Microbiota transfer therapy improves gastrointestinal and autism symptoms

Last Updated: 2017-01-26

By Will Boggs MD

NEW YORK (Reuters Health) - Extended-duration fecal microbiota transfer therapy improves gastrointestinal and behavioral symptoms in individuals with autism spectrum disorder (ASD), a small open-label study suggests.

"Many children with autism have chronic GI problems since infancy, and abnormal gut bacteria seem to be a major factor," Dr. Rose Krajmalnik-Brown from Arizona State University in Tempe told Reuters Health by email. "Hence modifying the gut microbiota is a promising future therapy."

Probiotics and vancomycin treatment aimed at altering the gut microbiome have yielded mixed results in individuals with autism, despite numerous studies showing that children with ASD have altered gut bacteria profiles.

Dr. Krajmalnik-Brown and colleagues investigated the effects of a prolonged daily treatment regimen in 18 children (ages 7-16 years) with GI problems and ASD (compared with 20 untreated neurotypical children).

Microbiota transfer therapy (MTT) included 14 days of oral vancomycin treatment followed by 12-24 hours fasting with bowel cleansing and then repopulation of the gut microbiota with high initial doses of standardized human gut microbiota followed by daily lower doses for seven to eight weeks.

Abdominal pain, indigestion, diarrhea, and constipation improved significantly after MTT, and these improvements were maintained after eight additional weeks without treatment, according to the January 23rd Microbiome online report.

ASD-related behavior also improved significantly following MTT, with 22% decreases in core ASD symptoms at the end of treatment and 24% decreases eight weeks later without further treatment.

Average developmental age of the ASD patients increased by 1.4 years after treatment.

MTT treatments were generally well tolerated with only temporary adverse behavioral effects at the beginning of vancomycin treatment.

Gut bacteria before treatment were significantly less diverse in children with ASD than in controls, but after MTT, gut bacterial diversity increased and by week 18 (eight weeks after treatment stopped) it was not significantly different from that of controls.

"FDA treats this as an Investigational New Drug (IND), and more research is needed before this can be used for treatment," Dr. Krajmalnik-Brown cautioned. "Families should not try this at home; microbiota should be very carefully screened and the treatment should be done under medical supervision."

"We are now planning a larger Phase 2 study (randomized, double-blind, placebo-controlled, multi-site) as next step towards FDA approval," she said.

Dr. Alper Evrensel from Uskudar University, Istanbul, Turkey, who recently reviewed the use of fecal microbiota transplantation (FMT) in neuropsychiatric disorders, told Reuters Health, "Gut dysbiosis and intestinal leakage may cause neuropsychiatric and metabolic disorders. And microbiota dysbiosis can be restored with FMT."

"FMT is a reasonable approach in the treatment of neuropsychiatric disorders," he concluded. "FMT is like a gold mine for neuropsychiatrists."

Kirsten Berding Harold and Dr. Sharon M. Donovan from Division of Nutritional Sciences, University of Illinois, Urbana, Illinois recently reviewed microbiome and nutrition in ASD. Harold told Reuters Health by email, "In light of the research published to date, treating symptoms of ASD by manipulating the gut microbiota might be a promising approach for a subgroup of individuals, especially those with severe comorbid GI symptoms. However, a deeper understanding of the contribution of the gut microbiota to symptoms of ASD as well as the influence of environmental factors (e.g., diet, medication) on the gut microbiota are necessary to develop more targeted, less invasive approaches."

"As acknowledged by the authors, studies investigating the effect of MTT in a more homogenous group of individuals (i.e., similar symptom severity), as well as in a subgroup without GI symptoms, are warranted to decipher the efficacy of such an approach," she concluded.

SOURCE: http://bit.ly/2jj1djM

Microbiome 2017.