AANS Neurosurgeon | Volume 26, Number 3, 2017

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Drug Trial Shows Promise for Deadly Neurological Disorder

Cyclodextrin slows progression of Niemann-Pick type C

 

Results of a small clinical trial show promise for treating a rare neurodegenerative condition that typically kills those afflicted before they reach age 20. The disease, called Niemann-Pick type C (NPC), causes cholesterol to build up in neurons, leading to a gradual loss of brain function. In the drug trial, researchers have shown that treatment with a type of sugar molecule called cyclodextrin slows progression of the disease.

“We were surprised to see evidence that this therapy could slow progression of the disease and, in some cases, get back some function — speech in particular,” said first author Daniel S. Ory, MD, the Alan A. and Edith L. Wolff Professor of Cardiology at Washington University School of Medicine in St. Louis. “In a neurodegenerative disease, therapies can’t recover neurons that have died. But if some brain cells are dysfunctional rather than dead, it seems this drug can recover some of that function.”

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