Prof. Dr. Daniel Rauh and Prof. Dr. Sebastian Bauer and their teams have licensed out a promising substance against the rare cancer “gastrointestinal stromal tumor”.

Around 1,200 people in Germany are diagnosed with gastrointestinal stromal tumors, or GIST for short, every year - a rare type of cancer in which the tumors develop in the walls of the digestive organs and quickly develop resistance to common precision drugs. Scientists at TU Dortmund University, the West German Tumor Center at Essen University Hospital and the Max Planck Institute of Molecular Physiology in Dortmund have identified a promising active substance against GIST, applied for a patent and licensed it to a US pharmaceutical company, which now wants to develop it to market maturity - an important step on the path from basic research to clinical application.

The teams led by Prof. Dr. Daniel Rauh from the Faculty of Chemistry and Chemical Biology at TU Dortmund University, Prof. Dr. 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), identified a chemical substance that proved to be highly effective against drug-resistant GIST cells in preclinical laboratory tests. The universities and the MPI filed a patent application for the active substance and with the help of PROvendis, the collecting society of 29 NRW universities, it was immediately licensed out to a US pharmaceutical company, which will drive forward clinical development.

“This success shows the enormous potential of drug research within the University Alliance Ruhr,” says Prof. Dr. Daniel Rauh. “The development of a new drug usually takes ten years or longer. The special thing about this success is that the compound was already tested years ago with a different focus. At that time, however, it was not convincing. Now we were lucky enough to have rediscovered this substance.”

The cell cultures for preclinical testing of the potential active substances were set up at the West German Tumor Center: “Modern molecular biology methods such as gene scissors enable us to recreate molecular variants of the tumors of our GIST patients in the laboratory in a very short time. We have thus established a dynamically growing model library that is unique in this form worldwide,” says Prof. Dr. Sebastian Bauer. “The identification of our substance is therefore the result of integrated innovation cycles within the UA Ruhr that have grown over the years.”

As GIST is a very dynamic disease, resistance to the rediscovered substance is also likely to develop. The interdisciplinary team from the fields of molecular genetics, cell biology, high-throughput screening, structural biology and organic synthesis is therefore 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 and funds from the Sarcoma Tour and the David Foundation.