Childhood Cancer Research

Innovation Grants

These grants are designed to provide critical and significant seed funding for experienced investigators with a novel and promising approach to finding causes and cures for childhood cancers. A Letter of Intent is required. The Innovation Award amount totals $250,000 over two years. The Award may not be renewed, however, one no cost extensions are allowable.

Download the 2026 Innovation Grant Full Application Guidelines

All Accelerator Programs All ALSF Funded Projects
2025
Unraveling the Origins and Therapeutic Vulnerabilities of RBM15-MKL1-Driven AMKL
Diane Krause, MD/PhD
Yale School of Medicine
2025
Targeting DIPG through therapeutic dietary intervention
Chao Lu, PhD
Columbia University Medical Center
2025
Boosting antitumor immunity to treat pediatric choroid plexus carcinoma
Weibo Luo, PhD
University of Texas Southwestern Medical Center
2025
Dissecting the disease evolution of myeloid leukemia of Down Syndrome using iPSC models
Hanna Mikkola, MD/PhD
University of California, Los Angeles
2025
Efficient Derivation of Isogenic Hepatoblastoma Cell Lines Bearing Genetically-Defined Driver Mutations
Edward Prochownik, MD/PhD
University of Pittsburgh
2025
Using a novel epigenetic therapy to drive differentiation of atypical teratoid rhabdoid cells and facilitate immunotherapy
Eric H. Raabe, MD/PhD
The Johns Hopkins University School of Medicine
2025
A Novel Zebrafish Model of Diffuse Midline Glioma
Owen Tamplin, PhD
University of Wisconsin - Madison
2025
The NSL histone acetyltransferase complex as a therapeutic target for N-MYC-driven neuroblastoma
William Tansey, PhD
Vanderbilt University
2025
Multiplex Engineered Gamma Delta CAR T Cell Therapy for DIPG
Beau Webber, PhD
The Regents of the University of Minnesota
2025
Extracellular vesicle cargo as biomarkers and molecular drivers of germ cell cancers
Robert Weiss, PhD
Cornell University
2025
Tumor-Targeted Nanocarriers for Dual-Selective Therapy of Chemoresistant Neuroblastoma
Michael Chorny, PhD
Children’s Hospital of Philadelphia
2025
Targeting DIPG with engineered macrophages
Mingye Feng, PhD
Beckman Research Institute of City of Hope
2025
Targeting core malignant programs in pediatric high-grade glioma
Marco Gallo, PhD
Baylor College of Medicine
2025
Exploiting the Retina to Study Angiogenesis and Immunoprotection in Alveolar Soft Part Sarcoma
Kevin Jones, MD
University of Utah
2024
Direct targeting of oncoprotein assembly using mimetic peptide interference
Alex Kentsis, MD/PhD
Memorial Sloan-Kettering Cancer Center
2024
Development of CAR-T therapy for Ewing sarcoma using zebrafish in vivo models
James Amatruda, MD/PhD
Children’s Hospital Los Angeles
2024
Pathological Condensates and Transcription Dysregulation in Rhabdomyosarcoma at Single-Molecule Resolution
Shasha Chong, PhD
California Institute of Technology
2024
Targeting of a putative unresolved wound healing process in childhood ependymoma
Nicholas Foreman, MD
University of Colorado Denver
2024
Illuminating Choroid Plexus Carcinoma Vascularization in Real Time
Maria Lehtinen, PhD
Boston Children’s Hospital
2024
Unraveling the Mechanisms of Inherited Predisposition to Childhood Leukemia
Vijay Sankaran, MD/PhD
Boston Children’s Hospital
2024
The role of tissue kallikrein 1 in hematopoietic stem cell transplant-associated thrombotic microangiopathy
Sarah Sartain, MD
Baylor College of Medicine
2024
RAF Dimer Inhibitor Treatment in Pediatric Acute Myeloid Leukemia
Kevin Shannon, MD
The Regents of the University of California San Francisco
2024
DMG: From SWI/SNF’s role in cholesterol biosynthesis to new therapeutic opportunities
Capucine Van Rechem, PhD
Board of Trustees of the Leland Stanford Junior University
2024
Targeting and imaging immunometabolic vulnerabilities in diffuse midline glioma
Pavithra Viswanath, PhD
The Regents of the University of California San Francisco
2024
The role of reactive gliosis in response to Immunotherapy in Diffuse Midline Glioma
Christina von Roemeling, PhD
University of Florida
2024
Development of allogeneic off-the-shelf gamma delta T cell product for pediatric AML
Norihiro Watanabe, PhD
Baylor College of Medicine
2024
Targeting the immunosuppressive effects of Myc-hyperactivation in high-risk osteosarcoma
Jason Yustein, MD/PhD
Emory University
2024
Targeting cancer-specific non-protein molecular signatures for immunotherapy of pediatric AML
Johannes Zakrzewski, MD
Hackensack Meridian Health Center for Discovery and Innovation
2024
