Measurable cognitive or adaptive gains occur in approximately 40–60% of treated children in published series. 'Success' varies: some show IQ point increases (5–15 points); others demonstrate improved social engagement or reduced behavioural symptoms without IQ change. Younger children (2–5 years) may respond better than older cohorts. Placebo effect and natural developmental variation are difficult to exclude in uncontrolled designs. Failure to improve is common, and no predictive biomarker currently identifies responders beforehand.
Two completed trials and one recruiting study form the current evidence base. Early-phase research focuses on safety in children aged 2–8 years with non-progressive intellectual disability. Published case series report modest improvements in adaptive behaviour and gross motor milestones over 6–12 months post-infusion. Functional MRI in a small cohort suggested increased activation in key learning circuits. No randomised controlled trials have been completed; comparisons are limited to historical natural history data. Effect magnitude remains unclear; blinded, sham-controlled designs are absent, limiting causal inference.
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由 StemCellAtlas 的编辑团队进行医学审查，与Kiian Nadiia, MD, PhD (Paediatric Neurologist · Medical Director, CSM Clinic Network · 12+ yrs in Autism Spectrum Disorders) 合作诊所 Stem Plus（索菲亚）的，根据 ISSCR、FDA 和 EMA 指南。 Educational information, not medical advice; figures indicative.
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