Gene therapy and CAR-T cells: feedback and description of the planned circuit at the Oncopole
Context and objectives
CAR-T cells (chimeric antigen receptor T cells) are described in the treatment of hematological malignancies. CAR-T cells are autologous T lymphocytes genetically modified to express a chimeric receptor, which is composed of an extracellular domain specific of a tumor antigen and intracellular signaling domains for lymphocyte activation. Currently in clinical trials, CAR-T cells are gene therapy medicinal products (GTMPs), and are classified as class 1 GMO by the High Council for Biotechnologies. The objective of this work is to propose a circuit adapted to this type of therapies.
Materials and methods
A prior risk analysis allowed us to identify and control GTMPs inherent risks. This analysis was conducted according to the preliminary risk analysis (PRA) method. The regulations as well as the current recommendations guided us to describe implemented actions step by step.
Results and conclusions
Because our hospital pharmacy workspace were not adapted to host GTMPs, we were in charge of controlling risks and guaranteeing GMO containment at the different stages of the circuit. The entire pharmaceutical team were trained to set up activities usually done by the cell therapy unit: liquid nitrogen storage, bags thawing and aseptic live cells manipulation. In this context, two agreements have been signed: the first with a research unit for the access of a L2 depression room dedicated to advanced therapy medicinal products (ATMPs), and the second with the pathology service concerning liquid nitrogen containers reserved to pharmacy within a monitored storage area. Faced with the medical challenge brought by these promising new treatments, the Oncopole pharmacy had to think and set up a new circuit. CAR-T cells are now individualized and research is underway for standardization and making it accessible to as many people as possible. The specialization of pharmacists in this area is essential and represents a real challenge for this most recent activity.