Autologous BM-MSC therapy combining Psychological education treating Autism

Cell & Gene Therapy Clinical Phase 2 Neurological diseases

Project Title: Autologous bone marrow mononuclear cell administration combined with educational intervention in the treatment of autism spectrum disorder: a randomized, open-label, controlled phase II clinical trial

Principal Investigator (PI): Prof. Nguyen Thanh Liem

Objective:

To evaluate the effectiveness of intrathecal autologous bone marrow mononuclear cell (BMMNC) therapy combined with education compared with education alone for the treatment of autism spectrum disorder (ASD).

Project Description (main page – 250 words):

This study evaluated the effectiveness of intrathecal autologous bone marrow mononuclear cell infusion combined with educational intervention compared with educational intervention alone in the treatment of autism spectrum disorder. A total of 54 children with autism spectrum disorder aged 3 to 7 years were randomly assigned, with 50 children completing the study, 25 in each group. The cell therapy group received two bone marrow mononuclear cell infusions six months apart in combination with educational intervention, while the control group received educational intervention only. Outcomes were assessed at baseline and at 2, 6, and 12 months based on autism severity (DSM-5), the Childhood Autism Rating Scale (CARS), and the Clinical Global Impression-Severity (CGI-S) scale scores and (2) improvements in social interaction, adaptive behavior, and daily living skills measured by the Vineland Adaptive Behavior Scales (VABS-II) and Clinical Global Impression-Improvement (CGI-I) scale scores. After 12 months, the intervention group showed a markedly greater reduction in the proportion of children at the most severe level compared with the control group; autism severity and clinical severity scores decreased more substantially, while adaptive functioning and daily living skills improved to a greater extent than in the education only group, indicating that the combination of bone marrow mononuclear cell infusion and educational intervention was more effective than educational intervention alone.