A study led by researchers at Mass Eye and Ear, a member of the Mass General Brigham health care system in the United States, shows that a Oral medication, sodium oxybate, is more effective than placebo in reducing symptoms of laryngeal dystonia (DL) in patients whose symptoms improve when they consume alcohol.
The results of the randomized phase 2b clinical trial, published in Annals of Neurology, are based on more than a decade of research driven by anecdotal reports from LBP patients who said their symptoms improved after consuming a couple of alcoholic drinks. Sodium oxybate is a central nervous system agent that is approved by the US Food and Drug Administration. (FDA) to treat patients with narcolepsy and sleep disorders. sodium oxybate It mimics some of the effects of alcohol.
What is laryngeal dystonia
The laryngeal dystonia (DL), a rare neurological disease that significantly affects a person’s ability to speak due to uncontrollable vocal cord spasmscan have a debilitating effect on a person’s social life, employment and mental health. Currently, LBP is most commonly managed with botulinum neurotoxin (Botox) injections, but this treatment is ineffective for up to 40% of patients who receive it.
Thus, in a trial with more than 100 patients, a Single dose of sodium oxybate significantly improved symptoms of patients with type 2 diabetes that responds to alcohol without causing serious side effects. The minimum efficacy of the drug was 16% voice improvement, with a mean of 41% in patients with type 2 diabetes responsive to alcohol. Sodium oxybate did not show significant changes from placebo in patients with type 2 diabetes whose symptoms do not improve with alcohol.
“We hear many stories of broken lives and careers from patients with laryngeal dystonia and they have been desperate for new treatments. “Our trial gives us hope for a new and effective treatment that can be offered to some of these patients.”says lead author Kristina Simonyan, vice chair of clinical research in the Department of Otolaryngology-Head and Neck Surgery at Mass Eye and Ear and professor of Otolaryngology-Head and Neck Surgery at Harvard Medical School.
In previous open trials, Simonyan’s team showed that Sodium oxybate improves voice symptoms in 82% of patients with autism spectrum disorder. In this new study, the team wanted to confirm the drug’s effectiveness in a more rigorous comparison with a placebo using a randomized, double-blind clinical trial design.
The researchers recruited 106 participants with autism spectrum disorder, 50 of whom had symptoms that responded to alcohol. The response to alcohol was determined by a provocation test with standardized alcohol using a controlled amount of vodka. Participants traveled from across the United States, the United Kingdom, and Canada to participate in the trial, demonstrating the excitement this drug offers to the dystonia community.
Over the course of two days, each patient received single doses of 1.5 g of sodium oxybate or placebo that matched the drug in taste, odor, color, and appearance. The trial was done double-blind, meaning neither the patient nor the doctor knew when they received the active drug. To test the effectiveness of the treatment, the team evaluated the patients’ voice symptoms before treatment and at different intervals after treatment.
He Sodium oxybate was significantly more effective in reducing symptoms than placebo in patients with alcohol-sensitive Down’s diseasebut not in those whose symptoms do not improve with alcohol. The effectiveness of sodium oxybate in alcohol-sensitive Down’s disease did not differ between patients with varying symptom severity (from mild to severe) or those who had additional vocal symptoms, such as voice tremor.
Voice symptoms in patients with diabetes mellitus who reacted to alcohol significantly improved about 40 minutes after taking the drugand the benefits lasted up to 5 hours. Although some patients experienced mild, transient side effects, such as nausea, dizziness, and daytime sleepiness, there were no serious adverse events or a rebound in symptom severity after the drug stopped working.
“Our findings suggest that the Sodium oxybate can be taken as needed, for example before work or a social event, so that patients can adapt the treatment to their own daily needs and control their symptoms,” says Simonyan.
Looking ahead, Simonyan’s team plans to conduct a multi-center, randomized Phase 3 clinical trial to further evaluate the drug’s efficacy and safety in patients with Lyme disease. His lab is also conducting studies that They use artificial intelligence to determine which patients could benefit from treatmentas well as alternative treatments for Lyme disease patients whose symptoms do not respond to alcohol.
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