Niemann-Pick Type C is a rare childhood disease that only affects about 500 people worldwide. But the impact of Niemann-Pick disease may be much more powerful than that statistic indicates. The same genetic error that causes heartbreak to Niemann-Pick families gives hope to millions of others facing Alheimer's, Parkinson's, lysosomal storage disorders, and other neurodegenerative diseases.
Niemann-Pick Type C (NPC) prevents cells from processing cholesterol and other lipids. In the brain, cholesterol is used to produce the chemicals required for neurons and other cells to function properly. When cholesterol processing is blocked, the brain begins to fail. Only recently have researchers realized that other neurodgenerative disorders, like Alzheimer's, are also affected by defects in the cholesterol pathway. Scientists now recognize that NPC research can provide important insights to speed identification of treatments for many disorders.
The National Niemann-Pick Disease Foundation is the second largest nonprofit funder of Niemann-Pick research in the world. Since 1992, the Foundation has sponsored ground breaking research in neurology and cell biology. If you would like your charitable contribution to have a big impact, please consider donating to the Foundation.
What do researchers say?
"the data showed interesting similarities between NPC and AD" T. Ohm (Pharmacopsychiatry)
"The regulation of lipid metabolism . . . could be a therapeutic and preventive target for Alzheimer's disease." K. Kamino (Shinkei Seishin Yakurigaku Zasshi)
"AD and Niemann-Pick type C disease [may] share a common cascade" M. Michikawa (Current Alzheimer's Research)
"cholesterol [has] been implicated in the formation of amyloid plaques in
Alzheimer's disease" J. Vance (Seminars in Cell and Developmental Biology)
"intriguing parallels exist in the cellular pathology of these two diseases, including neurofibrillary tangle formation, prominent lysosome system dysfunction, and influences of apolipoprotein E" R. Nixon (American Journal of Pathology)
"Neurofibrillary tangles are seen both in senile dementia of Alzheimer's disease and in juvenile dementia of Niemann-Pick type C disease" R. Distl (Acta Neuropathologica)
NPC is one of 40 lysosomal storage diseases that together affect thousands of people, mostly children. Lysosomal storage diseases share many common characteristics and research advances made for one disease often can be applied to other diseases. Other lysosomal diseases include Tay-Sachs and Gaucher.
Niemann-Pick research can also benefit other diseases, such as Parkinson's, stroke, heart disease, and cancer. More work needs to be done to fully understand how Niemann-Pick affects these common diseases. A lack of money is the main obstacle. Even small donations make a big difference. Join us in funding Niemann-Pick research today.
Tommorrow's Hope and HFTP Florida Gold Coast have been consistent benefactors of NP research. We are very appreciative of their faith in supporting a rare disease. Their support has helped identify ties between Niemann-Pick and diseases that affect millions of people. Also, thanks to Charleston Orwig who donated their services to create the ad (shown above) that highlights these ties.
Niemann-Pick Type C is a rare childhood disease that only affects about 500 people worldwide. But the impact of Niemann-Pick disease may be much more powerful than that statistic indicates. The same genetic error that causes heartbreak to Niemann-Pick families gives hope to millions of others facing Alheimer's, Parkinson's, lysosomal storage disorders, and other neurodegenerative diseases.
The National Niemann-Pick Disease Foundation is the second largest nonprofit funder of Niemann-Pick research in the world. Since 1992, the Foundation has sponsored ground breaking research in neurology and cell biology. If you would like your charitable contribution to have a big impact, please consider donating to the Foundation.