Under trail cancer drug may help reverse autism's behavioral symptoms

A new study has revealed that a drug undergoing test as a treatment for cancer could also help reverse autism's behavioral symptoms.

Researchers from the University of Edinburgh and McGill University have identified a chemical pathway that goes awry in the brains of Fragile X patients, stated that a cancer drug candidate could reverse their behavioral symptoms and found that a naturally occurring anti-fungal called cercosporamide can block the pathway and improve sociability in mice with the condition.

The team identified a key molecule - eIF4E - that drives excess protein production in the brains of Fragile X patients. This can cause behavioural 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.

The team found that treatment with cercosporamide blocks the activity of eIF4E, and therefore reduces the amounts of MMP-9, and reverses the behavioural symptoms in mice with a version of Fragile X Syndrome. The new findings suggested that it could have a use as a treatment for patients with Fragile X Syndrome.

Christos Gkogkas, of the University of Edinburgh's Patrick Wild Centre for Research into Autism, Fragile X Syndrome and Intellectual Disabilities, said that by designing treatments that block just this pathway, it is hoped that they can limit the potential side-effects and develop therapies that are more efficient than general treatment approaches.

The study is published in the journal Cell Reports.