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Hematology/Oncology/BMT

Modifying NK Cells With CRISPR/Cas9
 Modifying NK Cells With CRISPR/Cas9 969 533 Mary Bates, PhD
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Illustration of NK Cells, T Cells, other immune cells floating across white background

A proof-of-concept study shows success in modifying NK cells for multiple myeloma. In a new proof-of-concept study, researchers from Nationwide Children’s Hospital used CRISPR/Cas9 technology to genetically modify natural killer immune cells, which they then showed are able to address a recognized hurdle in immunotherapy of multiple myeloma. “It was an amazing feeling when we…

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Protecting an ‘Open Future:’ Improving Fertility Preservation Decision-Making for Young Males With Cancer
 Protecting an ‘Open Future:’ Improving Fertility Preservation Decision-Making for Young Males With Cancer 1024 575 Katie Brind'Amour, PhD, MS, CHES
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Color photo of Black father holding infant on shoulder in front of nursery background with clouds on the wall

Evidence from a new study highlights the importance of family-centered decision processes and clinician facilitation in improving fertility preservation uptake prior to cancer treatment. Numerous cancer treatments can reduce fertility or render some patients entirely infertile, but fertility preservation (FP) services have historically had low rates of uptake. When a young person is diagnosed with…

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Inverse Relationship Between Age and Dose of Blood Thinners for Children
 Inverse Relationship Between Age and Dose of Blood Thinners for Children 1024 495 Natalie Wilson
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blood cells

Infants and young children require higher doses of enoxaparin to treat blood clots than do older children. A blood clot, or thrombosis, isn’t a common problem in children, but it can be a dangerous one. Without proper treatment, a thrombosis can cause a host of long-term problems up to and including permanent damage to patients’…

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Novel Genetic Driver Discovered for Pediatric Meningiomas Using Molecular Profiling
 Novel Genetic Driver Discovered for Pediatric Meningiomas Using Molecular Profiling 1024 575 Katie Brind'Amour, PhD, MS, CHES
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Researchers have uncovered a rare subset of meningiomas with a genetic driver shared by another cancer type, opening the door to new therapeutic considerations. When an interesting or intractable cancer case arises at Nationwide Children’s Hospital, researchers and clinicians involved in the Brain Tumor Protocol through The Steve and Cindy Rasmussen Institute for Genomic Medicine…

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CLABSI in Hematology and Oncology: Progress Toward Zero
 CLABSI in Hematology and Oncology: Progress Toward Zero 1024 576 Katie Brind'Amour, PhD, MS, CHES
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Blood cells

A thorough review of five years of CLABSI data reveals key gaps in clinical knowledge that must be addressed to further reduce infection rates. For some time, any bloodstream infection (BSI) in a patient with a central venous catheter was considered a central line-associated bloodstream infection (CLABSI). In 2013, refined definitions enabled a distinction between…

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Boosting Natural Killer Cells for the Treatment of COVID-19
 Boosting Natural Killer Cells for the Treatment of COVID-19 150 150 Lauren Dembeck
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Natural killer cells — critical for immune surveillance and host defense — have an important role in cancer immunotherapy and antiviral responses. While driving to the laboratory one day, Brian Tullius, MA, MD, a fourth-year bone marrow transplant and cell therapies fellow at Nationwide Children’s in the Lee Lab, had an epiphany. “I thought, ‘wait…

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Can Smartphone Apps Keep AYA Oncology Patients Plugged in to Their Medication Routines?
 Can Smartphone Apps Keep AYA Oncology Patients Plugged in to Their Medication Routines? 1024 684 Natalie Wilson
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teen girl using cell phone

Studies show that suboptimal medication adherence may be a barrier to increased survival rates among adolescents and young adults with cancer. Anthony Audino, MD, explores the utility of medication adherence apps to meet the unique needs of these patients. The incidence of cancer among adolescents and young adults (AYA), or those 15-39 years old, has…

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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…

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Managing Lymphatic Flow Disorders: Expanding Care Through Collaboration
 Managing Lymphatic Flow Disorders: Expanding Care Through Collaboration 1024 683 Abbie Roth
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Alteration in the Lymphatic System Leading to Flow Disorders The lymphatic system plays a critical role in circulating a clear to yellow-colored fluid called lymph, which contains proteins, immune factors and cells throughout the body. It is also responsible for collecting fats and fat-soluble vitamins from the gastrointestinal tract in cloudy-appearing chylous fluid. This fluid…

