Niemann-Pick disease type C, long thought to be tied to cholesterol metabolism, may eventually be treated with immune inhibitors
UT Southwestern researchers have identified an immune protein tied to the rare neurodegenerative condition known as Niemann-Pick disease type C. The finding could offer a powerful new therapeutic target for Niemann-Pick disease type C, a condition that was identified more than a century ago but still lacks effective treatments.
“Niemann-Pick disease has never been considered an immune disorder,” says study leader Nan Yan, PhD, associate professor of immunology and microbiology. “These findings put it in a whole new light.”
Niemann-Pick disease type C, which affects about 1 in every 150,000 people worldwide, has long been considered a disease of cholesterol metabolism and distribution, a topic well-studied at UT Southwestern, where faculty members Michael Brown, MD, and Joseph Goldstein, MD, won the Nobel Prize in 1985 for their discovery of low-density lipoprotein (LDL) receptors, which led to the development of statin drugs.
