Cancer-fighting gene therapy: from research to application

Whether repairing faulty genes or introducing characteristics by delivering new genes into cells, gene therapy holds great potential for personalized medicine. However, safe, efficient and cost-effective therapies of this kind are currently few and far between for clinical use. Researchers at the MDC now want to translate a new cancer therapy involving reprogrammed immune cells from the laboratory scale to the clinic, and are cooperating with industry partners to achieve this. The Helmholtz Association is supporting the transfer process by setting up a Helmholtz Innovation Lab at the MDC.

The transfer of knowledge from basic research to clinical application allows research projects to become medicines and therapies available to society as a whole. In summer 2016 the Helmholtz-funded Max Delbrück Center Cell Engineering Lab (MD-CEL) will open at the MDC premises, creating a bridge between MDC research and industry partners and facilitating joint research and development. Competing with 27 other applicants, the MDC was successful in obtaining funding together with six other projects.

At the new laboratory, MDC research groups under Dr. Zsuzsanna Izsvák and Prof. Wolfgang Uckert will work on developing a new technique to equip a large number of cells from the immune system with new genes in a safe and reproducible way. This will enable cells to be personally tailored to the patient in whose body they are intended to fight tumors.

Therapeutic genes are delivered into the cells using gene transfer vectors, for example viruses. As an alternative, the Izsvák’s team uses non-viral vectors called transposons. “The transposon system known as ‘Sleeping Beauty’ delivers genes into cells and integrates them in the genome,” explains Izsvák. “We have optimized the system; our version SB100X now integrates itself up to a hundred times more efficiently as before and is thus comparable to viral delivery methods.”

Izsvák is working with Uckert, a professor at the Humboldt-Universität zu Berlin who is developing a promising strategy for fighting cancer. Uckert reprograms T cells from the immune system to detect and eliminate tumor cells. “The T cells target and destroy only the diseased tissue, so the likelihood of side effects is low,” explains Uckert. “This form of therapy has already been extremely successful in mice.”

The two groups are already working together with other groups from the MDC, the Charité and The University of Chicago on reprogramming T cells for tumor therapy. Compared to the popular gene delivery technique using retroviruses, this new technique is technically much easier to conduct, and it's cheaper and faster. The cooperation of Izsvák, Uckert and other groups is also supported by the Berlin Institute of Health (BIH) which is funding a big research program on T cell therapy.

At MD-CEL this reprogramming of T cells will now be transferred into clinical scale. The three industry partners Miltenyi Biotec GmbH, PlasmidFactory GmbH & Co. KG and Formula Pharmaceuticals Inc. have already been gained for the venture, bringing with them experience in the development of medical production devices, reagents and therapies.

MD-CEL could also prove useful for similar transfer projects further down the line. “Cell modification is at the heart of MD-CEL’s activities,” says Dr. Felix Lorenz, who will head the new laboratory. “This new technique can also be used to investigate other questions in gene therapy, so we are always open to further collaborative projects between MDC and industry.”

In three years, the current transposon-based immunotherapy project should be far enough along for cell products to be manufactured for clinical use in patients. This will enable studies on humans to be carried out, bringing wide-scale application of the technique in the fight against cancer one step closer.


With the Innovation Lab at the MDC there will be an open platform for cell engineering projects. Image: Katharina Bohm/MDC.


Vera Glaßer
Max Delbrück Center for Molecular Medicine (MDC) Berlin-Buch
Head of Communications department (interim)
Tel: 030/94 06 - 2120

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