Research

Unlocking the Structure of Biofilms
Unlocking the Structure of Biofilms 1024 575 Kevin Mayhood
Illustration showing the precise pattern of a biofilm - a perfect, 3D matrix

Researchers characterize a component that stabilizes biofilms, a step toward learning ways to disrupt protection of harmful bacteria. In the extracellular DNA lattice of bacterial biofilms, nature appears to reprise the functional equivalent of Holliday junction (HJ) intermediates — cross-shaped structures formed during the process of genetic recombination, researchers at Nationwide Children’s Hospital report in Proceedings…

Study Shows Promising Host-Targeted Approach for the Prevention and Cure of Gonorrhoea in Women
Study Shows Promising Host-Targeted Approach for the Prevention and Cure of Gonorrhoea in Women 1024 683 Nationwide Children's

In research published in mBio, researchers from the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital and Griffith University’s Institute for Glycomics have discovered non-antibiotic (host-targeted) therapies for the effective treatment of Neisseria gonorrhoeae infections by repurposing existing drugs. Gonorrhoea is the second most commonly reported sexually transmitted infection (STI). The Centers for Disease Control and…

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…

Could Restoring CD4+ T Cell Immunity Help Control Hepatitis C Infection?
Could Restoring CD4+ T Cell Immunity Help Control Hepatitis C Infection? 1024 683 Kevin Mayhood

Following childbirth, some women with chronic infection experienced a recovery of HCV-specific CD4+ T cells and a reduction in viral replication. In chronic hepatitis C (HCV) infections, CD4+ T cells that recognize the virus have been so hard to detect, some researchers thought they may be completely depleted. But research led by investigators at Nationwide Children’s…

From Clinician Ideas to Commercially-Available Clinical Devices
From Clinician Ideas to Commercially-Available Clinical Devices 150 150 Kevin Mayhood

Pressure wounds were a common complication following a tracheostomy, often resulting in advanced-stage wounds, national studies showed. Nationwide Children’s Hospital was no different but Kris Jatana, MD, and Charles Elmaraghy, MD, surgeons in the Department of Otolaryngology, knew they could improve these outcomes. Brendan Boyle, MD, and Alex Green, DO, were fellows in the Division…

Asymptomatic Infants With Congenital Cytomegalovirus May Still Have Detectable, Significant Abnormalities
Asymptomatic Infants With Congenital Cytomegalovirus May Still Have Detectable, Significant Abnormalities 1024 575 Katie Brind'Amour, PhD, MS, CHES
Color photo of Black father holding infant on shoulder in front of nursery background with clouds on the wall

More than half of high-risk CMV-positive newborns may have abnormalities not detected by a physical exam alone. A study in 34 infants with a normal physical exam despite a positive diagnosis of congenital cytomegalovirus (CMV) — a viral infection that can lead to neurodevelopmental delays and permanent hearing loss — found that in more than…

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…

Percutaneous Occlusion Linked to Respiratory Improvements in Low-Weight Infants with PDA
Percutaneous Occlusion Linked to Respiratory Improvements in Low-Weight Infants with PDA 1024 575 Abbie Miller
Nurse caring for infant in Neonatal Intensive Care Unit (NICU)

Percutaneous occlusion for patent ductus arteriosus (PDA) in infants weighing less than 6 kg is associated with potential longer term improvements in respiratory health, research by investigators at Nationwide Children’s Hospital shows. “A new device for PDA closure recently approved by the FDA has potential to reduce the incidence of device-related complications. This important work…

A Deep-Learning Computer Model for Predicting Pediatric Patient Health Care Risks and Costs
A Deep-Learning Computer Model for Predicting Pediatric Patient Health Care Risks and Costs 150 150 Kevin Mayhood

The model, which requires fewer resources and less time to develop, outperformed traditional risk-prediction models in prospective hospitalization prediction. A deep-learning computer model was better at predicting the health care risk of 112,000 children in a Medicaid accountable care organization (ACO) than traditional risk prediction models, researchers at Nationwide Children’s Hospital have shown. “Using this…

A Major Milestone in Cystic Fibrosis Treatment
A Major Milestone in Cystic Fibrosis Treatment 1024 575 Mary Bates, PhD
Illustration of lungs on blue silhouette of upper chest on black background

