Metformin and cannabidiol (CBD) show promise for treating two genetic conditions with a high incidence of autism spectrum disorder. The corresponding research was presented at the Federation of European Neuroscience Societies (FENS) Forum 2024.
Fragile X Syndrome is the most common inherited form of intellectual disability. It is also the most common monogenetic cause of autism. The condition affects around 1 in 4,000 males and 1 in 6,000- 8,000 females in the US. Current treatments include special education, speech, occupational, and sensory integration training, and behavior modification programs.
“During my career, I often meet parents of children with Fragile X and hear how they try to help their children to live a more comfortable life. So, with my experience in behavioral neuroscience, I want to look for safe treatments that might improve behavioral difficulties in children with these conditions,” said study author, Dr. Ilse Gantois from McGill University, Montreal, Canada, in a press release.
In previous work, Gantois and colleagues used the diabetes drug metformin to treat adult mouse models of Fragile X syndrome. In the current study, they tested the drug on newborn mouse models of the condition. Metformin was able to rectify a biochemical difference in their brains thought to underlie the condition. It also stopped the young mice from developing impaired speech, characterized by high-pitched squeaking and repetitive behavior.
In another experiment, the researchers tested CBD on mouse models of Fragile X syndrome from three weeks after birth. By the time the mice reached adulthood, they behaved similarly to control mice in terms of repetitive and social behaviors. The researchers are now studying how CBD interacts with the mouse brain to better understand its behavioral effects.
In the same study, the researchers also tested metformin and CBD on mouse models of Phelan-McDermid Syndrome, a rare genetic condition that causes developmental and speech delays, behavioral problems, and a weakened or no ability to feel pain or sweat. Both drugs improved symptoms of the condition.
“This research and the outcome of the clinical trials could have a major impact on behavioural difficulties that people with Fragile X syndrome and Phelan-McDermid syndrome experience. The major strengths of these studies are that we are using approved drugs that can be taken throughout lifetime and have minimal side effects. Taken at an early age, these drugs could ultimately improve speech delay, social interaction and repetitive behaviour in these developmental conditions,” said Gantois.
“Using mouse models makes it possible to look in detail at what is happening in the brain, define the underlying cause of these conditions, and study behaviour and the effect of targeted drugs. Of course, mouse brains are not the same as human brains, however we can often translate the mouse behaviour we study to human behaviours,” continued Gantois.
Sources: EurekAlert