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MUSC Center for Cystic Fibrosis

Like David facing down the much-larger Goliath, the MUSC Center for Cystic Fibrosis (CF), which includes both adult (Director: Patrick Flume, M.D.) and pediatric (Director: Isabel Virella Lowell, M.D.) programs, packs a lot of punch for its size. It was awarded the 2011-2012 Quality Care Award at the North American CF Conference for its ongoing dedication to developing and implementing quality improvement projects that use clinical outcomes data to improve patient care. It was chosen as one of 74 therapeutics development centers by the CF Therapeutics Development Network (TDN), the largest CF clinical trials network in the world, because of its demonstrated expertise in clinical research. Of those 74 centers, it is ranked 58th in size but enrolls the second most patients into clinical trials and has been ranked number one for overall performance for the past few years. It was instrumental in collecting the data that led to the approval in January 2012 of ivacaftor (Kalydeco,™ Vertex Pharmaceuticals), the first drug to target one of the underlying causes of cystic fibrosis—reduced airway surface liquid resulting from a defective gating protein. It is also participating in phase 2 and 3 trials to test the efficacy of ivacaftor, in combination with other therapies, in CF patients with other genetic mutations.

With such support for clinical research, CF patients, many of whom were once fated to die in their twenties, now live to a median of 40 years. Because the CF Center at MUSC treats both pediatric and adult patients, it can help ensure continuity of care for these patients. The Center has also recently received a grant to develop a telehealth patient education program. Instead of being overwhelmed with too much information during a clinic visit, CF patients can meet with a respiratory therapist regularly via teleconference from the comfort of their own home, providing them more time to perfect their self-care (airway clearance) skills.