In 2017, the US American Food and Drug Administration FDA approved a gene therapy for the first time. The therapy with CAR-T cells achieved impressive success in the treatment of cancer. It is also the focus of a research project that the Proof-of-Concept initiative will be supporting with about 2.8 million euros.
Non-viral gene transfer for gene therapy
The chimeric antigen receptors (CAR) developed at the University Hospital of Würzburg detect a certain molecule (ROR1). It is barely present on healthy cells, but which occurs all the more on cancerous cells from leukemia, breast or lung cancer.
In the case of the research project now being funded by the PoC initiative, the gene transfer does not take place with viruses, but by means of the Sleeping Beauty transposon system. The gene vector called SB100X has been developed by the lab of MDC researcher Zsuzsanna Izsvák. The funding is to be used to complete pre-clinical studies on the safety and efficacy of the ROR1 CAR-T cells and to achieve the clinical translation into a Phase I study (First-in-Man).
Project participants include the, Medical Clinic and Polyclinic II; the Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC); and the .
An initiative to foster translation
Translation of scientific results is full of obstacles. The path from basic research to new drugs, therapies and medicinal products is hard and long-winded. That's why, in the next three years, Fraunhofer-Gesellschaft, Helmholtz Association and Deutsche Hochschulmedizin will be providing up to twelve million euros for the Proof-of-Concept initiative. A high-ranking, specialist jury from science, industry and regulatory authorities has now determined the most promising research projects for funding.