Huntington's disease drug clears initial hurdles - raising hopes

Huntington's disease drug clears initial hurdles - raising hopes

Huntington's disease drug clears initial hurdles - raising hopes

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.

A "GROUND-breaking" new drug has stopped Huntingdon's disease in its tracks for the first time, experts have revealed.

Current available treatments only help the symptoms, rather than slow down the progression.

Prof Sarah Tabrizi, executive of University College London's Huntington's Disease Center who drove the stage 1 trial, said the outcomes were "past what I'd ever trusted ..."

Prof Giovanna Mallucci, who discovered the first chemical to prevent the death of brain tissue in any neurodegenerative disease, said the trial was a "tremendous step forward" for patients and there was now "real room for optimism".

The results caused ripples across the scientific world as the drug, which is a synthetic strand of DNA, could potentially be adapted to target the incurable brain disorders such as Alzheimer's and Parkinson's.

Despite more needing to be done to test its effectiveness, the pharmaceutical industry has quickly taken an interest, with Swiss company Roche putting down a $45m fee to launch the larger trial.

The first symptoms, which typically appear in middle age, include mood swings, anger and depression. Later patients create uncontrolled jerky developments, dementia and at last loss of motion.

A genetic error disables the protein and makes it kill brain cells.

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The drug, called Ionis-HTTRx, works by intercepting a messenger molecule and destroying it before the harmful protein can be made, effectively silencing the effects of the mutant gene.

To convey the medication to the cerebrum, it must be infused into the liquid around the spine utilizing a four-inch needle.

UCL neuroscientist Professor John Hardy, who was not involved in the research, says the same approach might be possible in other neurodegenerative diseases such as dementia and Parkinson's. "The fact that it does work is really remarkable".

Sarah Tabrizi, the lead researcher and director of the Huntington's Disease Centre at UCL, said: "I've been seeing patients in clinic for almost 20 years, I've seen many of my patients over that time die".

The experimental drug, IONIS-HTTRx, was injected into patients' spinal fluid.

Injected directly into spinal fluid, the drug has been shown to actually lower the levels of the toxic huntingtins protein that devastates the nervous system. The gene in question codes for a protein known as huntingtin that builds up in the brain, causing the progressive degeneration of the nervous system and significantly harming movement, learning, thinking, and emotions.

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

"I have prayed every day for a breakthrough - living with this illness, you cannot put into words how devastating it is to suffer, how devastating it is for a family, especially children who know that this can be store in them".

An worldwide 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.

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