In this new study published in Stem Cell Translational Medicine 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 of symptoms, indicating the merits of larger studies that look at the use of mesenchymal stem cells 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 very 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 only involves non-human subjects. Phase I 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 looked at.

The current trial was a mixed Phase I/II trial involving 20 children with ASD. They received stem cell infusions every 12 weeks for a total of four treatments over the course of 9 months. These children were then followed for 1 year and they symptoms were assessed at 3 months and 12 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
  • It lends support to further study of umbilical cord mesenchymal stem cells for neurological conditions like ASD.