ASU to lead new FDA approved autism treatment study

ASU to lead new FDA approved autism treatment study

August 19, 2014

August 19, 2014

Arizona State University (ASU) will lead a new study examining a novel treatment for gastrointestinal problems in children with autism. This research study has been approved by the U.S. Food and Drug Administration (FDA).

About half of children and adults with autism suffer from chronic gastrointestinal problems, which cause pain, discomfort, and irritability. Recent research at Arizona State University (ASU) has suggested that those gastrointestinal (GI) problems may be due in part to abnormal gut bacteria.

The study involves a novel treatment, called fecal microbiota transplant (FMT). FMT involves the transfer of live gut bacteria from a healthy donor, containing around 1000 different species of gut bacteria that act like a broad-spectrum probiotic treatment to restore normal gut bacteria.  FMT is used to treat serious Clostrium difficle infections, which kill 15,000 people per year in the US.

Researchers at ASU applied to the FDA to investigate FMT for treating GI problems in children with autism.  The FDA has approved a pilot treatment study of 20 children with autism, ages 7-17 years, and moderate to severe gastrointestinal problems.

The purpose of the study is to determine safety and tolerability of FMT in a 10-week treatment study.

The ASU team is led by professor Rosa Krajmalnik-Brown, an expert on evaluating the composition of gut bacterial communities, and professor James Adams, director of the ASU Autism/Asperger’s Research Program.  They published a scientific paper last year demonstrating that children with autism were missing several hundred species of gut bacteria compared to typical children. 

“Our initial work found major differences in the gut bacteria of children with autism compared to typical children, and our subsequent work has confirmed those findings.  Children with autism seem to be missing hundreds of beneficial gut bacteria,” said Krajmalnik-Brown.

“Many children and adults with autism have chronic gut problems, sometimes lasting for many years, and seriously affecting their quality of life.  We think this treatment may be helpful,” said Adams.

Their hypothesis is that FMT will “reseed” the gut with beneficial bacteria, and reduce gastrointestinal problems and possibly reduce autistic symptoms. 

There are also several studies showing that FMT may also be helpful for treating other GI problems, including ulcerative colitis, Crohn’s disease, inflammatory bowel disease, irritable bowel syndrome, and chronic constipation.

The human gut normally contains over 1,000 different species of bacteria.  Most of those are beneficial, and help with digesting food, making certain vitamins, improving GI function, and protecting against pathogenic bacteria.  However, there are a few dangerous bacteria like Clostidium difficile (C. difficile), which can cause serious, life-threatening infections. While C. difficile kills about 15,000 people per year in the US, but a single dose of FMT has been shown to cure C. difficile with 92 percent effectiveness, usually within a few days. 

The ASU team is working with collaborators at Northern Arizona University (NAU) and University of Arizona (UA). The ASU team will lead the treatment portion of the study, with the help of Sharon McDonough-Means, M.D. FAAP a developmental pediatrician involved in the care of children with autism and previous research studies.  Greg Caporaso at NAU, an expert in computational and statistical methods for studying communities of microorganisms, will analyze the effect of FMT on gut bacterial communities, and Matthew Sullivan at UA will investigate the viruses that infect gut bacteria and thereby affect bacterial populations in the gut. 

The new initiative is a follow-up to a previous study that demonstrated that treatment with a powerful oral antibiotic, vancomycin, led to a temporary improvement in both gut symptoms and symptoms of autism, presumably because it killed off harmful bacteria in the gut. However, when the treatment was stopped, the benefits were lost, presumably because there was insufficient “reseeding” of the gut with beneficial bacteria.

The study is funded primarily by the Arizona Board of Regents, with additional funding from the Autism Research Institute.

For more information on the study, go to:


http://autism.asu.edu or http://krajmalnik.environmentalbiotechnology.org/

Media sources:

Rosa Krajmalnik-Brown, Ph.D., is currently an associate professor in the Fulton School of Engineering's School of Sustainability Engineering and the Built Environment and a researcher at the Center for Environmental Biotechnology in the Biodesign Institute. 

James Adams, Ph.D., is a President's Professor in the School of Engineering of Matter, Transport, and Energy (SEMTE) in the Ira A. Fulton School of Engineering.

Media contact:

Joe Caspermeyer
Biodesign media relations
480-727-0369
joseph.caspermeyer@asu.edu