Center for Childhood Cancer and Blood Diseases

Assessing the Impact of COVID-19 on Pediatric Cancer Research
Assessing the Impact of COVID-19 on Pediatric Cancer Research 1024 575 Lauren Dembeck

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…

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Epigenetics, Chromatin Architecture and a Judo Mechanism to Attack Cancer
Epigenetics, Chromatin Architecture and a Judo Mechanism to Attack Cancer 1024 575 Abbie Roth

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…

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Understanding Drug Interactions in Diffuse Midline Gliomas
Understanding Drug Interactions in Diffuse Midline Gliomas 1024 683 Lauren Dembeck

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…

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Targeting Alternative RNA Splicing in Pediatric Cancers
Targeting Alternative RNA Splicing in Pediatric Cancers 150 150 Mary Bates, PhD

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…

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The Search to Identify Tumor Cells Evading Chemotherapy
The Search to Identify Tumor Cells Evading Chemotherapy 150 150 Sanjana Rajan

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…

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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

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…

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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
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…

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An Infant. A Virus. An Emergency IND. A Life Saved.
An Infant. A Virus. An Emergency IND. A Life Saved. 150 150 Abbie Roth

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…

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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

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…

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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

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…

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Two Classes of GGAA-Microsatellites in a Ewing Sarcoma Context
Two Classes of GGAA-Microsatellites in a Ewing Sarcoma Context 150 150 Abbie Roth

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…

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A New Paradigm for Treating Transcription Factor-Driven Cancers
A New Paradigm for Treating Transcription Factor-Driven Cancers 150 150 Abbie Roth

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…

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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 Roth

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…

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Combining Immunotherapies Effective Against Mouse Model of Cancer
Combining Immunotherapies Effective Against Mouse Model of Cancer 150 150 Mary Bates, PhD

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…

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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 Roth

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…

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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 Roth

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…

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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 Roth

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…

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