When we talk about the first man to walk on the moon, we immediately think of Neil Armstrong. But he was just one person among thousands who made that first step possible. In this year's second plenary the speakers talked about the CF community's efforts to “reach the moon.” And, like the Apollo 11 mission, it will not be one or even a few individuals but a collective group of thousands of dedicated people who enable us to bring transformative CF therapies forward and one day have a cure for every person with CF.
The importance of everyone working together toward a common purpose, and the role clinical trials will play in achieving this goal, was underscored today throughout the plenary, entitled Clinical Research: A Worldwide CF Community Effort.
The first speaker, Dr. Patrick Flume, professor of medicine and pediatrics at the Medical University of South Carolina, encouraged us to shoot for the moon -- a goal that seems far away but, with new scientific advances, is getting closer than ever. How close was highlighted by his descriptions of the promising, novel therapies currently in the pipeline, including ones that treat the underlying cause of CF.
Whereas the currently approved CFTR modulators benefit over 50 percent of the CF population, new modulators about to start clinical trials have potential not only to benefit more people than ever before, but also to be significantly more effective. It is the increased effectiveness of these drugs that may enable them to treat people with CF who have a single F508del mutation, which would dramatically increase the number of people with CF who could benefit from modulators. Some 86% of people with CF have either one or two F508del mutations.
Dr. Flume also profiled new potential therapies currently in clinical trials that could be used to complement CFTR modulators. Because of the continued importance of developing these new symptomatic treatments, researchers are conducting more clinical trials than ever before and are exploring the effectiveness of potential drugs for everything from anti-inflammatories and antibiotics to medicines that very efficiently break down mucus.
However, as exciting as it to have the most clinical trials for CF underway ever, enrollment is a serious challenge, Dr. Flume said. With only 33,000 people with CF in the United States, it is difficult to recruit enough people who meet all the eligibility criteria for a trial.
Discussing the importance of patients, Dr. Flume said, “We owe a tremendous gratitude to all of those persons who have participated in clinical trials.”
In an effort to expand the pool of people who can enroll in trials and accelerate drug development, the CF Foundation is also focusing on helping the performance of clinical trials in the United Kingdom, Europe and Australia. These countries have diverse concentrations of people with specific CF mutations, making it easier to find people who are eligible for certain trials, said Kris De Boeck, M.D., Ph.D., director of the Pediatric Pulmonary and Infectious Diseases program at the University of Leuven in Belgium and the second speaker at today's plenary. The European Cystic Fibrosis Society region alone represents people with 1,300 different CF mutations.
“We need global participation to advance as fast as possible,” Dr. De Boeck told the audience.
Worldwide collaboration by the entire CF community is essential to the effort to develop better treatments faster, she said. Currently, there are clinical trials at 43 sites in 15 countries within the European Cystic Fibrosis Society's clinical trials network. De Boeck said the Society plans to use funding from the CF Foundation to recruit more research coordinators and boost enrollment in clinical trials. This year, the CF Foundation also helped fund the Cystic Fibrosis Trust's Clinical Trial Accelerator Platform, which will help research sites hire staff and increase enrollment in clinical trials in the United Kingdom.
Finally, in the high point of the session, the audience also heard a powerful panel discussion moderated by Patricia Burks, M.A., R.N., C.C.R.C., Director of Clinical Trial Affairs at the CF Foundation, about the importance of clinical trials from members of the CF community via live video link. The speakers included Kristin Dunn and Brandon Erhart, two people with CF who have participated in clinical trials, and Elizabeth Bishop, the mother of 11-year-old twins with CF. The speakers particularly focused on why they think it is important that everyone with CF has the opportunity to participate in clinical trials.
Elizabeth, whose twins have participated in multiple clinical trials, explained that when two of her children were diagnosed with CF she initially felt helpless -- how do you fight something you cannot see? She then explained how clinical trials give their family the chance to do just that, an opportunity to do something about their disease, an opportunity to have some power.
Explaining how her twins feel about clinical trials, Elizabeth said, “Participating in the trials has given them some power back. My twins don't want to see another child go through what they have gone through. It gives them a way to do something about their illness and empower them.”
The panel also emphasized the impact that clinical trials have on the lives of the CF community by bringing hope. They empower CF community members to make decisions and, as Brandon explained, to go ahead and invest in the future. Kristin, who traveled the world as a financial analyst before having to leave her job because of her health, explained how clinical trials give her the hope that she will one day return to the workforce.
Talking about how they now have to plan for adulthood, Brandon said, “There is now this idea of being an adult. Who thought having a mortgage could be so exciting!”
Today's speakers captured perfectly the critical importance of everyone coming together to focus on our common goal. I certainly came away inspired! It will only be through the entire CF community's collective efforts that we will one day reach the moon and conquer this disease -- and the moon is getting closer every day!
To find out more about clinical trials near you as well as eligibility, please visit the CF Foundation's Clinical Trial Finder.