New Autism Treatment Could Come from Altering Gut Microbiota

Researchers take novel approach in the search for effective autism treatments by focusing on improving the gut microbiome through fecal microbial transplants. [dimdimich/Fotolia]

Getting to the guts of the matter for autism is exactly what a group of investigators, led by scientists at Arizona State University (ASU), hopes to accomplish with its novel approach to effective autism treatments by focusing on improving the gut microbiome through fecal microbial transplants. While initial results from a small human study are promising, additional testing will be necessary before an FDA-approved therapy would be available or recommended to the public.

The research team—which included collaborators from Northern Arizona University, Ohio State University, and the University of Minnesota—completed a study involving 18 participants with autism spectrum disorders who ranged in age from 7 to 16 years old. The results of the study were recently published in the journal Microbiome.

Expanding upon previous research done by the ASU scientists that showed ties between autism symptoms and the composition and diversity of a person's gut microbes, the participants from the current study underwent a 10-week treatment program involving antibiotics, a bowel cleanse, and daily fecal microbial transplants over 8 weeks.

Remarkably, the new therapy seemed to provide some long-term benefits, including an 80% improvement of gastrointestinal symptoms associated with autism spectrum disorders and roughly a 20% to 25% improvement in autism behaviors, including improved social skills and better sleep habits.

“The results are very compelling,” noted senior study investigator James Adams, Ph.D., professor of materials science and engineering at ASU. “We completed a Phase I trial demonstrating safety and efficacy, but recommending such treatment and bringing it to market requires Phase II and Phase III trials. We look forward to continuing research on this treatment method with a larger, placebo-controlled trial in the future.”

Fecal microbial transplants involve the transfer of live gut bacteria from a healthy donor to a recipient. The donor materials contain around 1000 different species of gut bacteria that act as a broad-spectrum probiotic treatment to restore normal gut bacteria in recipients.

“We saw a big increase in microbe diversity and a big increase in certain bacteria, especially Prevotella [a Gram-negative bacterial genus], which we previously found was low in children with autism spectrum disorders,” explained co-author Dae-Wook Kang, Ph.D., assistant research scientist at the Swette Center for Environmental Biotechnology at ASU.

Interestingly, the investigators found that many of the microbes added through the treatment program remained after treatment stopped—an important part of the therapy if researchers hope to obtain lasting effects within patients.

“That is compelling, because not only did we provide good microbes, but the microbes we provided changed the gut environment in a way that helped the host recruit beneficial microbes and allowed them to stay around,” remarked senior study investigator Rosa Krajmalnik-Brown, Ph.D., associate professor of fundamental and applied microbiomics in The Biodesign Institute at ASU.

While the research team was excited and optimistic about their results, they urged caution against overinterpretation of results and families attempting to replicate the treatment on their own.

“Although we see promise in this treatment, it is important that parents and children consult their physicians,” Dr. Krajmalnik-Brown stated. “Improper techniques can result in severe gastrointestinal infection.”