Experimental Treatment May Reverse Symptoms of Huntington's Disease

7:39 PM, Jun 21, 2012   |    comments
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WASHINGTON, D.C. (WUSA) -- So far, Huntington's disease can't be cured - the symptoms can only be lessened with medication. But with the help of an experimental treatment, scientists hope they can reverse the symptoms of the progressive degenerative condition.  

Huntington's disease is hereditary, and it is caused by a defect in a single gene. Over time, it breaks down the nerve cells in the brain. According to the Mayo Clinic, this degeneration typically affects one's functional abilities and can result in movement, cognitive and psychiatric disorders.

The experimental treatment aims to reverse these symptoms. It could result in movement and neurological benefits in people who have moderate to severe forms of Hungtingon's disease. The study, published in the June 21 issue of the journal "Neuron," found that these improvements can last months after the initial treatment.

The treatment uses "antisense oligonucleotides," or ASOs. These ASOs first find the mutant gene and bind to it. Once attached, they destroy the molecular instructions that tell the gene to make the toxic huntingtin protein (which breaks down nerve cells).

The study involved testing the treatment on mice and non-human primates, both of which had positive results. Just one injection of the treatment caused animals to move better within one month and begin functioning normally within two months.

These improvements lasted for nine months, even after the treatment had left the animals' systems and production of the huntingtin protein had begun again. Researchers are hoping this can occur in humans, as well. 

"For diseases like Huntington's, where a mutant protein product is tolerated for decades prior to disease onset, these findings open up the provocative possibility that transient treatment can lead to a prolonged benefit to patients," study author Dr. Don W. Cleveland, chair of the UC San Diego department of cellular and molecular medicine, told CBS News in a statement. "This finding raises the prospect of a 'huntington holiday,' which may allow for clearance of disease-causing species that might take weeks or months to re-form." If so, Cleveland said, just one drug treatment could "reset the disease clock" and provide long-term benefits.

According to CBS News, Cleveland told UT San Diego that his team anticipates testing the treatment on humans in 18 months.

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