Preclinical efficacy and safety assessments of Adult human neural stem cells (AhNSCs) for spinal cord injury

Spinal cord injury (SCI) is a severe and devastating condition that leads to irreversible damage to neural tissues, creating significant medical, economic, and social challenges. The ability to differentiate into multiple neural cell types and to regulate immune response makes neural stem cells (NSC) a promising strategy for treating SCI. In this study, we investigated the therapeutic potential, safety profile, and tumorigenic risk of intrathecally transplanted adult human neural stem cells (AhNSCs) produced under clinical-grade standards in a Good Manufacturing Practice (GMP) facility, in rat SCI models, thereby laying the foundation for future clinical trials. Functional tests, including the Basso, Beattie, and Bresnahan (BBB) locomotor rating, rotarod, and von Frey tests, showed significant improvements in motor function and mechanical sensitivity in rats with SCI. Histological analysis revealed reduced tissue loss, glial scar formation, and increased axonal regeneration. Biodistribution studies indicated that the transplanted AhNSCs are primarily localized within the spinal cord, with minimal systemic distribution. Toxicity studies found no significant adverse effects, suggesting a favorable safety profile. Long-term tumorigenicity studies reported no treatment-related deaths or signs of tumor formation in either gender. In conclusion, the study demonstrates that AhNSCs offer promising therapeutic potential for treating SCI, contributing to improved motor function and sensory recovery. These findings support further investigation and potential clinical applications of AhNSCs for treating SCI and related neurological disorders.