Narcolepsy is a rare, debilitating neurological disorder that impacts the brain's ability to control sleep-wake cycles, leading to excessive daytime sleepiness with uncontrollable sleep attacks.
In addition to excessive daytime sleepiness, narcolepsy patients also experience sleep paralysis, hallucinations, and in some cases episodes of cataplexy (partial or total loss of muscle control, often triggered by a strong emotion such as laughter). The symptoms appear in childhood or adolescence, but many people have symptoms of narcolepsy for years before getting a proper diagnosis.
Narcolepsy with cataplexy is caused by the loss of a chemical in the brain called hypocretin. Hypocretin acts on the alerting systems in the brain, keeping us awake and regulating sleep wake cycles. In narcolepsy, the cluster of cells that produce hypocretin—located in a region called the hypothalamus—is damaged or completely destroyed. Without hypocretin, the person has trouble staying awake, and also experiences disruptions in the normal sleep-wake cycles.
Currently in the US, there are limited FDA approved medications for narcolepsy, with no new treatments approved in over a decade. Medications and behavioral treatments can improve symptoms for people so they can lead productive lives, but many patients are still not well controlled. No approved therapies address the underlying causes of the disease and there is no cure for narcolepsy.