Autologous Bone Marrow Cell Therapy for Autism: An Open Label Uncontrolled Clinical Trial

Nguyen Thanh Liemn 1 *, Nguyen Hoang Phuong1, Anh Tuan Nguyen1, Chinh Duy Vu2 and Anh V Bui1

1 Department of Stem Cell and Immune Cell, Vinmec Research Institute of Stem Cell and Gene Technology, Vietnam

2 Department of Rehabilitation, Vinmec International Hospital, Vietnam

Aim: The aim of this study is to assess the safety and effectiveness of autologous bone marrow mononuclear stem cell (BMMNC) transplantation in patients with autism.

Materials and Methods: An uncontrolled clinical trial was carried out on 24 children with autism aged 3 to 16 years. The intervention consisted of two intrathecal administrations of stem cells.

Results: Improvements in behaviors, emotional and intellectual functions were seen in 19 patients (79.2%) while 2 cases (8.3%) showed no changes and 3 other cases (12.5%) presented with more severe symptoms. The overall severity of autism lessened after transplantation. The total CARS score was significantly improved. Of the 15 CARS domains, two domains (Body Use and Intellectual Response) showed statistically significant improvement 3 months after transplantation. After 6 months, significant improvement was also observed in 3 additional domains.

Conclusion: Autologous BMMNC transplantation appears to be a safe and effective therapy for patients with autism.

Trial Registration: ClinicalTrials.gov Identifier: NCT02627131 Registered on December 08, 2015