Investigating mechanistic roles of a novel oncofusion-driven transcript isoform in Ewing sarcoma
Poul Sorensen, MD/PhD
BC Cancer (Provincial Health Services Authority)
2023
Novel Resilience Genes Required for Cerebellum Development and Medulloblastoma Survival
Stephen Fancy, PhD, DVM
University of California San Francisco
2023
Targeting Neurons in the Osteosarcoma Microenvironment with Non-Opioid Analgesic Lipid Nanoparticles
Aaron James, MD/PhD
The Johns Hopkins University School of Medicine
2023
Desmoplastic Small Round Cell Tumors: Investigation of its Pathogenesis and Development of Novel Therapies
Andrea Ventura, MD/PhD
Memorial Sloan-Kettering Cancer Center
2023
CDK12 Links Radiation Response to PARPi Sensitivity in Pediatric Glioma
Nathan Dahl, MD
University of Colorado Denver_ Anschutz Medical Campus
2023
Targeting Chromatin Remodeling Complexes for Rhabdomyosarcoma (RMS) Therapy
Jun Qi, PhD
Dana-Farber Cancer Institute
2023
Stapled Oncolytic Peptides for Treatment-Resistant Pediatric Leukemias
Loren Walensky, MD/PhD
Dana-Farber Cancer Institute
2023
Development of RADARS-based Therapy to Treat Fusion Molecule Driven Pediatric Malignancies
Alan Cantor, MD/PhD
Boston Children’s Hospital
2023
Mechanisms to Redirect Tumor Associated Macrophages to Target Retinoblastoma Cells
Timothy Hallstrom, PhD
The Regents of the University of Minnesota
2023
Ectopic Neuroligin Signaling in Pediatric Glioblastoma
Alex Shcheglovitov, PhD
University of Utah
2023
The Impact of the Intestinal Microbiome on Sarcoma Metastasis
David Loeb, MD/PhD
Albert Einstein College of Medicine
2023
Inducing Neuroblastoma Differentiation In Vivo
Ankur Saxena, PhD
University of Alabama at Birmingham
2023
Structure-guided de novo design of molecules that selectively target EWS-FLI1
David Libich, PhD
University of Texas Health Science Center at San Antonio
2023
In Situ CD19.CAR-T Generation Using TRIDENT System for Pediatric B Cell Malignancies
Masataka Suzuki, PhD
Baylor College of Medicine
2022
Isoform-specific TNC-redirected T cell Therapy for DIPG
Stephen Gottschalk, MD, Giedre Krenciute, PhD
St. Jude Children’s Research Hospital
2022
Impact of STAG2 loss on DNA Damage and Immunobiology in Ewing sarcoma
Kelly Bailey, MD/PhD
University of Pittsburgh
2022
Targeted nanoparticle-mediated “cold”-to-“hot” reprogramming of the tumor microenvironment of high-risk neuroblastoma
Prabhani Atukorale, PhD, Jason Shohet, MD/PhD
University of Massachusetts Amherst, University of Massachusetts Chan Medical School
2022
Blocking the Evil Messenger in MYCN-driven High-risk Neuroblastoma
Hui Feng, Md, PhD
Boston University
2022
Developmental origins of leukemia in Down Syndrome
James Palis, MD, Laurie Steiner, MD
University of Rochester
2022
Molecular circuitry of fibrolamellar carcinoma
Nabeel Bardeesy, PhD
Massachusetts General Hospital
2022
Epigenetic Enhancement of MHCI to Augment Neuroblastoma Immunotherapy
Paul Sondel, MD/PhD, Amy Erbe-Gurel, PhD
University of Wisconsin - Madison
2022
Developing Novel Transcription Factor PROTACs to Disrupt the HMGA1 Epigenome in Relapsed Leukemia
Linda Resar, MD, Jian Jin, PhD
Icahn School of Medicine at Mount Sinai, The Johns Hopkins University School of Medicine
2022
Inhibition of translation initiation as a novel therapeutic strategy against MYCN-amplified neuroblastoma
Rani George, MD/PhD
Dana-Farber Cancer Institute
2021
Targeting CCAAT/enhancer binding proteins CEBPB and CEBPD in neuroblastoma
Darrell Yamashiro, MD/PhD
Columbia University
2021
In-vivo characterization of MYCN-driven immunosuppression
William Weiss, MD/PhD
University of California San Francisco
2021
Choline Kinase and Alkylating Agent Resistance in Refractory Childhood Leukemias
Alejandro Gutierrez, MD
Boston Children’s Hospital
2021
Loss of ER-mitochondria contacts drives therapy resistance in neuroblastoma
Michael Hogarty, MD
Children’s Hospital of Philadelphia
2021
Targeting tumor associated macrophages in metastatic Ewing sarcoma (ES)
Mitchell Cairo, MD
New York Medical College
2021
A leukemia organoid platform for dissection and targeting of tumor-niche interactions
Adolfo A. Ferrando, MD/PhD
Columbia Institute for Cancer Genetics
2021
Phosphopeptides as shared targets for donor derived T cell therapy of AML
Richard O'Reilly, MD
Memorial Sloan-Kettering Cancer Center
2021
Role of H3.3-G34R mutation in pediatric high-grade glioma
Jian Hu, PhD
University of Texas M.D. Anderson Cancer Center
2021
Unraveling metabolic dependencies in childhood supratentorial ependymomas.
