A NEW Phase I clinical trial has demonstrated the long-term safety and feasibility of neural stem cell transplantation for chronic spinal cord injuries, with sustained neurological improvements observed in two out of four participants.
Chronic spinal cord injuries are life-altering, often resulting in permanent paralysis and significant loss of function. Current treatments focus on symptom management rather than reversal of damage. In this study, led by researchers at the University of California San Diego School of Medicine, human spinal cord-derived neural stem cells (NSI-566) were implanted into the damaged spinal cord tissue of four patients with thoracic spinal cord injuries. The primary goal was to evaluate the safety and tolerability of the procedure while gathering preliminary data on potential therapeutic effects. Participants were followed for five years to monitor outcomes.
The transplantation procedure was well tolerated by all four participants, with no serious adverse events reported. Notably, two patients exhibited significant and durable neurological improvements. These included enhanced motor function, increased sensory scores, and improved electromyography (EMG) activity. Additionally, some patients reported reductions in chronic pain. Despite the small sample size, the results underscore the potential of neural stem cell therapy to partially restore function in patients with chronic spinal cord injuries. The findings pave the way for further clinical studies to determine efficacy and refine the therapeutic approach.
This study highlights the promise of neural stem cell transplantation as a regenerative treatment strategy for spinal cord injuries. By leveraging the ability of human-derived stem cells to integrate into the nervous system and repair damaged tissue, this emerging therapy could significantly alter the treatment landscape for spinal injuries. Building on these findings, the researchers plan to initiate a Phase II clinical trial to evaluate treatment efficacy in a larger cohort. While the Phase I trial was not designed to assess full clinical benefits, these results offer a critical foundation for future advancements in spinal cord injury management and underscore the importance of continued research in this field.
Katrina Thornber, EMJ
Reference
Martin JR et al. Long-term clinical and safety outcomes from a single-site phase 1 study of neural stem cell transplantation for chronic thoracic spinal cord injury. Cell Reports Medicine. 2024;5(12):101841.
