A First-in-Human, Phase I Study of Neural Stem Cell Transplantation for Chronic Spinal Cord Injury

Cell Stem Cell. 2018 Jun 1;22(6):941-950.e6. doi: 10.1016/j.stem.2018.05.014.

Abstract

We tested the feasibility and safety of human-spinal-cord-derived neural stem cell (NSI-566) transplantation for the treatment of chronic spinal cord injury (SCI). In this clinical trial, four subjects with T2-T12 SCI received treatment consisting of removal of spinal instrumentation, laminectomy, and durotomy, followed by six midline bilateral stereotactic injections of NSI-566 cells. All subjects tolerated the procedure well and there have been no serious adverse events to date (18-27 months post-grafting). In two subjects, one to two levels of neurological improvement were detected using ISNCSCI motor and sensory scores. Our results support the safety of NSI-566 transplantation into the SCI site and early signs of potential efficacy in three of the subjects warrant further exploration of NSI-566 cells in dose escalation studies. Despite these encouraging secondary data, we emphasize that this safety trial lacks statistical power or a control group needed to evaluate functional changes resulting from cell grafting.

Keywords: SCI; spinal cord injury; spinal surgery; stem cell therapy.

Publication types

  • Case Reports
  • Clinical Trial, Phase I

MeSH terms

  • Adult
  • Animals
  • Cell Line
  • Chronic Disease
  • Female
  • Humans
  • Male
  • Neural Stem Cells / cytology
  • Neural Stem Cells / transplantation*
  • Rats
  • Rats, Nude
  • Spinal Cord Injuries / pathology*
  • Spinal Cord Injuries / surgery
  • Spinal Cord Injuries / therapy*
  • Stem Cell Transplantation* / adverse effects
  • Young Adult