A triple medication combination could be life-changing for the majority of patients with cystic fibrosis. A Phase III clinical trial shows that elexacaftor added to ivacaftor and tezacaftor improves lung function and quality of life in cystic fibrosis patients with the most common genetic mutation, F508del. The triple therapy, known as Trikafta, could effectively treat 90%…

A New Therapeutic Era in Pediatric Functional and Motility Disorders
A New Therapeutic Era in Pediatric Functional and Motility Disorders 150 150 Katie Brind'Amour, PhD, MS, CHES

20 years ago, treatment options were limited for children with functional gastrointestinal (GI) and motility disorders. Now, a growing list of drugs, behavioral and dietary care plans, and an advanced, forward-looking technique – neuromodulation – are transforming pediatric GI care. Gastrointestinal (GI) disorders are notoriously difficult to diagnose. Virtually all functional and motility-related GI problems…

Finding the Reasons Why: Looking for Answers in Trends of Child and Youth Suicides
Finding the Reasons Why: Looking for Answers in Trends of Child and Youth Suicides 1024 575 Kevin Mayhood

Epidemiological studies are the first step to learn how to prevent suicide attempts and deaths. Suicide is the second-leading cause of death among 10- to 24-year-olds. And even as awareness grows, the suicide rate continues to climb, according to national statistics. But those national statistics don’t tell the whole story. For decades, researchers around the…

Nation’s First Clinical Trial for Pediatric Stroke Rehabilitation
Nation’s First Clinical Trial for Pediatric Stroke Rehabilitation 1024 575 Mary Bates, PhD
Toddler playing with toys

A novel movement-based therapy is being evaluated in infants who suffered a stroke as newborns or in the womb. Nationwide Children’s Hospital is participating in the nation’s first multicenter pediatric stroke recovery trial. The Phase III clinical trial, called I-ACQUIRE, will evaluate an innovative therapy to increase motor skills in 8-month-old to 24-month-old infants who…

Largest Study of Glomerular Diseases Sheds Light on Pediatric Subtypes
Largest Study of Glomerular Diseases Sheds Light on Pediatric Subtypes 150 150 Cyndie Trapasso

In one of the first papers published from the largest study of glomerular diseases, researchers have found significant differences between patients who have IgA nephropathy and a subtype of the disease, IgA vasculitis, and between children and adults with these diagnoses. The paper is from Cure Glomerulonephropathy (CureGN) — a longitudinal observational study of nearly…

Preventing the Development of Muscular Dystrophy Through Surrogate Gene Therapy
Preventing the Development of Muscular Dystrophy Through Surrogate Gene Therapy 1024 683 Lauren Dembeck

Researchers identify a key regulatory protein implicated in Galgt2 overexpression and begin to elucidate its protective mechanism against muscular dystrophy. Researchers at Nationwide Children’s Hospital have identified a small, soluble protein that can activate a human gene known to inhibit the development of muscular dystrophy when overexpressed in muscle. That is, when there is a…

Uroplakin Plaque Key to Protecting Kidney During Urinary Tract Obstruction
Uroplakin Plaque Key to Protecting Kidney During Urinary Tract Obstruction 150 150 Kevin Mayhood

Plaque appears to help remodel urothelium, allowing kidney to hold excess urine without damage, Urinary tract obstruction (UTO) is the leading cause of chronic kidney disease and end-stage renal disease in children. But the extent of damage caused by obstruction differs from child to child. Researchers at Nationwide Children’s Hospital studying why suggest that uroplakin…

Development of Innovative Scoring Systems for Sickness and Tissue Damage From Clostridium difficile Colitis
Development of Innovative Scoring Systems for Sickness and Tissue Damage From Clostridium difficile Colitis 1024 575 Lauren Dembeck

In a new publication, the team reported the development of two novel scoring systems to consistently and efficiently assess sickness and tissue injury during antibiotic-associated C. difficile colitis in a murine model. “My laboratory at Nationwide Children’s Research Institute has collaborated for several years with two other laboratories, those of Dr. Steven Goodman and Dr. Michael Bailey.…

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…

Hey Google, Siri and Alexa, How Do We Bring Voice Technology Into Health Care?
Hey Google, Siri and Alexa, How Do We Bring Voice Technology Into Health Care? 1024 576 Katie Brind'Amour, PhD, MS, CHES

