

Survival in BRAF V600–Mutant Advanced Melanoma Treated with Vemurafenib

(2012) Jeffrey A. Sosman et al.   NEW ENGLAND JOURNAL OF MEDICINE

Improved Survival with MEK Inhibition in BRAF-Mutated Melanoma

(2012) Keith T. Flaherty et al.   NEW ENGLAND JOURNAL OF MEDICINE

Mesenchymal Transition and PDGFRA Amplification/Mutation Are Key Distinct Oncogenic Events in Pediatric Diffuse Intrinsic Pontine Gliomas

(2012) Stephanie Puget et al.   PLoS One

KRASness and PIK3CAness in Patients with Advanced Colorectal Cancer: Outcome after Treatment with Early-Phase Trials with Targeted Pathway Inhibitors

(2012) Ignacio Garrido-Laguna et al.   PLoS One

Mechanisms of Acquired Crizotinib Resistance in ALK-Rearranged Lung Cancers

(2012) R. Katayama et al.   Science Translational Medicine

Functional Characterization of an Isoform-Selective Inhibitor of PI3K-p110β as a Potential Anticancer Agent

(2012) Jing Ni et al.   Cancer Discovery

Genomic Determinants of PI3K Pathway Inhibitor Response in Cancer

(2012) Britta Weigelt et al.   Frontiers in Oncology

Pathogenesis of NUT Midline Carcinoma

(2011) Christopher A. French   Annual Review of Pathology-Mechanisms of Disease

Sensitivity of malignant rhabdoid tumor cell lines to PD 0332991 is inversely correlated with p16 expression

(2011) Yoshiki Katsumi et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

MEK1 mutations, but not ERK2 mutations, occur in melanomas and colon carcinomas, but none in thyroid carcinomas

(2011) Avaniyapuram Kannan Murugan et al.   CELL CYCLE

A Phase I Trial of Liposomal Doxorubicin, Bevacizumab, and Temsirolimus in Patients with Advanced Gynecologic and Breast Malignancies

(2011) J. W. Moroney et al.   CLINICAL CANCER RESEARCH

The Selective Class I PI3K Inhibitor CH5132799 Targets Human Cancers Harboring Oncogenic PIK3CA Mutations

(2011) H. Tanaka et al.   CLINICAL CANCER RESEARCH

Oncogene Mutation Profiling of Pediatric Solid Tumors Reveals Significant Subsets of Embryonal Rhabdomyosarcoma and Neuroblastoma with Mutated Genes in Growth Signaling Pathways

(2011) N. Shukla et al.   CLINICAL CANCER RESEARCH

Phase II trial of the mTOR inhibitor, temsirolimus and evaluation of circulating tumor cells and tumor biomarkers in persistent and recurrent epithelial ovarian and primary peritoneal malignancies: A Gynecologic Oncology Group study

(2011) Kian Behbakht et al.   GYNECOLOGIC ONCOLOGY

Identification of human triple-negative breast cancer subtypes and preclinical models for selection of targeted therapies

(2011) Brian D. Lehmann et al.   JOURNAL OF CLINICAL INVESTIGATION

Biology, Risk Stratification, and Therapy of Pediatric Acute Leukemias: An Update

(2011) Ching-Hon Pui et al.   JOURNAL OF CLINICAL ONCOLOGY

Phase II Study of the Mammalian Target of Rapamycin Inhibitor Ridaforolimus in Patients With Advanced Bone and Soft Tissue Sarcomas

(2011) Sant P. Chawla et al.   JOURNAL OF CLINICAL ONCOLOGY

Common BRCA1 and BRCA2 mutations in breast cancer families: a meta-analysis from systematic review

(2011) Furu Wang et al.   MOLECULAR BIOLOGY REPORTS

PIK3CA Mutations in Patients with Advanced Cancers Treated with PI3K/AKT/mTOR Axis Inhibitors

(2011) F. Janku et al.   MOLECULAR CANCER THERAPEUTICS

Improved Survival with Vemurafenib in Melanoma with BRAF V600E Mutation

(2011) Paul B. Chapman et al.   NEW ENGLAND JOURNAL OF MEDICINE

PIK3CA mutation, but not PTEN loss of function, determines the sensitivity of breast cancer cells to mTOR inhibitory drugs

(2011) B Weigelt et al.   ONCOGENE

Critical oncogenic mutations in newly diagnosed pediatric diffuse intrinsic pontine glioma

(2011) Jacques Grill et al.   PEDIATRIC BLOOD & CANCER

Targeting MYC dependence in cancer by inhibiting BET bromodomains

(2011) J. A. Mertz et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Recurrent BRAF mutations in Langerhans cell histiocytosis

(2010) G. Badalian-Very et al.   BLOOD

Response and determinants of cancer cell susceptibility to PI3K inhibitors: Combined targeting of PI3K and Mek1 as an effective anticancer strategy

(2010) Ker Yu et al.   CANCER BIOLOGY & THERAPY

Isoform-Specific Phosphoinositide 3-Kinase Inhibitors Exert Distinct Effects in Solid Tumors

(2010) K. A. Edgar et al.   CANCER RESEARCH

Pharmacologic Inhibition of Cyclin-Dependent Kinases 4 and 6 Arrests the Growth of Glioblastoma Multiforme Intracranial Xenografts

