Pediatric Research: Asthma & Cystic Fibrosis

Nemours Children's researchers and clinicians in the Delaware Valley and Florida are working hard to develop new ways to help children breathe easier.

By focusing on asthma and cystic fibrosis (CF), our work has the potential to help improve the lives of millions of children who struggle to breathe.

Active Research

Our research centers explore ways to improve life for children with breathing problems.

Asthma and Dietary Supplements

Can dietary supplements help control asthma symptoms in children who are overweight? Nemours researchers are working to find answers.

We hope our work may help shed some light on several interrelated issues:

The link between obesity and asthma
The value of certain supplements as ways to treat asthma in children
The importance of a child’s genetic makeup in determining how well a particular asthma therapy or treatment will work

Genetics and Asthma

We know tobacco smoke makes it harder for anyone to breathe — especially children with asthma. But some children have a harder time than others. Likewise, we know some medications work better than others in children with asthma.

What accounts for these differences? Does genetics play a role? Could a child’s genes help doctors predict how sensitive they are to second-hand smoke? And could genes help determine what treatments might work best for each child?

Nemours researchers recently finished gathering data for two studies that focus on the effects of tobacco smoke in children with asthma. When the results are analyzed, we hope to have answers that can help kids everywhere breathe easier and better.

Treating CF Complications

For children with cystic fibrosis, one of the most common complications is a chronic lung infection that’s hard to control. Typically it is treated with an antibiotic, which many patients use every other month.

Researchers at Nemours facilities in Delaware and Florida are investigating whether there is a benefit to switching back and forth each month between two different antibiotics. This regimen may keep the infection “in check” and improve lung function in those with chronic lung infections.

Treating an Underlying Cause of CF

CF is caused by the dysfunction or complete lack of function of a protein called CFTR. The faulty protein results from an inherited abnormality in a particular gene. What if the faulty version of the protein could be corrected or activated (repaired) before any damage to the body can occur?

Nemours researchers in Delaware and Florida are participating in studies that test this idea. They’re investigating whether new therapies might have potential to allow this faulty protein to function more normally — so CF symptoms won’t have a chance to develop.

Noninvasive Respiratory Support of the Neonate/Continuous Quality Improvement

When newborns need help to breathe, what are the best ways to administer assistance? Researchers at Nemours Children’s Hospital are performing clinical studies to investigate and optimize neonatal respiratory care, in particular noninvasive respiratory support including nasal continuous positive airway pressure (CPAP) and humidified high-flow nasal cannula therapy.

They are involved in ongoing trials related to noninvasive respiratory support, including the neurodevelopmental follow-up study of those infants enrolled in the Vermont Oxford Network Delivery Room Trial, one of the largest multicenter randomized controlled trials to date on CPAP in neonates.

Improving the Effectiveness of Inhaled Medications for Cystic Fibrosis and Asthma

For children with lung diseases such as CF and asthma, taking medications by inhaling them can improve how well the drugs work and reduce side effects. Many new drug therapies are being developed with this in mind, including inhaled antibiotics, airway wetting and clearance agents, long-acting bronchodilators, antioxidants, gene-modifying agents, and gene replacement therapy.

With so many treatments requiring inhalation, the time-burden on the patient is so great that nonadherence with therapy becomes a real problem. The Nemours Aerosol Research Lab in Orlando, founded in 1999, has been exploring efficient new technologies to shorten treatment times, and has participated in clinical research projects designed to improve treatment adherence and study new therapies. Nemours Children’s Hospital, Florida is in a unique position to advance this work. Researchers can test various drugs and delivery devices in the lab and have the clinical population and experience to carry that research forward into phase 1 through phase 3 testing.

The Lab opens up new avenues for patients to experience cutting-edge therapies and gives them hope for a better tomorrow. It also has allowed Nemours to participate on national and international levels to affect the care of children with CF and respiratory diseases.

CF Foundation Patient Registry

Nemours Children’s Clinic locations in the Delaware Valley and Florida participate in the CF Foundation Patient Registry. The registry collects data anonymously on individuals with CF to better understand the illness and ultimately improve the care and survival of patients with CF. The Patient Registry was established in 1966 as a means to monitor important trends in CF and accomplish these goals.

How it’s used: The CF Patient Registry is used by both clinicians and researchers to better understand this disease and to improve care of individuals with CF. Nemours care team members use the registry data in a variety of ways. For instance, they can search the registry to determine if a patient is due for routine testing. They can also look at the data to see who might be eligible for a new study.

Annual report: On the national level, the data in the Patient Registry is analyzed to create an annual report of CF health trends. The annual report is usually available about 12 months after the close of the calendar year.

Learn more about the CF Patient Registry

Our Centers

Center for Pharmacogenomics and Translational Research (CPTR)

At the Center for Pharmacogenomics and Translational Research (CPTR), our multidisciplinary team focuses on asthma and other diseases, with an eye toward tailoring treatments to fit each child’s individual needs.