Sriram Venneti, MD/PhD
Regents of the University of Michigan
2021
Hematopoietic stem cell function in familial platelet disorder therapy
Lucio Castilla, PhD
University of Massachusetts Medical School
2021
Targeting EZHIP as a therapeutic strategy for infantile ependymomas
Peter Lewis, PhD
University of Wisconsin - Madison
2021
Novel targeting approach for pediatric B-cell malignancies
Christine Eischen, PhD
Thomas Jefferson University
2019
Pediatric Osteosarcoma: Identifying the elusive molecular signature and its relationship to this disease
Linda Hendershot, PhD
St. Jude Children’s Research Hospital
2019
Novel Therapy of Heterogeneous B-cell Acute Lymphocytic Leukemia by Targeting Convergent Oncogenic Mediators STATs
Hasan Uludag, PhD
University of Alberta
2019
Targeting microenvironment-induced TGFB signaling to overcome drug resistance in acute lymphoblastic leukemia
Charles Mullighan, MBBS(Hons), MSc, MD, FRACP, FRCPA
St. Jude Children’s Research Hospital
2019
Norepinephrine Transporter-Targeted Pharmacotherapy of Aggressive Neuroblastoma
Michael Chorny, PhD
Children’s Hospital of Philadelphia
2019
Therapeutic Targeting of the Disrupted Metabolic State in DIPG
Costas Lyssiotis, PhD, Sriram Venneti, MD/PhD
University of Michigan
2019
The anti-tumor immune microenvironment in the Sonic Hedgehog subclass of medulloblastoma
Anna Marie Kenney, PhD
Emory University
2019
Targeting mechanisms of Siglec15-mediated immune evasion in hematologic malignancies
Christopher Porter, MD
Emory University
2019
Using Splice Switching Oligonucleotides (SSOs) to target MDM2 for rhabdomyosarcoma therapy
Dawn Chandler, PhD
Research Institute at Nationwide Children's Hospital
2019
Identify Novel Druggable Targets to Treat Philadelphia chromosome-like B-Acute Lymphoblastic Leukemia
Wei Tong, PhD
Children’s Hospital of Philadelphia
2019
The use of hematopoietic stem and progenitor cells to reverse treatment resistance to PD-1 blockade
Catherine Flores, PhD
University of Florida
2019
Developing drugs to treat NT5C2 mediated resistance in pediatric ALL
William Carroll, MD
New York University School of Medicine
2019
CD36-Sphingosine 1-Phosphate Axis in Osteosarcoma Progression
Meenal Mehrotra, MD/PhD
Medical University of South Carolina
2019
Identifying targeted treatments for DICER1-associated sarcomas
William Foulkes, MD/PhD, Sidong Huang, PhD
Research Institute of the McGill University Health Centre
2019
Targeting symmetric division in pediatric cancers
Rosalind Segal, MD/PhD
Dana-Farber Cancer Institute
2019
Epigenetic regulation of genomic instability and its therapeutic implications in T-cell acute lymphoblastic leukemia
Birgit Knoechel, MD/PhD
Dana-Farber Cancer Institute
2019
Selectively targeting EP300 with small molecules for neuroblastoma (NB) therapy
Jun Qi, PhD
Dana-Farber Cancer Institute
2019
Predicting and Overcoming Resistance to Immunotherapy in Pediatric High-Grade Glioma
Oren Becher, MD, Robert Wechsler-Reya, PhD
Ann & Robert H. Lurie Children’s Hospital of Chicago
2019
Role of the macro lncRNA KCNQ1OT1 in embryonal rhabdomyosarcoma tumorigenesis
Corinne Mary Linardic, MD/PhD
Duke University
2019
Saturating CRISPR gene body scans in MLL-rearranged leukemia
Chun-Wei Chen, PhD
Beckman Research Institute of City of Hope
2018
Olfactory Ensheathing Cells for Pediatric Glioma Gene/Cell Therapy
Bakhos Tannous, PhD
Massachusetts General Hospital
2018
Nuclear Receptor Tyrosine Kinases Mediating Chromatin Remodeling & Checkpoint Adaption
Charles Keller, MD
Children’s Cancer Therapy Development Institute
2018
Circulating Repetitive Element RNAs as an Osteosarcoma Marker
David Cobrinik, MD/PhD
Children’s Hospital Los Angeles
2018
Energy Balance Strategies for Decreasing Chemotherapy-Induced Cardiotoxicity
Eugenie Kleinerman, MD
University of Texas M.D. Anderson Cancer Center
2018
Targeting KDM6B in Pediatric Leukemia
Grant A. Challen, PhD
Washington University
2018
Molecular Landscape for Targeted Therapy in Retinoblastoma
James Harbour, MD
University of Miami
2018
Modeling and Therapeutic Targeting of p21-Activated Kinase Members in High-Risk Rhabdomyosarcoma
Jason Yustein, MD/PhD
Baylor College of Medicine
2018
Identifying New Therapeutic Targets in Fibrolamellar Hepatocellular Carcinoma
Julien Sage, PhD
Stanford University
2018
Targeting Oncogenic RAS in Pediatric Leukemia
Loren Walensky, MD/PhD
Dana-Farber Cancer Institute
2018
Is SIRT5 a Therapy Target in Acute Lymphoblastic Leukemia?
Michael Deininger, MD/PhD
University of Utah
2018
RNA Methylation in Metabolically Disrupted Pediatric Cancers
Patricia Dahia, MD/PhD
University of Texas Health Science Center at San Antonio
2018
Targeting Glutamine Addiction in Diffuse Intrinsic Pontine Gliomas (DIPGs)
Sriram Venneti, MD/PhD
University of Michigan
2018
RNA-ECS to Quantify Rare Clonal RNA Species at Diagnosis, Remission and Relapse from the COG AAML1031 Study
Todd Druley, MD/PhD
Washington University
2018
Alternative Splicing Driven by MYCN Activates RAS-MAPK in Neuroblastoma
William Weiss, MD/PhD
University of California San Francisco
2018
Targeting the ALT Pathway to Induce Synthetic Lethality and Treat Poor Outcome Pediatric Tumors
Wolf-Dietrich Heyer, PhD
University of California, Davis
2018
Physical Properties of Drug-Resistant Leukemia Cells
Yong-mi Kim, MD/PhD
Children’s Hospital Los Angeles
