Lamotrigine, a drug commonly used to treat epilepsy and certain mood disorders, has been shown to be an excellent treatment option for a rare genetic neuromuscular disease known as non-dystrophic myotonia, in a world-first trial led by University College London researchers. The study, published in The Lancet Neurology , detailed the "head-to-head" trial implemented by the researchers to test two drugs, mexiletine and lamotrigine , on people with the condition. The trial, which was conducted at the UCL Queen Square Multidisciplinary Centre for Neuromuscular Diseases and the National Hospital for Neurology and Neurosurgery, UCLH, involved 60 adults with confirmed non-dystrophic myotonia.
Patients were randomly assigned to receive either mexiletine for eight weeks followed by lamotrigine for eight weeks, or the reverse order, with a seven-day break in between treatments. Neither the participants nor the researchers knew which treatment was being given at any time. At the end of the trial, lamotrigine was found to reduce stiffness—the main symptom of non-dystrophic myotonia—by around the same amount as mexiletine.
Chief investigator, Dr. Vino Vivekanandam (UCL Queen Square Institute of Neurology and consultant neurologist), said, "Approximately one in 17 people in the UK have a rare disease and the majority have no treatment. Many are neurological diseases and rarity makes clinical trials to develop treatments very difficult.
"Head-to-head trials comparing drugs are important.