(2010) K. Michaud et al.   CANCER RESEARCH

Imatinib Mesylate as a Preoperative Therapy in Dermatofibrosarcoma: Results of a Multicenter Phase II Study on 25 Patients

(2010) D. Kerob et al.   CLINICAL CANCER RESEARCH

Predictive Biomarkers of Sensitivity to the Phosphatidylinositol 3' Kinase Inhibitor GDC-0941 in Breast Cancer Preclinical Models

(2010) C. O'Brien et al.   CLINICAL CANCER RESEARCH

Phase II Study of Everolimus (RAD001) in Previously Treated Small Cell Lung Cancer

(2010) A. Tarhini et al.   CLINICAL CANCER RESEARCH

Prognostic but not predictive role of platelet-derived growth factor receptors in patients with recurrent glioblastoma

(2010) Janna Paulsson et al.   INTERNATIONAL JOURNAL OF CANCER

Deregulation of the PI3K and KRAS signaling pathways in human cancer cells determines their response to everolimus

(2010) Federica Di Nicolantonio et al.   JOURNAL OF CLINICAL INVESTIGATION

Whole-Genome Profiling of Pediatric Diffuse Intrinsic Pontine Gliomas Highlights Platelet-Derived Growth Factor Receptor α and Poly (ADP-ribose) Polymerase As Potential Therapeutic Targets

(2010) Maryam Zarghooni et al.   JOURNAL OF CLINICAL ONCOLOGY

ATM Deficiency Sensitizes Mantle Cell Lymphoma Cells to Poly(ADP-Ribose) Polymerase-1 Inhibitors

(2010) C. T. Williamson et al.   MOLECULAR CANCER THERAPEUTICS

Molecular Pharmacology and Antitumor Activity of PHT-427, a Novel Akt/Phosphatidylinositide-Dependent Protein Kinase 1 Pleckstrin Homology Domain Inhibitor

(2010) E. J. Meuillet et al.   MOLECULAR CANCER THERAPEUTICS

Clinical efficacy of a RAF inhibitor needs broad target blockade in BRAF-mutant melanoma

(2010) Gideon Bollag et al.   NATURE

Selective inhibition of BET bromodomains

(2010) Panagis Filippakopoulos et al.   NATURE

Anaplastic Lymphoma Kinase Inhibition in Non–Small-Cell Lung Cancer

(2010) Eunice L. Kwak et al.   NEW ENGLAND JOURNAL OF MEDICINE

Therapeutic CDK4/6 inhibition in breast cancer: key mechanisms of response and failure

(2010) J L Dean et al.   ONCOGENE

Pattern of retinoblastoma pathway inactivation dictates response to CDK4/6 inhibition in GBM

(2010) W. R. Wiedemeyer et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

AZD8055 Is a Potent, Selective, and Orally Bioavailable ATP-Competitive Mammalian Target of Rapamycin Kinase Inhibitor with In vitro and In vivo Antitumor Activity

(2009) C. M. Chresta et al.   CANCER RESEARCH

Biochemical, Cellular, and In vivo Activity of Novel ATP-Competitive and Selective Inhibitors of the Mammalian Target of Rapamycin

(2009) K. Yu et al.   CANCER RESEARCH

An ATP-competitive Mammalian Target of Rapamycin Inhibitor Reveals Rapamycin-resistant Functions of mTORC1

(2009) Carson C. Thoreen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

MEK/ERK inhibitor U0126 affects in vitro and in vivo growth of embryonal rhabdomyosarcoma

(2009) F. Marampon et al.   MOLECULAR CANCER THERAPEUTICS

Specific apoptosis induction by the dual PI3K/mTor inhibitor NVP-BEZ235 in HER2 amplified and PIK3CA mutant breast cancer cells

(2009) Saskia M. Brachmann et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

NVP-BEZ235, a Dual PI3K/mTOR Inhibitor, Prevents PI3K Signaling and Inhibits the Growth of Cancer Cells with Activating PI3K Mutations

(2008) V. Serra et al.   CANCER RESEARCH

Novel MEK1 Mutation Identified by Mutational Analysis of Epidermal Growth Factor Receptor Signaling Pathway Genes in Lung Adenocarcinoma

(2008) J. L. Marks et al.   CANCER RESEARCH

Phase II Study of Imatinib in Patients With Recurrent Gliomas of Various Histologies: A European Organisation for Research and Treatment of Cancer Brain Tumor Group Study

(2008) Eric Raymond et al.   JOURNAL OF CLINICAL ONCOLOGY

Identification of ALK as a major familial neuroblastoma predisposition gene

(2008) Yaël P. Mossé et al.   NATURE

Essential roles of PI(3)K–p110β in cell growth, metabolism and tumorigenesis

(2008) Shidong Jia et al.   NATURE

Somatic and germline activating mutations of the ALK kinase receptor in neuroblastoma

(2008) Isabelle Janoueix-Lerosey et al.   NATURE

Breast Tumor Cells with PI3K Mutation or HER2 Amplification Are Selectively Addicted to Akt Signaling

(2008) Qing-Bai She et al.   PLoS One

PTEN-deficient cancers depend on PIK3CB

(2008) S. Wee et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA