12 December, 2017
IONIS-HTTRx is the first pharmaceutical agent created to target the root cause.
A trial led by researchers from University College London (UCL) has demonstrated for the first time that a treatment is capable of suppressing the genetic defect which causes toxic proteins to build up in the brain.
"The key now is to move quickly to a larger trial to test whether [it] ... slows disease progression", she said in a statement.
"The results of this trial are of groundbreaking importance for Huntington's disease patients and families", the chief investigator of the research, Professor Sarah Tabrizi, said. The Swiss pharmaceutical goliath Roche has paid a $45m permit expense to take the medication forward to clinical utilize.
They found that IONIS-HTTRx could successfully lower the levels of the mutant huntingtin.
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. Later patients develop uncontrolled jerky movements, dementia and ultimately paralysis.
Results from the five-year study show that the Huntington's sheep model, which are symptom-free but carry the mutant form of the human Huntingtin gene, have the same increase in urea in the brain as the post-mortem brains of people who have died of Huntington's.
Those blueprints have to be carried to a cell's protein-making factories and that job is done by a short strand of genetic code, called messenger RNA.
Ionis said Roche would now be responsible for all IONIS-HTT (Rx) development, regulatory and commercialization activities and costs.
To deliver the drug to the brain, it has to be injected into the fluid around the spine using a four-inch needle.
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John Hardy, who was awarded the Breakthrough Prize for his work on Alzheimer's, described it as "potentially, the biggest breakthrough in neurodegenerative disease in the past 50 years". "The fact that it does work is really remarkable".
The trial involved 46 men and women from the UK, Germany and Canada. This kind of closely matched relationship normally indicates a drug is having a powerful effect.
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.
"This is probably the most significant moment in the history of Huntington's since the gene", added Prof Tabrizi.
The surprising achievement raises the tempting probability that a comparative approach may work for other degenerative cerebrum issue. "The drug's like Lego", said Wild. "You can target any protein".
An global research team led by Professor Russell Snell and Dr Renee Handley from the University of Auckland's School of Biological Sciences, Centre for Brain Research, have identified elevated levels of the chemical urea in the brain of a transgenic sheep model which matches those of human brains affected by Huntington's.
"My family and my children, they are very excited, and very supportive".
But she said the transferability to Alzheimer's treatment is not so "clear-cut".
Finding a cure for any disease is a complex process.