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Center for Childhood Cancer

Coexistence of Different Telomere Maintenance Mechanisms in Pediatric High-Grade Glioma Tumors
 Coexistence of Different Telomere Maintenance Mechanisms in Pediatric High-Grade Glioma Tumors 1024 504 Lauren Dembeck
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Due to within-tumor heterogeneity, telomere-based therapeutic interventions will likely need to target both known telomere maintenance mechanisms to prevent resistance and recurrence.   Normal differentiated somatic cells can divide only a limited number of times due to loss of the physical ends of their chromosomes, the telomeres, with each replication. When the telomeres reach their…

Mapping the 3D Structure of Rhabdomyosarcoma Chromatin
 Mapping the 3D Structure of Rhabdomyosarcoma Chromatin 1024 575 Abbie Miller
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yellow squiggly lines representing chromatin strands in the nucleus

Researchers published the first comprehensive, 3D analysis of the complete rhabdomyosarcoma genome. In a paper published in Nucleic Acids Research Cancer (NAR Cancer), researchers from the Center for Childhood Cancer Research at Nationwide Children’s and their collaborators report a comprehensive 3D chromatin structural analysis and characterization of rhabdomyosarcoma (RMS). RMS is a pediatric soft-tissue tumor…

Featured Researcher — Meisam Naeimi Kararoudi, DVM, PhD
 Featured Researcher — Meisam Naeimi Kararoudi, DVM, PhD 150 150 Katie Brind'Amour, PhD, MS, CHES
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Meisam Naeimi Kararoudi, DVM, PhD, director of the CRISPR/Gene Editing Core and principal investigator in the Center for Childhood Cancer Research at Nationwide Children’s, has taken a winding, international road to his current role at Nationwide Children’s Hospital. From his native Iran to Italy, England, Sweden and the United States, Dr. Naeimi Kararoudi has collected…

Going Viral: The AAV Approach to Curing Cancer
 Going Viral: The AAV Approach to Curing Cancer 1024 256 Emily Siebenmorgen
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According to Timothy Cripe, MD, PhD, chief of the Division of Hematology and Oncology at Nationwide Children’s Hospital, it’s an incredible time to be working on cancer treatment – and now, targeted cancer prevention. “There’s so much going on in the cancer world these days,” says Dr. Cripe, who is also a principal investigator in…

Cancer-Causing Gene and Treatment Target for Ultra-Rare Rhabdomyosarcoma Confirmed Via Multiple Models
 Cancer-Causing Gene and Treatment Target for Ultra-Rare Rhabdomyosarcoma Confirmed Via Multiple Models 1024 764 Katie Brind'Amour, PhD, MS, CHES
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An international team has validated a cancer-causing gene fusion — and therapeutic targets — for an unusual presentation of muscle cancer in infants. In 2016, researchers first identified a novel gene mutation and fusion in rare cases of infants with rhabdomyosarcoma, a type of skeletal muscle-like cancer, normally only seen in school-age children and teenagers.…

Ewing Sarcoma Transcription Factor Mediates Changes in Transcription by Reprogramming Chromatin Structure
 Ewing Sarcoma Transcription Factor Mediates Changes in Transcription by Reprogramming Chromatin Structure 1024 575 Abbie Miller
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yellow squiggly lines representing chromatin strands in the nucleus

By binding to DNA in the nucleus, the Ewing sarcoma-specific transcription factor EWS/FLI changes chromatin organization, leading to altered gene expression and the development of Ewing sarcoma.   A new report, published in Nucleic Acids Research, focuses on how the Ewing sarcoma-specific transcription factor EWS/FLI changes spatial organization of DNA in the nucleus of Ewing…

Optimizing the Body’s Natural Cancer Killers
 Optimizing the Body’s Natural Cancer Killers 1024 649 Katie Brind'Amour, PhD, MS, CHES
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Recent advances in the expansion and production of natural killer cells offers pediatric patients new hope for remission after high-risk cancer diagnoses. Natural killer (NK) cells are the innate immune system’s first line of defense for viral infections. Although these white blood cells don’t have the antigen-specific “memory” that characterizes T cells or the antibody-producing…

Engineered Viruses Help the Immune System Target Cancer Cells
 Engineered Viruses Help the Immune System Target Cancer Cells 1024 623 Lauren Dembeck
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The seemingly unlikely “partnership” leads to strong antitumor responses in pediatric tumor models. A recent study from researchers at Nationwide Children’s Hospital, published in the Journal for Immuno Therapy of Cancer, describes how a type of viroimmunotherapy activates the immune system to preferentially target tumor cells. “Pediatric tumor typically lack neoantigens – new proteins that…

