The Damaged Spinal Cord Is a Suitable Target for Stem Cell Transplantation

Area of Science:

• Neuroscience
• Regenerative Medicine
• Clinical Trials

Background:

• Spinal cord injury (SCI) affects millions, necessitating regenerative repair options.
• Human central nervous system stem cells (HuCNS-SC) demonstrate engraftment and differentiation potential in preclinical models.
• Previous animal studies showing improved locomotor function informed the first human trial of HuCNS-SC cells.

Purpose of the Study:

• To assess the feasibility and long-term safety of surgical transplantation of HuCNS-SC cells in human SCI patients.
• To evaluate preliminary efficacy, including sensory and motor function changes, and neuroimaging outcomes.

Main Methods:

• A Phase I/IIa multisite trial involving 12 adult subjects with traumatic thoracic SCI (AIS A or B).
• Surgical transplantation of 20 million HuCNS-SC cells into the thoracic spinal cord.
• Six-year follow-up including sensory assessments, neuroimaging, and clinical evaluations.

Main Results:

• Demonstrated short- and long-term surgical and medical safety with well-tolerated immunosuppression.
• Preliminary efficacy indicated reliable sensory improvements in 5 out of 12 subjects.
• Neuroimaging showed no tumor formation or lesion malformation; no changes in lower limb motor scores were observed.

Conclusions:

• Short- and long-term safety and feasibility support considering HuCNS-SC cell transplantation for SCI patients.
• This study is a crucial step in advancing cell transplantation therapies for human SCI.