A quarterly publication of the Autism Research Institute

The Autism Research Review International is quarterly publication of the Autism Research Institute

Winter, 2017 | Number 1, Volume 31

Pilot study: microbiota transplant improves behavior, GI symptoms of children with ASD

Fecal transplants may improve the gastrointestinal (GI) symptoms and behavior of children with autism spectrum disorders (ASD), a pilot study suggests. 

Fecal transplants have already gained attention as a treatment for C. difficile infection and inflammatory bowel disease. Dae-Wook Kang and colleagues are interested in the potential of this approach for treating children with ASD, who have high rates of diarrhea, constipation, and other bowel problems that may be connected to their behavioral issues. 

In the study, 18 participants with ASD and GI problems underwent Microbiota Transfer Therapy or MTT. They first received two weeks of antibiotic treatment and a bowel cleanse to prepare the GI tract. Next, they received one high dose of microbiota administered either orally or rectally, followed by a lower oral daily maintenance dose for 7 to 8 weeks. The researchers used a standardized product containing more than 99% bacteria obtained from healthy individuals and carefully tested to ensure safety. The children also took an acid pump inhibitor in order to reduce stomach acidity and increase the survival rate of the microbes. The researchers followed up on the children for eight weeks after treatment ended.

Participants in the study were between the ages of 7 and 16. The researchers also enlisted 20 neurotypical children (who did not receive treatment) as controls. 

Using multiple measures, the researchers detected marked changes in GI symptoms and behavior in the children with ASD. Among their findings: 

• The average score on the Gastrointestinal Symptom Rating Scale (GSRS) dropped 82% from the beginning to the end of treatment. At the eight-week follow-up, it remained 77% lower than at baseline. 

• Only two of the 18 children with ASD showed less than a 50% reduction in the average GSRS score. 

• There was a significant decrease in the number of days with abnormal or no stools, and this improvement also was maintained at the eight-week follow-up. 

• The Parent Global Impressions-III (PGI-III) scale revealed significant improvements in behavior, and there was a significant correlation between GSRS scores and PGI-III scores. 

• Scores on the Childhood Autism Rating Scale, which measures core autism symptoms, decreased by 22% from the beginning to the end of the study and were 24% lower than baseline at the eight-week follow-up. 

• Participants with ASD also showed improvements on the Social Responsiveness Scale and the Autism Behavior Checklist. 

• On the Vineland Adaptive Behavior Scale II, the average developmental age of participants with ASD increased by 1.4 years and improved across all domain areas. 

The researchers also detected significant changes in the gut bacteria of the children with ASD. While these children initially had significantly less diverse gut bacteria than controls, the researchers say that “median richness at week 18 was statistically indistinguishable between the ASD and control groups.” Importantly, they say, “the donor bacterial community was at least partially engrafted in the recipient gut,” indicating that their therapy overcame “colonization resistance.” 

The researchers also studied changes in phages, which are viruses that play a role in gut health. They detected no significant changes but did see evidence that phage communities also responded to MTT. 

The researchers conclude that MTT “appears to be a promising approach to alter the gut microbiome and virome and improve GI and behavioral symptoms of ASD.” They caution, however, that their study was small and non-blinded, and that researchers need to follow up with randomized, double-blind, placebo-controlled studies.

Scores on the Childhood Autism Rating Scale, which measures core autism symptoms, decreased by 22% from the beginning to the end of the study, and were 24% lower than baseline at the eight-week follow-up. On the Vineland Adaptive Behavior Scale-II, the average developmental age of participants with ASD increased by 1.4 years and improved across all domain areas.


Citations

“Microbiota Transfer Therapy alters gut ecosystem and improves gastrointestinal and autism symptoms: an open-label study,” Dae-Wook Kang, James B. Adams, Ann C. Gregory, Thomas Borody, Lauren Chittick, Alessio Fasano, Alexander Khoruts, Elizabeth Geis, Juan Maldonado, Sharon McDonough-Means, Elena L. Pollard, Simon Roux, Michael J. Sadowsky, Karen Schwarzberg Lipson, Matthew B. Sullivan, J. Gregory Caporaso, and Rosa Krajmalnik-Brown, Microbiome, January 2017 (free online). Address: Matthew B. Sullivan, [email protected]