2017
Novel Approaches for Epigenetic Therapy for Relapsed Acute Lymphoblastic Leukemia
Linda Resar, MD, Andrei Thomas-Tikhonenko, PhD
The Johns Hopkins University School of Medicine
2017
Mechanisms of OTX2 Mediated Chromatin Regulation in Medulloblastoma
Miguel Rivera, MD
Massachusetts General Hospital
2017
Targeted Degradation of Proliferative Transcription Factors in Pediatric Cancers
Seth Rubin, PhD
University of California at Santa Cruz
2017
3-D Printed Self-fitting Shape Memory Bone Grafts for Smart Pediatric Skeletal Reconstruction
Jie Song, PhD
University of Massachusetts Medical School
2017
De-escalation of Radiotherapy for Medulloblastoma by a Novel DNA Damage Checkpoint Inhibitor
Marc Symons, PhD
Feinstein Institute for Medical Research - North Shore
2017
Alternative Splicing in the Pathogenesis of Acute Lymphoblastic Leukemia
Andrei Thomas-Tikhonenko, PhD
Children’s Hospital of Philadelphia
2017
Characterization of Novel OTX2-semaphorin Gene Signaling Pathways Regulating the ‘Grow and Go’ Arms of Highly Aggressive Medulloblastomas
Tamra Werbowetski-Ogilvie, PhD
University of Manitoba
2017
Long Non-Coding RNAs as Novel Biomarkers and Therapeutic Targets in Pediatric Leukemia
Iannis Aifantis, PhD
New York University School of Medicine
2017
Targeting BRD9 in Sarcoma
Scott Armstrong, MD/PhD
Dana-Farber Cancer Institute
2017
Development of Nanoparticle-Encapsulated Chemo/Radio-Sensitizers for Intrathecal Delivery
Ranjit S. Bindra, MD/PhD, Mark Saltzman, PhD
Yale School of Medicine
2017
Targeting Pediatric Brain Tumors Using PTPmu Nanochains with Radiofrequency-Releasable Therapeutics
Susann Brady-Kalnay, PhD, Efstathios Karathanasis, PhD
Case Western Reserve University
2017
Genome-Wide 5hmC Profiling of Neuroblastoma Tumors and Patient Cell-Free DNA
Susan Cohn, MD, Chuan He PhD
University of Chicago
2017
Predictive Modeling of MDS Disease Progression From Inherited Bone Marrow Failure Syndrome Using Novel Branching Process and Next Generation Sequencing to Improve Patient-Specific Survival
Seth Corey, MD, Marek Kimmel, PhD
Cleveland Clinic Foundation
2017
Defining the Cell Surface Landscape of Diagnostic and Relapsed Neuroblastoma: Immunotherapeutic Target Identification
Sharon Diskin, PhD
Children’s Hospital of Philadelphia
2017
Targeting Metabolic Vulnerabilities in ETP-ALL
Adolfo A. Ferrando, MD/PhD
Columbia University
2017
Development of Mithramycin Analogs for Ewing sarcoma
Patrick Grohar, MD/PhD
Van Andel Research Institute
2017
Role of Protocadherin-9 in Enabling Leukemia Cell Colonization of the CNS
Jordan Jacobelli, PhD
National Jewish Health
2017
Targeting Childhood Brain Cancer in a Dish to Catalyze New Therapies
Paul Knoepfler, PhD
University of California, Davis Medical Center
2017
Establishing the Function of Novel MicroRNAs Associated with Resistance and Relapse in Pediatric AML
Vivian Oehler, MD, Soheil Meshinchi, MD/PhD
Fred Hutchinson Cancer Research Center
2016
CD47-blocking Oncolytic Vaccinia Viruses as Biotherapeutics for Pediatric Solid Tumors
Stephen Gottschalk, MD, Nino Rainusso, MD
St. Jude Children’s Research Hospital
2016
Targeting the Function of Ezrin in Rhabdomyosarcoma - Neetu Gupta, PhD & Peter Anderson, MD (2016)
Neetu Gupta, PhD, Peter Anderson, MD
The Cleveland Clinic Foundation
2016
Targeted Combination Therapy to Sensitize High-Risk Acute Lymphoblastic Leukemia to Methotrexate via Restoration of Ikaros Tumor Suppression
Sinisa Dovat, MD/PhD, Chunhua Song, MD/PhD
The Pennsylvania State University
2016
HOX Genes As Developmental Regulators Of Sarcomagenesis
Elizabeth Lawlor, MD/PhD, Deneen Wellik, PhD
University of Michigan
2016
MYC as a Driver and Anti-Cancer Target in Malignant Rhabdoid Tumors
William Tansey, PhD
Vanderbilt University Medical Center
2016
Enhancing Immunotherapy of Neuroblastoma
Alice Yu, MD/PhD, Mitchell Diccianni, PhD
The Regents of the University of California, San Diego
2016
Development of an anti-KIR3DL1 Antibody to Promote Innate Immunity Against Neuroblastoma
Katherine Hsu, MD/PhD, Nai-Kong Cheung, MD/PhD
Memorial Sloan-Kettering Cancer Center
2016
Targeting Microenvironmental Support of Acute Lymphoblastic Leukemia
Craig Mullen, MD/PhD
University of Rochester
2016
DICER1-Driven Cancers: Models, Mechanisms and Therapies
James Amatruda, MD/PhD
University of Texas Southwestern Medical Center at Dallas
2016
Modeling Pediatric Spinal Gliomas Using Murine Spinal Neural Stem Cells
Charles Eberhart, MD/PhD, Eric H. Raabe, MD/PhD
The Johns Hopkins University School of Medicine
2016
GeneEditing in T cell Leukemia to Promote Immunomodulatory Forms of Cell Death
Dr. Ben Croker, PhD
Boston Children’s Hospital
2016
Targeted Inhibition of Lin28/let-7 Binding to Treat Pediatric Malignancy
George Q. Daley, MD/PhD
Boston Children’s Hospital
2016
Circulating Exosomes as Biomarkers of Medulloblastoma Progression, Metastasis, and Recurrence
David Lyden, MD/PhD, Parveen Raju, MD/PhD
Weill Cornell Medical College
2016
Redefining the Cellular Architecture of Diffuse Intrinsic Pontine Glioma through Large-Scale Single-cell RNA-seq Analyses
Mario Suva, MD/PhD
Massachusetts General Hospital
2016
Image Guided Diffuse Intrinsic Pontine Glioma Drug Delivery and Design
Mark Souweidane, MD, Richard Ting, PhD
Weill Cornell Medical College
2016
Enhancing Pediatric Sarcoma Immunotherapy by Drug Induced Expression of Tumor-Specific Cryptic Neoantigens
