Potential of cell therapy and challenges in the product development

Abstract

Cell therapy to regenerate organ function lost in the body is becoming real in the world. Based on their pluripotent and proliferative characteristics, induced pluripotent stem cells (iPSCs) can provide great opportunities to save patients suffering from diseases without effective treatments, and several iPSC-derived differentiated cells have already progressed into clinical trials. However, multiple challenges still exist in the development of iPSC-derived cell therapy product with respect to the demonstration of safety, efficacy and quality with scalable manufacturing process tolerable to the implantation in human. In safety perspective, absence of undifferentiated iPSCs and malignant transformed cells after differentiation needs to be proven with long term tumorigenicity study. In efficacy studies, translatability of immunocompromised animals used as recipients should be considered regarding immune responses and dosage. For quality control, precise, stable and reliable parameters are required to realize the ideal cell product although differentiated cells are often heterogeneous. In this presentation, the potential of cell therapy and challenges in the product development are introduced by showing some actual cases of iPSC-derived pancreatic islet cells (iPIC), a novel cell therapy product candidate for type 1 diabetes.