Stem Cell Treatment and Cerebral Palsy Report Summary

Major Points and Findings:

1. Background: Cerebral palsy (CP) is a severe motor disability that can result from perinatal complications. It is a non-progressive disease with a prevalence of 1-2 per 1000 live births in developed countries. Given the extensive and multidisciplinary care CP demands, it poses a challenge to the health system and is burdensome for patients and their families. Recently, stem cell therapy has been viewed as a potential treatment option, offering hope to patients and their families.
2. Aim: The study aimed to evaluate the efficacy and safety of stem cell treatment in children with CP through a systematic review and meta-analysis.
3. Methods: A systematic literature search was conducted on PubMed and EMBASE to find randomized controlled clinical trials (RCT) that investigated the effect of stem cell transplantation in children with CP. The primary focus was on the change in gross motor function, quantified using the gross motor function measure.
4. Results: Out of the identified RCTs, 8 were selected for a qualitative review, and 5 met the criteria for inclusion in the meta-analysis. These trials encompassed a patient population ranging from 0.5 to 35 years, with a total of 282 participants. The findings revealed a significant improvement in gross motor function in the stem cell group, with a pooled standard mean difference of 0.95 (95% confidence interval: 0.13-1.76). However, there was high heterogeneity (I^2 = 90.1%). Serious adverse events were rare and were distributed equally between the intervention and control groups.
5. Conclusion: Compared to symptomatic standard care alone, stem cell therapy for CP showed a significant positive effect on gross motor function. However, the magnitude of improvement is limited. The short-term safety of the treatment is confirmed, but further high-quality RCTs are needed to solidify these findings.
6. Core Tip: CP results from perinatal complications and is a severe motor disability. Stem cell therapy has recently emerged as a promising treatment option, raising hopes for patients and their families. The meta-analysis of available trials indicates a slight but significant positive effect on gross motor function in favor of the stem cell group.