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SUCCESS FOR DRUG RESEARCHERS FROM UA RUHR

Highly Promising Substance Against Rare Cancer Licensed Out

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The graphic drawing is intended to represent an active substance that binds to its cellular target structure. © Prof. Daniel Rauh​/​TU Dortmund University
Symbolic image: An active substance binds to its cellular target structure.
Each year, around 1,200 people in Germany are diagnosed with a gastrointestinal stromal tumor (GIST) – a rare type of cancer where the tumors form in the walls of the digestive system and that rapidly develops resistance to the therapies commonly used in precision medicine. Researchers at TU Dortmund University, the West German Cancer Center at University Hospital Essen and the Max Planck Institute of Molecular Physiology in Dortmund have identified a highly promising active substance against GIST, filed a patent application and licensed it to a US-American pharmaceutical company, which now wants to develop it further and make it ready for the market – an important step on the path from basic research to clinical application.

Together with their teams, Professor Daniel Rauh from the Department of Chemistry and Chemical Biology of TU Dortmund University, Professor Sebastian Bauer from the Faculty of Medicine at the University of Duisburg-Essen (UDE) and Dr. Sonja Sievers, head of the Compound Management and Screening Center at the Max Planck Institute (MPI), have identified a chemical substance that has shown in preclinical laboratory trials to be highly effective against drug-resistant GIST cells. The universities and the MPI have filed a patent for the substance and, with the help of PROvendis, the IP commercialization agency of 29 universities in North Rhine-Westphalia, it was immediately licensed out to a US-American pharmaceutical company, which will now drive its clinical development forward.

“This success shows the tremendous potential of drug research within the University Alliance Ruhr,” says Professor Daniel Rauh. “The development of a new drug usually takes ten years or longer. What’s special about this success is that the compound was already tested years ago, but with a different focus. At that time, however, it was unconvincing. We were now fortunate enough to rediscover it.”

The cell cultures for the preclinical testing of the potentially active substance were prepared at the West German Cancer Center: “Modern molecular biology methods such as gene scissors enable us to reproduce molecular variants of our GIST patients’ tumors in the laboratory in a very short time. In this way, we have established a library of models that is growing dynamically and unique in this form worldwide,” says Professor Sebastian Bauer. “The identification of our active substance is thus the result of integrated innovation cycles within the UA Ruhr that have grown over the years.”

As GIST is a very dynamic disease, it is likely to develop resistance to the rediscovered substance, too. That is why the interdisciplinary team of scientists from molecular genetics, cell biology, high-throughput screening, structural biology and organic synthesis is already working on successor substances.

The joint research was supported by the German Research Foundation (DFG), the Drug Discovery Hub Dortmund (DDHD), the CANTAR research network funded by the State of North Rhine-Westphalia, the IGNITE project funded by the Mercator Research Center Ruhr as well as funds from the Sarkomtour sponsored race and the David Foundation.

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