ABSTRACT

The transplantation of in vitro expanded human neural precursor cells (hNPCs) and their differentiated progeny (e.g., neurons) represents a potential new treatment alternative for individuals suffering from incurable, central nervous system (CNS) disorders, such as Parkinson’s disease, Huntington’s disease, and chronic neuropathic pain. However, for CNS-targeted cell restorative therapies to have widespread therapeutic significance, it will be necessary to generate large quantities of clinical grade hNPCs and their differentiated progeny using standardized manufacturing processes. This chapter focuses on the development of robust protocols for the large-scale expansion and differentiation of therapeutically effective hNPC populations for the treatment of neurodegenerative disorders. It describes proper hNPC handling and characterization methods, the large-scale expansion of hNPC populations under controlled conditions in suspension bioreactors, and animal model studies where differentiated cells derived from bioreactor-generated hNPCs were transplanted to effectively treat Huntington’s disease and spinal cord pain (allodynia).