Global Challenges/Chemistry Solutions

Promoting Public Health: Toward new medication for chronic brain diseases

July 11, 2011

Chronic brain disorders such as Parkinson's
disease may become more manageable
using a new substance that can sneak
through the brain's protective barrier and
block cholesterol formation.
Credit: iStock

Summary

A needle-in-the-haystack search through nearly 390,000
chemical compounds has led scientists to a substance
that can sneak through the protective barrier surrounding
the brain with effects promising for new drugs for
Parkinson’s and Huntington’s disease. They report on
the substance, which blocks formation of cholesterol
in the brain, in the journal, ACS Chemical Biology.

A needle-in-the-haystack search through nearly 390,000 chemical compounds has led scientists to a substance that can sneak through the protective barrier surrounding the brain with effects promising for new drugs for Parkinson’s and Huntington’s disease. They report on the substance, which blocks formation of cholesterol in the brain, in the journal, ACS Chemical Biology.

Here’s the study’s lead author Aleksey G. Kazantsev, Ph.D., of the Mass General Institute for Neurodegenerative Disease in Charlestown, Massachusetts …

“We 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 the team launched a search for so-called “small molecules” — substances ideal for developing into medicines — capable of blocking formation of cholesterol. Kazantsev and colleagues discovered a small molecule that blocks the activity of a key protein involved in cholesterol production.

“The small molecule 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, 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.”

Smart Chemists/Innovative Thinking

Smart chemists. Innovative thinking. That’s the key to solving global challenges of the 21st Century. Please check out more of our full-length podcasts on wide-ranging issues facing chemistry and science, such as promoting public health, developing new fuels and confronting climate change, at www.acs.org/GlobalChallenges. Today’s podcast was written by Katie Cottingham. I’m Adam Dylewski at the American Chemical Society in Washington.

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Aleksey G. Kazantsev, Ph.D.,
Mass General Institute for
Neurodegenerative Disease
Charlestown, Massachusetts