How can clinical safety and efficacy concerns in stem cell therapy for spinal cord injury be overcome?

Expert Opin Biol Ther. 2023 Jul-Dec;23(9):883-899. doi: 10.1080/14712598.2023.2245321. Epub 2023 Aug 20.


Introduction: Spinal cord injury (SCI) can lead to severe neurological dysfunction. Despite scientific and medical advances, clinically effective regenerative therapies including stem cells are lacking for SCI.

Areas covered: This paper discusses translational challenges related to the safe, effective use of stem cells for SCI, with a focus on mesenchymal stem cells (MSCs), neural stem cells (NSCs), Schwann cells (SCs), olfactory ensheathing cells (OECs), oligodendrocyte precursor cells (OPCs), embryonic stem cells (ESCs), and induced pluripotent stem cells (iPSCs). We discuss approaches to enhance the efficacy of cell-based strategies by i) addressing patient heterogeneity and enhancing patient selection; ii) selecting cell type, cell source, cell developmental stage, and delivery technique; iii) enhancing graft integration and mitigating immune-mediated graft rejection; and iv) ensuring availability of cells. Additionally, we review strategies to optimize outcomes including combinatorial use of rehabilitation and discuss ways to mitigate potential risks of tumor formation associated with stem cell-based strategies.

Expert opinion: Basic science research will drive translational advances to develop stem cell-based therapies for SCI. Genetic, serological, and imaging biomarkers may enable individualization of cell-based treatments. Moreover, combinatorial strategies will be required to enhance graft survival, migration and functional integration, to enable precision-based intervention.

Keywords: Clinical trials; regenerative therapies; safety and efficacy; spinal cord injury; stem cells.

Publication types

  • Review

MeSH terms

  • Embryonic Stem Cells
  • Humans
  • Induced Pluripotent Stem Cells* / transplantation
  • Neural Stem Cells*
  • Spinal Cord
  • Spinal Cord Injuries* / pathology
  • Spinal Cord Injuries* / therapy
  • Stem Cell Transplantation / adverse effects
  • Stem Cell Transplantation / methods