New Therapies Being Studied for the Treatment of Autism


Recent statistics from the Centers for Disease Control and Prevention suggested that the diagnosis of autism spectrum disorders among children has increased 57% since 2002. Approximately 1 in 110 8-year-olds in the Unites States has the condition. With these findings, researchers and drug companies are searching for better treatments.

At the federal level, autism is a priority for research. Among pediatric disorders, only diabetes, AIDS, and asthma draw more funding from the National Institutes of Health. President Barack Obama has allocated $60 million of stimulus funds to the autism research pool and earmarked $1 billion for future studies extending through 2018.

Drug companies are also on board to fight autism. Pfizer has formed a 14-person autism research group at its Connecticut laboratory location to conduct early-stage clinical trials. They are first focusing on genes that may affect the synapses in the brain that transmit information. Brain scan data suggest that autistic patients may process spoken language more slowly than normal patients, which leads to a theory of non-typical neural connections.

Another research group, Seaside Therapeutics in Massachusetts, is studying a rare brain disorder called Fragile X syndrome which is known to cause a hereditary form of autism. The condition is caused by a mutation in the X chromosome. Fenobam, a drug in phase 1 clinical trials, calmed behavior and reduced hyperactivity in a small study of 12 people.

The most difficult task with autism research is the complexity of the disorder. It is described as a “spectrum” because symptoms vary widely in both nature and severity. Dr. Eric Hollandar of the Seaver Autism Center for Research and Treatment in New York says, “Early on we felt that if we could collect large enough samples of families with multiple affected individuals, we would find the autism gene. As the sample size got bigger, there were more genes popping up that had minor effects."


Geri Dawson, Chief Science Officer of the advocacy and research group Autism Speaks, says “We’re really beginning to understand that autism is not one disease but that there are many different autisms. The treatment could be very different, depending on the cause.”

There are not any medications that treat the core condition of the autism spectrum disorder. Most drugs focus on the secondary symptoms, such as anxiety and anticonvulsants. The most recent medication on the market is an Alzheimer’s drug from Forest Laboratories. Namenda treats the brain’s glutamate system, which is involved in learning and memory. Oxytocin is also being studied in Asperger’s patients to treat social bonding difficulties and decrease repetitive behaviors.

A 2007 study showed that as many as 70% of children with autism are prescribed psychoactive medications. Abilify, by Bristol-Myers Squibb, has recently been approved as a treatment for the irritability and aggression associated with autism in children ages 6 to 17.

Nutritional treatments are also being studied. Researchers from MIND recently tested injections of methyl B12 in a controlled study of 30 children, since recent findings have shown that some autistic children have altered biomarkers for oxidative stress. Nine of the children did improve in language and socialization. The group is also studying omega-3 fatty acids, which may have an anti-inflammatory effect.

Another promising treatment is a drug called CM-AT by Curemark. Dr. Joan Fallon, the company's founder and CEO, observed that many autistics show a strong preference for foods high in carbohydrates and low in protein because of a lack of enzymes that digest protein. As a result, these children produce fewer of the essential amino acids that are the building blocks for brain development and neuroreception. Fallon believes this deficiency is linked to the most severe symptoms of autism. An early observational study of CM-AT, an orally ingested powder that contains protein-digesting protease, showed "significant improvements." Curemark is enrolling patients in phase III clinical trials at 10 to 12 sites—the largest autism trial to date.

Parents of autistic children are warned against relying on untested and unproven treatments, which include high doses of vitamin B6 and magnesium, intravenous immune globulin therapy, and chelation therapy.



Please don't refer to people with autism as "autistics".