The Riley Hospital for Children Cystic Fibrosis Center is the first site in the nation to enroll patients ages 6-11 for the first part of a 48-week clinical trial of a promising investigational treatment for cystic fibrosis (CF).
Cystic Fibrosis is an inherited chronic disease that affects the lungs and digestive system of about 30,000 children and adults in the United States and 70,000 people worldwide. CF is caused by a defect in the CFTR (cystic fibrosis transmembrane conductance regulator) gene which makes a protein that controls the movement of salt and water in and out of a body’s cells. The defect in this gene causes thick mucus and very salty sweat. The predicted median age of survival for a person with CF is more than 37 years.
The Riley Hospital Cystic Fibrosis Center is enrolling children ages 6-11 who carry a G551D mutation of CF, of which 3-4% of the CF population has the mutation. Participants will receive a pill twice daily of either a placebo or a drug called VX-770. This clinical trial will enroll approximately 30 participants worldwide including 3-5 at Riley Hospital.
VX-770 represents one possible strategy to treat CF by increasing the activity of defective CFTR proteins at the cell surface. VX-770 was discovered by Vertex Pharmaceuticals as part of a collaboration with the Cystic Fibrosis Foundation.
“We are excited to take the lead in this very important step toward a cure for CF patients and families. The credit must be shared with these brave kids and their tireless families,” said Michelle Howenstine, MD, director of the Riley Cystic Fibrosis Center, and professor of Clinical Pediatrics, Indiana University School of Medicine.