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From bench to bedside

How basic research leads to medical progress

Before a new drug is available, it is subjected to a rigorous set of tests. This benefits us all because it means we can trust the safety and efficacy of new medicines. However, we could make the process faster and more efficient without compromising quality. Two researchers from the University of Cologne know how to turn good ideas into new solutions. 

Anke Henrich

A healthy kidney is around ten centimetres long and weighs 100 grams. Cystic kidneys, as seen in polycystic kidney disease, can grow to over thirty centimetres and weigh more than 10 kilograms. If the overtaxed kidneys fail, it affects all the other organs in the human body. Many kidney patients, regardless of the cause of their kidney failure, end up aging prematurely and dying from cardiovascular diseases. 

‘Cystic kidney disease is the most common genetic cause of loss of kidney function. In Germany alone, around 80,000 people suffer from this illness,’ says Professor Dr Roman-Ulrich Müller. Before recently relocating to Düsseldorf, Müller worked as professor of Translational Nephrology in Cologne until the end of 2025, and continues to serve as a principal investigator at the CECAD Cluster of Excellence for Aging Research. As spokesperson for the Centre for Rare and Hereditary Kidney Diseases, he not only researches the causes of the disease and its progression, but also conducts clinical trials at University Hospital Cologne to develop therapies for affected patients. His conclusion: ”Research into new drugs must be translated into therapies much faster than before.”

A science news video in which Roman-Ulrich Müller explains the benefits of a ketogenic diet for polycystic kidney disease

At the CECAD Cluster of Excellence, many of the research groups are not only concerned with finding the causes of aging. They also want to translate key findings from basic medical and biological research into clinical practice. CECAD has set up its own translational infrastructure for this purpose. Translational medicine is the transfer of the latest scientific findings from the laboratory to the patient: ‘from bench to bedside’, as the medical profession calls it.

Professor Dr Oliver Cornely is director of Translational Research at CECAD and scientific director of the Clinical Trials Centre at the University Hospital. The expert in fungal diseases not only supports other researchers there. His own trials also benefit from the interdisciplinary campus concept. “With our data, my team and I enabled the approval of the drug isavuconazole for mucormycosis, a rapidly progressing fungal infection for which no medication has ever been approved before,’ says Cornely. 

His colleague Müller has good news for kidney patients: “The development of dietary interventions for kidney disease and age-related conditions has greatly benefited from the campus structures. We carried out the very first randomized controlled study on a nutritional intervention for cystic kidneys. The results are very promising and form the basis for the final clinical trial, which must now follow.” Such successes are achieved through the collaboration of experts from a wide range of disciplines, who still have to contend with the occasional setback.

Hope and reality

It all starts with basic research. Ideally, physicians, biologists, or chemists recognize a medical problem and develop a solution for it. Or an approach to combating a disease may emerge by chance whilst working on a completely different issue. In each case, researchers then test their potential under laboratory conditions, often on cells, tissue, or in animal models. After initial successes, study leaders plan the necessary Phase I to III trials on humans and recruit participants and doctors. Researchers then analyse the study results. Only after these steps is approval by a national or international supervisory authority possible. Then, many years after the start of clinical trials, the pharmaceutical industry can produce and sell the new drug. 
 

Phase I to III trials – Three trial phases are required before a new drug or procedure can be approved by German, European, or international authorities. These phases are designed to establish the efficacy and safety of the substance or procedure. Phase I trials initially recruit healthy volunteers. If the drug progresses to Phase II, it is tested on a small group of patients. Finally, the Phase III trial involves a large group of patients. Following approval, which is granted after successful completion of Phase III, Phase IV trials often follow, designed to monitor therapeutic use in practice. 

However, the reality is even more complex. Physicians across Germany are complaining about a lack of staff and infrastructure at every level. At the same time, they are under cost pressure. New therapies also lead to new, labour-intensive requirements for reimbursement, implementation, documentation, liability, and data protection. New digital applications must be evaluated and regulated by law. The human factor also plays a role. Those involved in research, quality assurance, authorities, IT, nursing, and medical services do not always work hand in hand. 

Bringing knowledge and funds into the system 

All things considered, it usually takes at least ten to fifteen years for a promising active ingredient or a new procedure to reach medical practice. The translation platform in Cologne cannot move mountains, but it can try to remove obstacles along the way. Dr Cornely, for example, advocates for accelerating funding approval for clinical trials. Moreover, in the case of international collaborations, conditions imposed by funding institutions should not prevent the best consortium from being formed. Another critical issue is legal matters. CECAD therefore always provides researchers with the latest information through seminars and databases. This may include information on criminal liability risks associated with animal testing as well as national and international regulations governing clinical trials.

Oliver Cornely, for example, advocates for practical data protection: ”For patients, data protection combined with the desire for absolute legal certainty is a burden.” The nature of the study can be explained in a matter of minutes. If a person then expresses an interest in participating, they are presented with roughly thirty to fifty pages of documentation to sign. Much of this covers legal matters, but not the nature of the study. Cornely has little patience for this: “My patients are seriously ill, and time is of the essence.”

To speed up the journey from basic research to patient care, kidney specialist Dr Müller is campaigning for better-regulated collaboration with the pharmaceutical industry. Pharmaceutical companies calculate every penny. “Commercial enterprises fail to ask themselves: What is the greatest medical benefit of a new drug? They ask themselves: Is this a profitable business case for us?” says Müller, describing the conflict of interest between medicine and business. 

Something else that baffles medical researchers is that many manufacturers are unwilling to produce even the small quantities of drugs required for a study. The academic sector, on the other hand, lacks this expertise. “It would be enough if the industry simply said: ‘You medical professionals conduct the study and we’ll supply you with the drug free of charge,’” says Müller, appealing to the concept of the common good. For this to happen, however, there must be clear direction from the regulatory authorities in order to define when and in what form the industry supports academic studies.

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.


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