A research on stem cell transplantation for treatment of autism has been done by Prof. MD. Nguyen Thanh Liem – Director of Vinmec Institute of Stem cell and Gene technology (VRISG). Results have been presented and discussed at the 3th Vinmec International Conference on Cell and Gene Therapy (VCGT-2019), on 31/10/2019 in Hanoi.
Nguyen Thanh Liem, MD.PhD.Prof.
ABSTRACT
Aim: To evaluate the safety and efficacy of autologous bone marrow mononuclear cell transplantation combined with educational intervention for children with autism spectrum disorder (ASD).
Methods: An open-label clinical trial was performed from July 2017 to August 2019 at Vinmec International Hospital, Hanoi, Vietnam. Thirty children with autism according to DSM-5 criteria with CARS scores ≥ 37 were selected. Bone marrow was harvested through anterior iliac crest puncture under general anesthesia. The volume collected was as follows: 8 ml/kg for patients under 10 kg; [80 ml + (body weight in kg - 10) × 7 ml] for patients above 10 kg. Mononuclear cells were isolated by Ficoll gradient and then infused intrathecally. The same procedure was repeated 6 months later. After the first transplantation, all patients underwent 8 weeks of educational intervention based on Early Start Denver Model (ESDM).
Results: There was no severe adverse event associated with transplantation. The severity of ASD was significantly reduced with CARS score from 49.9 ± 4.3 to 45.2 ± 5.4 (p<0.05). The adaptive capacity increased with change of VABS scores from 52.4 to 60.4. Social communication, language, and daily skills improved markedly at 18 months after transplantation. Conversely, repetitive behaviors and hyperactive manifestation decreased remarkably.
Conclusion: Autologous bone marrow mononuclear cell transplantation in combination with educational intervention was safely tolerated and improved clinical manifestations of children with ASD.
Trial registration: ClinicalTrials.gov identifier: NCT03225651. Registered on July 21, 2017
Key words: Autism spectrum disorder, bone marrow mononuclear cell, stem cell transplantation, educational intervention
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