Could a Sequence-Specific Splice Switching Oligonucleotide and Antibody Therapy Combination Successfully Treat Rhabdomyosarcoma?
 Could a Sequence-Specific Splice Switching Oligonucleotide and Antibody Therapy Combination Successfully Treat Rhabdomyosarcoma? 1024 683 Jessica Nye, PhD
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Blocking alternative splicing may be a viable therapeutic intervention strategy for reducing tumor growth in rhabdomyosarcoma (RMS).   RMS is the most common pediatric soft tissue sarcoma and is associated with poor prognosis, a high rate of metastasis and recurrence. RMS tumors overexpress insulin growth factor (IGF)-2, making the IGF pathway a potential interventional target.…

Study Supports Virotherapy as a Potential Treatment for Brain Tumors
 Study Supports Virotherapy as a Potential Treatment for Brain Tumors 1024 575 Nationwide Children's
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A new study provides additional evidence of the efficacy of virotherapy for glioblastoma, the deadliest type of brain tumor. The research findings, published in Clinical Cancer Research, indicate that an oncolytic herpes simplex virus, G207, appears to boost immune response and that this is associated with better overall survival for patients with glioblastoma. The study,…

An Epigenetic Vulnerability for Rhabdomyosarcoma Among Children?
 An Epigenetic Vulnerability for Rhabdomyosarcoma Among Children? 150 150 Jessica Nye, PhD
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A deep classification of epigenetic machinery in rhabdomyosarcoma (RMS) tumors finds that the mammalian SWItch/Sucrose Non-Fermentable (mSWI/SNF) complexes are essential for the stabilization of RMS.   A recent study, published in Nature Communications, by Ben Stanton, PhD, and colleagues focused on rhabdomyosarcoma (RMS), a lethal pediatric soft tissue malignancy. Fusion positive RMS (FP-RMS) has properties…

Distinct Transcriptional Regulatory Domain Identified in Ewing Sarcoma Fusion Protein
 Distinct Transcriptional Regulatory Domain Identified in Ewing Sarcoma Fusion Protein 1024 1024 Lauren Dembeck
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Illustration of split DNA - Single strand ribonucleic acid

A better understanding of a newly defined region in the fusion protein that causes Ewing sarcoma may lead to novel approaches for therapeutic targeting. Ewing sarcoma is an aggressive pediatric bone cancer defined by the presence of a single genetic abnormality: a chromosomal translocation. The translocation splits two genes and joins them abnormally, creating a…

A Novel FUS/ETV4 Fusion and Other Rare FET/ETS Fusion Proteins Are Bona Fide Ewing Sarcoma
 A Novel FUS/ETV4 Fusion and Other Rare FET/ETS Fusion Proteins Are Bona Fide Ewing Sarcoma 1024 575 Jessica Nye, PhD
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Researchers conduct a molecular comparison between the established EWS/FLI translocation with rarer FET/ETS fusions, broadly supporting the inclusion of FET/ETS chimeric proteins in Ewing Sarcoma diagnosis and treatment.   Ewing sarcoma, most commonly caused by a chromosomal translocation of the amino-terminal domain of EWSR1 with the transcription factor FLI (EWS/FLI), presents as an aggressive pediatric…

Unraveling the Role of Pioneer Factors in Childhood Cancer
 Unraveling the Role of Pioneer Factors in Childhood Cancer 150 150 Abbie Miller
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New study implicates PAX3-FOXO1, an oncogenic driver of rhabdomyosarcoma, as a chimeric pioneer factor. Transcription factors are proteins that read the DNA sequences and can direct the transcription of that sequence into mRNA for translation into a protein. Transcription can become deregulated in cancer. Normally, transcription factors read sequences of DNA that are described as…

Featured Researcher — Genevieve Kendall, PhD
 Featured Researcher — Genevieve Kendall, PhD 150 150 Natalie Wilson
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Genevieve Kendall, PhD, is a principal investigator in the Center for Childhood Cancer and Blood Diseases and an assistant professor of Pediatrics at The Ohio State University College of Medicine. Dr. Kendall and her lab focus on developing basic and translational models of pediatric sarcomas—devastating and aggressive solid tumors with limited treatment options. Specifically, they…

Leaving No Stone Unturned: Nationwide Children’s Joins Children’s Oncology Group Pediatric Early Phase Clinical Trial Network
 Leaving No Stone Unturned: Nationwide Children’s Joins Children’s Oncology Group Pediatric Early Phase Clinical Trial Network 1024 683 Natalie Wilson
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The Children’s Oncology Group (COG), the world’s largest and most recognized pediatric cancer research collaborative, recently selected Nationwide Children’s Hospital to join its Pediatric Early Phase Clinical Trial Network (PEP-CTN) through a peer review process. The COG PEP-CTN is a re-iteration of the COG Phase 1/Pilot Consortium and is comprised of 21 core member sites…

