A clinical trial has shown that sodium oxybate, a medication that mimics some effects of alcohol, can significantly improve speaking ability in people with laryngeal dystonia who experience symptom relief from alcohol.
Published in Annals of Neurology | Estimated reading time: 6 minutes
Laryngeal dystonia affects over 50,000 people in the US and Canada, causing uncontrollable vocal cord spasms that can devastate a person’s ability to speak and seriously impact their social life, employment, and mental health. While botulinum toxin injections are the current standard treatment, they don’t work for up to 40% of patients.
Led by researchers at Mass Eye and Ear, this new study emerged from patients’ reports that their symptoms improved after consuming alcohol. The team conducted a rigorous trial involving 106 participants from across the US, UK, and Canada to test sodium oxybate, a medication already approved for narcolepsy that mimics some of alcohol’s effects.
“We hear many stories of broken lives and careers from patients with laryngeal dystonia and they have been desperate for new treatments,” says lead author Dr. Kristina Simonyan. “Our trial gives us hope for a new, effective treatment that can be offered to some of these patients.”
The trial found that a single 1.5g dose of sodium oxybate significantly improved symptoms in patients whose condition responds to alcohol. Voice improvements began around 40 minutes after taking the medication and lasted up to 5 hours. The minimum improvement was 16%, with an average improvement of 41% in alcohol-responsive patients.
The condition disproportionately affects women and typically begins in their 40s. Currently, diagnosis takes an average of 5.5 years, and treatment options are limited. The study determined whether patients were alcohol-responsive through standardized testing using controlled amounts of vodka before enrolling them in the double-blind trial.
While some participants experienced mild side effects like nausea, dizziness, and sleepiness, there were no serious adverse events or symptom rebounds after the medication wore off. “Our findings suggest that sodium oxybate can be taken on an as-needed basis, such as before work or a social event, so patients can tailor treatment to their own daily needs and get in control of their symptoms,” said Dr. Simonyan.
The researchers are now planning a larger phase 3 trial to further assess the drug’s safety and effectiveness. They’re also using artificial intelligence to help identify which patients might benefit from the treatment and exploring alternative options for those whose symptoms don’t respond to alcohol.
Glossary
- Laryngeal Dystonia
- A neurological condition causing uncontrollable vocal cord spasms that significantly impact speaking ability.
- Double-blind Trial
- A study design where neither the participants nor the researchers know who receives the actual treatment versus placebo.
- Sodium Oxybate
- A central nervous system medication that mimics some effects of alcohol, currently approved for treating narcolepsy.
Test Your Knowledge
How long did the medication’s effects last?
Answer: Up to 5 hours
What was the average symptom improvement in alcohol-responsive patients?
Answer: 41%
How many people in the US and Canada are affected by laryngeal dystonia?
Answer: Over 50,000 people
How long does it typically take to receive a correct diagnosis?
Answer: 5.5 years on average
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