As voice assistant technology plays an increasing role in everything from home security to baking, researchers at the vanguard of medical innovation must figure out how to appropriately adapt it for the future of health care. Voice assistant technology has advanced dramatically in the last couple of decades, moving from almost comical talk-to-text tools in…

Bacteria Hiding Out Inside Epithelial Cells May Promote Recurring Ear Infections
Bacteria Hiding Out Inside Epithelial Cells May Promote Recurring Ear Infections 1024 575 Rachael Hardison

Middle ear infections, also known as otitis media (OM), remain a health care concern for children in the United States and across the world, despite recent therapeutic and technological advances. For the subset of children with chronic or recurring infections, OM becomes a significant socioeconomic burden for caregivers. Chronic or recurrent episodes of OM can…

Neonatal Abstinence Syndrome: Transforming Care for Newborns and Their Families
Neonatal Abstinence Syndrome: Transforming Care for Newborns and Their Families 1024 575 Abbie Miller

If there’s a success story to be told at this point in the history of the opioid crisis, it’s in the newborn intensive care unit. From changing attitudes to standardizing treatment, clinical care for infants with neonatal abstinence syndrome (NAS) and their families is markedly different than it was 10 years ago.   CHANGING ATTITUDES…

Could Earlier Spirometry Get CF Inpatients Home Sooner?
Could Earlier Spirometry Get CF Inpatients Home Sooner? 150 150 Katie Brind'Amour, PhD, MS, CHES

A retrospective chart review suggests early spirometry is associated with shorter length of stay. As multicenter studies examine whether length of hospital stay and duration of treatment can be safely shortened for cystic fibrosis-related pulmonary exacerbations, physician-researchers at Nationwide Children’s Hospital have been exploring another unresolved angle related to these admissions: when to do pulmonary…

Crafty Hepatitis E Virus Uses Decoy to Evade Immune System
Crafty Hepatitis E Virus Uses Decoy to Evade Immune System 1024 683 Kevin Mayhood

Researchers find a related protein cloaking the virus has differences that could become treatment targets. A protein that doctors use as a marker of hepatitis E virus (HEV) infection is not directly involved in the virus lifecycle but acts as a decoy drawing away antibodies, helping HEV evade the immune system and thrive, researchers in…

Solving the Puzzle of Transfusion-Related Immune Reactions
Solving the Puzzle of Transfusion-Related Immune Reactions 576 367 Katie Brind'Amour, PhD, MS, CHES

Now that physicians have the “how” of blood transfusions mastered, they are starting to explore the “what” — and they’re learning that the effects of sharing human blood may be even more far-reaching and complex than previously imagined. With the initial safety challenges addressed and the technical barriers of donor blood storage and cleaning improved,…

Phase 1 Study Shows Promise of Gene Replacement Therapy for Spinal Muscular Atrophy Type 1
Phase 1 Study Shows Promise of Gene Replacement Therapy for Spinal Muscular Atrophy Type 1 1024 575 Abbie Miller

The phase 1 clinical study shows that gene therapy extends survival of patients and supports achievement of milestones previously unseen in the natural course of the disease. A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1), according to a study published in…

On the Road to Eliminating RSV
On the Road to Eliminating RSV 150 150 Abbie Miller

With the recent publication of two papers, researchers shed light on factors that influence disease severity and immune response to respiratory syncytial virus infection in infants and young children. “RSV is a very common infection in infants and young children – almost everyone will be infected by age 2. In the United States, 2-3 percent…

Could Nutritional Supplements Impact Autism Symptoms in Toddlers Born Preterm?
Could Nutritional Supplements Impact Autism Symptoms in Toddlers Born Preterm? 150 150 Rachael Hardison

Supplementing with omega-3 and -6 fatty acids impacts language development in preterm toddlers at risk for developing autism. Delay in language development is often an early indicator in children at risk for developing autism spectrum disorder (ASD), yet clinicians are still trying to understand the best practices for how and when to implement early intervention…

Children With Autism and Abdominal Pain Have Distinctive Bacterial Profiles
Children With Autism and Abdominal Pain Have Distinctive Bacterial Profiles 150 150 Kevin Mayhood

