Laryngeal dystonia (LD), a rare neurological disease that significantly impacts a person's ability to speak due to uncontrollable vocal cord spasms, can have a debilitating effect on a person's social life, employment and mental health. Currently, LD is most commonly managed with botulinum neurotoxin (Botox) injections, but this treatment is ineffective for up to 40% of patients who receive it. Now, a study led by researchers from Mass Eye and Ear, a member of the Mass General Brigham healthcare system, shows that an oral medication, sodium oxybate, is more effective than placebo at reducing LD symptoms in patients whose symptoms improve when they consume alcohol.
The results from the phase 2b randomized clinical trial, published November 20th in Annals of Neurology , build on more than a decade of research spurred by anecdotal reports from patients with LD who said their symptoms improved after consuming a couple alcoholic drinks. Sodium oxybate is a central nervous system agent that is FDA-approved to treat patients with narcolepsy and sleep disorders. Sodium oxybate mimics some of the effects of alcohol.
In the trial of more than 100 patients, a single dose of sodium oxybate significantly improved symptoms of patients with alcohol-responsive LD without causing serious side effects. The minimum efficacy of the drug was 16% of voice improvement, with the average of 41% in patients with alcohol-responsive LD. Sodium oxybate showed no significant changes from placebo in LD pat.