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Sleep researchers at SRI International identify promising new treatment for narcolepsy
Date:5/7/2014

w DTA model and the Ataxin model to compare R-baclofen against gamma-hydroxybutyrate (GHB). Sodium oxybate, the sodium salt of GHB, was approved by the FDA in 2002 as the only therapeutic for narcolepsy that simultaneously alleviates cataplexy, excessive daytime sleepiness and nocturnal sleep disruption. However, it remains unclear how this drug exerts its beneficial effects.

It was suspected that GHB works by affecting brain cells that respond to a neurotransmitter known as gamma-aminobutyric acid (GABA), which primarily functions to inhibit excitability and regulate muscle tone. To study the mechanism of action of GHB, SRI Biosciences' researchers tested R-baclofen, which blocks the GABA receptors suspected to be the target of GHB.

The research team found that R-baclofen promoted sleep time and longer bouts of wakefulness during the appropriate times for mice and also suppressed cataplexy. GHB modestly reduced cataplexy and increased sleep intensity, but did not improve other symptoms of narcolepsy to the extent that R-baclofen did. "The improvement in wakefulness that we observed after R-baclofen was a particularly unexpected and important finding," said Black.

"R-Baclofen works better than GHB in these two mouse models, but it remains to be determined whether it will work better in humans," cautioned Kilduff. "Although baclofen is already known to be safe for use in humans, the dose that is effective for spasticity may be different than the dose of R-baclofen that has the potential to treat narcolepsy."


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Contact: Dina Basin
dina.basin@sri.com
650-859-3845
SRI International
Source:Eurekalert

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