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Intrathecal autologous bone marrow stem cell therapy in children with autism: A randomized controlled trial
Narges Sharifzadeh1, Ali Ghasemi2, Jalil Tavakol Afshari3
1Psychiatry and Behavioral Sciences Research Center, Mashhad University of Medical Sciences, Mashhad, Iran.
Insights
Autologous bone marrow mesenchymal stem cell (BMMSC) injections showed safety and feasibility in children with autism spectrum disorder (ASD). However, this treatment demonstrated limited clinical efficacy compared to standard care over 12 months.
Area of Science:
- Neuroscience
- Regenerative Medicine
- Pediatric Neurology
Background:
- Autism spectrum disorder (ASD) is a complex neurodevelopmental condition.
- Current treatments for ASD focus on symptom management and behavioral interventions.
- Investigating novel therapeutic approaches like stem cell therapy is crucial for improving ASD outcomes.
Purpose of the Study:
- To evaluate the safety and efficacy of autologous bone marrow mesenchymal stem cell (BMMSC) treatment in children with ASD.
- To compare BMMSC therapy combined with standard care against standard care alone.
Main Methods:
- A randomized controlled trial involving 32 children (aged 5-15) diagnosed with ASD.
- Participants received either BMMSC intrathecal injections plus rehabilitation and risperidone, or rehabilitation and risperidone alone.
- Assessments included the Childhood Autism Rating Scale (CARS), Gilliam Autism Rating Scale-II (GARS-II), and Clinical Global Impression (CGI) at baseline, 6, and 12 months.
Main Results:
- No significant differences in CARS, GARS-II, or CGI global improvement were observed between the BMMSC and control groups over 12 months.
- A significant improvement in CGI-severity of illness was noted in the BMMSC intervention group compared to the control group.
- The study found intrathecal injection of autologous BMMSCs to be safe and feasible in this pediatric population.
Conclusions:
- Intrathecal administration of autologous BMMSCs is a safe and feasible procedure for children with ASD.
- BMMSC therapy demonstrated limited overall clinical efficacy in improving core ASD symptoms over a 12-month period.
- Further research may be needed to optimize stem cell therapy protocols for ASD treatment.
Introduction:
This study aimed to determine the safety and efficacy of treatment with autologous bone marrow mesenchymal stem cell (BMMSCs) compared with the routine treatment in children with autism spectrum disorder (ASD).
Methods:
In this ethically approved randomized controlled trial, 32 ASD children aged 5-15 years were randomly assigned to receive either autologous BMMSC plus rehabilitation therapy and risperidone (intervention group) or rehabilitation therapy and risperidone (control group). Autologous BMMSCs were intrathecally injected in the intervention group twice in 4 weeks. Patients were assessed using childhood autism rating scale (CARS), Gilliam autism rating scale-second edition (GARS-II), and clinical global impression (CGI) at the baseline, as well as 6 and 12 months after intervention.
Results:
Overall, 32 patients in two groups of intervention (n = 14) and control (n = 18) completed the study, of which 27 (84.4%) were male. Mean age was 9.50 ± 2.14 years. The improvements in CARS total score, GARS-II autism index, and CGI global improvement showed no significant differences between the groups over 12 months. However, the main effect for time*group interaction was significant regarding the CGI-severity of illness, showing a significantly more pronounced improvement in the intervention group (F = 6.719; P = .002).
Discussion:
Intrathecal injection of autologous BMMSCs seems to be safe and feasible, but has limited clinical efficacy in treatment of children with ASD.
