Nonclinical Studies for Cell-Based Medicines

Cell-based therapies are being developed for the treatment of degenerative diseases, inflammatory conditions, cancer, and repair of damaged tissue. Safety depends on many factors including the cell type, the differentiation status and proliferation capacity of the cells, the intended clinical condition, and the long-term survival of the product. Understanding the characteristics and function of the cellular therapy is crucial to the development of the nonclinical safety program that will support translation to the clinic. In addition to traditional local and systemic safety assessments, the potential for tumorigenicity, biodistribution, and immunogenicity of the cell therapy need to be addressed. The nonclinical strategy is product specific and designed on a case-by-case basis. Regular interaction with the regulatory agencies is advised to ensure acceptability of the proposed nonclinical plans. This chapter aims to provide advice on the aspects to consider when designing the nonclinical programs, potential limitations and how to address these, and to introduce the concept of the risk-based approach.