Drug offers ray of hope for 'childhood Alzheimer's'

Not long before her 10th birthday, Hayley Koujaian began to have seizures. They weren't the first sign that something was amiss—for three years she was put in a variety of learning environments and programs but her cognition seemed to be slowly declining instead of progressing.

Finally, after a battery of tests, her family got a concrete and devastating answer in 2011, reports the Chicago Tribune. Hayley suffered from a rare genetic disorder called Niemann-Pick Disease Type C, sometimes dubbed "childhood Alzheimer's," which prevents her from processing cholesterol properly and leads to the gradual shutdown of the nervous system.

Of the 500 or so patients, most diagnosed in elementary school, few will live to be 20, reports Health Day News. "You've got kids who are dying, kids in wheelchairs, kids who can't eat, kids on feeding tubes," says dad Harry Koujaian.

"It's a cruel disease." "These children deserve a fighting chance at life," says Hayley's doctor, Elizabeth Berry-Kravis, who two years ago began giving Hayley bi-weekly shots of cyclodextrin, which seems to help cells process cholesterol, under compassionate use permission from the FDA.

Berry-Kravis is now part of a trial on an experimental new drug called VTS-270 that has slowed decline in mice and cats and is being injected into volunteer kids like Hayley with promising early results—results Berry-Kravis hopes will push VTS-270 through the FDA approval process.

Hayley, now 16, is improving in areas like walking and swallowing instead of simply maintaining her base line, which the Koujaians note on FightNPC.com is all they'd first hoped for.

Questions linger about dosage and more, but, "There is hope," mom Gail Koujaian tells the Tribune. (These parents are losing both their sons to a rare brain disease.)

This article originally appeared on Newser: Ray of Hope in Drug to Treat 'Childhood Alzheimer's'

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