Targeting DIPG: The Most Puzzling of Pediatric Brain Tumors
 Targeting DIPG: The Most Puzzling of Pediatric Brain Tumors 772 447 Katie Brind'Amour, PhD, MS, CHES
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Survival has dramatically improved for numerous pediatric cancers over the last several decades, with a notable and very deadly exception: diffuse intrinsic pontine glioma (DIPG). Now, a community of researchers and clinician-scientists have set the stage for a renewed — and better-armed — assault against this beast of a brain tumor. The past 50 years…

Assessing the Impact of COVID-19 on Pediatric Cancer Research
 Assessing the Impact of COVID-19 on Pediatric Cancer Research 1024 575 Lauren Dembeck
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Times of uncertainty offer an opportunity to reflect and reevaluate priorities. “This is a time that warrants resiliency and different approaches for dealing with difficulties, and pediatric cancer research is a field that reflects the resilient spirit of the kids that we are striving to find cures for,” says Jeffery Auletta, MD, director of the Blood…

Epigenetics, Chromatin Architecture and a Judo Mechanism to Attack Cancer
 Epigenetics, Chromatin Architecture and a Judo Mechanism to Attack Cancer 1024 575 Abbie Miller
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yellow squiggly lines representing chromatin strands in the nucleus

Researchers broaden the understanding chromatin architecture in human disease. Epigenetics is the study of how genetic information is context-dependent: it is organized so it can be repressed, but also read, repaired and replicated. For example, transcription factors can “communicate” with each other through the chromatin-DNA interface, and work in combinations to regulate which genes are…

Understanding Drug Interactions in Diffuse Midline Gliomas
 Understanding Drug Interactions in Diffuse Midline Gliomas 1024 683 Lauren Dembeck
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In a recent publication in Science Translational Medicine, researchers studied combinations of approved and investigational drugs to identify promising pairs, such as panobinostat and marizomib, for the treatment of diffuse intrinsic pontine glioma (DIPG) and other diffuse midline gliomas (DMG). In recent years, studies have shown that diffuse intrinsic pontine glioma (DIPG) subtypes are driven…

Targeting Alternative RNA Splicing in Pediatric Cancers
 Targeting Alternative RNA Splicing in Pediatric Cancers 150 150 Mary Bates, PhD
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Targeting alternative RNA splicing in tumor cells could lead to cancer therapies. RNA splicing, the enzymatic process of removing segments of premature RNA to produce mature RNA, is a key regulator of gene expression. Recent studies have identified variations in the transcriptomes of tumors due to alternative splicing changes, in addition to mutations in splicing…

The Search to Identify Tumor Cells Evading Chemotherapy
 The Search to Identify Tumor Cells Evading Chemotherapy 150 150 Sanjana Rajan
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Graduate research associate Sanjana Rajan shares why her work to label and track cells before and after chemotherapy is the next step to preventing tumor relapse. For a long time, the cells within a tumor were thought to be similar to one another, like a bowl of chocolate chips. However, scientific studies have identified that…

Understanding the Role of LSD2 in the Molecular Genetics of Ewing Sarcoma
 Understanding the Role of LSD2 in the Molecular Genetics of Ewing Sarcoma 150 150 Mary Bates, PhD
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Study reveals novel potential drug target for Ewing sarcoma. Ewing sarcoma is a bone-associated tumor of mostly children and young adults. Despite aggressive multi-modal treatment strategies, five-year survival remains at 75% for patients with localized disease and 20% for patients with metastases. “Our laboratory is organized to try to understand how Ewing sarcoma develops at a…

A Novel Method of Data Analysis Enables Identification of Genetic Drivers of Pediatric Cancer
 A Novel Method of Data Analysis Enables Identification of Genetic Drivers of Pediatric Cancer 1024 575 Bailey Dye
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conceptual art of DNA

An innovative approach to data analysis can more efficiently identify gene fusion events common to pediatric cancers, and inform clinical diagnoses and treatment decisions. The standard of care for cancer patients is changing. With the advent of personalized medicine, genetic testing is slowly becoming a routine clinical practice, and with it comes a better understanding…

An Infant. A Virus. An Emergency IND. A Life Saved.
 An Infant. A Virus. An Emergency IND. A Life Saved. 150 150 Abbie Miller
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Clinician scientists collaborate to use virus-specific T-cells from the mother to successfully treat a systemic adenovirus infection in a preterm infant. It’s not every day that researchers can say that they’ve written and submitted and emergency investigational new drug (EIND) application to the Food & Drug Administration (FDA) and saved a life. But that’s what…