David Largaespada, PhD
University of Minnesota
2016
Preclinical Testing of Candidate Therapeutics in a Pediatric Spinal Cord High-grade Glioma Model
Michelle Monje, MD/PhD
Stanford University
2016
Developing and Distributing a Pediatric Spinal Cord High-grade Glioma Model
Michelle Monje, MD/PhD
Stanford University
2016
Improving Immune-Mediated Oncolytic Viral Therapy: Engineering Tumor Vaccine Elements into the Virus
Kevin Cassady, MD, Christopher Walker, PhD
Research Institute at Nationwide Children's Hospital
2016
Detection of Circulating Tumor DNA in Pediatric Sarcomas
Alejandro Sweet-Cordero, MD, Maximillian Diehn, MD/PhD
The Regents of the University of California San Francisco
2016
Targeting MYCN-amplified Neuroblastoma Through RORalpha Activation
Eveline Barbieri, MD/PhD
Baylor College of Medicine
2016
Selective Epigenetic Regulation of Notch1 in T-cell Acute Lymphoblastic Leukemia
Mark Yat-Fung Chiang, MD/PhD
University of Michigan
2016
Pre-clinical Testing of Novel Glutamine Metabolic Inhibitors in MYC-driven Medulloblastoma
Eric H. Raabe, MD/PhD, Dr. Barbara Slusher, PhD
The Johns Hopkins University School of Medicine
2015
Characterizing Resistance Mechanisms to BET-Bromodomain Inhibition in MYC-amplified
Rameen Beroukhim, MD/PhD
Broad Institute
2015
Small Molecule Inhibitors of ERG for Pediatric Leukemia and Sarcoma
John Bushweller, PhD
University of Virginia, School of Medicine
2015
Enhancing Effectiveness of Immune Checkpoint Therapy in Neuroblastoma
Shahab Asgharzadeh, MD
Children’s Hospital Los Angeles
2015
Synergistic Differentiation Therapy in Neuroblastoma
Gerard Blobe, MD/PhD
Duke University
2015
Transcriptional Effectors of Activated RAS Signaling in Juvenile Myelomonocytic Leukemia
Alan Cantor, MD/PhD
Boston Children’s Hospital
2015
Single Cell Analysis of Leukemia Initiation
Michael Cleary, MD
Stanford University
2015
Pharmacogenomics and Drug Screening Lead to a Novel Targeted Therapy with Potent and Specific Activity Against Mouse Models of MYCN-amplified Neuroblastoma
Anthony Faber, MD, Yael Mossé, MD
Virginia Commonwealth University
2015
Preclinical Development of Atr Inhibitor VE-822, Delivered Systemically in Nanoparticles, for Medulloblastoma Therapy
Timothy Gershon, MD/PhD
University of North Carolina
2015
Antibody Targeted Natural Killer Cell-Derived Extracellular Vesicles for the Treatment of Childhood Acute Lymphoblastic Leukemia and Neuroblastoma
Robert Seeger, MD, Alan Wayne, MD
Children’s Hospital Los Angeles
2015
Identifying how Pre-existing Anti-Therapeutic Antibodies (PATA) are Associated with Better Outcome in a Clinical Trial of ADCC-inducing Anti-GD2 mAb
Paul Sondel, MD/PhD
University of Wisconsin - Madison
2015
Functional Analysis and Therapeutic Targeting of STAG2 Mutations in Ewing sarcoma
Todd Waldman, MD/PhD
Georgetown University
2015
Optimizing Bispecific Antibody Therapy for Acute Leukemias
Roland Walter, MD/PhD, James Olson, MD/PhD
Fred Hutchinson Cancer Research Center
2015
Identifying Epigenetic Mechanisms Impacting Neuroblastoma Genome Stability and Drug Response
Johnathan Whetstine, PhD
Massachusetts General Hospital
2015
Integrative Genomic and Immunogenomic Analysis of Neuroblastoma
David Haussler, PhD
University of California at Santa Cruz
2015
Developing a Novel Tumor Model to Screen for New Therapies for Spinal Ependymoma
Linda Resar, MD, Richard Schlegel, MD/PhD
The Johns Hopkins University School of Medicine
2015
High-dimensional Biochemical CyTOF Analysis of Pediatric T cell Leukemias
Jeroen Roose, PhD
University of California San Francisco
2015
Enhanced Drug Repurposing Screen in DSRCT Enabled by New In Vitro and In Vivo Models Based on CRISPR/Cas9 Genome Engineering
Marc Ladanyi, MD, Maria Jasin, PhD
Memorial Sloan-Kettering Cancer Center
2015
Identifying Mechanisms of Glucocorticoid Resistance in Pediatric Acute Lymphoblastic
Michelle Kelliher, PhD
University of Massachusetts Medical School
2015
Regulation of Tumor Debris Mediated Inflammation as a Therapeutic Modality in Medulloblastoma
Mark Kieran, MD/PhD, Dipak Panigrahy, MD
Dana-Farber Cancer Institute
2015
Histone H3.3 Targeted Therapy for Pediatric High-grade Gliomas
Paul Knoepfler, PhD
University of California, Davis Medical Center
2015
In Vivo Analysis of Early Neuroblastoma Development using Transgenic Quail
Paul Kulesa, PhD
Stowers Institute for Medical Research
2015
Cure Osteosarcoma Lung Metastasis by Disrupting Tumor VCAM-1 Signaling with Macrophages
Alex Huang, MD/PhD
Case Western Reserve University
2014
Targeting Tumor Suppressor MicroRNAs in Pediatric Acute Lymphoblastic Leukemia
Linda Resar, MD
The Johns Hopkins University School of Medicine
2014
Therapies for SHH-subtype Medulloblastoma
Rosalind Segal, MD/PhD
Dana-Farber Cancer Institute
2014
The Tumor Suppressive Role of LNK (SH2B3) in Acute Lymphoblastic Leukemia
Wei Tong, PhD
Children’s Hospital of Philadelphia
2014
Targeting the Accomplices of MYC in MYC-driven Medulloblastoma
Robert Wechsler-Reya, PhD
Sanford-Burnham Medical Research Institute
2014
Modeling Juvenile Myelomonocytic Leukemia with Patient-Derived Induced Pluripotent Stem Cells
Mitchell Weiss, MD/PhD
Children’s Hospital of Philadelphia
2014
Targeting eEF2 Kinase in Neuroblastoma
William Weiss, MD/PhD
University of California San Francisco
