Vienna, Austria: Studies of mouse models of Fragile X syndrome and Phelan-McDermid syndrome show that treatment with cannabidiol (CBD) and the diabetes drug metformin can alleviate behavioural difficulties, according to research presented at the Federation of European Neuroscience Societies (FENS) Forum 2024.
Fragile X syndrome and Phelan-McDermid syndrome are two genetic conditions that cause a variety of neurodevelopmental difficulties throughout life, including autism spectrum disorder symptoms such as speech delay, social challenges, and repetitive behavior.
Dr. Ilse Gantois from McGill University in Montreal, Canada, presented the research findings, which revealed promising results in using metformin and CBD to address these conditions. The studies aimed to identify safe treatments with minimal side effects to help individuals with Fragile X and Phelan-McDermid syndromes improve their quality of life.
The research team found that metformin corrected a biochemical imbalance in the brain of mice bred to model Fragile X syndrome, preventing the development of speech impairments and repetitive behaviors. Similarly, metformin showed positive effects in mice modeling Phelan-McDermid syndrome, improving speech, behavior, learning, and memory.
CBD treatment in young mice with Fragile X and Phelan-McDermid syndromes resulted in significant improvements in repetitive and social behaviors as they reached adulthood. Researchers are investigating how CBD interacts with the brain to enhance behavior in these conditions.
Clinical trials of metformin and CBD for Fragile X syndrome are underway in Canada and the United States, raising hopes for improved treatments for individuals with neurodevelopmental conditions. The studies offer insights into the neurological mechanisms underlying these syndromes and the potential benefits of targeted drug therapies.
Experts emphasize the importance of further research to deepen our understanding of Fragile X and Phelan-McDermid syndromes and develop effective treatments to support those affected. Mouse models provide valuable insights into the mechanisms of these conditions, guiding the exploration of new therapeutic approaches for developmental disorders.
