Sodium oxybate (also known as the brand name Xyrem), frequently used to treat adults for symptoms of narcolepsy such as excessive daytime sleepiness and cataplexy, is also effective at treating pediatric patients, according to an abstract published in the journal Sleep and presented at the SLEEP 2017 conference.
The promising preliminary results, which indicate the efficacy of sodium oxybate in reducing cataplexy among children and adolescents, is hopeful news to young patients and their parents. Especially since early diagnosis and treatment can significantly improve the lives of those with narcolepsy, and it’s estimated that about half of narcolepsy patients first show symptoms of the disorder in their youth.
Emmanuel Mignot, MD, PhD, director of the Stanford Center for Sleep Sciences and Medicine, says the positive preliminary findings from the study were not surprising to him given he has used Xyrem off-label in children since 2009.
“Narcolepsy is especially hard in children, [especially since] the onset is explosive when they are young,” says Mignot, who also co-authored a 2010 study on clinical and therapeutic aspects of childhood narcolepsy. “There [tends to be] a 10 to 20 kg weight gain, they become irritable, cannot laugh without collapsing, cannot just go to school, and [often] feel very invalidated. But with Xyrem and other drugs, we can make a huge difference. I have had so many mothers who say that they found their kid again after we successfully treat them.”
Years ago, Mignot along with other researchers noted the paradox in which narcolepsy was being increasingly recognized in children and adolescents, yet there was not enough adequate research that helped illuminate some questions about narcolepsy in pediatric populations.
The treatment methods of narcolepsy in children was one area that hadn’t been explored enough at the time, but the retrospective study showed that sodium oxybate was self-reported by young patients as being an effective treatment option for all narcolepsy symptoms. But as with any other emerging treatment method in children, there were concerns around the potential side effects of giving Xyrem to children.
“One small thing that people have been worried was the effect on puberty and growth, but in general I believe it rather stabilizes things and improves development especially because the [medication] can reduce the obesity that goes with narcolepsy,” says Mignot. “The problem with Xyrem is that occasionally it has some bad complications, [such as] losing too much weight, having nausea, a loss of appetite, and most of all it can increase anxiety in some children. But this too can be addressed.”
In fact, the new study also revealed that sodium oxybate’s safety profile was similar to ones observed in adult patients and there was no indication that children and adolescents taking the medication would be prone to experience side effects not usually seen in adult patients.
As more research addresses outstanding questions on effective treatment options for pediatric patients, Mignot notes that the future for pediatric narcolepsy treatment looks promising. In fact, he looks to hypocretin replacement therapy as another promising lead for pediatric treatment.
“We know that the cause [of narcolepsy] is a lack of hypocretin, so drug companies are trying to find hypocretin and orexin agonists, but that has turned out to be more difficult technically than anticipated,” says Mignot. “It will eventually happen, but it could still take years because companies are not so interested in narcolepsy as a market. Drugs that block hypocretin have been found, and have been developed as hypnotic sleeping pills, such as Belsomra.”
Yoona Ha is a freelance writer and digital strategist.