Improved cystic fibrosis treatment is Science Breakthrough of the Year

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Improved cystic fibrosis treatment is Science Breakthrough of the Year

CHARITÉ – UNIVERSITÄTSMEDIZIN BERLIN

Prof. Dr. Marcus Mall © Charité | Wiebke Peitz

IMAGE: PROF. DR. MARCUS MALL © CHARITÉ | WIEBKE PEITZ

CREDIT: © CHARITÉ | WIEBKE PEITZ

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.

Vita Prof. Dr. Marcus Mall
Marcus Mall studied medicine at the University of Freiburg and University College London. Following his doctorate, he conducted research at the Cystic Fibrosis/Pulmonary Research and Treatment Center at the University of North Carolina at Chapel Hill. In 2004 he joined the Center for Pediatrics and Adolescent Medicine at Heidelberg University Hospital, where he established the EU-funded junior research group “Cystic Fibrosis/Chronic Respiratory Disease” and received his habilitation in 2007. That same year, he became director of the Cystic Fibrosis Center as a specialist in pediatric and adolescent medicine. Between 2012 and 2018 he was director of the Translational Lung Research Center (TLRC) and the Department of Translational Pulmology at Heidelberg University Hospital. In 2018, Charité, the Einstein Foundation Berlin and the Berlin Institute of Health at Charité (BIH) appointed him professor of Pediatric Respiratory Medicine and Immunology. In this role he is also the director of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine; the Pediatric Emergency Department; and the Christiane Herzog Center for Cystic Fibrosis at Charité. Prof. Mall is one of the founding directors of the German Center for Lung Research (Deutsches Zentrum für Lungenforschung, DZL); as of 2021 coordinator of the Berlin site of the German Center for Child and Adolescent Health (DZKJ); and deputy spokesperson of the newly established Collaborative Research Center 1449, Dynamic Hydrogels on Biointerfaces. He has received numerous research awards, including a Heisenberg Professorship in Translational Pediatric Pneumology from the German Research Foundation (DFG).

Falling Walls Science Summit
The Falling Walls Science Summit is an interdisciplinary and cross-sectoral forum for the world’s leading scientists with a focus on scientific breakthroughs. The Summit takes place every year on November 7 through 9 in Berlin. The event is organized by the Falling Walls Foundation, a nonprofit organization inspired by the fall of the Berlin Wall on November 9, 1989, and the dawn of a new era of freedom. The foundation promotes groundbreaking ideas, building a growing network of thought leaders in academia, business, and the public sector to tackle the great challenges of our time and make groundbreaking approaches available to society.

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