Trial shows Huntington's drug could slow progress of disease

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Professor Tabrizi, Director of the UCL Huntington's Disease Centre and IONIS-HTTRx Global Chief Investigator, said: "The results of this trial are of ground-breaking importance for Huntington's disease patients and families".

For the first time ever, an experimental drug has showed promise in lowering levels of brain toxins caused by Huntington's disease. It involved 46 patients with early Huntington's disease, who were treated and monitored at nine centres in the UK, Germany and Canada.

This work was supported by Brain Research New Zealand, Cure Huntington's disease initiative, the Neurological Foundation and the Freemasons of New Zealand.

The unstoppable death of brain cells in Huntington's leaves patients in permanent decline, affecting their movement, behaviour, memory and ability to think clearly.

The onset of the disease usually begins between the ages of 30 and 50 and continually worsens over a period of 10 to 25 years.

To deliver the drug to the brain, it has to be injected into the fluid around the spine once a month using a four-inch needle.

Ionis Pharmaceuticals, the company behind the drug, said in a statement on Monday that, following the trial, pharmaceutical giant Roche had exercised its right to license IONIS-HTT (Rx) at a cost of $45m.

"This study on Huntington's Disease is the final piece of the jigsaw which leads us to conclude that high brain urea plays a pivotal role in dementia", Professor Cooper said.

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There is now no treatment to stop or reverse the disease, and existing medication can only help manage its symptoms.

The defect that causes the neurodegenerative disease Huntington's has been corrected in patients for the first time, the BBC has learned. She doesn't know if she received the drug or placebo injections, but she is going to receive the actual medication starting February. "This is probably the most significant moment in the history of Huntington's since the gene [was isolated]".

The UCL trial is too small-scale to show whether the drop in toxic protein levels has alleviated the participants symptoms, but Swiss pharmaceuticals firm Roche has bought a licence to develop the drug further, The Guardian reports. Or, you can leave the gene alone, and alter the way the body codes for the misshapen protein.

The drug, called Ionis-HTTRx, works by intercepting a messenger molecule and destroying it before the harmful protein can be made, effectively silencing the effects of the mutant gene.

"Clearly, there will be much interest into whether can be applied to the treatment of other neurodegenerative diseases, like Alzheimer's", she added.

"For the first time we have the potential, we have the hope, of a therapy that one day may slow or prevent Huntington's disease".

The trial was to assess the safety as well as a the effectiveness of the new drug.

"Alzheimer's and Huntington's are at opposite ends of the dementia spectrum - so if this holds true for these types, then I believe it is highly likely it will hold true for all the major age-related dementias", Cooper said, in the paper published in the Proceedings of the National Academy of Sciences.