Background:
- Autism is a pervasive neurodevelopmental disorder with no defined mechanisms of pathogenesis, and therapy is mostly limited to behavioral interventions.
- Stem cell transplantation may offer a unique treatment strategy for autism due to the observed immune and neural dysregulation in the disease.
- This study is a non-randomized, open-label, single-center phase I/II trial that investigated the safety and efficacy of combined transplantation of human cord blood mononuclear cells (CBMNCs) and umbilical cord-derived mesenchymal stem cells (UCMSCs) in treating children with autism.
Methods:
- 37 subjects diagnosed with autism were enrolled and divided into three groups:
- CBMNC group (14 subjects) - received CBMNC transplantation and rehabilitation therapy.
- Combination group (9 subjects) - received both CBMNC and UCMSC transplantation and rehabilitation therapy.
- Control group (14 subjects) - received only rehabilitation therapy.
- Transplantations included four stem cell infusions through intravenous and intrathecal injections once a week.
- Treatment safety was evaluated with laboratory examinations and clinical assessment of adverse effects.
- The Childhood Autism Rating Scale (CARS), Clinical Global Impression (CGI) scale, and Aberrant Behavior Checklist (ABC) were used to assess therapeutic efficacy before and after treatment.
Results:
- No significant safety issues were related to the treatment, and no severe adverse effects were observed.
- Statistically significant differences were shown on CARS, ABC scores, and CGI evaluation in the two treatment groups compared to the control at 24 weeks post-treatment (p < 0.05).
Conclusions:
- Transplantation of CBMNCs demonstrated efficacy compared to the control group.
- The combination of CBMNCs and UCMSCs showed larger therapeutic effects than the CBMNC transplantation alone.
- No safety issues were noted during infusion and the monitoring period.