Home Niemann-Pick Disease Type C Niemann-Pick Disease Type C: A Rare Disease with Dedicated Clinical Investigators in...

Niemann-Pick Disease Type C: A Rare Disease with Dedicated Clinical Investigators in Hot Pursuit

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researcher working collection data in the research laboratory / researcher engineer working on scientific formulas in the chemical laboratory

Niemann-Pick Disease Type C disrupts the movement of cholesterol and other fats within cells. With 400 in the US afflicted with the rare disease, Niemann-Pick Type C happens to be the cellular receptor for the Ebola virus.

Interest in Niemann-Pick Disease Type C Interest

Stephen Sturley, a professor in the department of pediatrics at Columbia University is on the scientific advisory board for the National Niemann-Pick Disease Foundation. He first became intrigued by the disease due to his research focus on cholesterol and was one of the first researchers to be funded by the Ara Parseghian Medical Research Fund.

Connections and the Investigational Drug

Dr. Sturley notes “If you don’t have Niemann-Pick Type C protein, then one prediction would be that you’d be resistant to Ebola virus.” Put another way, if the medicine could cause a short-term case of Niemann-Pick Type C disease, it might prevent those exposed to Ebola from contracting the disease—a current area of research for Sturley reports WCAI Cape and Islands.

Another connection—the drug that is currently in FDA trials to treat Niemann-Pick Type C is beta-cyclodextrin—the active ingredient in Febreze. That’s right-the anti-odor spray! The cyclodextrin cage encloses cholesterol and supports the movement to its target destination within the cell. Based on pure serendipity, the researchers were testing another drug and were simply using the Febreze ingredient to dissolve the drug and make it available to the cells. However, Sturley notes that “After a series of experiments, it was discovered that the Febreze compound was the active compound rather than the drug candidate that was being looked at.”

Clinical Trials

There are a few clinical trials involving cyclodextrin including the sponsor CTD Holdings (2 active studies) and Washington School of Medicine.

CTD Holdings, Inc. organized as a Florida corporation in 1990 changed its name to Cyclodextrin Technologies, a wholly-owned subsidiary of CTD Holdings, Inc. They focus on the use of cyclodextrins in drug development. The FDA recently accepted the Type II Drug Master File for their lead drug candidate called Trappsol® Cyclo™, for filing. The company’s Neiman-Pick Type C disease focus started in 2015.

Lead Research/Investigator on the disease

Stephen Sturley

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