Researcher calls it "some of the most exciting research in my career."
A “wonder drug” commonly used to treat diabetes may be capable of treating autism.
According to a recent study, metformin — the most widely-used drug to treat type 2 diabetes – improved social, behavioral and morphological defects in mice models with Fragile X syndrome, an inherited form of intellectual disability and a cause of some forms of autism.
Fragile X syndrome, the genetic disease brought on by defects in the Fragile X Mental Retardation 1 gene (FMR1), triggers an excess production of protein in the brain, as well as a dysregulated connections between neurons and changes in behavior. It leads to impairments in speech, language, behavior, and social interaction, and is frequently co-diagnosed with autism, anxiety disorders, and seizures.
The mice models — whose symptoms of Fragile X syndrome include increased grooming and decreased socialization – showed normal brain connections and behavioral patterns of ten days of metformin injections.
The genetic disease that affects about 1 in every 5,000 boys and 1 in every 6,000 girls does not have a cure. Co-senior author of the study and James McGill University Biochemistry Department professor Nahum Sonenberg (pictured), PhD, called the study “some of the most exciting research work in my career.”
Sonenberg described metformin as a wonder drug because of its recently-found potential in treating forms of cancer, cardiovascular diseases, neurological diseases and aging.
Jean-Claude Lacaille, a major collaborator in the study and Canada Research Chair in Molecular Neurophysiology and professor in University of Montreal’s Department of Neurosciences, said the drug’s well-documented safety can push their research towards a quick clinical phase.
“This makes the drug an ideal candidate for fast-tracked clinical trials and, if all goes well, a readily available drug for the treatment of Fragile X syndrome,” Lacaille said.
The team found that metformin restores some of the molecular pathways disrupted in the absence of the FMR1. Results also suggested the treatment might be capable of treating other autism spectrum disorders, co-lead author and McGill University research associate Ilse Gantois said. Their research mostly focused on the autistic form of behavior in the Fragile X mouse model.
“We want to start testing other mouse models to see if the drug could also have benefits for other types of autism,” Gantois said.
The next round of research will entail learning what role the drug plays in the molecular pathways. The corrections allowed by the drug are already part of what Sonenberg called a “simple story.”
“What is more complicated is the molecular mechanism, how exactly it works,” Sonenberg said. “We need to study, in the lab, what molecules metformin interacts with and what cellular functions are affected.”
Sonenberg believes the drug could be tweaked to be “more efficient than metformin,” and repurposed for other disorders.
The study was recently published in Nature Medicine.
A press release regarding the study’s findings was made available.