Published: Tue, December 12, 2017
Culture&Arts | By Darrell Mcdonald

New drug may combat deadly Huntington's disease

New drug may combat deadly Huntington's disease

An global team of researchers, including Professor Garth Cooper from the University of Manchester, used both human brains, donated by families for medical research, and those of genetically modified sheep to investigate whether Huntington's disease is directly linked to brain urea levels.

Lead researcher Sarah Tabrizi, professor of clinical neurology at University College London, says the ability of the drug to tackle the underlying cause of Huntington's by lowering levels of a toxic protein was "ground-breaking", adding that the "key now is to move quickly to a larger trial to test whether IONIS-HTT (Rx) slows disease progression". "The results of this trial are of groundbreaking importance for Huntington's disease patients and families".

It's hoped this form of drug could be adapted to target proteins in other now incurable brain disorders, such as Alzheimer's disease. The Swiss pharmaceutical giant Roche has paid a $45m licence fee to take the drug forward to clinical use.

Huntington's is a hopeless degenerative disease caused by a solitary quality deformity that is gone down through families.

Huntington's symptoms typically manifest in middle age and include involuntary movements, changes in personality, mood swings and dementia-like cognitive problems.

They say this is the first time that this protein, which is known to trigger Huntington's disease, has been lowered in patients with the disease.

"A large portion of our patients realize what's in their future", said Ed Wild, a UCL researcher and specialist neurologist at the National Hospital for Neurology and Neurosurgery in London, who controlled the medication in the trial.

The protein associated with the disease is called huntingtin protein and this drug successfully lowered the levels of this protein and reduced the harm caused to the nervous system.

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Known as IONIS-HTTRx, the drug does not target the gene itself, but is instead a piece of synthesized genetic code that binds to the piece of messenger RNA that transports the information needed to build huntingtin around the cell.

The technique has already led to a drug for spinal muscular atrophy that was approved past year.

"I am a neuroscientist".

A tragic neurodegenerative disease, Huntington's is caused in most people by a single genetic mutation, although a small number of others do develop the genetic fault through random mutation. For those receiving the drug, the dose was increased over time, and safety was monitored throughout, according to a statement.

In trials, the experimental drug was shown to lower levels of the harmful protein responsible for the devastating inherited illness. This is coupled with the fact that the drug had no adverse effects and was seemingly safe. "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.

If the future trial is also successful, Tabrizi believes the drug could be used in people who have the gene, before they become ill, possibly stopping the symptoms before they even begin. "It's not a total cure but it's the next best thing at the moment", she said. In other neurodegenerative diseases, "there is at least one protein you would like to partially reduce", he 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.

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