Drug lowers harmful Huntington's disease proteins in humans, according to research

Lead researcher Professor Sarah Tabrizi, head of University College London's Huntington's Disease Centre, said: "The results of this trial are of ground-breaking importance for Huntington's disease patients and families".

The results showed the drug lowered the level of the toxic disease-causing protein in the nervous system and did so safely.

According to The Guardian, the phase one trial included 46 men and women with early onset Huntington's disease, who were given four spinal injections, one each month, with an increasing dose each time.

Ionis senior vice president of research Frank Bennett said the protein reductions observed in the study "substantially exceeded our expectations" and that the drug was also well tolerated.

Alzheimer's and Parkinson's are both caused by buildups of harmful proteins in the brain; if a similar technique could be used to stop the production of these proteins, it could slow or cure these diseases (which are far more common than Huntington's).

The Swiss pharmaceutical giant Roche paid a $45 million license fee to take the drug forward to clinical use.

The researcher noted, however, that a much larger, longer trial is needed to show that IONIS-HTTRx can slow the progression of Huntington's disease.

Now treatments only exist for the symptoms of Huntington's, which usually begin to appear between the ages of 30 and 50, leading to their gradual decline over the next 10 to 25 years.

Huntington's Disease is a inherited condition in which nerve cells in the brain break down over time.

The error is found in the huntingtin gene that tells the body to produce the huntingtin protein.

"What we have seen is a really strong, really encouraging piece of chemical evidence that the drug is doing what it's supposed to do, which is lowering the level of the toxic protein in the spinal fluid where it was injected".

Roughly a quarter of participants formed the control group and were given a placebo injection. "This is presumably the most noteworthy crossroads in the historical backdrop of Huntington's since the quality [was isolated]".

The new drug, developed by Ionis Pharmaceuticals, contains an "antisense" molecule consisting of a single strand of chemically-modified DNA. "One day we want to prevent the disease".

They created a snippet of synthetic DNA that messes with the messenger RNA responsible for translating DNA protein codes, effectively blocking the problematic huntingtin protein from every being made.

There is no treatment, but Russell Snell of the Centre for Brain Research said they had identified raised levels of urea in the brain of a transgenic sheep model that matched human brains affected by Huntington's.

A comparison of protein levels before and after the treatment showed "significant" drops after IONIS-HTTRx was administered, UCL said in a press release, suggesting the drug had successfully intercepted the rogue gene.

"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.


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