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30-Year-Old Antibiotic Offers Hope for Patients With Huntington's Disease


WebMD Health News

June 30, 2000 -- There is currently no treatment to slow disease progression in patients with Huntington's disease, the devastating hereditary brain disorder that killed folk-singer Woody Guthrie and afflicts some 30,000 Americans.

Victims' mental and physical abilities degenerate, leading to dementia, and they lose control of their movements and eventually die. But animal studies from Harvard University's Brigham and Women's Hospital offer hope that sufferers will soon have a weapon to help them fight the disease -- one that has been around for the last 30 years.

Mice bred as models for Huntington's lived 14% longer when treated with the common antibiotic minocycline, the July issue of the journal Nature Medicine reports. Harvard researchers say they will soon begin human trials of the antibiotic in patients with Huntington's disease.

"If the human safety trials go well, we should be able to recommend this for Huntington's patients within the year," study author Robert M. Friedlander, MD, tells WebMD. "Our studies showed that there were more minocycline side effects in the Huntington's mice compared to normal mice. This could pose a problem, but I don't think it will translate to humans."

If the antibiotic appears safe and effective in patients with Huntington's, Friedlander suggests it might be taken daily by those who know they will develop the disease, beginning perhaps five to 10 years before symptoms appear. Children who have a parent with Huntington's have a 50% chance of also developing the disease, and symptoms generally first develop between the ages of 35 and 45. A genetic test has been available since 1993.

"This is all hypothesis, because we have no human data at this point," Friedlander says. "What is real is that today there is no drug treatment for Huntington's. This is a very good candidate for a treatment, not a cure."

Friedlander and other experts say the antibiotic could be the first in a series of advances bringing the management of Huntington's out of the Dark Ages. They envision a drug cocktail approach similar to that now being used to treat AIDS patients, in which different agents target different mechanisms of this complex disease.

"I think the importance of this study is not necessarily that this drug will be useful in humans, although it may turn out to be, but it shows that drug compounds can potentially be helpful," says Christopher Ross, MD, PhD, who was not involved with the study.

"This drug by itself may not have a huge impact, but if you can get two or three drugs acting in different ways, the cocktail might be quite effective," says Ross, professor of psychiatry and neuroscience at Johns Hopkins University and chair of the Medical and Scientific Advisory Committee of the Huntington's Disease Society of America (HDSA).

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