The microbiome may harbor causes of abdominal and behavioral issues and potential targets for relief. Children with autism spectrum disorder (ASD) and functional abdominal pain may have a distinct microbiome-neuroimmune profile compared to kids with gastrointestinal disorders (GI) and those with no GI illness. Building on others’ research showing that Clostridia bacteria in the gut are altered…

Integrating Research Into the “Journeys”
Integrating Research Into the “Journeys” 1024 683 Jeb Phillips

Along with their work to build an innovative follow-up program, Nationwide Children’s faculty and staff members are international leaders in NICU follow-up research. A number of foundation and National Institutes of Health-funded follow-up studies are housed entirely or in part at the hospital, and The Research Institute at Nationwide Children’s is one of 17 member…

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

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…

A Narrow Focus: Perfecting Tissue Engineered Vascular Grafts
A Narrow Focus: Perfecting Tissue Engineered Vascular Grafts 1024 575 Abbie Miller
image of heart with fontan conduit highlighted

A pair of surgeon-researchers is perfecting tissue engineered vascular grafts through bench, clinical and computational modeling studies.

Two Genes Linked to Postpartum Immunity Revival in Women With Persistent Hepatitis C
Two Genes Linked to Postpartum Immunity Revival in Women With Persistent Hepatitis C 150 150 Kevin Mayhood

Research may provide a model for identifying immune factors needed to control chronic infections. Alternative forms of two genes are associated with a boost in immunity to hepatitis C after childbirth, a study led by a Nationwide Children’s Hospital physician-researcher shows. At three months postpartum, the number of viruses circulating in the blood declined sharply…

Best Practices for Research Recruitment and Retention
Best Practices for Research Recruitment and Retention 1024 575 Tiasha Letostak, PhD

You can’t obtain study data without participants. From initial design and promotion to communication tactics and patient satisfaction, here are some strategies to ensure success. Advancing pediatric research depends on successful recruitment and retention of study participants. Unfortunately, 9 out of 10 trials end up having to double their original timelines in order to meet…

Childhood Kidney Stones: Their Surprising Connection to Future Disease
Childhood Kidney Stones: Their Surprising Connection to Future Disease 150 150 Jeb Phillips

Once thought to be an adult condition, urinary stone disease is increasingly found in children – and may be related to the development of cardiovascular disease, chronic kidney disease and low bone density. By one well-regarded estimate, the risk of developing urinary stone disease in childhood doubled between 1997 and 2012. That’s worrying enough on…

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

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…

How Does Genomic Medicine Become a Reality for Children’s Health?
How Does Genomic Medicine Become a Reality for Children’s Health? 1024 575 Jan Arthur
conceptual art of DNA

Perhaps one of the most important initiatives to advance our understanding of pediatric disease is genomic analysis. Genomics encompasses all aspects of understanding the human genetic code, especially how genetic information contained in every human cell can be interpreted to prevent disease and customize treatments for nearly every type of illness. Genomic analysis allows us…

Achieving CLARITY
Achieving CLARITY 1024 575 Abbie Miller
conceptual art of DNA

Multidisciplinary team from Nationwide Children’s wins international genomics competition. Accurate, patient-centered, comprehensive. That’s how the judges and leaders of the CLARITY Undiagnosed Challenge described the work of a team from Nationwide Children’s Hospital. Those same words could be used to describe the highly motivated and diverse team who surprised themselves by winning the challenge. “When we entered the challenge, we knew we would…

Technology Expands Access to Translational Medicine
Technology Expands Access to Translational Medicine 150 150 Abbie Miller

When primary care physicians, specialists and all the lab work in their arsenal fail to provide a diagnosis for debilitating symptoms, patients earn the label of “undiagnosed.” These undiagnosed patients wait with unresolved symptoms for medical research to catch up with them. The Undiagnosed Diseases Program (UDP) at the National Institutes of Health sees 120-150 previously undefined…

Building the Modern-Day Vaccine
Building the Modern-Day Vaccine 150 150 Katie Brind'Amour, PhD, MS, CHES

Vaccine development used to be straightforward. Now, the challenges are many and the successes are few. What will it take to overcome the obstacles presented by both immunology and society? For 160 years, vaccine after vaccine succeeded at safely and effectively preventing its targeted illness using a set of standard strategies. Scientists knew they simply…