Improved cystic fibrosis treatment is Science Breakthrough of the Year

The Falling Walls Foundation has awarded Prof. Dr. Marcus Mall of Charité – Universitätsmedizin Berlin the title Science Breakthrough of the Year 2022 in Life Sciences for a new treatment approach to cystic fibrosis. The new treatment, developed by Prof. Mall and his team in cooperation with international partners, addresses the underlying causes of the as-yet-incurable disease and considerably improves the therapy. Prof. Mall, Director of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, will present this scientific breakthrough at the Falling Walls Science Summit in Berlin on November 9. Cystic fibrosis is one of the most common fatal inherited diseases worldwide. The condition is diagnosed in approximately one in 2,500 to 3,000 children of European descent. It is caused by a defective gene that leads to progressive loss of lung function and respiratory distress in affected individuals. Despite improved treatment of cystic fibrosis symptoms, the disease shortens the sufferers’ life expectancy significantly. Prof. Mall’s work has been instrumental in deciphering the cystic fibrosis disease mechanism and developing the first therapy that very effectively targets the disease’s root cause. Available in Europe since August 2020, the treatment, a triple combination of so-called CFTR modulators, significantly improves the lung function and quality of life of patients with the most common genetic defect F508del, i.e., almost 90 percent of all individuals with cystic fibrosis. As of early 2022, the triple combination therapy is approved for the treatment of children aged 6 and older. “The fact that we are now able to address not only the symptoms of cystic fibrosis but its underlying molecular defect is a milestone in the treatment of this serious inherited disease,” says Prof. Mall. “I am very pleased that the jury of the Falling Walls Foundation recognize this great advance.” The lung expert, who also heads the Christiane Herzog Center for Cystic Fibrosis Center at Charité, adds, “It is my hope and my goal to make cystic fibrosis a treatable disease. At the moment, we are working towards applying the triple combination therapy as early in childhood as possible. Hopefully, this will allow us to prevent even the early stages of lung damage in the future.” Prof. Dr. Heyo K. Kroemer, Charité’s Chief Executive Officer, commended the award winner, saying, “My congratulations to Prof. Mall for winning this prestigious award. His research has definitely led to an unprecedented breakthrough for cystic fibrosis patients and I am pleased that the jury recognized his achievement. Prof. Mall’s work is an excellent example of successful research translation from bench to bedside.” Prof. Mall will present the latest advances in cystic fibrosis treatment on Wednesday, November 9, at the Falling Walls Science Summit. The hybrid event is part of Berlin Science Week. Between 12:30 and 12:55 p.m., Prof. Mall will be available for questions and discussion during the interactive Breakthrough Conversations format; between 3:05 and 3:20 p.m., he will present his lecture (in English) online. The online event is free of charge, registration is required. The title Science Breakthrough of the Year is awarded annually by the Falling Walls Foundation to research projects in ten categories: Life Sciences, Physical Sciences, Engineering and Technology, Social Sciences and Humanities, Art and Science, Future Learning, Science and Innovation Management, Science Start-ups, Science Engagement, and Emerging Talents. The award in Life Sciences goes to internationally renowned scientists who are leaders in their field helping to solve the world’s greatest challenges with their groundbreaking work and effectively breaking down walls in science and society. This year, the jury selected the winners from over 1,000 project submissions from 105 countries. Read More