New research presented today at the International Society for Stem Cell Research (ISSCR) 2026 Annual Meeting highlights a significant step toward developing a stem cell-based treatment for chronic spinal cord injury (SCI), a condition for which no effective restorative therapy currently exists.
Hideyuki Okano, M.D., Ph.D., Keio University, Japan, presented new preclinical efficacy data using clinical-grade gliogenic neural stem/progenitor cells (gNS/PCs) for chronic incomplete SCI, along with newly updated plans for a physician-initiated clinical trial targeting patient recruitment in 2027. The findings build on the successful completion of a world-first, first-in-human clinical study in patients with subacute SCI that 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. Okano, who also is ISSCR's president. "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."
The upcoming clinical trial will focus on patients with chronic, incomplete spinal cord injuries who retain inactive but demyelinated nerve fibers and lack sufficient endogenous cellular support, making them appropriate candidates for this remyelination-focused strategy.
"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," said Okano. "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 preclinical studies, the clinical-grade gNS/PCs efficiently differentiated into neurons, astrocytes, and oligodendrocytes in vitro and, following transplantation into chronic injury models, safely promoted behavioral recovery without tumor-like tissue formation while remodeling the injured microenvironment.
If confirmed in future clinical studies, the approach could represent a significant advance for people living with chronic spinal cord injury. The physician-initiated clinical trial is expected to begin recruiting patients in 2027, with the long-term goal of developing a safe, standardized therapy capable of restoring voluntary movement and autonomic function for chronic patients.
To learn more about ISSCR 2026 visit www.isscr2026.org
