Scientists identify potential treatment for Huntington’s disease

November 14th, 2007 - 1:54 am ICT by admin  
The researchers say that a small molecule called C2-8 has shown potential to delay the loss of motor control, and to reduce neurological damage in a mouse model of the disorder.

“We found that C2-8 slows the progress of HD in a mouse model and might do the same thing in human patients, if it or its biochemical relatives can be translated into a drug,” says Dr. Steven Hersch of MIND and the Massachusetts General Hospital (MGH) Department of Neurology, who led the study to be published in the Proceedings of the National Academy of Sciences.

“What we don’t know yet is precisely how it works, what molecules it interacts with in cells and how potent it might be,” he added.

MIND researcher Aleksey Kazantsev was the first to identify C2-8 as a candidate treatment for HD, based on its ability to block the aggregation of the mutant huntingtin protein in yeast and animal tissue, and to improve function in a fruit fly model.

In the current study, the researchers found that oral doses of C2-8 could cross the blood-brain barrier, and were non-toxic in a mouse model of HD. They also found that C2-8 did not interact with a number of molecules predictive of negative side effects.

The mice were treated with C2-8 starting at the age of 24 days scored significantly better on tests of strength, endurance and coordination than did the subjects that did not receive the molecule.

Although the treatment significantly delayed progressive motor disability, the animals who received C2-8 did not live longer.

Upon examination of the animals’ brain cells from the striatum, the area of the brain where the deterioration of HD occurs, the researchers found that treated mice had less shrinkage of brain cells and smaller aggregates of huntingtin protein than did untreated HD mice.

“We’ve both validated that compounds reducing the aggregation of mutant huntingtin are potential HD drugs - so that strategy is one that other scientists should pursue - and shown that C2-8 has potential as the basis of a neuroprotective treatment,” says Hersch.

“We now need to confirm those results in a different mouse model, see whether similar compounds may be more potent than C2-8 and search for the enzyme or receptor it is binding to,” he added. (ANI)

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