Mice Study Suggests Cancer Drug May Combat Fragile X Autism

New research suggests people affected by a common inherited form of autism may be helped by a drug that is being tested as a treatment for cancer.

The most common genetic cause of autism spectrum disorders is Fragile X Syndrome, a condition that affects around one in 4,000 boys and one in 6,000 girls. Currently, there is no cure.

For Fragile X patients, a chemical pathway in the brain is altered causing excess protein in the brain. New research suggest a known naturally occurring chemical called cercosporamide can block the pathway and improve sociability in mice with the condition.

The team at the University of Edinburgh and McGill University in Canada identified a key molecule, eIF4E, that drives excess protein production in the brains of Fragile X patients.

This can cause behavioral symptoms that include learning difficulties. It can also lead to more serious intellectual disabilities, delays in speech and language development, and problems with social interactions.

“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,” said Dr. Nahum Sonenberg, a McGill professor and co-author of the study.

“Excess MMP-9 disrupts communication between brain cells, leading to changes in behavior.”

The team found that treatment with cercosporamide blocks the activity of eIF4E, and therefore reduces the amounts of MMP-9, and reverses the behavioral symptoms in mice with a version of Fragile X Syndrome.

The new findings suggest that it could have a use as a treatment for patients with Fragile X Syndrome. The study is published in the journal Cell Reports.

McGill postdoctoral researcher and co-first author of the study Dr. Arkady Khoutorsky said that “the enzyme MMP-9 has been implicated before in Fragile X Syndrome. What’s new in our research is the demonstration that the symptoms of the disease can be controlled by manipulating eIF4E activity with available drug candidates.”

“Our findings open the door to targeted treatments for Fragile X Syndrome,” said neuroscientist Christos Gkogkas, Ph.D., of the University of Edinburgh’s Patrick Wild Centre for Research into Autism, Fragile X Syndrome and Intellectual Disabilities.

“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.”

Source: McGill University

Brain pill photo by shutterstock.