Numerous research studies have demonstrated that stem cell therapy is safe, well tolerated and feasible in children with ASD. Stem cell therapy has been shown to not only reduce inflammatory markers and influence changes to the central nervous system but also improve behavior, socializing, nonverbal communication, and lethargy in children with ASD. These benefits and other therapeutic properties make stem cell therapy a promising option in the treatment of ASD.
Based on the early human clinical trials there appears to be a strong argument for the safe and effective use of stem cell therapy in the treatment of ASD. While we wait for phase II/III trials the current data is encouraging and supports use of stem cell therapy in the clinical setting.
Below we highlight and summarize selected studies demonstrating the safety and efficacy of stem cell therapy in children with ASD. All of the selected studies used intravenous administration but used different cell types and tissue types.
Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for the Treatment of Autism Spectrum Disorder in Children: Safety Profile and Effect on Cytokine Levels (Riordan et al 2019)
In 2019 Riordan et al conducted a clinical trial to assess the safety and efficacy of intravenous infusions of umbilical cord derived mesenchymal stem cells (UCMSCs) in patients with ASD. Patients aged 6-16 years old received a single infusion every 12 weeks over the course of 9 months for a total of 4 infusions. Over 12 month follow up period most of the adverse events were ‘not related’ or ‘not likely related’ to stem cell treatment. Adverse events related to treatment were mild or moderate and short in duration. They included mild inflammation, swelling and/or redness at infusion site, mild fatigue, headache, fever, etc. which are commonly reported in treatments with stem cells. There were no treatment related serious adverse events. 40% of children had improvements of symptoms achieving a lower ASD symptom category measured by standardized diagnostic tools; Childhood Autism Rating Scale (CARS) and Autism Treatment Evaluation Checklist (ATEC). Of these patients, 62.5% improved from mild or moderate autism symptoms to below threshold for autism and 37.5% improved from severe autism symptoms to below threshold. Laboratory results showed that intravenous stem cell therapy reduced inflammatory cytokines MDC and TARC, which are higher in individuals with ASD compared to general population by 20%. Riordan et al concluded that “The administration of repeated-dose UC-MSC infusions is safe and tolerable for patients with ASD” and “the trends observed in this study are indicative of potential therapeutic benefits, reflected in lower CARS and ATEC scores that may be associated with decreases in TARC and MDC levels.”
Safety and Observations from a Placebo-Controlled, Crossover Study to Assess Use of Autologous Umbilical Cord Blood Stem Cells to Improve Symptoms in Children with Autism (Chez et al 2018)
In 2018 Chez et al conducted a trial to assess the safety of a single intravenous infusion of autologous umbilical cord blood (AUCB) in patients aged 2 to 6 years old with ASD and to document changes in language, social behavior and learning. Umbilical cord blood contains a mixed population of cells and is rich in stem cells. The results of this study showed that treatment with intravenous AUCB was safe in children with ASD. Over the 24 week follow up period, there were minimal treatment related adverse events and none required treatment. There were no allergic reactions and no serious adverse events associated with the treatment. Chez et al used several standardized assessment tools to assess changes in language, social behavior and learning including Expressive One Word Picture Vocabulary Test (EOWPVT-4), Receptive One Word Picture Vocabulary Test (ROWPVT-4), Stanford-Binet Fluid Reasoning (SBFR) and Knowledge subtests (SBKN), Vineland Adaptive Behavior and Socialization Scales and others. Although there were no statistically significant differences in these measurements between treatment and placebo group, there were trends in improvement, specifically in socialization. Chez et al concluded that “The present study provided further evidence that treatment with AUCB is safe but there was minimal evidence of clinical effectiveness.”
