The principal objective of The ONE Study cooperative project is to establish whether purified haematopoietic regulatory cells can be used therapeutically to reduce the level of pharmacologic immunosuppression needed by recipients of transplanted organs.
To assess this, several regulatory cell products will be licensed for manufacture under good manufacturing practice (GMP) conditions. We have chosen to include in The ONE Study those cell preparations which, in our view, represent the most promising therapeutic agents and which are closest to clinical application: Tregs, Mregs, Tr1 cells and DCs. With the expertise of Pharmatching GmbH, particular emphasis will be placed on compliance with the recent European Medicines Agency (EMEA) regulations concerning therapeutic cell preparations, as well as ensuring that each cell product meets the local and national guidelines. The ability of the academic partners to translate their innovative ideas into clinical reality will be enormously strengthened by the involvement of Miltenyi Biotec GmbH. Miltenyi will provide our academic partners with the necessary tools for the in vitro isolation, and in some cases, expansion, of regulatory cell populations. Miltenyi will also develop new experimental tools for the quality control and monitoring of administered cell products. Furthermore, the introduction of flow cytometry-based cell sorter by select partners adds another new dimension that could prove effective for cell manufacturing. Such technologies should facilitate a more rapid and widespread use of cell-based immunosuppressive therapy, if a potential benefit is demonstrated by The ONE Study.
The ONE Study project will lead to a clear, statistically-valid conclusion as to whether further testing with each cell product is warranted. It is our ambition that, by end of The ONE Study program, the technology to produce and administer a therapeutically efficacious immunoregulatory cell product should be approved for dissemination throughout all EU member states.