A mother helps her son use a nebulizer. (Stock photo)
In the last few decades, scientific collaboration has played a vital role in improving quality of life and extending life expectancy for patients with cystic fibrosis (CF). Now, the members of a new cooperative hope that sharing their research and expertise will lead to more progress. The Southeast Cystic Fibrosis Research Cooperative connects specialists from about two dozen high-volume centers across the Southeast region, including Children’s of Alabama. Members have been meeting for the last year with the goal of advancing a sea change in available CF treatments.
Launched by the Cystic Fibrosis Foundation, the research cooperative blends a social component with the work of effectively treating CF, for which breakthrough therapies targeting gene defects have transformed care in just the last several years. Members of the cooperative—who, in many cases, have attended the same medical conferences over the years—have held virtual meetings in recent months but hope to gather in person as more join the group.
“Our region is big, and our patients are spread out—all the way from eastern Texas to the tip of Florida,” Children’s of Alabama pulmonologist and sleep medicine specialist Christopher “Miles” Fowler, M.D., said. “Every big center is working on different research and has different strengths to offer, so I think we’re all bringing something different to the table.”
About 600 CF patients are receiving treatment at any given time from the combined adult and pediatric program at Children’s and the University of Alabama at Birmingham (UAB). “There are more than 30,000 patients with CF in the United States, and the Southeast represents a big percentage of those patients,” Fowler said.
Accordingly, each CF center in the Southeast is tackling diverse aspects of CF, from groundbreaking gene therapy studies to clinical trials assessing new infection-fighting protocols or ways to optimize nutrition or exercise. UAB will soon be enrolling patients for gene therapy trials, but other centers in the region will be analyzing both similar and different gene therapy endpoints.
“There are going to be different options: The drugs will be different, and the risks will be different,” Fowler explained. “There’s a need to disseminate information about the trials being offered and educate providers about the risks and benefits of the drugs we’re still learning about. That’s been part of our focus.”
Ultimately, CF patients here and elsewhere should greatly benefit from the research cooperative’s efforts, Fowler said. The collaboration makes it easy for patients to learn about trial options and for centers to refer patients to other studies.
“There’s so much going on with cystic fibrosis that I think it’s impossible for any one center to do all of this,” he said. “We all have different resources and expertise that allow us to do the studies we do, so one way the regional cooperative helps is by making more types of studies available to patients in our region.”