Stem Cell Treatment for Autism Tested in Humans

Study in a Sentence: In a phase I clinical trial with 25 children under the age of 6, researchers recently demonstrated the safety and feasibility of using self-derived cord blood frozen and stored from birth to treat autism, an increasingly common neurodevelopmental disorder impairing children’s ability to learn and socialize.

Healthy for Humans: Although the study did not contain a control group, promising improvements in the patients’ symptoms and social communication skills were observed over the course of a year. The efficacy of this treatment will be better tested and quantified in follow-up clinical trials with control groups for comparison. 

Redefining Research: By conducting this phase I clinical trial in humans to test the safety of this treatment, there is no concern about the translatability of the results to humans. It also allowed the researchers to identify promising outcome measures for next phase clinical trials, thus accelerating the therapeutic pipeline without compromising patient safety.