BOSTON -- Imagine your doctor prescribing you a shot of vodka or whiskey to bring you some relief.
Well, a new study out of Boston shows the promise of an alcohol-mimicking drug for patients with the debilitating neurological voice disorder, laryngeal dystonia (LD).
The researchers from Mass Eye and Ear found that an oral medication, sodium oxybate, can give LD patients back their voice.
This groundbreaking clinical trial was borne out of anecdotal reports that LD patients experienced symptom improvements after having a couple alcoholic drinks.
Sodium oxybate mimics the effects of alcohol without the intoxication, and the researchers found that the drug is more effective than a placebo at reducing LD symptoms in patients whose symptoms improve when they consume alcohol.
"We hear many stories of broken lives and careers from patients with laryngeal dystonia, and they have been desperate for new treatments," said lead author Kristina Simonyan, vice chair for clinical research in the Department of Otolaryngology-Head and Neck Surgery at Mass Eye and Ear.
"Our trial gives us hope for a new, effective treatment that can be offered to some of these patients," added Simonyan, who's also professor of Otolaryngology-Head and Neck Surgery at Harvard Medical School. "There's a lot of interest from the dystonia community, and we get many calls from patients asking, 'When will this drug be available? How can I have a prescription?' "
Laryngeal dystonia, known previously as spasmodic dysphonia, is a rare condition that affects more than 50,000 people in the U.S. and Canada. Robert F. Kennedy Jr. has been public with his LD diagnosis.
The neurological disease 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 Botox injections in the throat, but that treatment is ineffective for up to 40% of patients who receive it.
In this recent Phase 2b 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 researchers found the drug improved symptoms by an average of 41% in patients whose symptoms were responsive to alcohol, which was verified first by a controlled test using vodka.
Voice symptoms in alcohol-responsive LD patients significantly improved about 40 minutes after drug intake, with the benefits lasting up to 5 hours.
Though some patients experienced mild side effects such as nausea, dizziness and daytime sleepiness, there were no serious adverse events and no rebound in symptom severity after the drug 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," Simonyan said.
Study participants traveled from across the U.S., U.K. and Canada to participate in the trial, showing the excitement that this drug offers to the dystonia community.
Moving forward, Simonyan's team is planning to conduct a Phase 3 clinical trial to further assess the drug's efficacy and safety in LD patients. Her lab is also leading studies using artificial intelligence to determine which patients might benefit from the treatment, as well as alternative treatments for LD patients whose symptoms are not responsive to alcohol.
Sodium oxybate is a central nervous system agent that is FDA-approved to treat patients with narcolepsy and sleep disorders.
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