Study - Possible treatment for Huntington's

WASHINGTON -- A bile acid the body produces in small amounts is able in laboratory studies to slow the progress of Huntington's disease, a fatal, inherited brain disorder that destroys the mind and has affected about 30,000 Americans.

"We found in mouse studies that this compound protects the animals' neurons (brain cells) from the effects of the Huntington's disease gene," said C. Dirk Keene, first author of a study appearing in the Proceedings of the National Academy of Sciences.

Keene said there are many other laboratory studies required before the drug could be considered for testing in humans, but he said the research is important because it offers the hint of a future treatment for a lethal disorder that now has no treatment.

"It is an interesting finding that needs to be followed up," said Dr. James F. Gusella, a Huntington's disease researcher at Massachusetts General Hospital and the Harvard Medical School. But he cautioned that although animal research provides clues about Huntington's, "They are still quite distant from being able to treat human patients."

Huntington's disease, or HD, is caused by a single defective gene that is inherited. About one in every 10,000 babies worldwide is born with the gene mutation and about 30,000 Americans have been diagnosed with HD.

The disease causes a progressive loss of neurons in the brain, leading to changes in personality, mood and judgment. As the disease progresses, patients develop slurred speech, an unsteady walk and difficulty in swallowing. Symptoms can appear as early as age 2 or as late as age 70 and will lead to death in 10 to 25 years.

Researchers discovered the gene that causes Huntington's in 1993, but there are still no approved drugs or treatments to slow its progression.