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Clinical trial looks at treating ASD using umbilical stem cells

Researchers show that injecting umbilical mesenchymal stem cells to children with ASD is safe and generally well tolerated. In their small study group, 40% of children showed improvement in symptoms, indicating the merits of more extensive studies to look at mesenchymal stem cells as a treatment option for ASD.

What is ASD?

Autism spectrum disorder (ASD) is characterized by developmental disabilities that result in deficiencies in communication, behaviour and social interactions. Symptoms of the disease vary from child-to-child and may be associated with other disorders such as anxiety, ADHD, sleep disorders and even gastrointestinal problems.

What’s a clinical trial?

Clinical trials are experiments that look at the effect of new treatments on humans. Clinical trials involving stem cells usually look at the safety and effectiveness of using stem cells to treat diseases like ASD.

At the discovery and preclinical stages, testing involves non-human subjects. Phase 1 trials are run on healthy volunteers to find out if a drug is safe in humans. If a therapy passes Phase 2 trials, where a small number of patients are tested, it can be advanced into Phase 3 trials where therapeutic effects can be examined.

The current trial was a mixed phase 1/2 trial involving 20 children with ASD. They received stem cell infusions every 12 weeks for four treatments over 9 months. These children were then followed for one year, and the symptoms were assessed at three months and twelve months after treatment.

What did the study find?

  • Repeated infusions of umbilical mesenchymal stem cells were safe and generally well tolerated.
  • In a subset of the children, it improved symptoms associated with ASD.

Based on their results, the researchers concluded that this small-scale study lends support for further investigating umbilical cord mesenchymal stem cells to treat neurological conditions like ASD.

Riordan, NH, Hincapié, ML, Morales, I et al. (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 Cell Translational Medicine. 8(10): 1008-1016. doi: https://doi.org/10.1002/sctm.19-0010.
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