From the Research
Metformin may be considered as a potential treatment for Fragile X syndrome, particularly for addressing cognitive and behavioral symptoms, based on recent studies showing its safety and efficacy in improving these areas 1, 2. The use of metformin for Fragile X syndrome is still experimental, but research suggests it may help normalize signaling pathways in the brain that are dysregulated in this condition. Key points to consider when using metformin for Fragile X syndrome include:
- Typical dosing for investigational use has ranged from 500-1000 mg twice daily in adults, with lower doses for children based on weight and age 3, 4.
- Treatment duration would likely be ongoing as Fragile X is a chronic genetic condition.
- Before considering metformin, patients should undergo baseline liver function tests and have kidney function assessed, with regular monitoring during treatment 5.
- Common side effects include gastrointestinal issues like diarrhea and nausea, which can be minimized by starting with a low dose and gradually increasing 2.
- Any use of metformin for Fragile X should be under close medical supervision, preferably by specialists familiar with both the medication and the condition, as part of a comprehensive treatment plan that includes behavioral therapies and educational interventions 1, 2. Recent studies, such as the longitudinal follow-up of metformin treatment in Fragile X Syndrome 1, have shown that metformin may help stabilize IQ and adaptive behavior in individuals with Fragile X syndrome, although the sample size and follow-up duration were limited. Another study 2 demonstrated the safety of metformin in normoglycemic patients with FXS and suggested its potential in modifying GABA-mediated inhibition, a hallmark of FXS pathophysiology. Overall, while metformin is not currently approved as a standard treatment for Fragile X syndrome, the available evidence suggests it may be a promising therapeutic option, particularly when used under close medical supervision and as part of a comprehensive treatment plan.