NIH awards Arkansas Children’s Research Institute $2.9 million for cystic fibrosis

The National Institutes of Health (NIH) has 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 think that the way the body handles the drug may help understand how a patient will respond to the drug. They will also explore the possibility that genetic variation may cause altered activity, which results in the drug being less effective and having different side effects.

ACH is home to the only Cystic Fibrosis Center in the state of Arkansas reviewed and approved by the national Cystic Fibrosis Foundation.