Research propels scientific discovery, new therapies for patients, new drugs and vaccines, and improved care. Simply put, research saves lives.
At OHSU Doernbecher Children’s Hospital, scientists work to translate discoveries made in the laboratory into new treatments for patients. And their patients are pint-sized. Or traversing adolescence. Or born prematurely.
Kids aren’t miniature adults. Their bodies, systems and brains are still developing and growing. Research conducted on adults can’t just shrink to fit their developing bodies and minds.
And because kids have their entire lives ahead of them, it’s especially important to minimize long-term side effects of any new therapy or treatment.
We asked three doctors at OHSU to share why pediatric research matters — to their patients and themselves.
Carl Eriksson, MD, MPH, is an associate professor of pediatrics in the division of critical care in the OHSU School of Medicine. He specializes in the care of critically ill infants and children.
Michael Powers, MD, is a professor of pediatrics in the pulmonology division at the OHSU School of Medicine. Powers is the Credit Unions for Kids Professor in Pediatrics, and he specializes in cystic fibrosis.
Cindy McEvoy, MD, MCR, is professor of pediatrics, obstetrics and gynecology and Credit Unions for Kids Endowed Professor in Pediatric Research. McEvoy is also the director of maternal child health research in the Department of Pediatrics and director of research in Neonatology. She specializes in infant lung health.
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Why is pediatric research important?
CE: “Children are different from adults in many ways. Many of the children we care for at Doernbecher Children’s Hospital have unique diseases that we rarely see in adults.
“Much adult-focused research (like studies focused on lung cancer or coronary artery disease) is not applicable to most children. Even when adults and children have the same disease, the body’s response to the disease or to treatment can be very different.
“Some of the medications used to treat adults simply aren’t safe in younger children, or may have significant side effects for developing bodies that we need to understand.”
MP: “Pediatric research is unique because children are developing and growing beings. New drug treatments must be carefully studied for safety in this vulnerable population. New therapies must be evaluated in the context of child’s unique metabolism as compared to adults.
“A fundamental concept in pediatric research is that we are improving or saving a life for a lifetime. We want our children to become fully functioning and productive adults, free from a premature death or lifelong health challenges.”
How is research vital to your work?
CE: “In the Pediatric Intensive Care Unit, we care for the sickest children, and our treatment choices can make the difference between life and death. Thanks to research and improved care, we take care of children today who would not have survived 20 years ago. Today, they walk out of the hospital after being incredibly sick.
“We are constantly learning and advancing in our understanding of how to treat critical illness in children, and research is key to that.”
MP: “My work is with cystic fibrosis (CF). During my career, I have seen basic and clinical research improve the median survival of CF from 24 years to 48 years by bringing a new understanding of the disease mechanisms, and thereby providing a foundation for new treatment approaches.
“Drugs are investigated in the labs, advanced through three phases of clinical trials, and then brought to the patient through a nationwide CF clinical network of care centers, including Doernbecher.
“Over the past 30 years, research advances have included medications to thin mucus in the lungs of CF patients, and specific antibiotics to fight infection in CF patients.
“Over the last 8 years, however, there have been breakthrough treatments that treat the underlying defect in CF. These novel therapies called modulators are small molecules that improve the function of defective chloride channel in CF patients — the underlying cause of thick mucus in the lungs and pancreas.
“Currently approximately 60 percent of patients are benefiting from these treatments, and we hope that over 90 percent of patients will benefit within the next year, with approval of newer and more effective modulators.
“Doenbecher’s CF Center has participated in a multitude of these phase 2 and 3 trials over the past 6 years, bringing novel treatments to our patients, and contributing to the approval of these lifesaving medications for all CF patients.”
CM: “The old adage is that children cannot be managed as young adults. But it is being increasingly realized that to understand, treat and potentially prevent disease in the adult, you need to know the child and the fetus and the womb that the fetus developed in – and even preconception conditions.
“This approach to health is exciting because it emphasizes the importance of perinatal and pediatric research. This area of research supports the idea that health trajectories are set very early. So through perinatal research and interventions, we have the potential to impact the individual for a lifetime and help them achieve lifelong health.”
What are some noteworthy studies or breakthroughs in pediatric research at OHSU Doernbecher?
CE: “There is amazing work happening in many areas at Doernbecher. As an example, Dr. Cydni Williams, one of my colleagues in pediatric critical care, is doing groundbreaking work to better understand outcomes for children with critical illness affecting the brain (like traumatic injuries, infections, and stroke).
“This is leading to some very important discoveries about how children’s brains recover from critical illness, and it’s helping us provide better treatment for patients and guidance for families in the intensive care unit. Dr. Williams’ program is also helping to identify areas where our patients are thriving after critical illness and areas where they need different treatment or more support.”
CM: “My team is working on two areas to improve lung health in infants, using therapies that are affordable and widely accessible but that can make an enormous impact.
First, we found that vitamin C supplementation to pregnant smokers who are unable to quit improved the infant’s pulmonary function and decreased wheeze through 12 months of age. This could improve the respiratory health of millions of children worldwide and is especially timely with the increase in e-cigarette use, which would have the same effect on the fetus if used during pregnancy.
“Second, we are working on ways to improve respiratory health in infants who are born preterm. Ten to 12 percent of all deliveries are born preterm, and these infants have abnormal lung development and lifelong respiratory problems. No current therapies exist to stimulate lung growth, improve lung function, and thus decrease respiratory morbidity after preterm birth.
“Continuous positive airway pressure (CPAP) is commonly used in preterm infants after delivery to help support their breathing. We published results of a trial in the Journal of Pediatrics showing that extending the duration of CPAP by two weeks in stable preterm infants stimulated their lung growth. This is very exciting since CPAP is a common therapy already used for preterm infants and may be a safe and non-pharmacologic strategy to improve the ultimate respiratory health of preterm infants.”
MP: “An evolving field of research is health disparities research, trying to understand difference in access and approach to care, based on ethnicity or socioeconomic status. Social determinates of health have been shown to have the biggest impact upon a child’s health. Dr. Katie Zuckerman at Doernbecher is researching the health disparities for Hispanic populations, in regards to diagnosis and access to care for childhood autism. Many lives can be improved in this area with Doernbecher on the forefront of this research.”
Doernbecher’s research mission sets it apart from other hospitals.
As part of Oregon’s only academic health center, Doernbecher is the only place in the state where breakthrough pediatric research quickly translates into new treatments and access to life-changing care, giving families new hope. Your gift funds this essential pediatric research.
