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A breakthrough approach designed to repair damaged spinal cord wiring is moving a step closer to human trials
Researchers from Keio University in Japan reported promising results from preclinical studies of specialized stem cells known as gliogenic neural stem/progenitor cells (gNS/PCs), which are intended to help restore function in people with long-standing spinal cord injuries.
The researchers are now planning a physician-initiated clinical trial, with patient recruitment expected to begin in 2027.
Building on Earlier Success
The latest work follows a first-in-human clinical study involving patients with subacute spinal cord injuries, which demonstrated a promising safety profile.
“We have already successfully completed a world-first, first-in-human clinical study targeting patients in the subacute phase, which demonstrated a promising safety profile,” said Dr. Hideyuki Okano of Keio University, who also serves as president of ISSCR.
“Our shift to the chronic phase is the next logical milestone, built upon that solid foundation. Since the cellular environment changes over time, we are evolving our strategy from just establishing safety to actively overcoming the stubborn, long-standing barriers of chronic paralysis.”
A Different Approach to Repair
Unlike some previous stem cell therapies that aimed to generate new nerve cells, the new strategy focuses on repairing existing nerve fibers that have lost their protective coating, known as myelin.
The upcoming trial will target people with chronic, incomplete spinal cord injuries who still have surviving nerve fibers, but whose nerves have become demyelinated and lack enough natural support cells.
“While previous approaches focused on transplanting ‘neuron-generating’ cells, chronic injuries present a different challenge: residual nerve fibers exist but have lost their protective insulation,” Okano said.
“Our unique strategy uses gliogenic cells (gNS/PCs), which are specifically tailored to generate the vital support cells—astrocytes and oligodendrocytes. Rather than trying to grow entirely new nerves, we are essentially ‘repairing and rebooting’ the existing wiring of the spinal cord.”
In simple terms, the therapy aims to help damaged nerve pathways work more effectively by restoring the cells that support and insulate them.
Promising Preclinical Results
In laboratory studies, the stem cells developed into neurons, astrocytes and oligodendrocytes—the key cell types needed to support nerve function.
When transplanted into models of chronic spinal cord injury, the cells safely promoted behavioral recovery without causing tumorlike tissue formation. The treatment also helped remodel the damaged environment around the injury site, according to the researchers.
Although the findings have not yet been tested in people with chronic spinal cord injuries, the results suggest the approach could potentially help restore movement and other functions if future clinical studies prove successful.
Looking Ahead
The researchers say their long-term goal is to develop a safe, standardized treatment capable of restoring voluntary movement and autonomic function in people with chronic spinal cord injuries.
If the upcoming clinical trial confirms the therapy’s safety and effectiveness, it could mark a significant advance for patients living with paralysis years after their original injury.
For now, however, the treatment remains experimental, with human testing in chronic spinal cord injury patients expected to begin in 2027.
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