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Relative Adrenal Insufficiency Observed Among Patients With High-Risk Neuroblastoma During Treatment
 Relative Adrenal Insufficiency Observed Among Patients With High-Risk Neuroblastoma During Treatment 1024 683 Jessica Nye, PhD
By: Jessica Nye, PhD

A subset of patients with high-risk neuroblastoma (NBL) and primary adrenal tumors develop an adrenal insufficiency (AI)-like phenotype during treatment. “We noticed that some of the patients with neuroblastoma had less hypotension when we added hydrocortisone while they were getting dinutuximab therapy. So, we said, I wonder if that has anything to do with the…

Breaking New Ground in Cancer Immunotherapy
 Breaking New Ground in Cancer Immunotherapy 1024 683 Pam Georgiana
By: Pam Georgiana

Nationwide Children’s researchers develop a scalable, targeted approach to overcoming cancer’s toughest challenges. Despite significant advances in treatments, cancer remains one of the leading causes of death globally. Traditional therapies, while improving patient outcomes, still have limitations — such as the need for frequent dosing, complex and costly manufacturing and severe side effects. These challenges…

Pivotal New Research Fuels Possibilities for Aggressive Pediatric Brain Cancer
 Pivotal New Research Fuels Possibilities for Aggressive Pediatric Brain Cancer 1024 414 Wendy Margolin
By: Wendy Margolin

A new umbrella trial of molecularly driven therapies for high-grade gliomas (HGG) and diffuse intrinsic pontine glioma (DIPG) aims to improve quality of live and extend survival rates for these aggressive tumors. The diagnosis of high-grade glioma (HGG), a type of aggressive, malignant brain tumor, in pediatric patients is particularly heartbreaking. HGGs are one of…

1 Trillion Cells: Nationwide Children’s Reaches New Milestone for Universal-Donor NK Cell Studies
 1 Trillion Cells: Nationwide Children’s Reaches New Milestone for Universal-Donor NK Cell Studies 1024 649 Abbie Miller
By: Abbie Miller

The Biologics Manufacturing Resource, Nationwide Children’s Cell and Tissue cGMP manufacturing facility, recently achieved a major milestone. The resource has now delivered more than 1 trillion universal donor natural killer (NK) cells to patients. These NK cells are being studied in clinical trials to learn if they can help children and adults with cancer. “This…

Oncolytic Viroimmunotherapy Improved by Enhanced Cytokine Transgene Expression
 Oncolytic Viroimmunotherapy Improved by Enhanced Cytokine Transgene Expression 1024 575 Lauren Dembeck
By: Lauren Dembeck

Researchers were able to suppress in vivo tumor growth with an oncolytic virus by enhancing the production of the cytokine interleukin-12 in the tumors. Malignant peripheral nerve sheath tumors (MPNSTs) are rare, aggressive sarcomas that exhibit resistance to non-surgical treatment and have high rates of metastasis and relapse. The prognosis for MPNSTs is poor, with…

Health-Related Quality of Life in Pediatric Cancer Survivors: Assessing the Impacts
 Health-Related Quality of Life in Pediatric Cancer Survivors: Assessing the Impacts 1024 683 Madison Storm
By: Madison Storm

Family factors have a significant impact on health-related quality of life. Childhood cancer is a life-changing diagnosis for children and their families. Children undergoing cancer treatment often experience impairment in health-related quality of life compared to children from healthy populations. The severity of impact can depend, in part, on social determinants of health, such as…

How Does Hodgkin Lymphoma Differ With Age?
 How Does Hodgkin Lymphoma Differ With Age? 150 150 Pam Georgiana
By: Pam Georgiana

A new study reveals age-related differences in pathology and medical management. When Anthony N. Audino, MD, a pediatric oncologist in the Division of Pediatric Hematology/Oncology/BMT at Nationwide Children’s, first met a preschool-aged patient with Hodgkin lymphoma, he was surprised. “It was startling to see someone so young with this disease because it’s typically diagnosed between…

Proton Therapy – A New Era of Cancer Therapy for Children
 Proton Therapy – A New Era of Cancer Therapy for Children 1024 683 Abbie Miller
By: Abbie Miller

Proton therapy offers benefits over traditional photon radiation therapy. Using proton therapy, doctors can give more specific, concentrated doses of radiation with less risk and damage to the surrounding tissue. Proton therapy is an advanced type of radiation treatment that uses protons instead of X-rays to kill cancer cells. Our proton therapy center uses the…

