New Developments in Cystic Fibrosis
Cystic fibrosis is a disorder affecting over 30,000 patients in the US and >70,000 worldwide. Mutations in the CF gene lead to defects in the CFTR protein, which works as a regulator of salt and water movement throughout the body. CF patients develop problems with mucus in the lungs and abnormal food absorption that lead to lung infections, poor growth, and death in early adulthood. Although improvements have been made in CF patient care for several decades, all previous therapies have targeted downstream symptoms of CF rather than early steps in the disease cascade.
2012 has been very exciting for the CF community in that the first drug that targets the root cause of CF gained FDA approval (kalydeco or ivacaftor, Vertex Pharmaceuticals). This pill helps restore activity to one of the mutant proteins that causes CF (G551D, found in about 5% of CF patients). Adult and pediatric patients with CF caused by this mutation that were treated with kalydeco had >10% improvements in lung function, gained more weight than the untreated patients, and reduced their risk of pulmonary exacerbations by >50%.
A second drug, Lumacaftor, completed phase II clinical trials in CF patients with the most common CF causing mutation (F508del, found in nearly 90% of CF Patients). These studies were led by Dr. Clancy at CCHMC and demonstrated its capacity to restore function in patients carrying this mutation. Phase II studies are in progress, and Phase III studies are targeted for late 2012.
NIH Training Grant Awarded
Our division was one of only two pediatric pulmonary centers in the United States to receive the National Institute of Health Ruth L. Kirschstein National Research Service Award (NRSA) program this year. The program provides funding for research training opportunities for medical students in basic or clinical research in pediatric pulmonary and sleep medicine areas. This research program is active in the summer between the first and second years of medical school. Eight applicants in the inaugural class were selected from students applying from medical schools in the South, Midwest and East Coast. Students were matched with mentors covering a variety of pediatric respiratory research areas including asthma, cystic fibrosis, lung cancer and sleep apnea. The strengths of our research programs, combined with an enthusiastic faculty and a well-organized summer program, provide an excellent opportunity to attract physician trainees at critical stages in their careers and increase the likelihood they will want to further pursue research as physician-scientists.
Carolyn Kercsmar awarded grant from Schroth Family Charitable Trust
In December 2011, the PNC Bank Charitable Trust Committee awarded the Asthma Center a grant of $150,000 from the John A. Schroth Family Charitable Trust. The funding supports a project to develop multimedia discharge instructions for patients with asthma, helping to improve asthma control and decrease re-admissions.
Carolyn Kercsmar, MD, Director of the Asthma Center, will lead this innovative project, focused on helping families with low health literacy. The specific aims of the study are to create and examine the acceptability of multimedia discharge instructions for caregivers of hospitalized children with asthma, particularly those with poor health literacy levels. The project will also determine the short-term efficacy of providing personalized, multimedia discharge instructions to caregivers of children hospitalized for treatment of asthma exacerbation. The study will feature the child and parent as “stars” in their own educational video to enhance viewing and increase understanding of asthma management skills. Overall goals are to reduce the use of emergency department and inpatient services due to asthma, and to improve the percentage of patients with well controlled asthma in the Greater Cincinnati community. Cincinnati Children’s is known to be a leader in innovative programs and local children can benefit from our advances in asthma care. Knowledge gained from an effective care model created in Cincinnati can also spread to other programs nationwide.