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

Orphan Disease Seeks Parents, Funding
 Orphan Disease Seeks Parents, Funding 1024 575 Kevin Mayhood
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Color photo, family portrait of Reagan, a young girl with an orphan disease, her parents, and their dog

Research on rare pediatric diseases often remains underfunded and obscure until motivated families give scientists — and their own children — a much-needed shot at potential therapies worthy of federal funding. Reagan McGee’s pediatrician couldn’t figure out why she had cold after cold. Her parents, Karin and Peter McGee, took her to an ear, nose and…

Fighting Fibrosis
 Fighting Fibrosis 1024 575 Katie Brind'Amour, PhD, MS, CHES
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Colorful illustration showing how fibrosis, or a spiky lumpy mass of scar tissue, forms over healthy liver tissue when the liver attempts to repair and replace damaged cells

Fibrosis is an unmet medical challenge with no satisfactory test and insufficient therapy. Now, one naturally occurring cellular component could simultaneously diagnose and heal patients. It’s much more than a million-dollar idea. The person who invents a simple blood or urine test that can accurately measure the severity of scarring in internal organs will have…

Preserving Biopreservation
 Preserving Biopreservation 1024 575 Katie Brind'Amour, PhD, MS, CHES
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Funding challenges, operational complexity and poor visibility threaten the field of human tissue biobanking. How sustainable are biorepositories? The Children’s Oncology Group (COG) boasts the collaboration of more than 9,000 pediatric cancer experts. They treat patients and research disease at more than 200 hospitals around the world. Nine out of every 10 U.S. children with cancer receive…

InSight: A Window to the Heart
 InSight: A Window to the Heart 720 530 Katie Brind'Amour, PhD, MS, CHES
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Cardiac MRI for Hypoplastic Left Heart Syndrome Hypoplastic left heart syndrome (HLHS) is a potentially fatal congenital heart defect affecting blood flow. It requires multiple palliative surgeries, starting within the first week of life. Nationwide Children’s employs a hybrid surgical approach. Hybrid Stage I (not shown) Bilateral branch pulmonary bands are placed to restrict blood flow.…

Beyond the Basics: Enrolling Children in Research
 Beyond the Basics: Enrolling Children in Research 150 150 Katie Brind'Amour, PhD, MS, CHES
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The ethics of pediatric research include far more than the concepts of autonomy and assent. Consent and assent. Competence and autonomy. Physicians are familiar with the catchphrases of ethical research, but the deeper researchers dig, the more they find that the field’s current understanding of the dimensions involved in pediatric investigations is still shallow. Numerous…

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

InSight: Working Up the Nerve
 InSight: Working Up the Nerve 150 150 Katie Brind'Amour, PhD, MS, CHES
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Using regional anesthesia to numb nerves reduces pain and speeds recovery in pediatric orthopedic surgery. Ultrasound-guided regional anesthesia has been used in adult patients for more than a decade but is now being used more regularly in pediatric patients, especially for orthopedic procedures. Femoral nerve block, in which the femoral nerve is numbed, is among…

Body, Heal Thyself: Harnessing Our Innate Immunity
 Body, Heal Thyself: Harnessing Our Innate Immunity 150 150 Katie Brind'Amour, PhD, MS, CHES
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What the urinary tract’s front-line defenses can teach us about our innate ability to self-heal …. and thwart antibiotic resistance. Antibiotic resistance is on the rise, health care-acquired infections are becoming harder to treat and even simple infectious illnesses account for billions of dollars per year in spending in the United States alone. As with…

Enteral Therapy on Trial
 Enteral Therapy on Trial 150 150 Katie Brind'Amour, PhD, MS, CHES
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Micah Cohen sat down at the dining room table in his family’s Columbus, Ohio, home and took the first sip of a new therapy he hoped would relieve the symptoms of his Crohn’s disease. The thick, sweet chocolate shake, rich with nutrients, felt heavy in the 14-year-old’s stomach. Can I really drink six of these a…

Thinking Outside the (Tool) Box: How Techniques From Alzheimer’s Research Illuminate the Pathophysiology of Preeclampsia
 Thinking Outside the (Tool) Box: How Techniques From Alzheimer’s Research Illuminate the Pathophysiology of Preeclampsia 150 150 Katie Brind'Amour, PhD, MS, CHES
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Irina Buhimschi, MD, has a habit of getting lost. In July 2007, that poor sense of direction proved to be a fortunate flaw. Having wandered into the wrong presentation at the Protein Society’s national conference, she hovered in the back to get her bearings. Up front, a speaker discussed how misfolded proteins accumulate in the…

Adult Congenital Heart Disease: Let’s Finish What We Started
 Adult Congenital Heart Disease: Let’s Finish What We Started 150 150 Curt Daniels, MD
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Congenital heart disease is the most common birth defect, diagnosed in nearly 1 percent of all births in the United States. Traditionally, life expectancy in many infants with severe CHD was limited to months. However, advances in medical and surgical care have led to remarkable improvements in survival: the median age of those living with severe…

Mentoring Junior Faculty: Fostering Tomorrow’s Leaders
 Mentoring Junior Faculty: Fostering Tomorrow’s Leaders 150 150 Katie Brind'Amour, PhD, MS, CHES
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Clinicians from institutions nationwide offer insight on how to piece together effective mentorship strategies in pediatrics. Mentoring may be a familiar concept to most pediatric professionals, but whether it occurs effectively in practice is less obvious. Research on the topic suggests a widespread lack of formal mentoring programs at academic medical institutions. Other studies point…

Gene Therapy’s Road to Redemption
 Gene Therapy’s Road to Redemption 150 150 Kelli Whitlock Burton
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Fifteen years ago, gene therapy suffered a highly visible fatality, leaving the field in shambles. Now, one team’s efforts at gene therapy for muscular dystrophy suggest the field may finally be on track to deliver on its initial promise. During the first few weeks of September in 1999, a 36-year-old air traffic controller from South…

Aiming for Zero
 Aiming for Zero 150 150 Kelli Whitlock Burton
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Efforts to eliminate preventable harm in pediatric care are making progress. But can we make it to zero? In October 2008, Richard Brilli, MD, stood in a silent conference room, waiting for his audience to digest the news he’d just delivered: hundreds of significant harm events are identified each year at Nationwide Children’s Hospital, and…

Evolution of an Atlas
 Evolution of an Atlas 150 150 Katie Brind'Amour, PhD, MS, CHES
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Adult brain atlases have existed for years. Why is it so crucial — and so difficult — to build one for preemies? Even experts need maps. They give perspective, scale and orientation. They can show both current location and the final destination. And in the world of premature brains, they can offer vital information about…

A Brave New World of Data
 A Brave New World of Data 150 150 Katie Brind'Amour, PhD, MS, CHES
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According to William C. Ray, PhD, of the Battelle Center for Mathematical Medicine in The Research Institute at Nationwide Children’s Hospital, novel techniques prototyped for understanding protein data could soon enable the visualization of large sets of demographic data, displaying contingency tables and group relationships through visual representations of positive and negative correlation, covariance and…

Printed Motion: Holding Molecular Movement
 Printed Motion: Holding Molecular Movement 150 150 Katie Brind'Amour, PhD, MS, CHES
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When student researcher Shareef Dabdoub discovered the appealing aesthetics of a transcriptional regulator from the Streptococcus family, he knew that cellular biology had handed him a work of art. And by using a computer program to predict the 3-D configuration of the molecule and its most probable pathway for rearranging from one state to another, he observed…