New Zebrafish Pipeline Enables Functional Evaluation of Novel, Rare, Fusion-Oncogenes in vivo
 New Zebrafish Pipeline Enables Functional Evaluation of Novel, Rare, Fusion-Oncogenes in vivo 1024 683 Jessica Nye, PhD
By: Jessica Nye, PhD

An innovative method that uses a zebrafish model to rapidly study novel fusion-oncogenes in vivo has been developed by investigators at Nationwide Children’s Hospital.   Fusion oncogenes are frequently observed in pediatric cancers. These fusion oncogenes are typically caused by a chromosomal translocation that produces gain-of-function chimeric gene activity. Although there has been experimental work…

Interactions Between Invading Tumor and Lung Cells Permit Metastatic Lung Colonization of Osteosarcoma
 Interactions Between Invading Tumor and Lung Cells Permit Metastatic Lung Colonization of Osteosarcoma 150 150 Lauren Dembeck
By: Lauren Dembeck

Survival signals elicited by lung tissue interactions promote osteosarcoma metastasis and represent a promising target for clinical trials in both human and canine patients.   Osteosarcoma, the most common primary tumor of bone, occurs predominantly in children, teens and young adults. Patient survival largely depends upon the presence or absence of metastasis. Within five years…

Transforming Treatment for Paratesticular Rhabdomyosarcoma in Adolescents through Robotics
 Transforming Treatment for Paratesticular Rhabdomyosarcoma in Adolescents through Robotics 1024 683 Erin Gregory
By: Erin Gregory

Retroperitoneal lymph node dissection (RPLND) has been a crucial component of treating testicular and paratesticular cancers for over a century. Recent advancements in minimally invasive techniques, such as robotic-assisted methods, have expanded the applicability of RPLND, offering reduced morbidity and faster recovery. However, concerns persist regarding the equivalence of oncological outcomes compared to traditional open…

Oncolytic Virus Immunotherapy Safe and Tolerable in Children With Relapsed or Refractory Solid Tumors
 Oncolytic Virus Immunotherapy Safe and Tolerable in Children With Relapsed or Refractory Solid Tumors 1024 683 Lauren Dembeck
By: Lauren Dembeck

The overall survival rates for children with childhood cancers is now over 80%; however, for children with relapsed and refractory tumors, the survival rates remain low. “For example, children with relapsed or refractory neuroblastoma have a five-year survival of less than ten percent,” explains Keri A. Streby, MD, director of the Neuroblastoma Program in the…

Targeted Muscle Reinnervation Prevents Neuroma and Phantom Limb Pain in Children After Amputation
 Targeted Muscle Reinnervation Prevents Neuroma and Phantom Limb Pain in Children After Amputation 1024 684 Lauren Dembeck
By: Lauren Dembeck

In certain cases, such as limb traumas and sarcomas, amputation may be chosen to optimize patients’ physical function. Persistent postamputation residual limb or “stump” pain and phantom limb pain can be debilitating and has been associated with prosthetic abandonment in patients who have undergone amputation. This pain is often secondary to symptomatic neuromas, tumor-like disorganized…

Is Growth Hormone Replacement Safe Following Central Nervous System Tumors?
 Is Growth Hormone Replacement Safe Following Central Nervous System Tumors? 1024 683 Mary Bates, PhD
By: Mary Bates, PhD

Research suggests the benefits of growth hormone therapy outweigh the risks for pediatric cancer survivors. In a new review paper, researchers from Nationwide Children’s Hospital evaluated the risks and benefits associated with administering growth hormone to pediatric patients after diagnosis of a central nervous system tumor. The authors conclude that the therapy has many important…

Chaotic Genomes in Childhood Bone Tumors Are Not Always Unstable
 Chaotic Genomes in Childhood Bone Tumors Are Not Always Unstable 1024 575 Jessica Nye, PhD
By: Jessica Nye, PhD
conceptual art of DNA

The structural genomic complexity that characterizes most osteosarcoma tumors in children was not evidence of an unstable genome and a driver of cell-to-cell variations in gene expression within each tumor. “We’ve been trying to understand how tumor cells change as they become resistant to therapy or as they metastasize and spread to different parts of…

1 Year In, the Molecular Characterization Initiative Has Analyzed Samples From Over 1,000 Patients
 1 Year In, the Molecular Characterization Initiative Has Analyzed Samples From Over 1,000 Patients 1024 614 Abbie Miller
By: Abbie Miller
abstract art of magnifying glass over DNA strand