Pharmacogenomics is the study of how genetic variations influence a person’s response to medication — why some drugs work better for some children than for others, and what part genes may play in a medication’s effectiveness. Translational research means taking what we learn in research and using that information to improve patient care — translating findings from the lab to the bedside.

The CPTR brings Nemours doctors, nurses, researchers and respiratory therapists together in a team approach to caring for children. Combining our research efforts with patient care allows Nemours doctors to achieve the best possible outcome for each child.

Our work is divided into four active research sections:

The American Lung Association - Airways Clinical Research Centers (ALA-ACRC)

The primary mission of this nationwide 18-center network is to conduct large clinical trials (in diverse populations of people with asthma) that will have a direct impact on patient care and asthma treatment.

Since 1999, Nemours and the University of Florida have partnered in an ACRC research consortium that has been supported by more than $3 million in research support funding from the American Lung Association, enabling our team to make significant research contributions that advance patient care. Nemours Children's Health, Jacksonville, is one of only two facilities in Florida belonging to the prestigious ACRC network.

Learn More About the ALA-ACRC

Center for Pharmacogenetics

Researchers at the Center for Pharmacogenetics study the relationship between individual genetics and response to treatment. By examining this relationship, Nemours doctors are better able to determine if a child is more likely to be at risk for developing asthma, based on his or her individual genetic code, and which treatment plans might work best.

Pharmacogenetics is a relatively new discipline, and much of our current research efforts focus on asthma treatment, as prior studies have shown that today’s asthma drugs are effective in only about 50% of patients. This difference in response to asthma drugs is related to genetic variation. Knowledge of the genes that are responsible for the inadequate drug response will enable us to predict which drugs work best to treat asthma, ultimately creating personalized therapies. A personalized medical approach may be the future of medicine and will likely improve treatment for children and reduce medication side effects.

Core Clinical Research Program

This important program supports Nemours scientists in diverse research areas, including studies relating to asthma and CF. CPTR research coordinators and staff are available for scientific consultation and support to Nemours clinical scientists interested in conducting research.

For consultations, call (904) 697-3925.

Nemours Network for Asthma Research

This targeted program involves research into the diagnosis and treatment of asthma in non-ACRC research projects. The research areas of our program include Comparative Effectiveness and Outcomes Research, Sickle Cell Disease-Related Asthma and Lung Disease, Obesity-Related Asthma and Lung Disease, and the Impact of Nutrition on Asthma.

Nemours scientists currently have a wide range of externally-funded and multiyear grants (including NIH/NHLBI, Thrasher Research Fund, James and Esther King Biomedical Research Program) to study these important areas in asthma care.

The Nemours Noninvasive Pulmonary Evaluation Lab (NIPEL) provides specialized respiratory testing that’s useful in the diagnosis of respiratory disease. The Center frequently collaborates with the departments of Pulmonology, Orthopedics, Pediatric Critical Care, Bone Marrow Transplant, Cardiology and Neonatalogy.

Under the direction of Thomas H. Shaffer, MSE, PhD, the team’s revolutionary work with the use of perfluorochemicals for liquid ventilation for respiratory distress syndrome, blood substitutes and imaging is known worldwide, as is their work in developmental airway physiology and clinical pulmonary function evaluation in neonates.

Additionally, NIPEL has introduced a new pulse oscillometry machine as a noninvasive way of performing pulmonary function testing on small children.

The Center for Pediatric Lung Research (CPLR)

The Center for Pediatric Lung Research uses progressive diagnostic and therapeutic procedures and techniques to help ease the work of breathing for infants and children challenged by acute and chronic lung disease. The Center takes a proactive role in developing and fostering opportunities for research in neonatal and pediatric lung disease. Two labs support the CPLR’s work:

Nemours Noninvasive Pulmonary Evaluation Lab (NIPEL)

The Nemours Noninvasive Pulmonary Evaluation Lab (NIPEL), provides specialized respiratory testing that’s useful in the diagnosis of respiratory disease. The Center frequently collaborates with the departments of Pulmonology, Orthopedics, Pediatric Critical Care, Bone Marrow Transplant, Cardiology and Neonatalogy. Under the direction of Thomas H. Shaffer, MSE, PhD, the team’s revolutionary work with the use of perfluorochemicals for liquid ventilation for respiratory distress syndrome, blood substitutes and imaging is known worldwide, as is their work in developmental airway physiology and clinical pulmonary function evaluation in neonates. Additionally, NIPEL has introduced a new pulse oscillometry machine as a noninvasive way of performing pulmonary function testing on small children.

Nemours Aerosol Research Lab

Many children with respiratory problems like asthma and CF inhale their medications in aerosol form — fine particles of medicine suspended in air or some other gas. Sometimes the nebulizers and inhalers they use to inhale these medications don’t work properly — because the medication particles are too large to get into the child’s lungs.

The Nemours Aerosol Research Lab has studied the delivery of inhaled therapies, focusing on getting more of the medicines where they’ll do the most good: in the lungs. By determining the right amount and size of particles that are needed, and finding ways to get the particles from their container into the lungs, the lab has helped children derive the most benefit from every dose of medication.