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Monday, December 5, 2011

Afinitor for Autism?

Autism May Involve Disordered White Matter in The Brain | Medindia

BOSTON,Dec. 5, 2011/PRNewswire-USNewswire/ -- It's still unclear what's different in the brains of people withautismspectrum disorders (ASDs), but evidence from genetic and cell studies points to abnormalities in how brain cells (neurons) connect to each other. A study at Children's HospitalBostonnow provides visual evidence associating autism with a disorganized structure of brain connections, as well as defects in myelin -- the fatty, insulating coating that helps nerve fibers conduct signals and that makes up the brain's white matter.

Researchers led byMustafa Sahin, MD, PhD, of the Department of Neurology,Simon Warfield, PhD, director of the Computational Radiology Laboratory, and first authorJurriaan Peters, MD, of both departments atChildren's, used advanced magnetic resonance imaging (MRI) to image the brains of 40 patients (infants to age 25) with tuberous sclerosis complex and 29 age-matched,healthycontrols. Tuberous sclerosis is a rare genetic condition often associated with cognitive and behavioral deficits, including ASDs about 50 percent of the time.

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Armed with thesedata, Sahin has launched a Phase II clinical trial of a rapamycin-like drug called Afinitor® (everolimus; formerly RAD001), sponsored byNovartis, the Tuberous Sclerosis Alliance and Autism Speaks. The trial will enroll 50 patients with TSC to test whether Afinitor improves neurocognition, autism, seizures and sleep disorders. "Specifically modulating neurocognition with a small molecule is only starting to be done," says Sahin. (For more on this trend, seethis related blog post.)

Read more:Autism May Involve Disordered White Matter in The Brain | Medindiahttp://www.medindia.net/health-press-release/Autism-May-Involve-Disordered-White-Matter-in-The-Brain-123572-1.htm#ixzz1fgxcOj9a



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