NIH Awards Arkansas Children’s Research Institute $2.9 Million to Explore How Critical Cystic Fibrosis Therapy Can Help More Patients

LITTLE ROCK, AR. (Oct. 15, 2024) – The National Institutes of Health (NIH) have awarded $2.9 million to Arkansas Children’s Research Institute (ACRI) to examine how a critical therapy for patients with cystic fibrosis (CF) might become even more effective at treating the genetic respiratory illness.

The five-year study will be led by Dr. Jennifer S. Guimbellot, chief of Pediatric Pulmonary and Sleep Medicine at Arkansas Children’s Hospital (ACH) and the University of Arkansas for Medical Sciences (UAMS), where she also serves as an associate professor of Pediatrics.

CF is a progressive disorder that severely damages the lungs, digestive system and other organs by affecting cells that produce mucus, sweat and digestive juices. For decades, CF therapies focused on reducing the problems the condition caused and made it easier to live with the symptoms. Improvements in screening and treatments helped people with CF live into their 40s and 50s.

A triple drug therapy approved by the FDA in 2019 revolutionized cystic fibrosis care by correcting the defective protein in many patients and helping them maintain the balance of salt and water in their affected organs. The triple combination therapy results in thinned mucus and reduces patients’ risk of infection and inflammation, often greatly improving quality of life and giving patients a chance to live decades longer than experts expected even five years ago.

“Triple combination therapy has changed the way we thought about life with cystic fibrosis,” Guimbellot said. “Patients are engaging in activities that may have been limited just a decade ago – running marathons, having long-term careers and families, and planning for a much longer lifespan.”

As the use of triple combination therapy has become widespread, pulmonologists have noted that a significant number of people with CF do not show the same robust response and others develop side effects that make continuing the therapy a challenge, such as drug-induced liver injury or neuropsychological side effects. Recent studies have shown the majority of patients will experience some kind of side effect, but the drug’s overall benefits drive people to continue using it.

Guimbellot and her research team feel that the way the body handles the drug may help us know how a patient will respond to the drug. They are also exploring the possibility that genetic variation may cause altered activity, which results in the drug being less effective and having different side effects.

The team will conduct an observational study of triple combination therapy in people with CF to determine if there is a precise dose that works best for each person. Guimbellot has assembled a unique team with decades of experience in clinical trials, pharmacology, genetics, statistics, pharmacogenetics, psychology and drug metabolism to run the study.

“Together, we will shift the paradigm of care for cystic fibrosis by improving the understanding of the optimal concentration and dosing for each patient’s lung function and tolerance of therapy,” Guimbellot said. “Our goal is to transition the standard of care to a truly personalized and patient-centered approach to modulator therapy.”

ACH is home to the only Cystic Fibrosis Center in the state of Arkansas reviewed and approved by the national Cystic Fibrosis Foundation. ACH’s Pediatric Pulmonary and Sleep Medicine program, recognized by U.S. News & World Report as one of the nation’s best pediatric pulmonary programs, also offers a state-of-the-art pediatric sleep center and specialized clinics for asthma, chronic lung disease of infancy (BPD) and pulmonary complications of neurological and neuromuscular disorders.

ABOUT ARKANSAS CHILDREN’S

Arkansas Children's is the only health care system in the state solely dedicated to caring for Arkansas' 850,000 children. The private, non-profit organization includes two pediatric hospitals, a pediatric research institute and USDA nutrition center, a philanthropic foundation, a nursery alliance, statewide clinics, and many education and outreach programs — all focused on fulfilling a promise to define and deliver unprecedented child health. Arkansas Children’s Hospital (ACH) is a 336-bed, Magnet-recognized facility in Little Rock operating the state’s only Level I pediatric trauma center; the state's only burn center; the state's only Level IV neonatal intensive care unit; the state's only pediatric intensive care unit; the state’s only pediatric surgery program with Level 1 verification from the American College of Surgeons (ACS); and the state's only nationally recognized pediatric transport program. Arkansas Children’s is nationally ranked by U.S. News & World Report in seven pediatric subspecialties (2023-2024): Cancer, Cardiology & Heart Surgery, Diabetes & Endocrinology, Nephrology, Neonatal Care, Orthopedics and Pulmonology & Lung Surgery. Arkansas Children’s Northwest (ACNW), the first and only pediatric hospital in the northwest Arkansas region, is a level IV pediatric trauma center. ACNW operates a 24-bed inpatient unit; a surgical unit with five operating rooms; outpatient clinics offering over 20 subspecialties; diagnostic services; imaging capabilities; occupational therapy services; and northwest Arkansas' only pediatric emergency department, equipped with 30 exam rooms. Generous philanthropic and volunteer engagement has sustained Arkansas Children's since it began as an orphanage in 1912, and today ensures the system can deliver on its promise of unprecedented child health. To learn more, visit archildrens.org.

ABOUT UAMS

UAMS is the state’s only health sciences university, with colleges of Medicine, Nursing, Pharmacy, Health Professions and Public Health; a graduate school; a hospital; a main campus in Little Rock; a Northwest Arkansas regional campus in Fayetteville; a statewide network of regional campuses; and eight institutes: the Winthrop P. Rockefeller Cancer Institute, Jackson T. Stephens Spine & Neurosciences Institute, Harvey & Bernice Jones Eye Institute, Psychiatric Research Institute, Donald W. Reynolds Institute on Aging, Translational Research Institute, Institute for Digital Health & Innovation and the Institute for Community Health Innovation. UAMS includes UAMS

Health, a statewide health system that encompasses all of UAMS’ clinical enterprise. UAMS is the only adult Level 1 trauma center in the state. UAMS has 3,275 students, 890 medical residents and fellows, and five dental residents. It is the state’s largest public employer with more than 12,000 employees, including 1,200 physicians who provide care to patients at UAMS, its regional campuses, Arkansas Children’s, the VA Medical Center and Baptist Health. Visit www.uams.edu or www.uamshealth.com. Find us on Facebook, X (formerly Twitter), YouTube or Instagram.

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