Autologous Cord Blood Infusions Are Safe and Feasible in Young Children with Autism Spectrum Disorder: Results of a Single-Center Phase I Open-Label Trial (Dawson et al)
In 2017 Dawson et al conducted a trial to assess the safety and efficacy of a single intravenous infusion of autologous umbilical cord blood (AUCB) in children aged 2-6 years old with ASD. The results of this study showed that treatment with intravenous AUCB was safe and well tolerated. Over the 12 month follow up period, all adverse events (AEs) were either mild or moderate and there were no serious adverse events or treatment related infections reported. The most common treatment related AEs was allergic reaction (Itchy skin and/or cough) and the most common unrelated AEs were agitation, skin changes and typical childhood infections. Dawson et al found significant improvements in behavior across a wide range of parent reported and clinician-rated behavioral assessment tools including VABS-II, Socialization, Communication and Adaptive Behavior Scores, PDDBI, and several others. Behavioral changes occurred during the first 6 months and were sustained at 12 months post-infusion. Dawson et al concluded that “intravenous infusion of autologous umbilical cord blood in young children with ASD is safe and feasible. We describe significant improvements in behavior observed in the first 6 months post-infusion and sustained at 12 months.”
Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism (Lv et al)
In 2013 Lv et al conducted a trial to investigate the safety and efficacy of intravenous and intrathecal (into spinal canal) administered cord blood derived mononuclear cells (CBMNCs) and umbilical cord derived mesenchymal stem cells (UCMSCs) in patients with Autism. Patients were divided into three groups, each group received rehabilitation. CBMNC group received CBMNCs via one IV treatment and 3 intrathecal treatments; Combination group received CBMNCs via 1 one IV treatment and one intrathecal treatment and UCMSCs via two intrathecal treatments; Control group received rehabilitation only. During the 24 week follow up period, there were no allergic reactions, no serious adverse events and no significant changes in laboratory tests between groups. Five patients experienced transient low grade fever and recovered without medical intervention. Compared to the control group, the CBMNCs and Combination group had improvements in numerous areas including visual, emotional and intellectual responses, adaptation to change fear or nervousness, nonverbal communication and activity levels as measured by Childhood Autism Rating Scale (CARS). The two treatment groups also had improvements in lethargy/social withdrawal, hyperactivity and inappropriate speech as measured by Aberrant Behavior Checklist (ABC). Furthermore, both treatment groups showed statistically significant differences in Clinical Global Impression scale (CGI) when compared to control group with the Combination group having more improvement. 88.89% of patients in Combination group and 50% in CBMNC group were ‘very much improved’ and ‘much improved’ on CGI-GI scale at 24 week follow up. Overall, the combination of CBMNCs and UCMCSs showed larger therapeutic effects than CBMNC alone. Lv et al concluded that “CBMNC and UCMSC transplantation may improve some behavioral symptoms and function observed in children with autism.”
SOURCES OF SELECTED STUDIES:
- Riordan, N. H., Hincapié, M. L., Morales, I., Fernández, G., Allen, N., Leu, C., … Novarro, N. (2019). Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for the Treatment of Autism Spectrum Disorder in Children: Safety Profile and Effect on Cytokine Levels. Stem Cells Translational Medicine. https://doi.org/10.1002/sctm.19-0010
- Chez, M., Lepage, C., Parise, C., Dang-Chu, A., Hankins, A., & Carroll, M. (2018). Safety and Observations from a Placebo-Controlled, Crossover Study to Assess Use of Autologous Umbilical Cord Blood Stem Cells to Improve Symptoms in Children with Autism. Stem Cells Translational Medicine, 7(4), 333–341. https://doi.org/10.1002/sctm.17-0042
- Dawson, G., Sun, J. M., Davlantis, K. S., Murias, M., Franz, L., Troy, J., … Kurtzberg, J. (2017). Autologous Cord Blood Infusions Are Safe and Feasible in Young Children with Autism Spectrum Disorder: Results of a Single-Center Phase I Open-Label Trial. Stem Cells Translational Medicine, 6(5), 1332–1339. https://doi.org/10.1002/sctm.16-0474
- Lv, Y. T., Zhang, Y., Liu, M., Qiuwaxi, J. na ti, Ashwood, P., Cho, S. C., … Hu, X. (2013). Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism. Journal of Translational Medicine, 11(1). https://doi.org/10.1186/1479-5876-11-196
Brought to you by:
Ahvie Herskowitz, MD, President of ACAM
Director of Anatara Medicine
Clinical Professor of Medicine at UC San Francisco (2014)
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