Autism is a complicated disorder that affects the way children socialize, communicate, and behave. Fragile X Syndrome is an inheritable form of autism, and a new discovery may mean cancer treatments can curb their behavior. Researchers from the University of Edinburgh collaborated with McGill University found a new pathway in the brain that may reverse certain autism symptoms, and published their findings in the journal Cell Reports.

Researchers discovered too much protein is produced in the brain of Fragile X patients, and molecule eIF4E is to blame. The molecule causes many of the behavioral problems seen in autism patients, such as serious intellectual disabilities, delays in speech and language development, and social interaction. Once they identified the molecule, they tested it in mice and found the naturally-occurring chemical cercosporamide can block the excess protein production. Their mice became more sociable as soon as eIF4E was blocked.

“Our findings open the door to targeted treatments for Fragile X Syndrome,” Dr. Christos Gkogkas, a Chancellor's fellow at the University of Edinburgh, said in a press release. “By designing treatments that block just this pathway, it is hoped that we can limit the potential side effects and develop therapies that are more efficient than general treatment approaches.”

The treatment has only been shown to work on patients with Fragile X syndrome, which is the most common known cause of inherited intellectual disability. According to the Centers for Disease Control and Prevention, one in 5,000 boys are born with the disorder, and females often have much milder symptoms than males. There is a similar disproportion of males to females in autism in general, which indicates something is developing incorrectly while the baby is still in the womb.

“Dr. Gkogkas is an important addition to the Patrick Wild Centre,” Peter Kind, the director of the Patrick Wild Centre at the University of Edinburgh said. “His research provides important insights into the brain processes underlying the symptoms of people with Fragile X Syndrome and other intellectual disabilities. Only with this knowledge can better medicines to treat these conditions be developed.”

The eIF4E molecules overproduce the protein MMP-9, which breaks down the brain’s connections and reorders the synapses. It’s exactly why the child has behavioral issues. The chemical that’s able to block all of the brain malfunction from happening is cercosporamide, and researchers are already testing it in patients with lung cancer and acute myeloid leukemia. Cercosporamide is actually a natural antifungal product that signals certain cell pathways. It was only discovered 10 years ago but already may be making serious strides in treating serious disorders and diseases.

“We found that eIF4E regulates the production of an enzyme called MMP-9, which breaks down and re-orders the connections between brain cells called synapses,” Nahum Sonenberg from McGill University said. “Excess MMP-9 disrupts communication between brain cells, leading to changes in behavior.”

Source: Gkogkas C, Kind P, and Sonenberg N, et al. Cell Reports. 2014.