Antibiotic Holds Promise as Huntington's Treatment

FRIDAY, Aug. 5 (HealthDay News) -- Preliminary research suggests a decades-old antibiotic may help Huntington''s disease patients by preventing mutant cells from wreaking havoc in the brain.

Studies in the laboratory and in mice suggest that Clioquinol could potentially become the first weapon against Huntington''s disease, which is incurable.

"There''s no really good treatment at this point," said study co-author Dr. Stephen Massa, a neurology researcher at the VA Medical Center in San Francisco. Clioquinol is "as promising as anything we''ve seen."

However, he cautioned that the drug, which is also being tested as a treatment for Alzheimer''s disease, has a long way to go before it could be used in clinical practice. "There are a lot of things that scientists try in animals and in (laboratory) cultures that never end up making it all the way to people."

There''s plenty at stake in Huntington''s research. An estimated one in every 10,000 Americans has the disease, which causes rapid, uncontrolled movements, like Parkinson''s disease, and can eventually lead to a variety of mental and physical problems. Death usually occurs within 10 to 25 years of diagnosis.

Perhaps the most famous American with Huntington''s was the late folk singer Woody Guthrie. While the disease is genetic, affecting as many as half of the children of people with the disease if they live long enough, his son, singer Arlo Guthrie, appears to have avoided the illness.

Doctors treat the disease with drugs that target its symptoms, Massa said, but "there''s been a lot of attempts with clinical trials and pre-clinical work to try to attack the underlying disease."

In the new study, Massa and colleagues tested Clioquinol on cells, and on mice genetically engineered to develop the disease. They report their findings in this week''s issue of the Proceedings of the National Academy of Sciences.

According to Massa, the drug appears to prevent the accumulation of protein blockages in the brain that contribute to the disease. However, internal use of the drug is banned in the United States because of side effects; according to Massa, it''s still used as an external treatment for skin infections.

The study results are "encouraging," said Dr. William Yang, an assistant professor and neurology researcher at the University of California, Los Angeles. "This is likely the first of a group of interesting, new avenues of treating this disease. However, this is a beginning, and there''s a lot of other work that needs to be done to confirm that this is really relevant."

More information

To learn more about Huntington''s disease, visit the Huntington''s Disease Society of America.

SOURCES: Stephen Massa, M.D., Ph.D., neurology researcher, VA Medical Center, San Francisco; William Yang, M.D., Ph.D., assistant professor, Neurobehavioral Genetics Research Center, David Geffen School of Medicine at University of California, Los Angeles; Aug. 1-5, 2005, Proceedings of the National Academy of Sciences