Prof. Dr. Annette Grüters-Kieslich, Eva Luise Köhler and Elke Büdenbender with the award winners Prof. Dr. Karl Kunzelmann, Prof. Dr. Thorsten Marquardt and Dr. Julien Park
"We see you, we know what you are going through and what you are achieving. And even more: we are doing everything in our power to help you," with these words Elke Büdenbender expressed her solidarity with those affected with rare diseases during the award ceremony for the 14th Eva Luise Köhler Research Prize for Rare Diseases in Berlin in the Berlin Brandenburg Academy of Sciences and Humanities.
Ceremony at the Berlin-Brandenburg Academy of Sciences and Humanities
Together with around 200 invited guests, she accepted the invitation of the Eva Luise and Horst Köhler Foundation to the Berlin Brandenburg Academy of Sciences on June 13, 2022. Professor Dr. Thorsten Marquardt and Dr. Julien Park from Münster University Hospital and Professor Dr. Karl Kunzelmann from the University of Regensburg received the award, presented jointly with the Alliance of Chronic Rare Diseases ACHSE e.V., for a promising repurposing approach to the treatment of the rare metabolic disease cystic fibrosis. The three scientists had already been awarded the prestigious research prize last year, but the award ceremony had to be postponed several times due to the pandemic. In a short lecture, Professor Marquardt used examples from his work at the Children's Hospital to illustrate that it sometimes takes medical detective work to turn research into concrete health, and presented the award-winning project. With the prize money of 50,000 euros, the researchers will investigate the extent to which the active ingredient niclosamide, which has so far been approved for the treatment of tapeworms, but is also suitable for the regulation of derailed mucus production in cystic fibrosis.
Pragmatic research approach with special patient proximity
Eva Luise Köhler and Prof. Dr. Annette Grüters-Kieslich, Chairman of the Foundation, explained why the innovative research project was able to convince not only with its pragmatic repurposing approach but also with its special closeness to patients: "With Professor Thorsten Marquardt and his young colleague Dr. Julien Park, we are experiencing two generations of physicians who embrace the forward-looking model of the clinician scientist in an almost ideal way. The cross-site collaboration with basic researchers around Prof. Dr. Kunzelmann impressively demonstrates, that successful research and care in the field of rare diseases are inseparable and must not know any institutional boundaries."
15 years Eva Luise and Horst Köhler Foundation
Looking back on 15 years of the Foundation's work for people with rare diseases, Eva Luise Köhler noted in her welcoming address: "Together, many things have already been achieved, and yet much remains to be done. We must make an effort to develop sustainability for what has been achieved for the future." The Chairwoman of the Foundation Board thanked companions such as the Alliance of Chronic Rare Diseases and the National Action Alliance for People with Rare Diseases (NAMSE) for their close cooperation and referred to important and hopeful successes such as the establishment of now 37 centers for rare diseases nationwide, the recently started certification of the centers and a variety of other initiatives in diagnostics and care. At the same time, she warned: "The enormous medical progress not only holds great opportunities but also the obligation to act quickly and decisively now so that everyone can really participate in the 'medicine of tomorrow'." There is still not enough knowledge about rare diseases within society and, unfortunately, also within medicine, although more than four million people are affected in Germany, she said. Eva Luise Köhler shared her concern that there is a lack of investment in research structures, sustainable funding and structured care.
Alliance4Rare: Future Model of Pediatric Research on Rare Diseases
Accompanied by a moving film by director Britta Wauer, she presented the Alliance4Rare, the new project of the Eva Luise and Horst Köhler Foundation: This alliance of charitable foundations, donors and scientists is building a bridge between the growing possibilities of modern precision medicine on the one hand and the extremely urgent need for effective therapeutic options for children and young people suffering from particularly serious rare diseases on the other. "At least one thousand children and adolescents die every year in Germany alone from a rare disease. At the same time, enormous medical progress is opening up unprecedented opportunities to save seriously ill children from premature death or lifelong disability," explained Eva Luise Köhler. Starting with three university children's hospitals associated as founding partners - Berlin, Dresden and Göttingen - the Alliance4Rare is to be rapidly developed into a nationwide care and research structure for rare diseases. Behind this is a common mission: to enable medical progress where it is most urgently needed. Eva Luise Köhler thanked Prof. Grüters-Kieslich as the "head and heart" of the project, the site partners, as well as Friede Springer and her foundation, who made it possible that the Alliance4Rare can start in July with the call for project funds in no less than three program lines. "We are convinced: the medicine of the future needs researching physicians, known as clinician scientists," Eva Luise Köhler concluded, adding with regard to the award winners: "You live our vision every day, your work gives courage and hope. From the bottom of our hearts, thank you for that."
At the end of the event, there was a happy surprise: Wera Röttgering, 1st chairwoman of the Herzenswünsche association, presented a symbolic donation check for 30,000 euros in support of Alliance4Rare to Eva Luise Köhler, who was as amazed as she was grateful.