2014
Development of Targeted Leukemia Inhibitors Using Nanoparticle Delivery
Lucio Castilla, PhD
University of Massachusetts Medical School
2014
Exploiting Epigenetic Dependencies of Notch1 for Clinical Benefit
Patricia Ernst, PhD
University of Colorado Denver
2014
Mechanisms and Targeted Inhibition of NT5C2 Mutations in Relapsed ALL
Adolfo A. Ferrando, MD/PhD
Columbia University
2014
Targeting the Six1/Eya Transcriptional Complex to Inhibit Pediatric Sarcomas
Heide Ford, PhD
University of Colorado Denver
2014
Converting the Glioblastoma Multiforme Tumor-Associated Macrophage Phenotype
Alan D. Friedman, MD
The Johns Hopkins University School of Medicine
2014
Targeting MYC/MYCN-driven Transcriptional Amplification in Pediatric Cancer
Rani George, MD/PhD
Dana-Farber Cancer Institute
2014
Development of AAV Vectors for Efficient Targeting and Elimination of Osteosarcoma Tumor-Initiating Cells
Steven Ghivizzani, PhD
University of Florida
2014
Two-pronged Cell Therapy: Engineering T Cells to Redirect Bystander T- and NK-Cells to Pediatric Cancer
Stephen Gottschalk, MD
Baylor College of Medicine
2014
Developing Personalized (precision) Medicine Strategies to Optimize Pediatric Brain Tumor Management
David Gutmann, MD/PhD
Washington University
2014
Modeling the Multi-hit Pathogenesis of t(12;21) Associated Pediatric ALL by CRISPR/Cas-mediated Genome Engineering
Hanno Hock, MD/PhD
Massachusetts General Hospital
2014
Mechanisms of LGR5 Regulation and Function in Ewing sarcoma Metastasis
Elizabeth Lawlor, MD/PhD
University of Michigan
2014
Identifying Drug Resistant Mutations in Ph-like Acute Lymphoblastic Leukemia
Mignon Loh, MD
University of California San Francisco
2014
Role of LMO1 in Neuroblastoma Initiation and Maintenance: Analysis in the Zebrafish model of Childhood Neuroblastoma
Thomas Look, MD
Dana-Farber Cancer Institute
2014
Correction of Hematopoietic Defects in Down Syndrome by Chromosome 21 Silencing
Peter Newburger, MD
University of Massachusetts Medical School
2014
Trib2 in the Pathogenesis of T-ALL
Warren Pear, MD, PhD
University of Pennsylvania
2013
DICER1, MicroRNAs and Pediatric Cancer: An Emerging Story
William Foulkes, MD/PhD
Research Institute of the McGill University Health Centre
2013
Therapeutic Targeting of LARG-RhoA Signaling Axis in Childhood Leukemia
Yi Zheng, PhD
Cincinnati Children's Hospital Medical Center
2013
Novel Therapeutic Targets in Relapse T-cell Acute Lymphoblastic Leukemia
David Langenau, PhD
Massachusetts General Hospital
2013
Mechanisms Regulating Medulloblastoma Progression
Nadia Dahmane, PhD
University of Pennsylvania
2013
Pre-Clinical Testing of T cells Coded with CARs for DIPG
Laurence Cooper, MD/PhD
University of Texas
2013
Next-Generation Therapies for Hedgehog Pathway-Dependent Tumors
James Chen, PhD
Lucile Packard Children’s Hospital at Stanford University School of Medicine
2013
Epigenetic Characterization of the Neuroblastoma Driver Genes ARID1A and ARID1B
Michael Hogarty, MD
Children’s Hospital of Philadelphia
2013
Therapeutically Targeting a Tumor Suppressor Complex in Aggressive Pediatric Cancers
Charles Roberts, MD/PhD
Dana-Farber Cancer Institute
2013
Reactivating Apoptosis in Refractory Pediatric Cancer by Molecular Inhibition of MCL-1
Loren Walensky, MD/PhD
Dana-Farber Cancer Institute
2013
Identification of TFAP4 (Transcription Factor AP-4/Activating Enhancer Binding Protein) as a Potential Master Regulator/Synthetic Lethal Gene of MYCN Amplified Neuroblastoma
Darrell Yamashiro, MD/PhD
Columbia University Medical Center
2013
Therapeutic Potential of the MST1/Hippo Tumor Suppressor Pathway in Alveolar Rhabdomyosarcoma
Corinne Mary Linardic, MD/PhD
Duke University
2013
Defining the Role of a Novel, Developmentally Restricted Hematopoietic Stem Cell in Pediatric Leukemias
Camilla Forsberg, PhD
University of California at Santa Cruz
2013
Novel Targeted Therapies for Rhabdomyosarcoma
Michael Dyer, PhD
St. Jude Children’s Research Hospital
2013
An Intersection of Cell Metabolism and Differentiation in Childhood Sarcomas
Sandra Orsulic, PhD
Cedars-Sinai Medical Center
2013
Multispecific T Cells for Adoptive Immunotherapy of Pediatric High Grade Glioma (HGG)
Nabil Ahmed, MD, MSc
Baylor College of Medicine
2013
Engager T cells for the Adoptive Immunotherapy of Pediatric Malignancies
Stephen Gottschalk, MD
Baylor College of Medicine
2013
Development of MicroRNA-Nanoparticles to Treat Childhood Acute Leukemia Carrying MLL Rearrangements
Jianjun Chen, PhD
University of Cincinnati College of Medicine
2013
Genetic Alterations in Intramedullary Spinal Cord Tumors
George Jallo, MD
The Johns Hopkins University School of Medicine
2013
Nanoparticle Delivery of Therapeutic Agents in Neuroblastomas
Garrett M. Brodeur, MD
Children’s Hospital of Philadelphia
2013
An Oncolytic Herpes Simplex Virus Restricted in Entry to Neuroblastoma Cells
Richard Longnecker, PhD
Northwestern University Medical School
2012
Development of the Next Generation FLT3 Inhibitors for Pediatric AML and Infant ALL - Donald Small, MD, PhD (2012)
Donald Small, MD/PhD
The Johns Hopkins University School of Medicine
2012
The role of vitamin D in hepatoblastoma formation and prevention
Wolfram Goessling, MD/PhD
Brigham and Women's Hospital
2012
Searching for genetic vulnerabilities in osteosarcoma through genome-wide RNAi screening
