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  4. Long-term clinical and safety outcomes from a single-site phase 1 study of neural stem cell transplantation for chronic thoracic spinal cord injury

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 · DOI: https://doi.org/10.1016/j.xcrm.2024.101841 · Published: December 17, 2024

Spinal Cord InjuryRegenerative Medicine

Simple Explanation

This study reports on the long-term (five-year) results of a phase 1 clinical trial that investigated the safety and potential benefits of transplanting neural stem cells (NSI-566) into the injured spinal cords of four patients with chronic thoracic spinal cord injury. The primary goal was to assess the safety and feasibility of the procedure. Secondary goals involved monitoring any neurological improvements, changes in pain levels, and overall quality of life in the participants. The researchers found that the stem cell implantation procedure was well-tolerated by all four subjects. Two subjects showed evidence of neurological improvement and increased motor and sensory scores five years after the transplantation.

Study Duration
60 Months
Participants
Four subjects with chronic ASIA-A thoracic spinal cord injury
Evidence Level
Phase 1 clinical trial

Key Findings

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    All four subjects tolerated the stem cell implantation procedure well, with no serious adverse events immediately following the procedure.
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    Two subjects showed durable electromyography-quantifiable evidence of neurological improvement, as well as increased neurological motor and sensory scores at five years post-transplantation.
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    Two of the four patients experienced overall decreased pain scores post-operatively.

Research Summary

This study reports the long-term results for a phase 1 study of neural stem cell transplantation for chronic spinal cord injury. Here, we report that all four subjects tolerated the stem cell implantation procedure well, and two subjects had durable electromyography-quantifiable evidence of neurological improvement as well as increased neurological motor and sensory scores at five years post-transplantation. Clinical data at five years post-NSC transplantation indicate some quantifiable and tractable responses and merit further investigation with dose-escalation studies in patients with chronic SCI.

Practical Implications

Safety and Tolerability

The study demonstrates the safety and tolerability of neural stem cell transplantation (NSI-566) in patients with chronic thoracic spinal cord injury.

Potential for Neurological Improvement

The findings suggest the potential for long-term neurological improvement, as evidenced by electromyography and increased motor and sensory scores in some patients.

Pain Reduction

The study indicates that neural stem cell transplantation may contribute to decreased pain scores in some patients post-operatively.

Study Limitations

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