First Human Trial of Stem Cell Therapy for Chronic Spinal Cord Injury

By Anna Tarasova‘19

Figure 3: A labeled diagram of the sensory pathways and ascending tracts in the spinal cord.

The therapeutic potential of stem cells has been widely examined by many researchers for more than two decades. Neurodegenerative damage is one of the areas in which the use of multipotent neural precursors (NPCs) has proven particularly useful. Although the use of human-derived NPCs in treating chronic spinal cord injury (SCI) has only been examined in animal models, Phase I and II trials have already been completed for patients with amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease. Depending on the extent of the spinal injury, the deficit can result in weakness or paralysis of either the lower limbs or all four limbs. No treatment currently exists that results in a significant improvement of motor or sensory function in SCI patients.

Dr. Erik Curtis and his team at the University of California-San Diego have conducted a Phase I study assessing the feasibility and safety of using the human-spinal-cord-derived stem cell line NSI-566 to treat SCI. Pre-clinical trials used mouse and rat models to determine the safety of NSI-566 cells. These studies provided the basis for designing a human Phase I trial. The Phase I trial used four subjects that received NSI-566 implantation and completed several assessments over the course of the next 27 months. These assessments included neurological and functional tests as well as surveys. Sensory and motor testing found improvements in three out of the four patients, and no surgery-related complications or follow-up pain occurred. Two of the subjects showed at least a one-level improvement on the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) scale, which is widely used for determining the gravity of spinal cord injury. While these results are promising, the researchers acknowledge that a larger sample size as well as a control group are necessary to further evaluate the effects of the NSI-566 treatment on SCI and other deficits.



  1. E. Curtis, et. al., A First-in-Human, Phase I Study of Neural Stem Cell Transplantation for Chronic Spinal Cord Injury. Cell Stem Cell (2018). doi: 10.1016/j.stem.2018.05.014
  2. Image retrieved from:

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