Spinal Cord Injuries Stem Cell Trial: Encouraging Phase I Results
A spinal cord injuries stem cell trial at the University of California San Diego School of Medicine has produced measurable improvement in three of four subjects, with no serious adverse effects. Researchers report these findings in the journal Cell Stem Cell, marking a significant milestone in the effort to develop safe and effective neural regeneration therapies for patients with chronic spinal cord injuries.
“The primary purpose of this first trial was to assess safety. And no procedure related complications were observed in any of the patients,” said Joseph Ciacci, MD, principal investigator and a neurosurgeon at UC San Diego Health. “Our results suggest the approach can be performed safely and early signs of efficacy warrant further exploration and dose escalation studies.”
How the Spinal Cord Injuries Stem Cell Trial Worked
The spinal cord injuries stem cell trial used a human spinal cord derived neural stem cell line developed by Neuralstem Inc., a biopharmaceutical company based in Maryland. Four trial participants with one to two year old permanent injuries to T2 through T12 thoracic vertebrae — located in the middle of the spine — received six injections, each containing 1.2 million neural stem cells.
The results, measured 18 to 27 months after transplantation, were not dramatic but were encouraging. Analysis of motor and sensory function and electrophysiology results showed improvement in three of the four participants.
Background: From Animal Studies to Human Trial
This spinal cord injuries stem cell trial builds on earlier preclinical research published in 2013 by Ciacci and co author Martin Marsala, MD, professor in the Department of Anesthesiology at UC San Diego School of Medicine. In that study, stem cells were transplanted into rats with spinal cord injuries, resulting in neuronal regeneration and improvement in the animals’ functioning and mobility.
The move from animal models to human Phase I trials represents a critical step in validating the safety and potential of neural stem cell transplantation as a therapeutic approach. To understand how this progression works across different phases of research, our introduction to clinical trials explains the full development pathway from Phase I through Phase IV studies.
What Makes This Trial Significant
“This is a small sample size, but the real strengths of this study are the extensive follow up period, electrophysiological assessments, and the timeline of treatment,” said Ciacci. “Everyone was treated after a year of injury, meaning there was essentially no chance of spontaneous recovery. Our primary objective was to provide proof of safety and tolerability of treatment. We’ve done that.”
These early signs of potential efficacy, combined with the promising results of earlier animal studies, argue for pressing ahead with new trials and greater doses to see if researchers can further accelerate repair and recovery in patients with spinal cord injuries.
Next Steps: Cervical Injury Trial
A second spinal cord injuries stem cell trial is currently in development, with its focus on subjects with cervical injuries — a higher injury level that affects a larger proportion of patients with complete paralysis.
This progression reflects the standard clinical research model: establish safety in a Phase I trial, then expand to larger populations and higher doses in Phase II and Phase III studies. Each step brings researchers closer to a therapy that could restore meaningful function to patients who currently have no effective treatment options.
This research also connects to broader advances in neurotechnology. For those interested in other approaches to restoring communication and function in patients with complete neurological impairment, our article on brain computer interface ALS covers parallel breakthroughs in neural interface research.
According to the National Spinal Cord Injury Statistical Center, approximately 18,000 new spinal cord injury cases occur in the United States each year, with an estimated 300,000 people currently living with a spinal cord injury — making advances in the spinal cord injuries stem cell trial field a critical area of medical research.