Aleksey G. Kazantsev and colleagues previously discovered that blocking cholesterol formation in the brain could protect against some of the damage caused by chronic brain disorders like Parkinson’s disease. Several other studies have suggested that too much cholesterol may kill brain cells in similar neurodegenerative diseases. So they launched a search for a so-called “small molecules” — substances ideal for developing into medicines — capable of blocking formation of cholesterol.
They describe discovery of a small molecule that blocks the activity of a key protein involved in cholesterol production. It successfully lowered cholesterol levels in isolated nerve cells and brain slices from mice. If the molecule proves to be a good target for developing new drugs, the scientists note, “it could have a broader application in other neurological conditions, such as Alzheimer’s disease, for which modulation of cholesterol and other associated metabolic pathways might be of therapeutic benefit.”
The authors acknowledge funding from the Carmen Foundation, the RJG Foundation, the Michael J. Fox Foundation for Parkinson’s Research, the CHDI Foundation, the Ecole Polytechnique Federale de Lausanne, and the Swiss National Science Foundation.