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huntingtons disease association

Antibiotic may treat Huntington's disease

NEW YORK (Reuters Health) - A common antibiotic holds promise as a treatment for Huntington's disease, a hereditary disorder for which there is no effective treatment or cure. In mice genetically engineered to develop a similar illness, minocycline, an antibiotic used to treat some forms of acne and arthritis, slowed down the development of symptoms and death from the disease, researchers report.

"The drug that we used is one that is in the pharmacy today," the study’s senior author, Dr. Robert M. Friedlander, of Brigham and Women's Hospital, in Boston, Massachusetts, told Reuters Health.

"It would be one of the first drugs that would be effective for Huntington disease," he said, cautioning that clinical trials need to be conducted to prove its effectiveness in humans.

There is currently no effective treatment for Huntington’s disease, a fatal hereditary disorder in which the death of brain cells eventually leads to involuntary movements and mental and behavioral changes. Symptoms usually begin when a person is 35 to 40 years old. Children of people with Huntington's have a 50% chance of developing the disease.

An enzyme called caspase-1 and a substance called nitric oxide are believed to be involved in Huntington’s disease. Since the antibiotic minocycline targets this enzyme and another one involved in the release of nitric oxide, the researchers tested its effects in mice with a disease similar to Huntington’s.

In mice who received daily treatment with minocycline, symptoms began later than in untreated mice, the researchers report in the July issue of the journal Nature Medicine. These mice also lived an average of 14% longer than untreated mice. In contrast, mice treated with another type of antibiotic did not appear to benefit at all.

In his comments to Reuters Health, Friedlander said that minocycline has been used over the long term in people without causing serious side effects. Before the drug is prescribed to people with Huntington's disease, however, he said that its effectiveness needs to be tested in clinical trials.

Assuming that the drug does slow down Huntington’s disease, it will probably be used in combination with other drugs that are developed to treat the disease, just as a multi-drug '"cocktail" is used to treat AIDS, according to Friedlander.

"It's not a cure for Huntington disease," said Friedlander, but "we're getting there."

  • SOURCE: Nature Medicine 2000; 6:797-801

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