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A paper announcing a breakthrough discovery in the fight againstNiemann-Pick Type C, coauthored by Olaf Wiest and Paul Helquist ofthe University of Notre Dame's Department Chemistry & Biochemistryand Frederick Maxfield, Chair of Biochemistry at Cornell UniversityWeill College of Medicine, appears in the Proceedings of the National Academy of Sciences this week. The paper shows how use of a histone deacetylaseinhibitor correct the damage done by the genetic disorder andallowed once-diseased cells to function normally. Niemann-PickType C (NPC) involves a genetic flaw that keeps cellsfrom using lipids appropriately and leaves the lipids trapped inthe cell. Brain cells are especially impacted, and destruction ofbrain cells typically kills victims by their teen years and thereis currently no treatment available in the U.S. NPC is an inherited cholesterol metabolism disorder that strikes one in every 150,000 children. Ithas been referred to by the National Institutes of Health as"childhood Alzheimer's" because of similarities in the brains ofNPC and Alzheimer's disease patients. Three of the four grandchildren of former Notre Dame head footballcoach Ara Parseghian died of NPC, and the University has beeninvolved in research on the disorder for years. Last year, itformally united with the Parseghian Foundation, which sponsoredthis work. Last summer, Notre Dame College of Science Dean Gregory Crawfordand his wife Renate bicycled 2,300 miles from Tucson to Notre Dameto raise awareness of the newly strengthened partnership with theParseghian Foundation. Notre Dame's Center for Rare and NeglectedDiseases works to develop therapies and outreach efforts for peoplesuffering from conditions, like NPC, that have bee3n largelyignored by pharmaceutical companies. A team of led by Wiest and Helquist at Notre Dame and Maxfield atCornell, uncovered evidence that histone deacetylase inhibitorscorrect NPC's genetic flaw. Detailed images obtained at Cornell byMaxfield's group gave vivid evidence of the drug's effectiveness,showing how NPC cells became indistinguishable from normal humancells after treatment with the drug. The histine deacetylaseinhibitors have a wide range of potential uses, from rare diseases,the focus at Notre Dame, to several forms of cancer , including leukemia , where they can increase the number of bone marrow cells. Several of the compounds studied are shown to be safe in advancedclinical studies of cancer and one compound is currently approvedby the FDA. "Our biggest single emphasis the last few years has beenNiemann-Pick among these rare diseases," Helquist said. "Wedeveloped several processes for the efficient preparation of thesetypes of drugs. There's a stream of publications and also a streamof patents starting in June 2007 and continuing this year." "If the results in human cells can be confirmed in clinical trials,the fact that the histone deacetylase inhibitors are already inadvanced clinical trials or even approved drugs could greatlyaccelerate the development of a treatment for this devastatingdisease." Source: Olaf Wiest University of Notre Dame Additional References Citations. I am an expert from ipllaserequipment.com, while we provides the quality product, such as Microdermabrasion Machines Manufacturer , China Body Shaping Underwear, Body Slimming Machine,and more.
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