Drug designed to treat lung cancer and leukaemia could also reverse common form of autism

A new drug being tested to treat cancer could help people affected by a common form of autism, scientists believe.

Researchers have identified a chemical pathway that goes awry in the brains of 'Fragile X' patients.

They say the cancer drug could block the pathway and reverse their behavioural symptoms.

Fragile X Syndrome is the most common genetic cause of autism spectrum disorders, The Daily Mail reports.

The team at Edinburgh University and McGill University in Canada identified a key molecule - eIF4E - that triggers 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.

They found that eIF4E regulates the production of a specific enzyme, which breaks down and re-orders the connections between brain cells called synapses.

This disrupts communication between brain cells, leading to changes in behaviour.

The scientists have found that a naturally occurring chemical - called cercosporamide - improved sociability in mice with the condition.

Treatment with cercosporamide blocks the activity of eIF4E, and therefore reduces the amounts of the enzyme, and reverses the behavioural symptoms in mice with a version of Fragile X Syndrome.

Cercosporamide is being tested as a treatment for lung cancer and acute myeloid leukemia.

The new findings suggest that it could also have a use as a treatment for patients with Fragile X Syndrome.

Dr Christos Gkogkas, of Edinburgh University, said: '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.'

The study was published in the journal Cell Reports.

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