Researchers at Rush-Presbyterian-St. Luke's Medical Center in Chicago have shown that some children suffering from autism may experience short-term but dramatic improvement during treatment with appropriate antibiotics. The study was published in the July issue of the Journal of Child Neurology. The researchers report that when a subset of patients whose autism may involve colonization of the bowel by certain bacteria that produce a toxin were treated with antimicrobial agents, most experienced short-term improvement of their autism symptoms.
The journal article is authored by Dr. Richard Sandler, director of pediatric gastroenterology at Rush Children's Hospital, part of Rush-Presbyterian-St. Luke's Medical Center in Chicago. Other key contributors are Dr. Sydney Finegold, professor of microbiology and molecular genetics at the University of California at Los Angeles Medical School and Ellen Bolte, mother of a child with autism. The Veteran's Administration Medical Center in West Los Angeles also contributed to the study. Autism typically occurs before two years of age and involves loss of language, social and play skills. According to a study just completed by the Centers for Disease Control indicates that autistic spectrum disorder affects an estimated 1 in 150 children.
"Autism is a devastating disorder with essentially no meaningful treatment," said Dr. Sandler, the paper's lead author. "Seeing most of these children apparently improve significantly was very exciting." However, Dr. Sandler cautioned that "the work needs to be repeated by others, although it presents intriguing evidence suggesting a fruitful line of research towards a possibly effective prevention or treatment strategy in a subset of autistic children." Sandler suggested that "the next step, besides repeating clinical studies, is to go to the lab and try and find out why these effects may have been observed."
The initial hypothesis was the result of years of research by Bolte, whose son developed autism at 19 months of age following several months of broad-spectrum antibiotic treatment for fluid within the middle ear. "It is possible that certain neurotoxin-producing bacteria may find favorable growth conditions if the protective intestinal flora has been disrupted by broad-spectrum antibiotic treatment," Bolte speculates. The hypothesis, although unconventional, may revolutionize the way autism is viewed. "It was not long ago that patients diagnosed with peptic ulcers were advised to watch their diet and reduce stress. Now, it is well-established that most peptic ulcers are caused by Helicobacter pylori (a bacteria) and can be cured by antibiotic treatment," she said.
"I didn't believe in Ellen's theory when she first presented it to me," said Sandler. "But I thought it's strange enough to be true, and decided to test it." Sandler treated Bolte's son, and he appeared to improve significantly. "After that," said Sandler, "we tried to design a careful study to test this hypothesis in other kids."
In this study, 9 of 11 children treated with the antimicrobial agent experienced improved cognitive function, behavior and social skills. The improvement was at times impressive. Unfortunately, regression usually occurred, often to the pre-treatment status.
"Additional research should address whether there are better drugs for this treatment, how long therapy should be continued, and what can be done to prevent relapse once treatment is stopped," explained Dr. Finegold. "Many questions need to be answered, " said Bolte. "These include identifying the possible mechanism of benefit, and properly identifying which autistic children follow this pattern."
Dr. Finegold also emphasized that the investigational therapy used in this trial, orally administered vancomycin, is not recommended for general use because it would encourage further development of resistance of various bacteria to it. "The growing resistance of microbes to this drug is already a major infectious disease and public health problem," said Dr. Finegold. "Autism is heartbreaking, " said Bolte. "These results indicate that further study of a possible connection between intestinal bacteria and autism is warranted. Additional research will hopefully lead to meaningful prevention or treatment in a subset of children afflicted with autism."
Journal
Journal of Child Neurology