New research by scientists in the United States carried out in mice has revealed a compound that releases excessive cholesterol accumulated inside cells.
According to the team their discovery could lead to a therapy for Niemann-Pick type C disease (NP-C), an inherited disorder characterized by abnormally high cholesterol levels in all body organs.
Patients suffering from NP-C carry a genetic mutation which causes excessive amounts of cholesterol to accumulate in lysosomes - vesicles in cells - which leads to liver disease, dementia and neurodegeration.
The disease is usually diagnosed in children from age 3 to 15, most of whom die before age 20 with some never reaching age 10.
About 10 percent of cases have the disease in its adult form which manifests itself especially through neurological and psychological symptoms - few people diagnosed with NP-C ever reach the age of 40.
Professor John Dietschy, senior author of the study says the study has shown that very quickly after administration of the compound, the huge pool of cholesterol that has just been accumulating in the cells is suddenly released and metabolized normally.
The researchers at the University of Texas Southwestern Medical Center at Dallas, injected a single dose of CYCLO, a cholesterol-binding agent, into mice aged 7 days that had the NP-C mutation and shortly after receiving the injection, the mice began to process cholesterol just like the healthy mice - 49 days later the mice continued to demonstrate substantially lower tissue cholesterol levels than mice who did not receive the treatment and the mice also had improved liver function and less neurodegeneration.