UPMC Video Rounds - Cystic Fibrosis

November 27, 2019


Daniel Weiner, MD, co-director, The Antonio J. and Janet Palumbo Cystic Fibrosis Center: 

Up until now, we've relied primarily on treating the effects of the disease with antibiotics and airway clearance treatments. Now, CF patients have the availability of new medications that include CFTR modulators. These modulators include both correctors, that increase the amount of protein that makes it to the cell surface, and CFTR potentiators, that increase the activity of the protein after it's made it to the cell surface.

Our hope is that these will eventually be available for all patients. The potentiators are currently utilized for patients that have mutations that result in protein that does get to the cell surface, and these be, may be milder patients. The correctors are treating more difficult to treat mutations that result in most of the protein being destroyed intracellularly.

So, at Children's Hospital of Pittsburgh, we have available infant lung function testing, that provides us the ability to do spirometry and measurement of lung volumes in infants with cystic fibrosis. We hope that this can guide how we utilize therapies best in these young children. We're also developing new testing called the multiple breath washout test for infants. And, these new tests will be very important for helping to target new therapies that are coming down the pike.

We pride ourselves on having a strong, multidisciplinary team that involves social workers, nutritionists, physical therapists, respiratory therapists, and nurse educators. We have a very strong working relationship with our adult team, which is the second oldest in the country. And, our adult team is physically integrated with the team at Children's.

I think in the next 15 or 20 years, the future seems to be more and more use of CFTR corrector and potentiator medications with the CFTR modulators. We are heavily involved in conducting clinical trials for these drugs. And, eventually, the hope is that we may have something that may be a one-time cure, and that may involve gene editing or gene therapy. 

Learn more about the Division of Pulmonary Medicine at UPMC Children's Hospital of Pittsburgh.