Autologous cellular therapy for cerebral palsy: a randomized, crossover trial

Area of Science:

• Neuroscience
• Regenerative Medicine
• Pediatrics

Background:

• Cerebral palsy (CP) is a group of movement disorders due to brain damage.
• Current CP treatments focus on managing symptoms, with limited options for neuroregeneration.
• Autologous stem cell therapy offers a potential regenerative approach for CP.

Purpose of the Study:

• To assess the safety of autologous mononuclear cell therapy in children with cerebral palsy.
• To evaluate the impact of this therapy on motor function using the Gross Motor Function Measure (GMFM).
• To investigate changes in corticospinal tract microstructure via radial diffusivity measurements post-treatment.

Main Methods:

• A Phase 1/2a, randomized, blinded, placebo-controlled, crossover study.
• Participants (children aged 2-10 with CP) received either autologous bone marrow-derived mononuclear cells or autologous umbilical cord blood.
• Follow-up included assessments at 6, 12, and 24 months, with crossover for placebo group at 12 months.

Main Results:

• No adverse events related to stem cell infusion were reported, indicating safety.
• No significant improvements in overall motor function (GMFM) were observed.
• Decreased corticospinal tract radial diffusivity, suggesting improved myelination, was noted in treated participants, correlating with handedness.

Conclusions:

• Autologous mononuclear cell therapy is safe for treating cerebral palsy in children.
• The therapy may promote microstructural improvements in the corticospinal tract, potentially related to myelination.
• Larger trials require advancements in cord blood banking for feasibility.