Production of autologous GMP-compliant T cells expressing a CAR specific for CSPG4 for clinical treatment of metastatic uveal melanoma

Institution: Hautklinik, Universitätsklinikum Erlangen
Applicant: Prof. Dr. Niels Schaft
Funding line:
Translational Research

Project partner:

Prof. Dr. Caroline Bosch-Voskens, Hautklinik, Universitätsklinikum Erlangen


Many cancer patients fail to generate T cells that effectively recognize tumor cells. Using the technique of RNA electroporation, the scientists can introduce the blueprint (RNA) for suitable, cancer-specific receptors into T cells, which are then able to kill cancer cells.
The hypothesis is that T cells expressing a CSPG4-specific chimeric antigen receptor (CAR) (hereinafter referred to as CSPG4-CAR-T cells) reduce metastases and prolong overall survival in metastatic uveal melanoma (UM). The tumorantigen CSPG4 is not only abundantly expressed, but also functions as an oncogenic driver in UM, making CSPG4 an ideal target antigen. The goal is therefore to initiate a single-center, open-label, phase I clinical trial to test the safety and efficacy of adoptively transferred ex vivo activated and expanded CSPG4-CAR-T cells. Extensive preclinical studies from the laboratory show that CSPG4-specific CAR-T cells generated by CAR-mRNA transfection are highly functional and have a favorable safety profile. This work led to the approval by the Bavarian government to produce CSPG4-CAR-T cells for clinical use in the GMP facility of the Department of Dermatology, Uniklinikum Erlangen.
The financial support by the ForTra gGmbH provides the resources to produce the T cells expressing a CSPG4-specific CAR for clinical use in uveal melanoma patients in a GMP-compliant manner.

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