Tags: huntington | drug | aso

Drug Reverses Huntington's Disease

Friday, 22 Jun 2012 01:04 PM


A single treatment with a promising new drug developed to treat Huntington’s disease completely reversed the symptoms of the neurological disorder in laboratory animal studies, new research has found.
Scientists with the University of California-San Diego School of Medicine found one-time injections of the new DNA-based drug treatment – known as ASO (short for antisense oligonucleotides) – blocked the activity of a gene linked Huntington's disease. As a result, the researchers were able to slow and partially reverse the progression of the fatal neurodegenerative disorder in mice and non-human primates.
"These findings open up the provocative possibility that transient treatment can lead to a prolonged benefit to patients," said Don W. Cleveland, who led the study published in the journal Neuron. "This finding raises the prospect [that] a single application of a drug …could 'reset the disease clock.' ”
He added that while the ASO therapy was tested in animals, it could produce sustained motor and neurological benefits in human adults with moderate and severe forms of the disorder. Currently, there is no effective treatment for the disease, which strikes about 30,000 Americans, whose symptoms include uncontrolled movements and progressive cognitive and psychiatric problems.
The disease is caused by the mutation of a single gene, which results in the production and accumulation of toxic proteins in the brain.
Cleveland noted that the findings of the new study are particularly promising because ASO therapy has already been proven safe in clinical trials and is the focus of drug development.


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A single treatment with a new Huntington's drug has promising results, in a laboratory study.
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2012-04-22
Friday, 22 Jun 2012 01:04 PM
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