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Novel Genetic Variant Identified in CARMIL2 Deficiency
 Novel Genetic Variant Identified in CARMIL2 Deficiency 1024 575 Lauren Dembeck
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International program facilitates the diagnosis of a rare immunodeficiency in two children caused by a novel genetic variant. A multidisciplinary team of physicians and scientists at Nationwide Children’s Hospital have identified a novel loss-of-function variant causing CARMIL2 deficiency, an inborn error of immunity, in two Saudi Arabian brothers born to consanguineous parents. The case was…

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Novel Use of Vedolizumab Resolves Steroid-Refractory Acute Graft-Versus-Host Disease in a Pediatric Transplant Recipient
 Novel Use of Vedolizumab Resolves Steroid-Refractory Acute Graft-Versus-Host Disease in a Pediatric Transplant Recipient 1024 575 Lauren Dembeck
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Vedolizumab is a monoclonal antibody that targets α4β7 integrin present on gut homing T cells and is indicated for adult inflammatory bowel disease Physicians at Nationwide Children’s Hospital have successfully treated steroid-refractory gastrointestinal acute graft-versus-host disease (SR GI aGVHD) with adjuvant vedolizumab therapy in a pediatric allogeneic hematopoietic cell transplant (HCT) recipient. The case was…

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Collaborative Study Seeks to Understand Gene Expression Changes During Acute Events in Patients With SCD
 Collaborative Study Seeks to Understand Gene Expression Changes During Acute Events in Patients With SCD 1024 575 Natalie Wilson
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Researchers explore gene responses among SCD patients hospitalized for acute complications to inform understandings of the under-studied disease. Sickle cell disease (SCD) is a chronic, genetic disorder characterized by structural changes in circulating red blood cells. According to the CDC, SCD affects approximately 100,000 individuals in the United States alone and accounts for 3,000 births…

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Preventing and Ameliorating Acute and Chronic Kidney Damage After Chemotherapy
 Preventing and Ameliorating Acute and Chronic Kidney Damage After Chemotherapy 720 480 Katie Brind'Amour, PhD, MS, CHES
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Research looking at mitochondrial targets in kidney health holds promise for cisplatin-induced kidney injury. Recent work exploring the mitochrondrial metabolism in the kidneys following exposure to cisplatin, a common chemotherapy, has revealed a key role of superoxide (O2•-, an indicator of oxidative stress in renal cells associated with cell damage and death) in both acute…

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Clinical Trials for Blood Cancers: Advancing Options for Children and Young Adults
 Clinical Trials for Blood Cancers: Advancing Options for Children and Young Adults 1024 683 Abbie Roth
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Most cancer treatments are first discovered, developed and approved for adult patients. But translating those therapies to pediatric and young adult patients with cancers can be more of a winding path than a superhighway. At Nationwide Children’s Hospital, the Division of Hematology, Oncology and Blood and Marrow Transplant is dedicated to participating in and supporting clinical…

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Hemophilia Gene Therapy Trials Aim to Reduce Patient Burden
 Hemophilia Gene Therapy Trials Aim to Reduce Patient Burden 1024 495 Katie Brind'Amour, PhD, MS, CHES
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blood cells

Clinical trials using gene therapy to boost the body’s production of clotting factor aim to remove the need for regular infusions — ideally giving patients years or even decades free from daily worry about their condition. Unlike most pediatric treatment centers, Nationwide Children’s Hospital is one of only a few dozen sites worldwide selected to…

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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
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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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Helping Children With Sickle Cell Disease Adhere to Their Medication
 Helping Children With Sickle Cell Disease Adhere to Their Medication 1024 575 Mary Bates, PhD
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Mobile intervention increases medication adherence in the children who engage with it. A study published in JMIR mHealth uHealth shows that an electronically delivered intervention can improve medication adherence in children with sickle cell disease. Hydroxyurea is a life-changing medication for children with sickle cell disease (SCD). However, many patients may not consistently receive their hydroxyurea. To…

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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…

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From Natural Killer Cells to Virus-Specific T Cells: What’s Next in Cellular Therapy
 From Natural Killer Cells to Virus-Specific T Cells: What’s Next in Cellular Therapy 969 533 Abbie Roth
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Illustration of NK Cells, T Cells, other immune cells floating across white background

Harnessing the power of the immune system to overcome cancer, improve outcomes of bone marrow transplants and fight viral infections in immunocompromised pediatric patients is at the heart of cell therapy research.   Natural Killers in Action Natural killer (NK) cells are a component of the innate immune system that mediates the death of cancer…

