Human Neural Stem Cell Therapy for Chronic Ischemic Stroke: Charting Progress from Laboratory to Patients

Stem Cells Dev. 2017 Jul 1;26(13):933-947. doi: 10.1089/scd.2017.0009. Epub 2017 May 18.


Chronic disability after stroke represents a major unmet neurologic need. ReNeuron's development of a human neural stem cell (hNSC) therapy for chronic disability after stroke is progressing through early clinical studies. A Phase I trial has recently been published, showing no safety concerns and some promising signs of efficacy. A single-arm Phase II multicenter trial in patients with stable upper-limb paresis has recently completed recruitment. The hNSCs administrated are from a manufactured, conditionally immortalized hNSC line (ReNeuron's CTX0E03 or CTX), generated with c-mycERTAM technology. This technology has enabled CTX to be manufactured at large scale under cGMP conditions, ensuring sufficient supply to meets the demands of research, clinical development, and, eventually, the market. CTX has key pro-angiogenic, pro-neurogenic, and immunomodulatory characteristics that are mechanistically important in functional recovery poststroke. This review covers the progress of CTX cell therapy from its laboratory origins to the clinic, concluding with a look into the late stage clinical future.

Keywords: clinical trials; mechanisms of action; neural stem cell therapy; preclinical studies; stroke disability.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Brain Ischemia / genetics
  • Brain Ischemia / physiopathology
  • Brain Ischemia / therapy*
  • Cell Differentiation / genetics
  • Cell- and Tissue-Based Therapy
  • Humans
  • Neural Stem Cells / transplantation*
  • Neurogenesis / genetics
  • Neurons / metabolism
  • Stem Cell Transplantation*
  • Stroke / genetics
  • Stroke / physiopathology
  • Stroke / therapy*