Data from the analysis helps clinicians confirm diagnoses and identify targeted treatments. The data also support new pediatric cancer research through the National Cancer Institute’s Childhood Cancer Data Initiative.   The CCDI Molecular Characterization Initiative (MCI) is a project that aims to collect, analyze and report clinical and molecular data and is currently supporting Children’s…

Going Viral: The AAV Approach to Curing Cancer
 Going Viral: The AAV Approach to Curing Cancer 1024 256 Emily Siebenmorgen
By: Emily Siebenmorgen

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…

Using Machine Learning in the Electronic Medical Record to Save Lives
 Using Machine Learning in the Electronic Medical Record to Save Lives 1024 683 Abbie Miller
By: Abbie Miller
physicians in white coats looking at a tablet

The deterioration risk index identifies patients at risk for deterioration and poor outcomes, triggering the care team to act before a crisis happens. In a report published in Pediatric Critical Care Medicine, a team from Nationwide Children’s Hospital describes a machine learning tool for timely identification of hospitalized children at risk for deterioration – a…

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
By: Katie Brind'Amour, PhD, MS, CHES

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.…

Genomic Medicine Translational Protocols – What Do They Mean for Patients Everywhere?
 Genomic Medicine Translational Protocols – What Do They Mean for Patients Everywhere? 1024 614 Elizabeth Varga
By: Elizabeth Varga
abstract art of magnifying glass over DNA strand

As part of the Journey to Best Outcomes, Nationwide Children’s Hospital has dedicated significant resources to genomic medicine, named as a key accelerator in the 2017-2021 strategic plan and as one of the three pillars that will be utilized to integrate research and clinical care over the next five years. In response, the Steve and…

New Initiative Aims to Bring Personalized Diagnosis and Treatment to Children With Solid Tumors
 New Initiative Aims to Bring Personalized Diagnosis and Treatment to Children With Solid Tumors 1024 614 Lauren Dembeck
By: Lauren Dembeck
abstract art of magnifying glass over DNA strand

Collaboration among National Cancer Institute, Children’s Oncology Group and Nationwide Children’s Hospital will offer molecular characterization of childhood cancers. The National Institutes of Health’s National Cancer Institute (NCI), Children’s Oncology Group (COG), and Nationwide Children’s Hospital are poised to change the direction of pediatric cancer diagnosis and care through a new initiative that is the…

Profiling the Tumor Microenvironment Offers New Insight for Hard-to-Treat Tumors
 Profiling the Tumor Microenvironment Offers New Insight for Hard-to-Treat Tumors 1024 269 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

A multi-faceted approach to precision medicine may allow clinicians to gain a better understanding of cancer and the complex ecosystem in which it resides. The field of precision medicine has evolved in leaps and bounds in the past two decades. Genomic profiling tools that were once cost-prohibitive and slow are now reasonably affordable and fast…

A Single-Dose Cancer Immunotherapy via Gene Transfer
 A Single-Dose Cancer Immunotherapy via Gene Transfer 150 150 Mary Bates, PhD
By: Mary Bates, PhD

Researchers at Nationwide Children’s Hospital report proof-of-principle results for a new gene therapy cancer treatment. The off-the-shelf, single-dose immunotherapy serves as an alternative to CAR-T therapy and can be engineered to be on-demand. T cells redirected to cancer cells either via a chimeric antigen receptor (CAR-T) or a bispecific molecule have been game-changers in treating…

Optimizing the Body’s Natural Cancer Killers
 Optimizing the Body’s Natural Cancer Killers 1024 649 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

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…

Featured Researcher — Diana Thomas, MD, PhD
 Featured Researcher — Diana Thomas, MD, PhD 150 150 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Diana Thomas, MD, PhD, joined the Biopathology Center (BPC) at Nationwide Children’s Hospital as its pathology operations director in 2021. Since then, she has been busy supporting research protocol development and the receipt, processing, banking and distribution of biospecimens for clinical and research investigators around the world. As a biorepository for some of the largest…

Exploring the RNA Cargo of Extracellular Vesicles in Malignant Pediatric Brain Tumors
 Exploring the RNA Cargo of Extracellular Vesicles in Malignant Pediatric Brain Tumors 150 150 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