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Kidney Biopsy Analysis Reveals High Risk for Disease in Patients With Sickle Cell Disease
 Kidney Biopsy Analysis Reveals High Risk for Disease in Patients With Sickle Cell Disease 1024 732 Mary Bates, PhD
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Patients with SCD are at high risk for significant kidney disease. A retrospective study of biopsy findings from the Pediatric Nephrology Research Consortium shows just how high. Kidney damage is a progressive complication of sickle cell disease (SCD), sometimes eventually leading to kidney failure. As the life expectancy of individuals with SCD has improved, the…

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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…

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What Can We Learn From the Co-occurrence of a Genetic Disorder and Cancer?
 What Can We Learn From the Co-occurrence of a Genetic Disorder and Cancer? 150 150 Mary Bates, PhD
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Researchers investigate an unusual case of concurrent genetic and somatic diagnoses. In a new paper in the European Journal of Medical Genetics, researchers from Nationwide Children’s Hospital discuss the case of a 4-year-old with both a rare genetic disorder and medulloblastoma. Genetic analyses allowed them to evaluate the cause of the genetic disorder and provided support…

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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…

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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
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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…

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Use of Cardiac MRI to Monitor Medical Treatment of Rhabdomyoma
 Use of Cardiac MRI to Monitor Medical Treatment of Rhabdomyoma 150 150 Mary Bates, PhD
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Case study shows success for medical therapy and MRI monitoring of a cardiac rhabdomyoma. In a new case study, researchers from Nationwide Children’s Hospital report treating cardiac rhabdomyoma in a newborn with an mTOR inhibitor called everolimus. They monitored the tumor’s regression using cardiac MRI. Cardiac rhabdomyoma is a common type of heart tumor in…

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Looking to the Future: Optimizing Fertility Preservation Decisions in Pediatric Patients Newly Diagnosed with Cancer
 Looking to the Future: Optimizing Fertility Preservation Decisions in Pediatric Patients Newly Diagnosed with Cancer 1024 575 Katie Brind'Amour, PhD, MS, CHES
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Parent and child holding hands

Family-centered research aims to improve fertility preservation uptake and decision satisfaction among adolescents newly diagnosed with cancer.

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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
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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…

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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
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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…

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Generating the Genome: How Scientists Changed the Face of Cancer Research
 Generating the Genome: How Scientists Changed the Face of Cancer Research 1024 683 Katie Brind'Amour, PhD, MS, CHES
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Color photo; environmental portrait ofDrs. Elaine Mardis and Richard Wilson

Team science. Ongoing innovation. Brilliant minds. Here’s how The Cancer Genome Atlas spawned a revolution in cancer research and technology. The Cancer Genome Atlas is wrapping up. Its data now lives online in the Genomic Data Commons, freely available to the public. Reports of the primary findings for each studied tumor type have been published, and…

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More Screening Needed for Young Females at Risk for von Willebrand Disease
 More Screening Needed for Young Females at Risk for von Willebrand Disease 1024 575 Mary Bates, PhD
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Black and white image of teen girl posing outside

A new study finds most girls with heavy menstrual bleeding are not screened for the bleeding disorder. Despite recommendations by the American College of Obstetricians and Gynecologists, approximately 90 percent of girls with heavy menstrual bleeding are not being screened for von Willebrand disease, according to a new study from Nationwide Children’s Hospital. “Von Willebrand disease is…

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Is Caregiver Education About Sickle Cell Trait Effective?
 Is Caregiver Education About Sickle Cell Trait Effective? 1024 575 Abbie Roth
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Color photo of Black father holding infant on shoulder in front of nursery background with clouds on the wall

Researchers evaluate the standardized education in Ohio to determine if it achieves high caregiver knowledge. Despite universal newborn screening that detects the presence of sickle cell trait (SCT), only 16 percent of Americans with SCT know their status. To address this issue, in Ohio, in-person education is offered to caregivers of referred infants with SCT.…

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How Can We Optimize Fertility Related Discussions With Male Survivors of Childhood Cancer?
 How Can We Optimize Fertility Related Discussions With Male Survivors of Childhood Cancer? 150 150 Abbie Roth
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A recent study suggests that fertility is a “later” issue for adolescents and young adults, rather than a “now” concern, leading to low rates of conversation with health care providers. Survival rates for pediatric cancers exceed 80 percent. But late effects can persist throughout the survivor’s lifetime. Among those late effects, infertility may have both…

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Starting the Conversation on Sickle Cell Disease and Reproductive Health
 Starting the Conversation on Sickle Cell Disease and Reproductive Health 1024 575 Katie Brind'Amour, PhD, MS, CHES
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Adolescents and young adults with sickle cell disease — and their caregivers — care about future fertility. But what should doctors tell them? Until just a few decades ago, sickle cell disease (SCD) was often fatal in childhood. Now that more patients reach adulthood, clinicians and researchers need to ask — and answer — questions…

