Press release

24.11.2020

Personalized medicine to treat cystic fibrosis

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Christiane Herzog Award to Charité´s young talent

Dr. Simon Gräber (photo: Andreas Süß/ Stiftung Charité)
Dr. Simon Gräber (photo: Andreas Süß/ Stiftung Charité)

Joint press release by the Christiane Herzog Foundation, CharitéUniversitätsmedizin Berlin and the Berlin Institute of Health (BIH)

Dr. Simon Gräber, an assistant physician in Charité’s Department of Pediatric Pulmonology, Immunology and Critical Medicine and a fellow in the BIH Charité Clinician Scientist Program sponsored by Stiftung Charité, is the 2020 Christiane Herzog Award winner of the Christiane Herzog Foundation. With the prize money of €50,000, Dr. Gräber will establish a care and research infrastructure at CharitéUniversitätsmedizin Berlin to examine cystic fibrosis patients who have rare mutations. His goal is to find an effective therapy for these patients by individually testing approved drugs on the patients’ cells, along with CFTR modulators currently being developed in the laboratory. In the long term, the aim is to establish personalized medicine to treat cystic fibrosis (CF).

Cystic fibrosis is a rare hereditary disease caused by genetic changes in what is known as the CFTR gene, coding for an ion channel in the cell membrane. Patients do not necessarily show the same changes. Depending on which section of the gene is mutated, the symptoms can be more or less pronounced. Recently, an international clinical study co-led by Prof. Dr. Marcus Mall, BIH Professor and Director of Charité’s Department of Pediatric Pulmonology, Immunology and Critical Medicine, demonstrated the effectiveness of a new triple therapy that helps about 90 percent of all cystic fibrosis patients. Dr. Gräber is now working on a solution for the remaining 10 percent.

“We plan to include a total of at least 50 cystic fibrosis patients with rare mutations in our project,” says Dr. Gräber, who conducts research at Charité’s Christiane Herzog Centre. The patients are being recruited at Charité and from surrounding centers and will be comprehensively examined. In this process, doctors and scientists want to measure lung function or visualize the lung with modern imaging techniques and record the activity of the CFTR channel in laboratory medical tests. “In addition, we are primarily relying on ‘ex vivo’ tests on tiny organoids, which we cultivate individually from the patients’ cell samples,” explains Dr. Gräber. “These enable us to test drugs for their efficacy, individually for each patient.” The substances used are already approved drugs, but modulators currently under development are also available to the research group through cooperation partners.

With his doctoral thesis in 2008, Dr. Gräber started his career as a CF researcher, which he continued along with his work as a doctor in cystic fibrosis care. Through his work, he shows that “bench to bedside” care is possible for patients. He has always oriented his research on CF care requirements, and he also generates new research questions from his daily work in the hospital. Since September 2018, Dr. Gräber has been supported by the BIH Charité Clinician Scientist Program, which is co-financed by Stiftung Charité.

Prof. Dr. Duska Dragun, director of both the BIH Charité Clinician Scientist Program and the BIH Biomedical Innovation Academy, is pleased about her successful participant: “Dr. Gräber covers the entire translational process with his scientific and clinical activities and works in extremely integrative fashion in his daily routine. In this way, he epitomizes an exemplary clinician scientist.”

The Christiane Herzog Award
The Christiane Herzog Award for the promotion of young scientists, which is endowed with €50,000, is awarded once a year to dedicated young researchers and enables them to carry out a project relevant to cystic fibrosis. The 2020 Christiane Herzog Award was presented at the Digital German Cystic Fibrosis Conference in November 2020.
(www.muko.info/dmt)

Cystic fibrosis
In Germany, up to 8,000 children, adolescents and adults are affected by the incurable hereditary disease cystic fibrosis. Due to a disturbance of the body’s sodium and water balance, those suffering from cystic fibrosis produce a viscous secretion that irreparably damages organs such as the lungs and pancreas. Every year, about 150 to 200 children in Germany are born with this rare disease.

Christiane Herzog Foundation
“Helping through Action.” Christiane Herzog’s advocacy for people with cystic fibrosis began in 1985 under this motto. In 1997, she bundled her commitment in the Christiane Herzog Foundation with the goal of making life easier for those affected by this tragic disease and their families, giving them hope and new strength in the fight against cystic fibrosis. The foundation makes people aware of the complex clinical symptoms and promotes understanding and solidarity with those affected. In doing so, it works closely with Mukoviszidose e.V.
Further information: www.christianeherzogstiftung.de

Mukoviszidose e.V.
Mukoviszidose e.V. networks patients, their relatives, doctors, therapists and researchers. It brings together different experiences, competencies and perspectives with the goal of enabling each patient to lead a self-determined life with cystic fibrosis. In order to achieve the common tasks and goals, the non-profit association depends on the support of committed donors and sponsors. Mukoviszidose Institut gGmbH is a fully-owned subsidiary of Mukoviszidose e.V.
Further information: www.muko.info

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Contact

Prof. Dr. Marcus A. Mall
Department of Pediatric Pulmonology, Immunology and Critical Medicine
Campus Virchow Klinikum
Charité – Universitätsmedizin Berlin        
Tel: +49 30 450 566 131



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