Intraspinal injections of human spinal cord-derived neural stem cells are well tolerated in a Phase I study of four patients with thoracic (T2–T12) ASIA-A grade spinal cord injury. This was the result recently published in Cell Stem Cell, supporting the potential of transplanted NSI-566 (Neuralstem), the stem cell line used, in benefitting patients with chronic spinal cord injury.
This first cohort of four patients received the intraspinal injections 12 to 24 months after injury, and all tolerated the procedure well, with no serious adverse events 18 to 27 months after grafting. Analysis of motor and sensory function and electrophysiology results showed improvement in three of the four patients after NSI-566 transplantation.
The primary objective of the study was to evaluate the safety of NSI-566 transplantation in subjects with stable thoracic spinal cord injury, but additional endpoints were also measured, including changes in neurologic deficits, neurophysiology, and neuropathic pain.
Senior author and study principal investigator Joseph D Ciacci (Department of Neurosurgery, VA San Diego Healthcare System; UC San Diego Health, San Diego, USA) comments, “Stem cell therapies have tremendous potential in addressing spinal cord injury, a condition for which there are currently no treatment options that can restore lost function. This study serves as a step in this direction, as it suggests the procedure is safe and has the potential for benefitting the patient.”
It is estimated that there are 12,500 new cases of spinal cord injury per year, and that, at any given time, there are between 240,000 and 337,000 people in the USA living with spinal cord injury.
Neuralstem has initiated the second cohort trial to test safety in patients with cervical spinal cord injury patients. “These data are encouraging and suggest that human spinal cord-derived neural stem cells may provide functional benefit to patients with spinal cord injury,” says Rich Daly, chief executive officer of Neuralstem. “We look forward to seeing the impact of this procedure on subjects with cervical injuries, who are the basis for the second cohort of the trial.”
In addition to chronic spinal cord injury, NSI-566 is also being tested for treatment of paralysis in stroke, amyotrophic lateral sclerosis (ALS), and traumatic brain injury. In a study published in Nature, NSI-566 was shown to have restorative function in a primate paralysis model.