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'Very promising' treatment for Huntington disease discovered


February 14, 2012 - Washington

Scientists have discovered a promising new therapy for Huntington disease that restores lost motor skills and may delay or stop the progression of the disease based on lab model tests.

Because the new therapy uses a molecule already being used in clinical trials for other diseases, it could be used in a clinical trial for Huntington disease within the next one to two years.

"We didn't expect to see such dramatic changes after administering this therapy," said principal Investigator, Simonetta Sipione, at the University of Alberta.

"We expected to see improvement, but not complete restoration of motor skills. When we saw this, we were jumping with excitement in the lab. This is very promising and should give hope to those with Huntington disease. I think it's a treatment that deserves to go to clinical trials because it could have huge potential," Sipione stated.

Those with this inherited brain disorder, where a mutant protein triggers brain cell death causing loss of motor and cognitive skills and eventually death, have slightly lower levels of a brain molecule known as GM1.

When U of A medical researchers restored GM1 to normal levels in lab models with the disease, motor skills in the lab models returned to normal within days, said Sipione, a researcher in the Department of Pharmacology and the Centre for Neuroscience, both within the Faculty of Medicine and Dentistry.

The molecule used in the lab tests at the U of A was produced both naturally and synthetically through chemical production.

This same molecule has been used in clinical trials for the treatment of Parkinson's and other neurodegenerative diseases, so using this molecule to treat patients with Huntington disease in a small first stage clinical trial could happen relatively quickly.

Details are still being worked out about where the trial would take place, but researchers are hoping it will be at the U of A and are in discussions with a University of Alberta Hospital neurologist.

The finding was published in the peer-reviewed journal Proceedings of the National Academy of Sciences today.

ANI

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