Molecule found that blocks cholesterol in brain

July 13, 2011

(Credit: ACS)

A “needle-in-the-haystack” search through nearly 390,000 chemical compounds has led scientists at the Mass General Institute for Neurodegenerative Disease to find a substance that can sneak through the protective barrier surrounding the brain, with promising effects for new drugs for Parkinson’s and Huntington’s disease.

Several other studies have suggested that too much cholesterol may kill brain cells in similar neurodegenerative diseases.  The researchers launched a search for “small molecules” — substances ideal for developing into medicines — capable of blocking formation of cholesterol. They discovered a brain-permeable SIRT2 inhibitor that blocks the activity of a key protein involved in cholesterol production.

The research is described in the July 11, 2011 episode of the American Chemical Society’s Global Challenges/Chemistry Solutions podcast series by the study’s lead author Aleksey G. Kazantsev, Ph.D., of the Mass General Institute for Neurodegenerative Disease.

Ref.: Aleksey G. Kazantsev, A Brain-Permeable Small Molecule Reduces Neuronal Cholesterol by Inhibiting Activity of Sirtuin 2 Deacetylase, ACS Chemical Biology, 2011; 54 (4): 1010 [DOI: 10.1021/cb100376q]