The team of 16 scientists, mostly drawn from the campuses of the University of Medicine and Dentistry of New Jersey, say their findings, the result of more than two years of study on how the body breaks down fatty acids, could be a breakthrough for what is the fastest-growing developmental disorder in the nation, with no known cause or cure.
The UMDNJ researchers say they have found that children with autism are unable to metabolize key fatty acids that help the body fight inflammation that causes damage to the brain and other organs.
"It\’s an exciting story that\’s unfolding," said George Lambert, coordinator of the 15-member research team.
The potential treatment, members of the team say, is a kind of "therapeutic cocktail" tailored to each child, which would give them a dose of a "good" fatty acid that they are not able to make on their own. Team member Bernd Spur of UMDNJ-Stratford created the chemical process to replicate one of those good fatty acids.
"The pathway doesn\’t work (in the body), so we circumvent it," said Spur, a chemist.
Currently, the only way to diagnose autism is by a clinical assessment of symptoms, which include difficulty with communication and social interaction, as well as obsessive behaviors and interests. New Jersey has a high incidence of the disorder, affecting 1 in 94 children in the state, compared with 1 in 150 in the U.S., according to the Centers for Disease Control and Prevention.
Researchers say that in the future a person\’s risk for autism could be measured with a simple urine test that would look for high levels of "bad" fat molecules, or a blood test that could reveal genetic problems, including the absence of a key gene, called GSTM1, which is responsible for metabolizing good fats. Many people with autism do not have this gene.
Xue Ming, a neuroscientist and a founding director of the Autism Center at UMNDJ-Newark, discovered that children with autism have higher levels of bad fat molecules in their urine than typical children.
No one understands yet why it is that so many children with autism have such metabolic differences, but Ming suggested it might be caused by an interaction between genes and the environment. It may be that having less of these key fats reduces the body\’s ability to deal with environmental and metabolic stress.
Since the 1990s, scientists have known about the potential for good fatty acids to treat inflammatory diseases such as Crohn\’s disease, ulcerative colitis, even asthma and Alzheimer\’s.
That potential has spurred a frenzy of research into lipids, the fatty compounds that include fatty acids. Studies at the University of Pennsylvania and Vanderbilt University have confirmed the New Jersey group\’s findings, said Spur.
At Harvard University, researchers are working on treatments for asthma and periodontal disease, while researchers at Louisiana State University are focusing on stroke.
Team members have been meeting every week for more than two years to discuss the results of their experiments. They include Lambert, a pediatric toxicologist who looks at the impact of the environment on children; Spur, the chemist who replicated the fatty acid in the lab; neurologist William Johnson, who associated the missing gene with autism; and Ming, a neuroscientist who tested for the presence of bad lipids in children.
So far, the scientists have obtained six patents for their research, Lambert said. They soon will meet with the Food and Drug Administration to discuss the requirements for producing and testing their substances, he added.
"Metabolic issues in autism are entirely understudied," said Sophia Colamarino, science director for Cure Autism Now, a major advocacy and research group in Los Angeles. "It\’s a very exciting area. There is accumulating evidence that would clearly tell me this is where I should look."
The New Jersey scientists are cautious, however, about their preliminary results, and warn families not to expect a miracle cure. Testing on humans, they say, could take a few years.
Meanwhile, the researchers are preparing a preliminary study to begin in September. Lambert hopes to work with 5- to 7-year-olds at the Douglass Developmental Disabilities Center in New Brunswick, a school for children with autism run by Rutgers.
Lambert will be giving the children doses of a good fatty acids to see if they have any noticeable effect on the children\’s cognitive, social and behavioral states. The study will use a control group of similar students.
"New Jersey is the perfect place to do this," said Lambert, director of the EPA-funded Center for Neurotoxicology. "We have a high incidence (of autism), a long history of activism and a strong community."