Aim: The objective of this research was to assess the effectiveness of combining autologous BMMNC intrathecal infusions with an educational intervention, comparing it to the sole utilization of educational interventions in the management of Autism spectrum disorder (ASD). Methods: This randomized phase II clinical trial was conducted from December 2019 to December 2023. Fifty-four children with ASD were randomly assigned to two groups: Group A received autologous BMMNC administration plus educational intervention, and Group B underwent educational intervention alone (control group). Group A received two rounds of autologous BMMNC intrathecal infusion at six-month intervals. Both groups received the same educational intervention. The efficacy outcomes were evaluated by the change in ASD severity according to DSM-5, Childhood Autism Rating Scale (CARS), the Vineland Adaptive Behavior Scales (VABS), Clinical Global Impression-Severity scale (CGI-S), and Clinical Global Impression-Improvement (CGI-I) scale. The follow-up duration was twelve months. Results: The average age was 4.8 ± 1.0 years. No severe adverse events were linked to the intervention. Noteworthy differences were observed between the groups over the 12-month period in terms of improvements in DSM-5, CARS, VABS, and CGI-S. The percentage of individuals categorized at level 3 (requiring very substantial support') decreased from 85.2% at baseline to 36.0% after 12 months in Group A, compared to a change from 85.2% to 76.0% in Group B (p<.01). In Group A, the CARS score significantly decreased from 46.7 ± 3.4 to 40.8 ± 5.1, while the control group experienced a change from 45.0 ± 4.7 to 43.7 ± 5.5 (p < .01). Similarly, in Group A, the VABS score increased from 56.8 ± 4.4 to 65.4 ± 6.2, whereas the control group showed a change from 58.3 ± 5.7 to 59.3 ± 6.7 (p < .01). Additionally, Group A demonstrated a more substantial change in CGI-S and CGI-I compared to the control group. Conclusion: Autologous bone marrow mononuclear cell transplantation in combination with behavioral intervention seems to be safe and has clinical efficacy in treatment of children with ASD.

Prof. Nguyen Thanh Liem is a pioneer and innovator in pediatric endoscopic surgery and stem cell transplantation.

He graduated from Hanoi Medical University in 1976 and completed medical residency program in Surgery at Viet Duc Hospital in 1979. He defended his PhD. thesis in 1991 and was appointed as a Medical Professor in Surgery in 2009.

He is internationally recognized inventor by his seven advanced operative techniques in the Pediatric Surgery. Regarding the stem cell studies, Prof. Liem is a pioneer of using stem cell therapy for unmet diseases in children such as cerebral palsy due to oxygen deprivation, due to neonatal icterus, due to intracranial hemorrhage, neurological sequelae after nearly death drowning, Autism, bronchopulmonary dysplasia, and liver cirrhosis due to biliary atresia.Throughout his career, he has been in charge of a number of important positions such as Director of Vietnam National Children Hospital from 2002 to 2012; Director of Vinmec International Hospital from 2013-2016; Vice president of Asian Association of Pediatric Surgery; President of Vietnam Association of Pediatric Surgery. Currently, Prof. Liem is the director of Vinmec Research Institute of Stem Cell and Gene Technology. 

He has published 125 studies on the international journals and over 200 papers on domestic journals. He is the Editor and author of six chapters of the Book titled “Cell Therapy: Stem Cells and Regenerative Medicine” which is shortly published by Springer Nature in early 2024. He is also the co-author of surgery pediatrics textbooks published in UK and US such as Operative Pediatric Surgery , Ashcraft’s Pediatric Surgery, and Pediatric Laparoscopic and Thoracospoc Surgery. 

Prof. Nguyen Thanh Liem has been invited to give lectures and performed demonstrative surgeries in many countries including the US, France, Netherlands, Italia, Australia, Japan, India, South Korea...

By his dedicated and delightful contributions to the medical practices and research, he has been honored many prestige awards conferred by the Vietnam government like Ho Chi Minh Award, National Labor Hero Medal, Award of Vietnam Talent.

In 2018, he has been awarded the 2018 Nikkei Asia Prize in the Science and Technology category thank to his contribution to develop and popularize pediatric endoscopic surgery in Asia and the world. He is categorized in top 100 scientists of Asia by Sigapore journal in 2019.