by Sharon Lovering
Do you have trouble falling asleep or staying asleep? Do you wake up groggy, sluggish or forgetful? Do you find yourself napping during the day? Are your sleep patterns different from most people you know? Do you find it hard to concentrate? Do you fight to stay awake during the day? Does the time your body wants to sleep seem to shift over time? Are you frustrated by this? If you answered "yes" to any of these questions, and you're totally blind, it could be Non-24-Hour Sleep-Wake Disorder.
What exactly is Non-24? It is a chronic circadian rhythm sleep disorder that affects more than 70 percent of totally blind individuals, or about 80,000 people in the United States alone. It occurs almost entirely in individuals who are totally blind and lack the light sensitivity necessary to reset the circadian clock. Without light perception, the brain's circadian rhythms, which guide many of the body's functions, including sleep, are not reset to a regular 24-hour cycle. Individuals with Non-24-Hour Disorder are unable to synchronize their internal clock to the 24-hour day-night cycle, which disrupts their sleep-wake cycle. The disorder was first diagnosed in 1948 by Dr. Rammler in Germany.
Enter Vanda Pharmaceuticals. "About 10 years ago, when I founded Vanda, we developed an interest, among other things, in developing drugs that address circadian rhythm disorders," stated Mihael Polymeropoulos, president and CEO of Vanda Pharmaceuticals. "We identified as the prototypical disorder, Non-24-Hour Sleep-Wake Disorder that almost exclusively occurs in the blind."
With Non-24, "blind patients who no longer have the time cue to reset their body clock begin to be in a pattern in sync with their endogenous clock, which happens to be about 24 1/2 hours per day," Polymeropoulos said. "It became apparent to us in 2004 that tasimelteon, a small-drug molecule that binds the melatonin 1 and 2 receptors in the human brain, it could act as a therapeutic for this disorder. ... The hope was that tasimelteon could act as the light cue to instruct the body clock in a small group of neurons in the brain."
Vanda began by studying the pharmacological properties of tasimelteon about 10 years ago. Clinical trials began in 2010. "We learned a lot about the blindness community," Polymeropoulos said. "All of us had some preconceived notions with blind people, what they can do and cannot do, and it has been an amazing education for us as well outside of Non-24."
Two key studies measured the drug's efficacy, one in the U.S. and a second that took place in both the U.S. and Germany, he added. Two longer-term safety studies are looking at potential side effects, one in the U.S. and one in France. More than 1,300 patients were treated during the studies. There are about 70 people still in treatment in the U.S. and 40 people in France who have been treated continuously over the last three years.
"The results of the studies suggested that tasimelteon, administered once a day at night, can indeed reset the body clock and give patients the ability now to rest at night," Polymeropoulos noted. "It is very exciting, especially responding to a medical need by thousands of blind patients who actually suffered and struggled ... for many, many years. ... What is more troubling is that ... these patients did not even know what they had." Patients were treated for insomnia, depression, or dementia; others were called lazy or "bad girls." Now we know that it's a circadian rhythm disorder that is treatable.
While developing Hetlioz (TM), Vanda worked for about 16 years, 6 of them with Bristol Myers Squibb, before getting approval for the drug from the Food and Drug Administration on Jan. 31, 2014. "This was an exceptional journey because of it being the first drug for this disorder," Polymeropoulos said. "They have never approved a drug before [for this disorder]."
Hetlioz (TM) (also known as tasimelteon) can help people with Non-24 regulate their body clocks. It will soon be available at pharmacies with a prescription from your doctor. In the U.S., the bottle even has braille on it. Polymeropoulos hoped that having braille on the bottle would inspire other manufacturers to develop accessible labels for blind patients. "There's a lot to be done ... but at least it's a step in the right direction," he added.
If you need more information about this medication, or physicians who can diagnose Non-24, call 1-844-HETLIOZ (1-844-438-5569) or visit www.hetlioz.com.