Stem Cell Therapy Enables Paralyzed Patient to Stand: A Milestone in Regenerative Medicine

In a significant advancement in the field of regenerative medicine, a clinical trial conducted at Keio University in Tokyo, Japan, has demonstrated promising outcomes for spinal cord injury (SCI) treatment. A Japanese man, previously paralyzed due to a severe SCI, has regained the ability to stand independently following an innovative intervention utilizing induced pluripotent stem (iPS) cells. This pioneering study, led by stem cell researcher Hideyuki Okano, MD, PhD, represents a critical step forward in the quest to restore function in patients with paralysis.

 

Clinical Trial Design and Methodology

The trial, initiated in December 2021, involved four adult male participants, all classified with grade A injuries on the American Spinal Injury Association (ASIA) Impairment Scale, indicating complete paralysis with no motor or sensory function below the injury site. Each patient received an injection of approximately two million neural precursor cells, derived from donor iPS cells, directly into the site of their spinal cord injury. These iPS cells, reprogrammed from adult cells into an embryonic-like state, were engineered to differentiate into neurons and glial cells to promote neural regeneration. To mitigate the risk of immune rejection, participants were administered immunosuppressive therapy for six months post-procedure.

Pr Okano, Hideyuki

Promising Outcomes in Functional Recovery

Remarkably, one patient achieved a dramatic recovery, progressing from ASIA grade A to grade D, enabling him to stand unassisted and initiate rehabilitation to regain walking ability. A second participant improved to ASIA grade C, regaining limited movement in his arms and legs. However, the remaining two participants exhibited minimal improvement, highlighting the variability in treatment response. While these results are encouraging, the study’s small sample size and lack of peer review underscore the need for cautious interpretation.

Safety Profile and Mechanisms of Action

iPS cell

The intervention’s safety profile was a key focus, with no serious adverse events reported, marking a significant milestone for iPS cell-based therapies in SCI. Imaging studies suggested that some transplanted cells survived, potentially contributing to neural repair by increasing nerve density or secreting regenerative factors. Nonetheless, challenges remain, including the low survival rate of transplanted cells, as many do not persist beyond the initial hours post-injection.

Building on Preclinical Foundations

This trial builds on prior preclinical research by Dr. Okano, who demonstrated neural regeneration in paralyzed monkeys using similar iPS cell techniques. The current findings, announced on March 21, 2025, have yet to undergo peer review but align with the growing body of evidence supporting stem cell therapies for SCI. Experts, including Toru Ogata, MD, PhD, from the University of Tokyo, have described the outcomes as “groundbreaking,” particularly for a grade A patient achieving such significant functional recovery.

No Serious Adverse Health Effects Observed 

Future Directions and Challenges

Despite the promise, researchers emphasize the necessity for larger, controlled trials to distinguish treatment effects from spontaneous recovery, which can occur in some SCI cases. K Pharma, a biotechnology startup founded by Dr. Okano, plans to seek approval for expanded clinical studies to further validate the therapy’s efficacy and optimize its application across diverse SCI patient populations.

A New Horizon for SCI Patients

This study represents a beacon of hope for the estimated 150,000 individuals in Japan and millions worldwide living with SCI-related paralysis. While not yet a definitive cure, these findings highlight the transformative potential of iPS cell therapy in restoring lost neurological function, paving the way for future innovations in regenerative medicine.