A team of international researchers are currently testing a drug for cancer that they believe can treat fragile X syndrome, an inherited form of autism.
The condition, also known as Martin-Bell syndrome, is triggered by a mutation in FMR1, a gene located on the X chromosome that makes fragile X mental retardation 1 protein (FMRP), which affects the development of synapses in the brain, according to according to Medical News Today.
Fragile X syndrome currently affects 1 in 4,000 men and 1 in 8,000 women in the U.S., and a cure has yet to be found for it.
The researchers, from the University of Edinburgh in the U.K. and McGill University in Canada, believe they have found the solution by studying the molecule eIF4E, which triggers excess protein production in the brains of patients with this condition, Science Daily reported. Too much protein production can lead to learning difficulties, intellectual disabilities, delays in speech and language development and issues with social interactions.
Nahum Sonenberg, McGill professor in the Faculty of Medicine and the Goodman Cancer Research Center and co-author of the study, said the enzyme created from eIF4E is called MMP-9, which breaks down and re-arranges connections between synapses, a type of brain cell.
"Excess MMP-9 disrupts communication between brain cells, leading to changes in behavior."
After treating mice with fragile X syndrome with the drug, called cercosporamide, the researchers were able to block the activity of eIF4E, lowing the production of MMP-9 and reversing the behavioral symptoms of fragile X syndrome in mice as a result, Medical News Today reported.
Arkady Khoutorsky, a post-doctoral student at McGill University and co-author of the study, says the team's research shows that drugs that already exist can be used to block eIF4E activity and control MMP-9 production.
Christos Gkogkas, co-author from the University of Edinburgh's Patrick Wild Centre for Research into Autism, Fragile X Syndrome and Intellectual Disabilities, said the results will allow for the development of new treatments for the condition, Science Daily reported.
"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 study was published in the journal Cell Reports.
