Müller’s perseverance may soon pay off for kidney patients. He and his team have recognized that a certain small RNA (microRNA) plays an important role in the regulation of the PKD1 gene, which is the most common cause of cystic kidneys. “We have been pursuing this approach for years and presented it at the German Congress of Nephrology in 2006,” he explains. Meanwhile, a working group in the US conducted parallel research and a small company produced microRNA inhibitors based on these findings. It became clearer than ever before: this seems to be the right path. The pharmaceutical giant Novartis promptly bought the company. Müller is delighted: “This means that money is now in the system and the Phase III trial is expected to start in 2026.”
AI as an accelerator
Oliver Cornely and Roman-Ulrich Müller are also working on minimizing bureaucracy. For example, urgent research proposals just sit in people’s inboxes. “We don’t want to accelerate development at the expense of patient safety,” they both agree. But to date, the German authorities and the Ethics Committee have not prioritized submitted studies; rather, they process them according to the date of receipt. “If such submissions were answered immediately, drugs could be developed more quickly,” Cornely warns. And Müller adds in general: “Purely in terms of the administrative process, we are experiencing major delays.”
Thankfully, artificial intelligence is speeding up processes elsewhere. This is where CECAD comes in. Researchers setting up a new trial will find tens of thousands of anonymized datasets from disease-specific cohorts as well as comparative values from healthy test subjects. The fund extends to ‘real-world evidence’, the practical testing of new therapeutic approaches. Artificial intelligence analyses and links image data, laboratory values, genomics, doctors’ letters, and progress data. This helps AI identify predictive markers, potential drug candidates, and possible side effects more quickly.
Cornely and his team also use AI to develop practical treatment guidelines. They want to understand how effective new therapies are for doctors and carers. “That’s why we develop handouts for each new guideline in the form of apps or pocket cards in multiple languages,” says Cornely. ”This allows us to measure the utilization and guideline compliance of all those involved, and to derive the quality of treatment.” Thanks to feedback provided directly in the app, his team can quickly identify which recommendations need to be readjusted in the workflow. This project is also making waves far beyond the Rhine. “We are in constant contact with colleagues in 96 countries through our scientific networks,” Cornely is proud to report.
Müller and Cornely and their teams are working tirelessly to improve the situation for patients. You could say they are blessed with great endurance: an ability to go the extra mile. “We write many trial or funding applications at night after work,” says Müller. “We don't have time for that during the day.” Ultimately, the transfer from bench to bedside depends on everyone's commitment. ‘Hard work and creativity, that’s our approach,” Cornely concludes.
CECAD Translational Platform
Professor Dr Oliver Cornely, Dr Ruth Hanssen and Professor Dr Roman-Ulrich Müller lead the translational activities of the Cluster of Excellence. The platform comprises the CECAD Institute of Translation (CIT), the Clinical Trials Centre Cologne (CTCC), the CECAD Clinical Study Design Lab and the CECAD Biobank. It also coordinates a large network of internal and external partners.