Cell Therapy for Spinal Cord Injury and ALS

Abstract

This chapter highlights recent advances in stem cell therapies for spinal cord injuries (SCI) and amyotrophic lateral sclerosis (ALS). Traditionally, the adult mammalian central nervous system’s limited regenerative ability has made SCI and ALS treatment challenging. Recent research, however, has focused on neural stem cells (NSCs) and neural progenitor cells (NPCs), particularly in SCI models. NPCs are effective when transplanted during the subacute phase after injury, promoting functional recovery through synapse formation with host neurons. NPC transplantation in SCI involves bystander effects and direct cell replacement. Techniques like optogenetics and chemogenetics are essential for understanding these cells’ role in recovery. In ALS research, NPCs expressing glial cell line-derived neurotrophic factor (GDNF) show promise in modulating disease progression. Clinical trials are ongoing to evaluate the safety and efficacy of iPSCs-based cell therapy for SCI. The first human transplantation of iPSCs-neural stem/progenitor cells (NS/PCs) in subacute SCI patients has been a significant step. Future trials aim to improve iPSCs-NS/PCs therapies for chronic SCI and ALS using gene therapy and other approaches. The application of GDNF-expressing NPCs in ALS trials represents a critical advancement in stem cell therapy, showcasing the potential of gene-modified progenitor cells in clinical settings.