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How to Identify and Treat Blood Clots in Pediatric Patients With Cancer
 How to Identify and Treat Blood Clots in Pediatric Patients With Cancer 1024 575 Mary Bates, PhD
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Black and white photo of a young patient with cancer wearing a mask and gown and holding an IV poll smiling and giving a thumbs up in the hospital

New guidelines will help doctors recognize and treat children at risk of venous thromboembolism. Prevention of blood clots is well described in adult cancer patients, but data on the problem in pediatric cancer patients is limited. In a new publication initiated at the request of the International Society of Thrombosis and Hemostasis, researchers provide guidance for…

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What Happens When Cancer Remission Is in Question?
 What Happens When Cancer Remission Is in Question? 1024 575 Mary Bates, PhD
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Photo of leukemia cells under microscope

A new study looks at outcomes for children with leukemia when two tests of remission disagree. In both clinical practice and clinical trials, remission in leukemia is determined by morphological assessment — a person counting cancer cells in a bone marrow sample under a microscope. But more sophisticated technologies, such as flow cytometry or polymerase…

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Large-Scale Genomics Study Identifies Children With High-Risk Cancer
 Large-Scale Genomics Study Identifies Children With High-Risk Cancer 150 150 Mary Bates, PhD
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Some subtypes of leukemia have a poor prognosis. Genomic studies are helping to identify these subtypes, leading to targeted therapies. Acute lymphoblastic leukemia (ALL), a malignancy of the white blood cells, is a common childhood cancer. Understanding the genomic changes underlying ALL has led to the development of new, targeted therapies, especially for patients with…

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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
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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…

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To Test or Not to Test: The Inherited Thrombophilia Question
 To Test or Not to Test: The Inherited Thrombophilia Question 150 150 Abbie Roth
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When a result provides questionable or limited clinical utility, physicians and genetic counselors must educate patients and providers on the pros and cons of genetic testing. Between the increased ease of clinical genetic testing and the direct-to-consumer genetic testing kits now available, physicians and genetic counselors face education challenges that simply did not exist a…

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Making Stem Cell Transplantation Work for Sickle Cell Disease
 Making Stem Cell Transplantation Work for Sickle Cell Disease 150 150 Mary Bates, PhD
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New regimen offers a promising approach for unrelated donor transplants. Sickle cell disease (SCD) is a blood disorder affecting 1 in every 500 African-American newborns. It is characterized by extreme pain and decreased longevity. Currently, the only cure for SCD lies in hematopoietic stem cell transplantation. A well-matched sibling donor is needed for the best…

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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
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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…

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The Evolution of Precision Medicine for Children With Aggressive Brain Tumors
 The Evolution of Precision Medicine for Children With Aggressive Brain Tumors 150 150 Jonathan Finlay, MB, ChB, FRCP
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The Head Start 4 clinical trial introduces risk stratification and builds on the success of bone marrow transplantation for young children with malignant embryonal tumors. Cancers in the brain and spinal cord (the central nervous system, CNS) are now the most common cause of deaths due to disease in children and adolescents. Among these tumors,…

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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
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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…

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“Impatient” Therapy: Physicians Too Aggressive in Treatment of ITP
 “Impatient” Therapy: Physicians Too Aggressive in Treatment of ITP 150 150 Brianne Moore
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Despite guidelines that advocate watching and waiting, physicians are still treating most patients with immune thrombocytopenia. When a child presents with easy bruising or bleeding, red skin spots and fatigue – symptoms similar to those for leukemia – families seek evaluation immediately. However, more often these cases are pediatric immune thrombocytopenia (ITP), an acquired disorder…

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Creative Reality: Using a New Platform Technology to Improve Patient Experience
 Creative Reality: Using a New Platform Technology to Improve Patient Experience 1024 575 Abbie Roth
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Amy Dunn, MD, had a problem. Some of her patients in the hematology clinic at Nationwide Children’s Hospital receive hundreds of needle sticks each year. “Needle phobia is very real for these patients and their families,” says Dr. Dunn, director of Pediatric Hematology at Nationwide Children’s. “In some cases, ports need to be implanted so that these…

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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
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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…

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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
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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…

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Assessing Romantic Lives of Adult Survivors of Childhood Cancer
 Assessing Romantic Lives of Adult Survivors of Childhood Cancer 150 150 Kevin Mayhood
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In some ways, the group appears more resilient than survivors of adult-onset cancer or those with other childhood-onset diseases. Adult survivors of childhood cancer appear, on the whole, to have comparable romantic lives to others their age, new studies show. Surprisingly, the group doesn’t seem to carry the same burdens as men and women 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
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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…

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