A panel of experts will discuss and debate whether clinical trials should reconsider standard practice and begin grouping bronchiectasis patients with and without cystic fibrosis.

The session, “Chair’s Symposium: Cystic Fibrosis and Non-CF Bronchiectasis — Time for the Twain to Meet in Clinical Trials?” takes place from 9:15 to 10:45 a.m. PT, Tuesday, May 20, in the Moscone Center, Room 24, Hall E (North Building, Exhibition Level).

Raksha Jain, MD, MSci, ATSF, session co-chair, describes the symposium as a chance for attendees to learn more about new research and theories in areas of pulmonary medicine that have been understudied and unrepresented at educational conferences.

“Bronchiectasis is underdiagnosed, and new therapies are on the horizon to treat people who suffer from cystic fibrosis (CF) and non-cystic fibrosis bronchiectasis (NCFB),” said Dr. Jain, a professor in the Department of Internal Medicine at the UT Southwestern Medical Center and the medical director of the Adult Cystic Fibrosis Program. “It is important for the pulmonary community to be aware of this area of research and potential breakthrough therapies so that they can take care of their patients with the highest standard of care.”

Studies on cystic fibrosis and bronchiectasis have traditionally been separated, but Dr. Jain noted that new cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies have blurred this distinction. These therapies, which treat the underlying genetic basis in the disease process for patients with CF, have affected inflammation so that it more closely resembles the inflammation found in non-CF bronchiectasis. These therapies have also mitigated some of the extra-pulmonary nutritional and absorption concerns that historically define CF, thus bringing the condition closer to non-CF bronchiectasis.

Furthermore, Dr. Jain noted, “non-CF bronchiectasis is already so heterogeneous, and some forms of NCFB share similar pathogens and inflammatory pathways to those observed in CF-related bronchiectasis.”

However, the proposal to merge the two groups for studies would go against current standard practice.

“The question we are coming back to is: Are we at a point where we should be trying to use a small number of people with CF who still have these signs and symptoms, such that they need inhaled antibiotics and anti-inflammatories, and combine them with the non-CF bronchiectasis groups and try to advance therapies for both groups at the same time?” said Jennifer L. Taylor-Cousar, MD, MSCS, ATSF, an adult and pediatric pulmonologist at National Jewish Health, and session co-chair.

Dr. Taylor-Cousar also noted that a small trial with dornase alfa (recombinant human DNase) led to the conclusion that results from CF-related bronchiectasis patient groups do not necessarily translate to results for patients with non-CF bronchiectasis, and in some cases, therapies might provide results in one group, but worsen the condition for another.

“There is still much to be learned as to whether and how CF and NCFB studies can be combined,” said Dr. Jain. “Among clinicians, researchers, and regulators across the globe, some would argue ‘Yes,’ while others would disagree. That is why this session will be so interesting as an opportunity to hear from experts around the world.”

Both cystic fibrosis and bronchiectasis are classified as orphan diseases, diseases that affect fewer than 200,000 people in the United States. However, the debate around their clinical trials has potentially significant impacts for patients with these diseases and has relevance for other fields, noted Dr. Jain.

“This is a rapidly evolving field with new therapies on the horizon that have the potential to be groundbreaking,” Dr. Jain said. “While there have been historical clinical trials studying therapies, such as dornase alpha, that had differing results for people with CF versus NCFB, this is not necessarily the case for all therapies. It is important for the community to understand why.”

For a full schedule of conference events, visit ats2025.d365.events. To see more conference-related news and coverage of sessions and events, visit atsconferencenews.org.