New medicine gives hope to Dutch narcolepsy sufferers

Some 8,000 Dutch people suffer from narcolepsy, a sleep disorder that leaves sufferers spontaneously falling asleep during the day.

Now, for the first time, a new drug approved earlier this month in the US is being touted as the first to target the disease’s roots rather than its symptoms.

“I have been trying to give people a reasonable life for 40 years,” Gert Jan Lammers, special professor of sleep disorders at Leiden University Medical Centre, told broadcaster NOS. “With this medicine, we finally saw real improvement.”

Several of his patients participated in the drug’s research trials, and he says the results are positive. “People feel normal again. I’ve been working on this for 40 years now and I haven’t seen this before. It doesn’t work perfectly for everyone, and it doesn’t work all day, but it’s fundamentally different from what we’ve been able to do so far.”

The drug, Orzeyful (oveporexton) still has to be approved by the European Medicines Agency before being prescribed in the Netherlands, which will likely not happen until at least the end of next year.

But for those who have taken it, including Dutch trial participants who can continue using it before its approval here, it’s been a life-changer.

“It’s like you’re finally getting a good pair of glasses,” says Leontien Sickenga, who has taken the drug for one and one-half years. “With other medication I saw the world a bit better, but with this medication the world is clear. When I’m awake, I’m really there.”

In addition to falling asleep in the day, Sickenga also suffered from muscle relaxation and sleep paralysis, common in narcolepsy patients. “It’s very drastic,” she said.

How it works

According to the US Food and Drug Administration, narcolepsy “is caused by the loss of brain cells that produce orexin, a chemical messenger that regulates wakefulness, sleep, and muscle tone.”

Without orexin, the brain has trouble controlling the boundaries between sleep and waking, resulting in narcolepsy’s debilitating symptoms. But the new drug, rather than trying to relieve those symptoms, “directly activates the same brain receptor that the body’s own orexin would normally stimulate, restoring the missing signal.”

Professor of sleep diagnostics Sebastiaan Overeem, who was not involved in the drug’s research, is positive. “This is fundamentally new and very promising,” he says. “Of course, there are no miracle cures, and we still have to see how this drug works in the long run. But in the field, everyone is impressed.”

Side effects of the drug include urinating more, and it’s not yet known how effective it will be in the long-term.

“I don’t want to be too positive, because some things we can’t know yet” says Lammers. “But so far, all the signals are on green.”