Researchers at McGill University in Montreal and the University of Edinburgh in Scotland say that a drug that is being studied as a possible treatment for cancer may help people with fragile X syndrome, an inherited form of autism.
Fragile X affects about 1 in 4,000 boys and 1 in 6,000 girls. The condition causes intellectual disability, especially in boys, and behavioral problems such as learning disabilities, repetitive movements, and problems with social interactions. Fragile X causes a chemical pathway in the brain to act abnormally.
The drug, cercosporamide, blocks this chemical pathway. Cercosporamide was shown to improve sociability in mice with a form of Fragile X syndrome. It has not been tested against fragile X syndrome in humans as yet.
The research team identified one molecule, called elF4E, which causes excess production of a brain enzyme called MMP-9 in people with fragile X. Cercosporamide apparently blocks the activity of elF4E which leads to a reduction in the amount of MMP-9 in the brain.
"Our findings open the door to targeted treatments for Fragile X Syndrome," said Christos Gkogkas of Patrick Wild Centre for Research into Autism, Fragile X Syndrome and Intellectual Disabilities at the University of Edinburgh. Studying cercosporamide and its effects may lead to the design of treatments that block just elF4E and the excessive production of MMP-9.
More research is needed to determine if the beneficial effects of cercosporamide will be seen in humans with fragile X syndrome or if there are any issues with side effects.
Cercosporamide is a chemical that is derived from a fungus. It is being investigated as a cancer drug, but human studies have not been completed as yet.
The McGill/Edinburgh study was published in the journal Cell Reports and was reported at ScienceDaily.com.
