'Groundbreaking' New Drug Gives Hope in Huntington's Disease

Huntington’s ‘game-changer’ gives sufferers hope			
   by Anna Thomson 

Huntington’s ‘game-changer’ gives sufferers hope by Anna Thomson Published

"The results of this trial are of groundbreaking importance for Huntington's disease patients and families".

"For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated", said Tabrizi.

The Ionis Pharmaceuticals drug IONIS-HTTRx managed to reduce the level of the mutant huntingtin protein during the trial and researchers were excited to discover that these reductions were "dose-dependent".

The hope is that this drug could be the key to slowing and potentially even stopping the debilitating disease in its tracks.

The Huntington's Disease Society for America describes the condition as "a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain".

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

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.

The drug known as IONIS-HTTRx is injected directly into the spinal fluid of the patient, this allows the drug to be sent straight to the brain.

The unstoppable progression of the inherited disease Huntington's could be slowed or even halted by an experimental new treatment which has proved "enormously successful" in early patient trials.

Current medication can only help alleviate the severity of symptoms and does not affect the progression of the disease.

Scientists believe they have identified one of the very earliest changes in the brain that results in Huntington's Disease and that the discovery holds a vital clue to developing the first-ever treatment for the disease. There is a problem at the genetic level especially at the huntingtin gene that codes for the huntingtin protein.

The results, hailed as the "biggest breakthrough in 50 years", saw 46 patients of the incurable disease being given an experimental drug into their spinal fluid at the National Hospital for Neurology and Neurosurgery. The authors report that these injections-the first of their kind tested in humans-lowered the amount of damaging huntingtin protein in participants' brains. "This is presumably the most noteworthy crossroads in the historical backdrop of Huntington's since the quality [was isolated]".

The trial was small and short and therefore could not aptly show whether patients' clinical symptoms improved, but Roche is now expected to launch a major trial aimed at testing this.

For example, a comparable engineered strand of DNA could be made to focus on the detachment that produces distorted amyloid or tau proteins in Alzheimer's.

Thanks to the success of the drug, the next stage will be a much larger trial.

But she said the transferability to Alzheimer's treatment is not so "clear-cut".

"This is a great day for the Huntington's disease community", said Huntington's Disease Association chief executive Cath Stanley. "Our findings very clearly point to brain urea and/or ammonia as a therapeutic target for Huntington's disease", Dr Handley says.

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