Phase 1 Safety Trial of Autologous Human Schwann Cell Transplantation in Chronic Spinal Cord Injury.

Abstract

A Phase 1 open-label, non-randomized clinical trial was conducted to determine feasibility and safety of autologous human Schwann cell (ahSC) transplantation, accompanied by rehabilitation, in participants with chronic spinal cord injury (SCI). MRI was used to screen eligible participants to estimate an individualized volume of cell suspension to be implanted. The trial incorporated standardized multimodal rehabilitation before and after cell delivery. Participants underwent sural nerve harvest, and ahSCs were isolated and propagated in culture. The dose of culture-expanded ahSCs injected into the chronic spinal cord lesion of each individual followed a cavity-filling volume approach. Primary outcome measures for safety and trend-toward efficacy were assessed. Two participants with ASIA Impairment Scale (AIS) A and two participants with incomplete (AIS B, C) chronic SCI were each enrolled in cervical and thoracic SCI cohorts (n=8 total). All participants completed the study per protocol, and no serious adverse events related to sural nerve harvest or ahSC transplantation were reported. Urinary tract infections and skin abrasions were the most common adverse events reported. One participant experienced a 4-point improvement in motor function, a 6-point improvement in sensory function and a 1-level improvement in neurological level of injury. Follow-up MR imaging in the cervical (6 months) and thoracic (24 months) cohorts revealed a reduction in cyst volume after transplantation with reduced effect over time. This Phase 1 trial demonstrated the feasibility and safety of ahSC transplantation combined with a multimodal rehabilitation protocol for participants with chronic SCI.