Outcomes of bone marrow mononuclear cell transplantation combined with interventional education for autism spectrum

Liem Nguyen Thanh1, Hoang-Phuong Nguyen1, Minh Duy Ngo2

  • 1Vinmec Research Institute of Stem Cell and Gene Technology (VRISG), Hanoi, Vietnam.

Insights

Autologous bone marrow mononuclear cell transplantation safely improved autism spectrum disorder (ASD) symptoms in children. This treatment, combined with educational intervention, significantly reduced ASD severity and enhanced adaptive behaviors.

Area of Science:

  • Regenerative Medicine
  • Neurodevelopmental Disorders
  • Clinical Trials

Background:

  • Autism spectrum disorder (ASD) presents significant challenges in social communication and behavior.
  • Existing interventions for ASD have varying degrees of efficacy, necessitating novel therapeutic approaches.
  • Autologous bone marrow mononuclear cells (BM-MNCs) offer potential regenerative properties.

Purpose of the Study:

  • To evaluate the safety and efficacy of autologous bone marrow mononuclear cell transplantation combined with educational intervention for children with autism spectrum disorder.
  • To assess the impact of this combined therapy on ASD severity and adaptive behaviors.

Main Methods:

  • An open-label clinical trial involving 30 children diagnosed with ASD.
  • Autologous bone marrow was harvested, mononuclear cells isolated, and infused intrathecally twice, 6 months apart.
  • Patients received 8 weeks of Early Start Denver Model educational intervention post-transplantation.

Main Results:

  • No severe adverse events were reported, indicating a safe treatment profile.
  • Significant reduction in autism severity, evidenced by a decrease in Childhood Autism Rating Scale (CARS) scores (P < .05).
  • Marked improvements in adaptive capacity (Vineland Adaptive Behavior Scales), social communication, language, and daily skills; reduction in repetitive behaviors and hyperactivity.

Conclusions:

  • Autologous bone marrow mononuclear cell transplantation combined with behavioral intervention is a safe and well-tolerated treatment for children with ASD.
  • The combined approach demonstrates significant efficacy in ameliorating core ASD symptoms and improving overall adaptive functioning.
  • This therapeutic strategy holds promise for improving outcomes in children with autism spectrum disorder.

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