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NIH clinical trial begins for treatment of rare, fatal neurological disorder
Date:1/23/2013

A clinical trial to evaluate a drug candidate called cyclodextrin as a possible treatment for Niemann-Pick disease type C1 (NPC), a rare and fatal genetic disease, will start today, researchers announced. Scientists from the NIH's National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) will conduct the clinical trial at the NIH Clinical Center. Reaching this trial stage required collaboration among government, industry, patient advocacy groups and academic researchers.

No therapies approved by the U.S Food and Drug Administration are available to treat NPC. The disease is characterized by the inability of cells to metabolize and dispose of cholesterol and lipids. It causes excessive amounts of cholesterol to accumulate within the liver, spleen and brain. NPC leads to progressive impairment of motor and intellectual function in early childhood. In childhood onset cases, life expectancy does not normally exceed a patient's teenage years.

"A crucial part of the NCATS mission is to collaborate within and beyond the NIH on projects to improve and accelerate the translational research process and deliver tangible improvements in human health," said NCATS Director Christopher P. Austin, M.D. "The cyclodextrin project is an important step in the development of both a potential treatment for a devastating disease that ravages the bodies and minds of its victims and a more efficient way to do translational projects."

In 2009, the NIH Therapeutics for Rare and Neglected Diseases (TRND) program, which is now led by NCATS, selected NPC cyclodextrin as one of its initial pilot projects to repurpose cyclodextrin from its conventional use as an ingredient in other drugs to a therapeutic for this rare disorder. TRND researchers work with project collaborators to conduct preclinical studies advancing potential treatments for rare and neglected diseases
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Contact: NCATS Office of Communications
info@ncats.nih.gov
301-435-0888
NIH/National Center for Advancing Translational Sciences (NCATS)
Source:Eurekalert

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