Recent studies in human cohorts and mouse models have demonstrated a link between the gut microbiota and Autism Spectrum Disorder (ASD) symptoms, suggesting that the gut microbiota may play a critical role in gastrointestinal and neurological symptoms associated with ASD. Using a Microbiota Transplant Therapy (MTT) approach, we modified the gut microbiome of children with autism in an open-label clinical trial. Following microbiome modification, gastrointestinal symptoms and behavior improved significantly, with benefits persisting after two years of follow-up. Using a multi-omic approach, we are analyzing microbes, pathways, genes, and metabolites that likely contributed to the trial’s success and to uncover candidate biomarkers or therapeutic targets. I will present results from this trial, as well as preliminary findings from a placebo-controlled MTT trial in adults with ASD.
Dr. Rosa Krajmalnik-Brown is the director of the Biodesign Center for Health Through Microbiomes and the Helmick Professor in Environmental Engineering, at the School of Sustainable Engineering and The Built Environment, at Arizona State University. She joins the 2025 class of the National Academy of Inventors. She came to the US with a Fulbright Scholarship to get a Ph.D. in Environmental Engineering from Georgia Tech. She was awarded an NSF CAREER award, was selected Fulton Engineering Exemplar Faculty, in 2020 she was awarded Arizona Researcher of the year by AZBio and has been recognized as highly cited researcher in her field by Web of Science in 2020, 2021, and 2023. She is a pioneer in research on gut microbial interactions. She is author of 13 US granted patents and more than 150 peer-reviewed publications. She is a co-owner and founder of two start-up companies. She specializes on the human intestinal microbial ecology and its relationship to human metabolism, and autism.