A lesser-known neurological disorder, Laryngeal dystonia (LD), can severely affect an individual’s speech due to involuntary spasms in the vocal cords. This condition can take a toll on a person’s mental well-being, social interactions, and career. The standard treatment for LD is injections of botulinum neurotoxin (Botox), but this method proves ineffective for nearly 40% of those who undergo it.
However, a recent study led by researchers from Mass Eye and Ear, a member of the Mass General Brigham healthcare system, has found that an oral medication, sodium oxybate, could be more effective than a placebo at easing LD symptoms in patients who find relief from alcohol consumption.
This research, published in the Annals of Neurology, is the culmination of over 10 years of study inspired by LD patients who reported symptom relief after moderate alcohol consumption. Sodium oxybate, an FDA-approved central nervous system agent used for treating narcolepsy and sleep disorders, replicates some of alcohol’s effects.
In a trial involving over 100 patients, a single dose of sodium oxybate significantly reduced symptoms in patients with alcohol-responsive LD without causing severe side effects. The least amount of voice improvement observed was 16%, with an average improvement of 41% among these patients. However, the drug did not show significant changes in LD patients whose symptoms didn’t improve with alcohol.
Laryngeal dystonia, previously known as spasmodic dysphonia, is a rare condition affecting over 50,000 individuals in the US and Canada. It is more prevalent in women and typically starts in their 40s, often significantly impacting their quality of life. The exact neurological cause remains unknown, and it takes an average of 5.5 years for patients to receive an accurate diagnosis. Once diagnosed, treatment options are limited to Botox injections every three to four months for life, provided they are effective.
In previous open label trials, Simonyan’s team demonstrated that sodium oxybate improves voice symptoms in 82% of patients with alcohol-responsive LD. In their latest study, they aimed to validate the drug’s effectiveness against a placebo in a more rigorous, double-blind randomized clinical trial.
A group of 106 participants with LD, 50 of whom experienced alcohol-responsive symptoms, were enrolled in the trial. Participants had to travel from across the U.S., the U.K., and Canada to partake in the trial, highlighting the potential this drug holds for the dystonia community.
The results showed that sodium oxybate was significantly more effective at reducing symptoms than a placebo for patients with alcohol-responsive LD, but not for those whose symptoms do not improve with alcohol. Voice symptoms in alcohol-responsive LD patients noticeably improved around 40 minutes after taking the drug, with the benefits lasting up to 5 hours. Some patients experienced mild and temporary side effects like nausea, dizziness, and daytime sleepiness, but no serious adverse events were reported, and there was no rebound in symptom severity after the drug wore off.
Simonyan’s team plans to conduct a phase 3 multi-site randomized clinical trial to further assess the drug’s efficacy and safety in LD patients. Additionally, her lab is spearheading studies using artificial intelligence to determine which patients might benefit from the treatment and to explore alternative treatments for LD patients whose symptoms are not responsive to alcohol.
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