UMG and SubRed Therapeutics aim to jointly develop a therapy for treatment of a rare, devastating childhood disease
In the Department of Pediatrics and Adolescent Medicine at UMG, scientists led by Dr. Lars Schlotawa have developed a therapeutic approach that may lead to the first effective treatment of the rare neurometabolic disease MSD (multiple sulfatase deficiency). Through the mediation of MBM ScienceBridge, the Australian pharmaceutical company SubRed Therapeutics, a subsidiary of Tarnagulla Ventures, was chosen as a partner for further development of the therapeutic approach. The common goal of UMG and SubRed is to jointly develop a therapy and achieve its approval as the first drug for the treatment of MSD.
The lysosomal storage disease MSD is a very rare, severe, pediatric disease based on a defect in the gene coding for a protein, the formylglycine-generating enzyme (FGE). In MSD patients, important recycling processes in all cells of the body are disturbed, because FGE does not activate the so-called sulfatases or activates them insufficiently. This leads to a malfunction in the degradation of cell waste, which then accumulates in the entire body. The consequences are impaired development and severe dysfunction of the central nervous system and other organs.
With a tailored screening method developed by UMG scientists Prof. Jutta Gärtner (left) and Dr. Lars Schlotawa, a compound was discovered that improves the function of multiple sulfatases in cells at the same time. Further experiments showed that treatment of MSD patient cells with this compound resulted in improved degradation of toxic cell waste and improvement of cell pathology. Dr. Schlotawa, as the driving force in this project, was able to raise EU-funds for the first ever clinical trial in MSD, which is to start in 2023/2024.
"We are proud to be involved in such an important program that has the potential to significantly help MSD patients for whom there are currently no disease-modifying treatments“, states William McNamee, Director of SubRed Thereapeutics. "The road to an approval is still long but the progress and commitment from Dr. Schlotawa to get this treatment to the verge of a clinical trial is impressive.“
Should UMG and SubRed succeed and jointly bring a drug to market, this would be an excellent example of a successful technology transfer from research to application in which all sides win. MSD patients would have access to the first effective treatment, but the university itself and research at UMG would also benefit, since a portion of all revenues generated by SubRed Therapeutics worldwide will flow back to the university.
This is possible because MBM has already protected the therapeutic approach for the UMG in 2019, which enables UMG now to offer an exclusive license for the treatment of MSD, providing SubRed a protection from competitors. Dr. Martin Andresen, patent manager at the MBM ScienceBridge emphasizes: „This protection is mandatory, especially in the pharmaceutical area, since companies need the possibility to generate revenue compensating the high investment they make“.