A team of researchers led by Dr Christos Gkogkas from the Patrick Wild Centre, together with colleagues from McGill University and Université de Montréal, Canada, have found that a commonly used diabetes drug, Metformin, has the potential to help people with a common inherited form of autism (fragile X syndrome).
Fragile X syndrome affects around 1 in 4,000 boys and 1 in 6,000 girls. It is the most common known cause of inherited intellectual disability. Affected children have developmental delays that impair speech and language, have problems with social interactions and are often co-diagnosed with autism, anxiety and seizures.
Fragile X syndrome is caused by inherited defects in a gene called FMR1, which leads to excess protein production in the brain. This results in the breakdown of connections between brain cells, leading to changes in behaviour.
The research shows that treating FMR1 mice with the diabetes drug metformin for 10 days reduced levels of protein production in the brain to normal levels, repaired neuronal connections, led to a reduction in seizure occurance, and restored the increased grooming and reduced social interaction behaviour usually displayed by FMR1 mice to normal patterns.
Researchers say that metformin could be repurposed as a treatment for Fragile X syndrome within a few years – if clinical trials prove successful. Metformin is already approved by the UK’s Medicines and Healthcare products Regulatory Agency and the US Food and Drug Administration as a therapy for type 2 diabetes.
The study was published in the journal Nature Medicine. Researchers say they next plan to investigate whether the drug offers any benefits for other types of autism.
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