New Drug Offers Hope For Treating Huntington's Disease

Huntington's breakthrough may stop disease

But a small trial of 46 men and women showed that the new drug silences the genetic mutation which causes Huntington's.

'The key now is to move quickly to a larger trial to test whether it slows disease progression'.

The trial began in the last months of 2015 and was led by Professor Sarah Tabrizi UCL Institute of Neurology, Director of the UCL Huntington's Disease Centre and IONIS-HTTRx Global Chief Investigator. The Swiss pharmaceutical giant Roche has paid a $45m licence 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.

Wellington film editor Bridget Lyon was just 21 when she found out her mother had the gene for Huntington's Disease and that it had been passed down to her and her siblings.

At every step patient safety and well being was assessed along with the effectiveness of the drug.

The mutant Huntington's gene has instructions for cells to create the toxic protein called huntingtin. There is a problem at the genetic level especially at the huntingtin gene that codes for the huntingtin protein.

Researchers in London have made a discovery that's being described as the biggest breakthrough in neuro-de-generative diseases in 50 years. "The fact that it does work is really remarkable".

Prof. Sarah Tabrizi of the University College London Institute of Neurology led a 46-patient trial involving Huntington's patients at nine study centres in Canada, the United Kingdom, and Germany.

They were each given four spinal injections of either the drug or a placebo. 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 presumably the most noteworthy crossroads in the historical backdrop of Huntington's since the quality [was isolated]".

Patients usually die within 20 years after the onset of symptoms. This could be because the use of energy is compromised in the brains of people with Huntington's and urea is produced as the damaged brain tries to find alternative energy sources. "They may simply require a heartbeat each three to four months", she said.

The drug, which was developed by the California biotech firm Ionis Pharmaceuticals, is a synthetic strand of DNA, customized to latch on to the huntingtin messenger molecule.

The unexpected success raises the tantalising possibility that a similar approach might work for other degenerative brain disorders. "The medication resembles Lego", said Wild. Huntingtin protein levels are markers of this disease. "I am very, very excited", he told the Guardian.

It's the first time that the defect that causes the fatal neurodegenerative disease has been corrected, and the results provide hope for a treatment, according to the University College London (UCL) scientists, BBC News reported Monday.

"Obviously, there will be much enthusiasm into whether it can be connected to the treatment of other neurodegenerative diseases, as Alzheimer's", she included.

Finding a cure for any disease is a complex process.

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