Microbiota Transfer Improves Gastrointestinal and Autism Symptoms: An OPEN Label Study

Friday, May 12, 2017: 5:00 PM-6:30 PM
Golden Gate Ballroom (Marriott Marquis Hotel)
D. W. Kang1, J. B. Adams1, A. C. Gregory2, T. Borody3, L. Chittick4, A. Fasano5, A. Khoruts6, E. Geis1, J. Maldonado1, S. McDonough-Means7, E. Pollard1, S. Roux4, M. J. Sadowsky6, K. Schwarzberg Lipson8, M. B. Sullivan9, J. G. Caporaso8 and R. Krajmalnik-Brown1, (1)Arizona State University, Tempe, AZ, (2)Ohio State University, colombus, OH, (3)Centre for Digestive Diseases, Five Dock, Australia, (4)Ohio State University, Colombus, OH, (5)Massachusetts General Hospital, Cambridge, MA, (6)University of Minnesota, Minneapolis, MN, (7)Integrative Developmental Pediatrics, Tucson, AZ, (8)Northern Arizona University, Flagstaff, AZ, (9)University of Arizona, Tucson, AZ
Background: Autism Spectrum Disorder (ASD) is a complex neurobiological disorder that impair social interactions and communication and lead to restricted, repetitive and stereotyped patterns of behavior, interests, and activities. The causes of this disorder remains poorly understood, but gut microbiota, the 1013 bacteria in the human intestines, have been hypothesized to impact ASD because ASD-afflicted children often suffer gastrointestinal (GI) problems that correlate with ASD severity, and many previous studies have reported abnormal gut bacteria in children with ASD. The gut microbiome-ASD connection has been tested in a mouse model of ASD where the microbiome was mechanistically linked to abnormal metabolites and behavior. Similarly, a study of children with ASD found that oral non-absorbable antibiotic treatment improved GI and ASD symptoms, albeit temporarily. Here, we conducted a small open label clinical trial to evaluate the impact of Microbiota Transfer Therapy (MTT) on GI and ASD symptoms of 18 ASD-diagnosed children.

Objectives: Determine the safety and possible efficacy of MTT for treating GI and ASD symptoms.

Methods: An open-label clinical trial of MTT involved a two-week antibiotic treatment, a bowel cleanse, and then an extended duration of fecal microbiota transplant (FMT) using a high initial dose followed by daily lower maintenance doses for 7-8 weeks.

Results: The Gastrointestinal Symptom Rating Scale (GSRS) revealed an approximately 80% reduction of GI symptoms at the end of treatment, including significant improvements in symptoms of constipation, diarrhea, indigestion, and abdominal pain, and all those benefits remained improved at 8 weeks after treatment. Similarly, clinical assessments showed that behavioral ASD symptoms improved significantly, and remained improved at 8 weeks after treatment ended.

Conclusions: This exploratory, extended-duration treatment protocol thus appears to be a promising approach to improve GI and behavioral symptoms of ASD. Improvements in GI symptoms and ASD symptoms persisted for at least 8 weeks after treatment ended, suggesting a long-term impact.