ACRI Launches Groundbreaking Study to Tailor Cystic Fibrosis Care for Moms and Babies

LITTLE ROCK, ARKANSAS (Sept. 18, 2025) — The Arkansas Children’s Research Institute (ACRI) has received $3.2 million from the National Institutes of Health (NIH) to identify how pregnant moms and their babies can better and more safely benefit from a revolutionary therapy for cystic fibrosis (CF).

ACRI will be the coordinating center for the five-year study, serving as the central hub for data management and leadership among seven academic research institutions across the nation. The study is the second NIH-funded project based at ACRI to lay the foundation for addressing why up to a quarter of patients do not respond optimally to standard CF treatment. The study is 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.

A new type of drug known as Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators have greatly improved the health of people with CF and have made it much easier for them to get pregnant. However, doctors don't know much about how these drugs affect pregnant women and their babies.

Triple-combination therapy, known as TC, is now a primary approach to CF therapy using CFTR modulator drugs and has been a life-changing discovery, improving lung function and overall health for  nearly 90% of eligible people with CF. Some estimates suggest about half of children who begin this therapy early will have a substantially longer life span.

The newest ACRI study aims to find out how the body uses and breaks down these CFTR modulator drugs during and after pregnancy.

“This funding will provide more evidence for the next step in CF care: Moving from uniform dosing to precision therapeutics dosing so all eligible children and adults, including those pregnant/postpartum, can experience the therapy’s benefits while limiting adverse effects,” Guimbellot said.

Researchers have found that for a significant number of patients, the therapy’s full benefits are not realized due to factors like variable drug absorption, metabolism and genetic differences. These factors are unknown in pregnant women with CF and their newborns.

The way a person's body uses and breaks down these therapies can change during pregnancy. This means the amount of medicine in a pregnant woman’s body might be different from a non-pregnant woman’s body. Dr. Guimbellot’s team aims to clarify those nuances.

ACH is home to the only accredited pediatric Cystic Fibrosis Center in the state of Arkansas. 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.

UAMS operates an accredited Adult Cystic Fibrosis Program. UAMS’ and ACH’s programs are members of the Cystic Fibrosis Foundation’s Therapeutics Development Network, which is made up of 80 nationwide clinical research centers conducting a variety of trials to evaluate the safety and effectiveness of new therapies for cystic fibrosis.

This research is supported by the Eunice Kennedy Shriver National Institute of Child Health and Human Development of the National Institutes of Health under Award Number R01HD117940-01.

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 with 361 licensed beds, 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 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 (2024-2025): Cancer, Cardiology & Heart Surgery, Neonatal Care, Nephrology, Neurology & Neurosurgery, 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 an inpatient unit that will expand in 2026; 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. 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.

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 3,485 students, 915 medical residents and fellows, and seven dental residents. It is the state’s largest public employer with more than 11,000 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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