Profile of a Cancer: Getting to Know Ewing Sarcoma
 Profile of a Cancer: Getting to Know Ewing Sarcoma 150 150 Katie Brind'Amour, PhD, MS, CHES
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Ewing sarcoma – a tumor type affecting the bone or soft tissue that primarily affects children and adolescents – has a 5-year survival rate of 70 percent among those with localized disease at diagnosis. Among children whose disease is metastatic, only 30 percent survive 5 years or longer. As a comparison, of all children diagnosed…

Conquering the Biological Politics of Cancer: Corruption, Coercion and Collusion
 Conquering the Biological Politics of Cancer: Corruption, Coercion and Collusion 150 150 Timothy Cripe, MD, PhD
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Understanding the “Three C’s” may provide the insights need to move the needle on the cancers with the bleakest prognoses. Broadly speaking, cancers fall into three categories: leukemias, brain tumors and other solid tumors. Since the dawn of chemotherapy in the 1940s, we’ve converted the most common type of leukemia, acute lymphoblastic leukemia, from incurable…

Two Classes of GGAA-Microsatellites in a Ewing Sarcoma Context
 Two Classes of GGAA-Microsatellites in a Ewing Sarcoma Context 150 150 Abbie Miller
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Characterization of GGAA-microsatellites provides insight in the role of noncoding DNA in cancer susceptibility and therapeutic development. In a study published in PLOS ONE, researchers describe two types of GGAA-microsatellites and their roles in EWS/FLI binding and gene regulation in Ewing sarcoma. Ewing sarcoma is the second most common pediatric bone malignancy. It is initiated by chromosomal…

A New Paradigm for Treating Transcription Factor-Driven Cancers
 A New Paradigm for Treating Transcription Factor-Driven Cancers 150 150 Abbie Miller
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Researchers describe how the association of the EWS/FLI transcription factor with GGAA-microsatellites drives Ewing sarcoma. In the current issue of Proceedings of the National Academy of Sciences, researchers from Nationwide Children’s Hospital describe a new paradigm for treating transcription factor-driven cancers. The study focuses on Ewing sarcoma and how the EWS/FLI transcription factor drives the malignancy…

Immune Profiling Leads to Implications for Immunotherapy for NF1-Associated Tumors
 Immune Profiling Leads to Implications for Immunotherapy for NF1-Associated Tumors 150 150 Abbie Miller
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Profiling reveals histologic subtype distinctions and heterogeneity of neurofibromatosis type 1-associated tumors. Neurofibromatosis type 1 (NF1) — an autosomal dominant disorder affecting approximately one in every 3,500 people — results in dysfunctional neurofibromin, a protein expressed throughout the body and involved in the RAS signaling pathway. Virtually all patients with NF1 develop benign peripheral nerve…

Combining Immunotherapies Effective Against Mouse Model of Cancer
 Combining Immunotherapies Effective Against Mouse Model of Cancer 150 150 Mary Bates, PhD
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Recent study results suggest that combining virotherapy and PD-1 blockades may be more effective than either approach alone. Rhabdomyosarcoma, a cancer made up of cells that normally develop into skeletal muscles, is the most common soft tissue cancer in children. If it is detected early and localized in certain areas, rhabdomyosarcoma is usually curable with…

The First Study of Oncolytic HSV-1 in Children and Young Adults With Cancer Indicates Safety and Tolerability
 The First Study of Oncolytic HSV-1 in Children and Young Adults With Cancer Indicates Safety and Tolerability 1024 575 Abbie Miller
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The phase 1 trial shows safety and tolerance of HSV1716 in the pediatric population; evidence of viral replication in the blood and acute inflammation on PET/CT scans suggest a phase 2 trial for dosage and efficacy is warranted. HSV1716 – an oncolytic herpes simplex virus-1 – has been studied in adults via injection into the…

Two Investigational Antitumor Agents Work Better Together Against MPNST and Neuroblastoma
 Two Investigational Antitumor Agents Work Better Together Against MPNST and Neuroblastoma 150 150 Abbie Miller
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The synergistic effects of a kinase inhibitor and an oncolytic herpes virus show promise for difficult-to-treat neuroblastomas and malignant peripheral nerve sheath tumors. Two investigational agents, Aurora A kinase inhibitor (alisertib) and HSV1716, a virus derived from HSV-1 and attenuated by the deletion of RL1, have shown some antitumor efficacy in early clinical trials as…

Harnessing the Immune System: Has the Cure for Cancer Been Within Us All Along?
 Harnessing the Immune System: Has the Cure for Cancer Been Within Us All Along? 1024 575 Abbie Miller
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By learning to manipulate the immune system to target cancer cells, clinician-scientists are ushering in a new era in cancer treatments. The advances in cancer immunotherapy have been headline-making, and some clinical studies have produced stories of near-miraculous recoveries. From the immunotherapy drug credited with curing former president Jimmy Carter’s cancer (pembrolizumab) to the promising…