A common drug used to treat epilepsy and certain mood disorders, called lamotrigine, has shown great promise as a new treatment for a rare muscle condition known as non-dystrophic myotonia.
This finding comes from a groundbreaking study led by researchers at University College London (UCL), making it the first trial of its kind in the world.
Non-dystrophic myotonia is a rare genetic disorder that affects the muscles, causing stiffness, pain, weakness, and fatigue.
These symptoms often begin in childhood and can lead to significant challenges in daily life, including reduced quality of life and difficulties with work. Unfortunately, there is currently no cure for this condition.
The study, published in The Lancet Neurology, involved a “head-to-head” comparison of two drugs: mexiletine, which is currently the first-choice treatment for non-dystrophic myotonia, and lamotrigine.
The trial took place at the UCL Queen Square Multidisciplinary Centre for Neuromuscular Diseases and the National Hospital for Neurology and Neurosurgery, UCLH, and included 60 adults with confirmed cases of the disorder.
Participants were randomly divided into two groups: one group received mexiletine for eight weeks followed by lamotrigine for eight weeks, while the other group received the drugs in the reverse order.
There was a seven-day break between treatments, and neither the participants nor the researchers knew which drug was being administered at any given time.
At the end of the trial, both drugs were found to be equally effective in reducing muscle stiffness, the primary symptom of non-dystrophic myotonia.
This is an exciting development because while mexiletine is effective, it does have some limitations.
Not all patients respond well to it, and about one-third of those who do experience significant side effects, such as gastrointestinal issues.
Moreover, mexiletine cannot be used during pregnancy, which is a time when myotonia symptoms often worsen.
Lamotrigine, on the other hand, was found to be well tolerated by patients with no serious side effects reported.
It also has additional benefits: it is safe to use during pregnancy and is more affordable, making it a valuable alternative, especially in countries where mexiletine is either too expensive or unavailable.
Dr. Vino Vivekanandam, the chief investigator of the study, emphasized the importance of such trials, particularly for rare diseases that often lack treatment options. Senior author Professor Michael Hanna highlighted that drug repurposing—using existing drugs for new treatments—is a crucial strategy for developing therapies for rare conditions.
The results of this trial are already being applied in clinical practice, with UCL developing a personalized treatment plan that considers the effectiveness and cost of both drugs.
This new approach is expected to improve the lives of people with non-dystrophic myotonia worldwide, offering them a safe and effective treatment option.
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