Research progress on the therapeutic efficacy of human umbilical cord mesenchymal stromal cells for cerebral palsy in children

Cerebral palsy (CP) is a non-progressive disorder of the central nervous system resulting from brain damage in the developing fetus or infant. It is primarily characterized by motor and postural abnormalities that severely impair motor function, speech, cognition, and mental health. Current comprehensive treatment approaches—including rehabilitation, pharmacotherapy, and surgical interventions—offer only limited symptomatic relief and fail to address the underlying neurodevelopmental deficits. Therefore, there remains an urgent clinical need for innovative therapies capable of fundamentally repairing neural damage and restoring functional neural circuitry. In recent years, umbilical cord mesenchymal stromal cells (UC-MSCs) have emerged as a promising therapeutic strategy for CP due to their unique biological properties, including low immunogenicity and potent paracrine activity. Preclinical studies have demonstrated that UC-MSCs can promote structural and functional reconstruction of damaged neural networks through multiple synergistic mechanisms, such as immunomodulation to reshape the immune microenvironment, neurotrophic support and neuroregeneration, and proliferative potential for cellular replacement. Notably, a recent study in Poland ( n = 152) provided large-sample validation of these effects. Following infusion of Human UC-MSCs (HUC-MSCs), the median increase in the Gross Motor Function Measure (GMFM) score was 1.9 points, with a significant negative correlation with age (Spearman's R = −0.38, p < 0.05). The median improvement in the 6-Minute Walk Test((6-MWT) was 75 m, which correlated with GMFM improvement (Spearman's R = 0.47, p < 0.05). The median reduction in Timed Up and Go test time was 2 s, and the magnitude of improvement was significantly correlated with baseline GMFM, 6-MWT gain, and age. The findings suggest that the functional benefits of HUC-MSCs are more pronounced in younger children and those with better baseline motor function. However, larger sample sizes and long-term follow-up studies are still required to validate the durability of these effects and establish long-term safety. This review systematically integrates the neurorestorative mechanisms of UC-MSCs, preclinical evidence, and the latest clinical data to provide a theoretical foundation and practical guidance for the future development of novel UC-MSC-based therapeutic strategies.

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Publication Details

Journal
Journal of Neurorestoratology
Published
2025-12-08
DOI
https://doi.org/10.1016/j.jnrt.2025.100266
Primary Topic
Mesenchymal stem cell research
Type
article
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article

Research progress on the therapeutic efficacy of human umbilical cord mesenchymal stromal cells for cerebral palsy in children

Yachen Wang, Jing Liu, Shujin Wang
Journal of Neurorestoratology
Mesenchymal stem cell research
article

Research progress on the therapeutic efficacy of human umbilical cord mesenchymal stromal cells for cerebral palsy in children

Yachen Wang, Jing Liu, Shujin Wang
article en

Abstract

Cerebral palsy (CP) is a non-progressive disorder of the central nervous system resulting from brain damage in the developing fetus or infant. It is primarily characterized by motor and postural abnormalities that severely impair motor function, speech, cognition, and mental health. Current comprehensive treatment approaches—including rehabilitation, pharmacotherapy, and surgical interventions—offer only limited symptomatic relief and fail to address the underlying neurodevelopmental deficits. Therefore, there remains an urgent clinical need for innovative therapies capable of fundamentally repairing neural damage and restoring functional neural circuitry. In recent years, umbilical cord mesenchymal stromal cells (UC-MSCs) have emerged as a promising therapeutic strategy for CP due to their unique biological properties, including low immunogenicity and potent paracrine activity. Preclinical studies have demonstrated that UC-MSCs can promote structural and functional reconstruction of damaged neural networks through multiple synergistic mechanisms, such as immunomodulation to reshape the immune microenvironment, neurotrophic support and neuroregeneration, and proliferative potential for cellular replacement. Notably, a recent study in Poland ( n = 152) provided large-sample validation of these effects. Following infusion of Human UC-MSCs (HUC-MSCs), the median increase in the Gross Motor Function Measure (GMFM) score was 1.9 points, with a significant negative correlation with age (Spearman's R = −0.38, p < 0.05). The median improvement in the 6-Minute Walk Test((6-MWT) was 75 m, which correlated with GMFM improvement (Spearman's R = 0.47, p < 0.05). The median reduction in Timed Up and Go test time was 2 s, and the magnitude of improvement was significantly correlated with baseline GMFM, 6-MWT gain, and age. The findings suggest that the functional benefits of HUC-MSCs are more pronounced in younger children and those with better baseline motor function. However, larger sample sizes and long-term follow-up studies are still required to validate the durability of these effects and establish long-term safety. This review systematically integrates the neurorestorative mechanisms of UC-MSCs, preclinical evidence, and the latest clinical data to provide a theoretical foundation and practical guidance for the future development of novel UC-MSC-based therapeutic strategies.

Journal of NeurorestoratologyVol. 14(2)
Openalex Percentile: Top 40%
Mesenchymal stem cell research
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