Fragile X syndrome and Phelan-McDermid syndrome are two genetic conditions that lead to various neurodevelopmental difficulties throughout life. Both conditions often include autism spectrum disorder (ASD) symptoms, such as speech delay, social challenges, and repetitive behaviours. These disorders can make life particularly tough for children and their families, and finding effective treatments has been an ongoing challenge.
Promising research unveiled
Recent studies presented at the Federation of European Neuroscience Societies (FENS) Forum 2024 offer new hope. Dr Ilse Gantois and her team from McGill University in Montreal, Canada, have been exploring how two substances – cannabidiol (CBD) and metformin – can alleviate behavioural difficulties in mouse models of Fragile X syndrome and Phelan-McDermid syndrome.
Metformin: More than a diabetes drug
Metformin is widely known as a treatment for type 2 diabetes. However, Dr. Gantois and her team have discovered that it can also correct certain biochemical differences in the brains of mice bred to model Fragile X syndrome. Their research shows that metformin can prevent these mice from developing impaired speech and repetitive behaviours, which are common issues in these conditions. When tested on newborn mice, metformin not only addressed these issues but also improved learning and memory behaviours in mice modelled after Phelan-McDermid syndrome.
CBD: The non-psychoactive component of cannabis
CBD is gaining attention for its potential therapeutic benefits. Unlike THC, the psychoactive component of cannabis, CBD does not induce a “high.” Dr Gantois’s research showed that when CBD was administered to young mice bred to model Fragile X syndrome, their repetitive and social behaviours improved significantly. Similar results were observed in mice modelled for Phelan-McDermid syndrome. These promising findings suggest that CBD could be an effective treatment for behavioural difficulties associated with these conditions.
The bigger picture: Ongoing and future research
Several research groups in Canada and the U.S. are now conducting clinical trials to test the effects of metformin and CBD on humans with Fragile X syndrome. The hope is that these substances, which are already approved for other uses and have minimal side effects, could provide new, effective treatments for these genetic conditions.
Expert insights
Dr Gantois believes that early intervention with these drugs could greatly improve speech delay, social interaction, and repetitive behaviours in children with Fragile X and Phelan-McDermid syndromes. Using mouse models allows researchers to closely examine what happens in the brain during these treatments, providing a detailed understanding of how these drugs work and their potential benefits.
However, it’s essential to remember that mouse brains are not identical to human brains. While there are similarities, translating these findings to human treatments requires careful and thorough research. Yet, the progress made so far is encouraging and could pave the way for more effective therapies.
Looking ahead: Broader applications
The researchers are also exploring the potential benefits of metformin and CBD for other autism spectrum disorder-related conditions. This could lead to broader applications, providing hope for many families dealing with these challenging conditions.
The importance of continued research
Many medical experts are emphasising the need for ongoing studies. They highlight the importance of understanding conditions like Fragile X and Phelan-McDermid syndrome and finding effective treatments to improve the lives of affected children. Mouse models offer valuable insights into the brain’s mechanisms and the potential benefits of new treatments.
A brighter future
The research presented by Dr Gantois and her team is a significant step forward in the quest to find effective treatments for Fragile X and Phelan-McDermid syndromes. By repurposing existing drugs like metformin and exploring the potential of CBD, scientists are opening new doors to improving the quality of life for those affected by these conditions. While more research is needed, the future looks promising, offering hope to many families worldwide.
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