New Cancer Drug Opens Treatment Avenues For Fragile X
A new drug being tested for cancer treatment has the potential to help individuals who have common inherited form of autism, according to researchers at the University of Edinburgh and McGill University,
A genetic syndrome that causes several developmental complications, including learning disabilities and cognitive impairment, is also the common cause for autism. This condition is called Factor X syndrome. It affects around 1 in 4,000 boys and 1 in 6,000 girls. There is no known cure for now.
According to the University of Edinburgh press release, the scientists, who have identified a chemical pathway that goes awry in the brains of Fragile X patients, said a cancer drug candidate could reverse their behavioural symptoms. The researchers have found that a naturally occurring anti-fungal called cercosporamide can block the pathway.
Cercosporamide is being tested to treat lung cancer and leukaemia. Autism-related findings were based on the test done on mice.
The research team was able to identify the molecule (elF4E) that triggers protein overproduction in the brains of the Fragile X patients. This can cause behavioural problems and could lead to graver intellectual inabilities, speech delays and social integration difficulties.
Professor Nahum Sonenberg of McGill University said, "We found that eIF4E regulates the production of an enzyme called MMP-9, which breaks down and re-orders the connections between brain cells called synapses. Excess MMP-9 disrupts communication between brain cells, leading to changes in behaviour."
In the study conducted, cercosporamide was able to block elF4A activity. Thus, MMP-9 production was reduced and behavioural symptoms in mice with Fragile X Syndrome was reversed.
These findings suggest that the drug can be used in developing a treatment for patients with the syndrome. Details on this study are published in the journal Cell Reports.