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Scientists Uncover New Weapon Against Huntington's

Treatment could save brain cells from ravages of neurodegenerative disease

FRIDAY, April 3 (HealthDay News) -- Harvard scientists say they've found a possible way to prevent brain cells from falling victim to the ravages of Huntington's disease.

The work is still in the preliminary stages, and the researchers don't know if the strategy will work in humans. Still, a Huntington's disease expert said the findings could lead to a way to combat the incurable condition.

"They suggest a new way that you could approach developing treatments. I think it's going to emerge as a theme," said University of California at Irvine professor Leslie Thompson.

Huntington's disease is inherited, and children of parents with a single faulty gene have at least a 50 percent chance of developing it. The condition typically develops in middle age, causing the body to move involuntarily, leading to symptoms such as balance and coordination problems, slurred speech, swallowing problems and dementia.

Death typically occurs within 10 to 30 years.

There's no treatment to stop the progression of Huntington's disease or cure it, although patients can take drugs to control their symptoms.

"It's like when you have a rotten tooth," said study co-author Dr. Dimitri Krainc. "You can take painkillers so you don't feel the pain, but the tooth is still rotten."

Krainc, an associate professor of neurology at Harvard Medical School, and his colleagues tried to find a way to reduce the build-up of proteins in brain cells stricken by the disease.

Essentially, Krainc said, proteins pile up in the cells and cause problems. "It's like trash that doesn't get picked up in a crowded apartment," he said, "If you don't remove trash, it eventually suffocates you."

Indeed, clogged cells either stop functioning properly or die, Thompson said.

In the new study, the researchers tinkered with the troublesome proteins through genetic modification. They report their findings in the April 3 issue of Cell.

According to Krainc, the researchers found that their approach allowed proteins within the cells to degrade as they're supposed to. The strategy worked in worms, mice and the brain cells taken from dead people.

Drugs known as HDAC inhibitors have the same effect and are already being tested as treatments for Huntington's disease and cancer, Krainc said. The drugs are currently used to treat psychiatric disorders.

There are many unknowns, however. Researchers don't know whether the treatment will work in humans, and the potential costs of drugs are unclear.

Still, research may take a matter of years, not decades, Krainc said.

In the big picture, the new research "could have broad implications for a number of neurodegenerative diseases," Thompson said.

Alzheimer's disease, for example, is also caused when cells become clogged and fail to function properly.

"It's an extremely exciting and promising area of research," Thompson said, "and new and novel."

More information

To learn more about Huntington's disease, try the U.S. National Institutes of Health.

SOURCES: Dimitri Krainc, M.D., Ph.D., professor, neurology, Harvard Medical School, Boston; Leslie Thompson, Ph.D., professor, psychiatry and human behavior, University of California at Irvine; April 3, 2009, Cell

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