The RNA cargo within the extracellular vesicles of medulloblastoma and diffuse infiltrative pontine glioma can provide valuable insight into diagnosing and treating these malignant pediatric brain tumors. Recent studies have shed light on extracellular vesicles’ ability to use their cargo, which includes small noncoding RNA (ncRNA), to facilitate cell-to-cell communication. Extracellular vesicles (EVs) derived from…

Short- and Long-Term Kidney Issues in Children Treated for Leukemia
 Short- and Long-Term Kidney Issues in Children Treated for Leukemia 720 480 Mary Bates, PhD
By: Mary Bates, PhD

While outcomes for childhood acute lymphoblastic leukemia (ALL) have improved, researchers are finding long-term effects on kidney function that require monitoring. In a new study published recently in Pediatric Nephrology, researchers from Nationwide Children’s report the incidence of hypertension and kidney issues in pediatric patients diagnosed with acute lymphoblastic leukemia over a recent nine-year period.…

Therapy With Double-Bright Natural Killer Cell Infusions Increases Response Rates Among Frail Patients With Acute Myeloid Leukemia
 Therapy With Double-Bright Natural Killer Cell Infusions Increases Response Rates Among Frail Patients With Acute Myeloid Leukemia 969 533 Jessica Nye, PhD
By: Jessica Nye, PhD
Illustration of NK Cells, T Cells, other immune cells floating across white background

A case series of 13 patients with relapsed/refractory acute myeloid leukemia (AML) finds that a novel approach using repeated infusions of cultured natural killer (NK) cells increases overall and complete response rates. NK cells are lymphocytes – white blood cell components of the innate immune response – that have anti-leukemic properties. Although efforts to incorporate…

Distinct Transcriptional Regulatory Domain Identified in Ewing Sarcoma Fusion Protein
 Distinct Transcriptional Regulatory Domain Identified in Ewing Sarcoma Fusion Protein 1024 1024 Lauren Dembeck
By: Lauren Dembeck
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…

Risk for Serious Complications From Vaccine-Preventable Infections After Hematopoietic Cell Transplant
 Risk for Serious Complications From Vaccine-Preventable Infections After Hematopoietic Cell Transplant 1024 683 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Clinician-scientists reveal the burden of vaccine-preventable infections among children post-transplant, when immunity is low and risk is high. When a hematopoietic cell transplant (HCT) recipient at Nationwide Children’s Hospital was diagnosed with a vaccine-preventable infection (VPI), treating clinicians decided to evaluate the burden of VPI in HCT patients at Nationwide Children’s and elsewhere. The team…

The Impact of Genomic Medicine: Carter’s Story
 The Impact of Genomic Medicine: Carter’s Story 1024 685 Natalie Wilson
By: Natalie Wilson

In 2013, at just 1 year old, Carter Daggett was diagnosed with a rare brain tumor. Now, in 2021, at 8 years old, he celebrated three years cancer free — thanks to his clinical teams and cutting-edge research by the team at the Steve and Cindy Rasmussen Institute for Genomic Medicine at Nationwide Children’s Hospital.…

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
By: Natalie Wilson

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…

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

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
By: Mary Bates, PhD

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…

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
By: 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…

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
By: Katie Brind'Amour, PhD, MS, CHES
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…

What Happens When Cancer Remission Is in Question?
 What Happens When Cancer Remission Is in Question? 1024 575 Mary Bates, PhD
By: Mary Bates, PhD
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…

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
By: Mary Bates, PhD

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…

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
By: 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…

Adolescents, Young Adults and Cancer: What Are the Issues?
 Adolescents, Young Adults and Cancer: What Are the Issues? 150 150 Abbie Miller
By: Abbie Miller

Adolescents and young adults with cancer have unique needs that may explain their plateau in survival rates, despite improved survival rates in other age groups. At an age when achieving independence and experiencing life milestones are most important, adolescents and young adults facing a cancer diagnosis must meet unique challenges. These challenges may contribute to…

A Watershed Moment for Cancer Virotherapy
 A Watershed Moment for Cancer Virotherapy 150 150 Timothy Cripe, MD, PhD
By: Timothy Cripe, MD, PhD

A recent decision may define the future of virotherapy’s role in the clinical treatment of cancer. At 4:50 pm on Wednesday, April 29 2015, the votes were cast: 22 in favor, 1 against. With this overwhelming majority, an advisory committee sent a clear message to the FDA recommending the first marketing approval for a live virus cancer…