Safety and Feasibility of Intravenous Administration of Autologous Stem Cells from Human Exfoliated Deciduous Teeth (SHED) in Children with Cerebral Palsy: A First-in-Human Phase I Clinical Trial (Interim Analysis)

Background Cerebral palsy (CP), often resulting from perinatal hypoxic-ischemic encephalopathy (HIE), is a lifelong neurological disorder with no curative treatment. Stem cells from human exfoliated deciduous teeth (SHED), derived from the neural crest, have demonstrated therapeutic potential in preclinical CP models, improving motor function even in the chronic phase. We conducted a first-in-human Phase I clinical trial to evaluate the safety and feasibility of autologous SHED therapy in children with CP. Methods This single-arm, open-label Phase I trial was conducted at Nagoya University Hospital. Eligible participants were children aged 6–11 years with CP (Gross Motor Function Classification System level III) associated with perinatal HIE. Three patients received a single intravenous infusion of autologous SHED at a target dose of 3 × 10⁶ cells/kg. The primary outcome was the occurrence of any adverse events (AEs within 4 weeks post-infusion. Exploratory efficacy outcomes, including the Gross Motor Function Measure-66 (GMFM-66), were assessed at 12 weeks. Results This interim analysis reports on the three enrolled patients. All patients completed the SHED infusion without acute infusion-related reactions. No adverse events possibly related to SHED administration were observed during the 4-week follow-up period. The AEs observed (e.g., acute gastroenteritis) were Grade 1, transient, and assessed by investigators as unrelated to the cell infusion. Laboratory tests, electrocardiograms, and chest radiographs revealed no clinically significant abnormalities. At 12 weeks, GMFM-66 scores improved in all three patients (Case 1: 45.3 to 50.3; Case 2: 41.6 to 43.4; Case 3: 47.3 to 50.1), with an average increase of 3.2 points. Conclusions This interim analysis suggests that the intravenous administration of autologous SHED is safe and feasible in children with CP in the chronic phase. No serious adverse events were observed, and preliminary signs of motor improvement were noted. These findings provide a basis for further investigation in larger, controlled trials. Trial registration Japan Registry of Clinical Trials (jRCTb040230042)