Stuart Orkin, MD
Dana-Farber Cancer Institute
2012
Developing Immune-modulatory Strategies Against Primary and Metastatic Tumors in the Central Nervous System
Alex Huang, MD/PhD
Case Western Reserve University
2012
Novel STAT3 drug development for childhood osteosarcoma therapy using drug repositioning
Jiayuh Lin, PhD
Research Institute at Nationwide Children's Hospital
2012
Mechanisms of LYL1-associated T-ALL development
Margaret Goodell, PhD
Baylor College of Medicine
2012
V alpha-invariant NKT cells as a novel platform for cancer immunotherapy
Leonid Metelitsa, MD/PhD
Baylor College of Medicine
2012
Casein Kinase 1 alpha Agonists as Novel Medulloblastoma Inhibitors
David Robbins, PhD
University of Miami Miller School of Medicine
2012
A novel mouse model of rhabdomyosarcoma
Chin Chiang, PhD
Vanderbilt University Medical Center
2012
Identification of predisposition genes for Ewing sarcoma segregating in high-risk pedigrees
Lisa Cannon-Albright, PhD
Huntsman Cancer Institute
2012
Identification and validation of new therapeutic targets in pediatric leukemia through an aneuploidy-based synthetic lethality screen
David Pellman, MD
Dana-Farber Cancer Institute
2012
Modeling Pediatric Leukemia in the Zebrafish to Enable Discovery of New Anti-Leukemic Compounds
David Traver, MD
University of California, San Diego
2012
Targeting Neovascularization in Graft-Versus-Host Disease - Marcel van den Brink, MD/PhD (2012)
Marcel van den Brink, MD/PhD
Memorial Sloan-Kettering Cancer Center
2012
In-UTR mutations in neuroblastoma: Functional consequences and therapeutic implications - Andrei Thomas-Tikhonenko, PhD (2012)
Andrei Thomas-Tikhonenko, PhD
Children’s Hospital of Philadelphia
2011
In vivo Analysis of Neuroblastoma Pathogenesis using Transgenic Zebrafish
Alfred Tom Look, MD
Dana-Farber Cancer Institute
2011
Investigating the Role of Lin-28 in Germ Cell Tumorigenesis
George Daley, MD/PhD
Boston Children’s Hospital
2011
Racial and Ethnic Disparities in Survival of Children with Neuroblastoma
Susan Cohn, MD
University of Chicago
2011
Blockade of MYCN in neuroblastoma
William Weiss, MD/PhD
University of California San Francisco
2011
The role of primary cilia in rhabdomyosarcoma
Brian Dynlacht, PhD
New York University School of Medicine
2011
Targeting inhibitory phosphatase signaling in high-risk childhood ALL
Markus Muschen, MD/PhD
University of California San Francisco
2011
Studying the clonal dynamics and evolution of leukemia using molecular barcodes
Matthew Porteus, MD/PhD
Lucile Packard Children’s Hospital at Stanford University School of Medicine
2011
Predicting Outcome and Ameliorating Toxicities of the Immunotherapy of Neuroblastoma
Alice Yu, MD/PhD
University of California, San Diego
2011
Strategies for Epigenetically Reprogramming Neuroblastoma Cancer Stem Cells
Jason Shohet, MD/PhD
Baylor College of Medicine
2011
Clinical Development of RNA Nanoparticle Vaccines Targeting Recurrent Pediatric Brain Cancers
Duane Mitchell, MD/PhD
Duke University
2011
Modeling Anaplastic Medulloblastoma using Cerebellar Stem Cells
Robert Wechsler-Reya, PhD
Sanford-Burnham Medical Research Institute
2011
BH3 Profiling to define therapy resistance classes in neuroblastoma
Michael Hogarty, MD
Children’s Hospital of Philadelphia
2010
Modeling Pediatric Leukemia in Zebra Fish to Enable Discovery of New Anti- Leukemic Compounds
David Traver, MD
University of California, San Diego
2010
Targeting Neovascularization in Graft-Versus-Host Disease - Marcel van den Brink, MD/PhD (2010)
Marcel van den Brink, MD/PhD
Memorial Sloan-Kettering Cancer Center
2010
Elucidating the Initiation Mechanism of the Fanconi Anemia Pathway of DNA Damage Repair
Yong Xiong
Yale School of Medicine
2010
Treating Brain Tumor by Changing Cell Division Pattern
Weimin Zhong
Yale School of Medicine
2010
A novel compound for treatment of T cell leukemia
Nikolaus Trede
Huntsman Cancer Institute
2010
Understanding the Role of a Novel Hereditary Cancer Gene
Patricia Dahia
University of Texas Health Science Center at San Antonio
2010
Pathogenesis of T-cell Acute Lymphoblastic Leukemia from Myeloproliferative Disorder
Motonari Kondo
Duke University
2010
Novel Therapeutic Target in Leukemia Stem Cells
James Mulloy
Cincinnati Children's Hospital Medical Center
2010
Discovery of Inhibitors of MLL Fusion Proteins as Targeted Therapies for Pediatric Acute Leukemia
Andrew Napper
Nemours Center for Childhood Cancer Research
2010
Identification of New Therapeutic Targets in Pediatric Acute Lymphoblastic Leukemia Through Mosaic Trisomy and Aneuploidy-Based Synthetic Lethality Screens
David Pellman, MD
Dana-Farber Cancer Institute
2010
Predicting Outcome for Patients with Metastatic Neuroblastoma by Analysis of Bone Marrow and Blood for a Neuroblastoma Gene Expression Signature
Robert Seeger, MD
Children’s Hospital Los Angeles
2010
Identification of the Molecular Targets of MLL Translocation-Based Childhood Leukemia
Ali Shilatifard
Stowers Institute for Medical Research
2010
In-UTR Mutations in Neuroblastoma: Functional Consequences and Therapeutic Implications - Andrei Thomas-Tikhonenko, PhD (2010)
Andrei Thomas-Tikhonenko, PhD
Children’s Hospital of Philadelphia
2009
Investigating the role of lin-28 in germ cell tumorigenesis
George Daley
Boston Children’s Hospital
2009
Epigenetic manipulation of leukemia.
