Ceremony with Elke Büdenbender at the Berlin-Brandenburg Academy of Sciences
“We see you, and we know what you are going through and what you achieve. More than that: We do everything in our power to help you,” said Elke Büdenbender during the 14th Eva Luise Köhler Research Award for Rare Diseases in Berlin, expressing her solidarity with people affected by cystic fibrosis and all other rare diseases. Afterwards, Eva Luise Köhler honored Professor Dr. Thorsten Marquardt and Dr. Julien Park from University Hospital Münster, as well as Professor Dr. Karl Kunzelmann from University of Regensburg, with the 14th Eva Luise Köhler Research Award for Rare Diseases for their promising repurposing approach for cystic fibrosis treatment.
Ceremony at the Berlin-Brandenburg Academy of Sciences
Together with about 200 invited guests, she attended the Eva Luise and Horst Köhler Foundation’s invitation to the Berlin-Brandenburg Academy of Sciences on June 13, 2022. Professor Dr. Thorsten Marquardt and Dr. Julien Park from University Hospital Münster and Professor Dr. Karl Kunzelmann from the University of Regensburg received the award, jointly presented with the Alliance for Chronic Rare Diseases (ACHSE e.V.), for a promising repurposing approach to treat the metabolic disease cystic fibrosis. The three scientists had already been awarded the prestigious research prize last year, but the official celebration had to be postponed several times due to the pandemic. In a short talk, Professor Marquardt explained, using examples from his work at the children’s hospital, how true “medical detective work” is sometimes needed for research to translate into real health benefits, and presented the award-winning project. With the €50,000 prize money, the researchers will study to what extent the drug Niclosamide, so far approved for treating tapeworms, can be used to regulate uncontrolled mucus production in cystic fibrosis.
Pragmatic research approach for cystic fibrosis with special patient focus
Eva Luise Köhler and Foundation board member Prof. Dr. Annette Grüters-Kieslich explained why the innovative research project stood out not just for its pragmatic repurposing, but also for its particular closeness to patients: “With Professor Thorsten Marquardt and his young colleague Dr. Julien Park, we see two generations of physicians embodying the pioneering model of the clinician scientist in the most exemplary way. The cross-site collaboration with basic science researchers around Prof. Dr. Kunzelmann demonstrates impressively that successful research and care in the field of rare diseases are inseparable and must not be limited by institutional boundaries.”
15 years Eva Luise and Horst Köhler Foundation
Looking back on 15 years of foundation work for people with rare diseases, Eva Luise Köhler remarked in her welcome speech: “Together we have already achieved a lot, yet much remains to be done, and we must make every effort to secure what has been achieved for the future.” The Chair thanked partners like the Alliance for Chronic Rare Diseases and the National Action Alliance for People with Rare Diseases (NAMSE) for their close cooperation, and highlighted important and hopeful progress like the establishment of 37 centers for rare diseases nationwide, the recently introduced certification of these institutions, and a variety of initiatives in diagnostics and care. At the same time, she warned: “Huge medical advances bring not only great opportunities, but also the responsibility to now act swiftly and decisively, so everyone can truly participate in the ‘medicine of tomorrow’.” Knowledge about rare diseases is still not widespread enough within either society or medicine, even though more than four million people in Germany are affected. There is a lack of investment in research infrastructure, sustained funding, and structured care, Eva Luise Köhler cautioned.
Alliance4Rare: A future model for pediatric research
Accompanied by a moving film from director Britta Wauer, she introduced the Eva Luise and Horst Köhler Foundation’s new project Alliance4Rare: This alliance of non-profit foundations, donors, and scientists is building a bridge between the growing possibilities of modern precision medicine and the urgent needs for effective therapies for children and adolescents with particularly severe rare diseases. “At least one thousand children and adolescents die every year in Germany alone due to rare diseases. Yet, enormous medical progress is opening unprecedented possibilities to save critically ill children from early death or lifelong disability,” Köhler said. Starting with three university pediatric clinics – Berlin, Dresden, and Göttingen – as founding partners, Alliance4Rare will soon expand into a national network for care and research in rare diseases. The joint mission: enabling medical progress where it is needed most. Eva Luise Köhler thanked Prof. Grüters-Kieslich as the “heart and mind” of the project, the partner sites, as well as Friede Springer and her foundation, whose support will allow Alliance4Rare to launch project funding in three program lines starting July. “We are convinced: The medicine of the future needs physician-scientists,” Eva Luise Köhler concluded, addressing the awardees: “You live our vision every day; your work inspires hope and confidence. Thank you from the heart.”
At the end of the event, there was a joyful surprise: Wera Röttgering, chairwoman of the Herzenswünsche association, presented a symbolic donation check for €30,000 to the surprised and grateful Eva Luise Köhler to support Alliance4Rare.
