RESUMO
The Editor-in-Chief of the Journal of Translational Medicine is issuing an editorial expression of concern to alert readers that concerns have been raised regarding the ethics of this study [1] and the potential association of the risk of teratoma formation with the transplantation of embryonic stem cells. Appropriate editorial action will be taken once this has been fully investigated. The authors disagree with this notice.
RESUMO
BACKGROUND: The present study evaluated the efficacy and safety of human embryonic stem cell (hESC) therapy in patients with CP. MATERIALS AND METHODS: This analysis included patients (30 days-18 yr) with documented diagnosis of CP. The study consisted of four treatment phases (T1, T2, T3, T4) separated by gap phases. Efficacy of hESC therapy was evaluated based on Gross Motor Function Classification Scores Expanded and Revised (GMFCS-E & R; 1-good to 5-bad). RESULTS: Ninety one patients were included and all received hESC therapy in T1, 66 patients returned for T2, 38 patients for T3, and 15 patients for T4. Overall, 30.2% patients achieved GMFCS-E & R score 1 during the study with different number of patients achieving GMFCS score 1 by the end of each treatment phase (T1: 6 [6.6%]; T2: 7 [10.6%]; T3: 11 [28.9%]; and T4: 5 [33.3%]). All patients in up to 2 yr (n = 10), 2-4 yr (n = 10), 4-6 yr (n = 9), and 6-12 yr (n = 8) age groups except one of the 5 patients in the age group of 12-18 yr transitioned from GMFCS-E & R score 5 to lower scores by end of T1. Most patients transitioned to GMFCS-E & R score 2 (n = 34) from higher scores by end of T2. Eleven patients achieved GMFCS-E & R score 1 by end of T3. No serious adverse events were observed. CONCLUSION: Use of hESC therapy in patients with CP is effective and safe. hESC therapy has demonstrated significant improvement in GMFCS-E & R scale.