H. Leighton Grimes
Cincinnati Children's Hospital Medical Center
2009
Metabolic control of p53 activation in T-ALL.
Jeffrey Rathmell
Duke University
2009
Modeling anaplastic medulloblastoma using cerebellar stem cells
Robert Wechsler-Reya, PhD
Duke University
2009
Targeted regulation of acetylation as novel therapy for Ewing sarcoma.
Jeffrey Toretsky
Georgetown University
2009
Molecular diagnostic, prognostic, and therapeutic approaches toward Ewing sarcoma.
Stephen Lessnick
Huntsman Cancer Institute
2009
Linking mitogenic sonic hedgehog signaling to the oncogene Yap 1 in neural stem/progenitor cells and medulloblastoma.
Anna Marie Kenney, PhD
Memorial Sloan-Kettering Cancer Center
2009
Pharmacogenomics of childhood AML susceptibility and treatment response.
Richard Aplenc, MD/PhD, MSCE
Children’s Hospital of Philadelphia
2009
BH3 profiling to define therapy resistance classes in neuroblastoma.
Michael Hogarty, MD
Children’s Hospital of Philadelphia
2009
Adaptation of acute lymphoblastic leukemia (ALL) to the post-transplant allogeneic environment.
Craig Mullen
University of Rochester
2009
Targeting wnt in synovial sarcoma models.
Josiane Eid
Vanderbilt University Medical Center
2008
The role of immunogenetics in natural killer cell targeting of neuroblastoma
Katharine Hsu
Memorial Sloan-Kettering Cancer Center
2008
Blockade of  Mycn in neuroblastoma
William Weiss, MD/PhD
University of California San Francisco
2008
Therapeutics In Ovo
Charles Keller, MD
Oregon Health & Science University
2008
Generation of CHD5 Antibodies and Know-out Mouse Model
Garrett M. Brodeur, MD
Children’s Hospital of Philadelphia
2008
Inhibition of Cathepsins to Treat Neuroblastoma
Robert Mason
Alfred I duPont Hospital for Children
2008
The role of OCT4 in self-renewal signaling and drug-resistance of childhood acute lymphoblastic leukemia
Markus Muschen
Children’s Hospital Los Angeles
2008
HMG-I/Y in Pediatric Leukemia and Lymphoma
Linda Resar, MD
The Johns Hopkins University School of Medicine
2008
Preclinical Studies of Gli1 Inhibition in Ewing Family Tumors
William May
Children’s Hospital Los Angeles
2008
PI-3 kinase as a novel and attractive target in pediatric cancer and neuroblastoma
Donald Durden
Emory University
2008
Molecular Regulation of CNS Involvement in Pediatric ALL
Iannis Aifantis, PhD
New York University School of Medicine
2008
Parallel sequencing to identify the causes of childhood cancer
Sharon Plon
Baylor College of Medicine
2007
Adoptive immunotherapy using clinical-grade T cells.
Laurence Cooper, MD/PhD
University of Texas M.D. Anderson Cancer Center
2007
Rac in p190-BCR-ABL0Induced Leukemia.
Jose Cancelas, MD/PhD
Cincinnati Children's Hospital Medical Center
2007
The role of overexpressed microRNA’s in the pediatric cancer rhabdomyosarcoma
Frederic Barr, MD/PhD
University of Pennsylvania
2007
Establishing surrogate markers of angiogenesis in pediatric oncology phase I and II clinical trials: A Children’s Oncology Group study
Sylvain Baruchel, MD
Hospital for Sick Children
2007
TrkB Signaling in the Genesis of Murine Neuroblastoma
Rae Nishi, PhD
University of Vermont
2006
Tumor cell hybridoma vaccine for recurrent neuroblastoma and Ewing sarcoma
Ken Lucas, MD
The Pennsylvania State University
2006
Anti-Angiogenic (Metronomic) Chemotherapy in Children with Poor Prognosis.
Mark Kieran, MD/PhD
Dana-Farber Cancer Institute
2006
Targeting Pediatric Brainstem Glioma with Oncolytic Polioviruses.
Matthias Gromeier, MD
Duke University Medical Center
2006
HMG-I/Y in pediatric Leukemia and Lymphoma.
Linda Resar, MD
The Johns Hopkins University School of Medicine
2006
Parallel sequencing to identify the causes of childhood cancer.
Sharon Plon, MD/PhD
Baylor College of Medicine
2006
Molecular characterization of OTX2 as a novel therapeutic target.
Hai Yan, MD
Duke University Medical Center
2006
Tyrosine Kinase Mutation Discovery in Neuroblastoma.
Matthew Meyerson, MD/PhD
Dana-Farber Cancer Institute
Innovation Grant - LOI
Innovation Grant - with approved LOI