Repeated intrathecal injections of autologous bone marrow-derived mesenchymal stem cells for spastic cerebral palsy: Single-arm safety and preliminary efficacy clinical trial

Cerebral palsy (CP) is the most prevalent motor disability among children, for which effective treatment options remain limited. This research sought to evaluate the safety and initial effectiveness of multiple intrathecal administrations of autologous bone marrow-derived mesenchymal stem cells (MSCs) in children with spastic CP.

A phase Ⅰ, prospective, single-arm, open-label clinical trial was conducted on 16 children aged 2–12 years with spastic CP. Patients received four intrathecal injections of MSCs at monthly intervals. Safety was evaluated based on the occurrence of adverse events, while efficacy was assessed using various clinical scales, including the Gross Motor Function Classification System (GMFCS), Gross Motor Function Measure (GMFM), Berg Balance Scale (BBS), and the Modified Ashworth Scale (MAS). Assessments were conducted at baseline before treatment, and at a 12-month post-treatment follow-up.

Safety assessment revealed no severe adverse events. Mild adverse events included nausea (18.75%), fatigue (12.5%), and headache (12.5%). Statistically significant improvements were observed in motor function, balance, and spasticity. GMFCS levels improved, with the proportion of patients in Level Ⅴ decreasing from 62.5% to 43.75% (p = 0.0047). Total GMFM scores increased significantly from 47.01 ± 21.45 to 54.69 ± 22.62 (p < 0.001), with domain-specific improvements in lying (p = 0.002), sitting (p = 0.001), crawling (p = 0.002), standing (p = 0.004), and walking (p = 0.043). BBS scores improved from 6.75 ± 10.88 to 9.88 ± 13.60 (p = 0.001), while MAS reduced from 2.25 ± 1.18 to 1.62 ± 0.96 (p = 0.004). Functional independence improved significantly, with FIM motor scores increasing from 38.63 ± 24.35 to 47.44 ± 25.27 (p = 0.001) and cognitive scores from 57.06 ± 29.15 to 70.88 ± 26.96 (p = 0.001).

Repeated intrathecal injections of autologous MSCs are safe and significantly improve motor function, balance, and spasticity in children with spastic CP. These findings suggest that MSC therapy enhances functional outcomes and quality of life, though larger randomized controlled trials are needed.