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 new 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 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. 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.