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New Data Point To Huntington's Disease Hope

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A study being published Monday offers hope for those with Huntington's disease. The Archives of Neurology has a report about a drug aimed at the serious cognitive deficits that people with Huntington's also suffer.


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Huntington's disease, which killed folk musician Woody Guthrie 43 years ago, has long been a no hope disease, no hope of treatment, no hope of a cure. Diagnosis generally means a slow and cruel decline to death within about 20 years.

But as NPR's Joanne Silberner reports, a new study out today suggests there may yet be reason to hope.

JOANNE SILBERNER: Earlier this year the Food and Drug Administration approved a drug that reduces the jerky movements of Huntington's disease. It was the first Huntington's drug to get an okay from the agency.

Now the Archives of Neurology has a report about a drug aimed at the serious cognitive deficits that people with Huntington's also suffer - the problems in memory and thinking.

Neurologist Karl Kieburtz of the University of Rochester headed the study.

Dr.�KARL KIEBURTZ (Neurologist, University of Rochester): This is a relatively short and small study looking to see about safety and tolerability, and secondly, about any signal or any sign of impact on cognition.

SILBERNER: Kieburtz and colleagues at medical centers in the U.S. and U.K. gave an experimental drug known as Dimebon to 46 people with Huntington's disease. Another 45 got a placebo. At the end of three months, the health of the two groups was about the same, but though the experiment was primarily designed to look at the drug's safety, the researchers also had the patients do a special test, a mental status test. The placebo group showed a slight drop; the treatment group showed a small improvement.

Dr.�KIEBURTZ: Which was a little surprising because it's usually thought as a tough scale to make better.

SILBERNER: Dimebon appears to beef up mitochondria, the little powerhouses inside cells that provide energy and that have recently been implicated in Huntington's disease.

Nancy Wexler, head of the Hereditary Disease Foundation, says the new study looks good.

Ms.�NANCY WEXLER (President, Hereditary Disease Foundation): On the safety factors, it really came out marvelously. You know, people who had taken the drug had a little bit more of a tendency to have either, you know, headache or sleepiness, but not really very much. And they had just as many or as few side effects as the people on placebo.

SILBERNER: Some of the same researchers are testing two other drugs on people, coenzyme Q and creatine, which have also been shown to beef up the mitochondria, but a lot more testing needs to be done to see if they're truly effective.

Huntington's has proved to be a terrifically difficult research problem. The gene for the disease was discovered in 1993, but exactly how the disease fouls the workings of the brain remains unknown. What is being learned about Huntington's disease, which is very rare, could help with other more common neurological conditions, says researcher Yvette Bordelon of the University of California at Los Angeles.

Dr.�YVETTE BORDELON (Researcher, University California at Los Angeles): There are many common features with other disorders like Alzheimer's disease, like Parkinson's disease and other neurologic disorders. So what we can learn in Huntington's will be applied to these other diseases and other disorders.

SILBERNER: The mitochondria that are the target of Dimebon are also implicated in Alzheimer's and Parkinson's. The advantage of studying Huntington's is that because it can be predicted pretty reliably by a genetic test and diagnosed early on in the disease, experimental treatments can be started before the disease really grabs hold, when they have a better chance of working. Meanwhile, a larger study of Dimebon involving 350 people and lasting six months has just gotten under way.

Joanne Silberner, NPR News.

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