Cancer Drug May Play Role In Treating Fragile X
A potential cancer treatment may also relieve behavioral symptoms of a condition associated with autism and intellectual disability, researchers say.
A drug known as cercosporamide is currently being tested as a treatment for lung cancer and acute myeloid leukemia, but a new study suggests it also holds promise for alleviating symptoms of fragile X syndrome, the most common cause of inherited intellectual disability and a condition that often co-occurs with autism.
In the study, scientists identified a molecule called eIF4E that prompts excess protein production in the brains of individuals with fragile X, leading to behavior problems, learning difficulties and other issues.
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However, treatment with cercosporamide appeared to mitigate the overproduction and reverse behavioral symptoms in mice with fragile X, researchers said in the journal Cell Reports.
“Our findings open the door to targeted treatments for fragile X syndrome. 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,” said Christos Gkogkas of the University of Edinburgh who led the study.