New Drug Offers Hope For Treating Huntington's Disease
12 December, 2017, 16:06 | Author: Joy Garcia
Some people die from the disease within decades.
Huntington's is a disorder that causes irreversible damage to the brain, affecting patients' movements, behaviour and ability to think clearly.
But the first in-human trial showed the drug was safe, well tolerated by patients and crucially reduced the levels of huntingtin in the brain. Therefore, the main aim of the UCLstudy was determining whether the experimental drug - given to the majority of the 46 men and women from the UK, Germany and Canada with early stages Huntington's who took part - is safe. The improvements increased with each larger dose, indicating its benefits in treating the disease.
Lead researcher Sarah Tabrizi, professor of clinical neurology at University College London, said the ability of the drug to tackle the underlying cause of Huntington's by lowering levels of a toxic protein was "ground-breaking". "This is probably the most significant moment in the history of Huntington's since the gene [was isolated]".
The outcomes have additionally caused swells of energy over the logical world in light of the fact that the medication, which is an engineered strand of DNA, could conceivably be adjusted to target other hopeless mind issue, for example, Alzheimer's and Parkinson's.
"The question is whether this is enough to make a difference to patients and their clinical course, and for that we will have to wait for bigger trials", said Roger Barker of the University of Cambridge, which was also involved in the research.
To deliver the drug to the brain, it has to be injected into the fluid around the spine once a month using a four-inch needle.
The UCL trial is too small-scale to show whether the drop in toxic protein levels has alleviated the participants symptoms, but Swiss pharmaceuticals firm Roche has bought a licence to develop the drug further, The Guardian reports.
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.
It is a synthetic single strand of DNA customised in order to latch the Huntington messenger molecule.
"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".
But she said the transferability to Alzheimer's treatment is not so "clear-cut". "I would prefer not to exaggerate this excessively, yet in the event that it works for one, for what reason wouldn't it be able to work for a considerable measure of them?"
Researchers working in other fields of degenerative disease are giddy with excitement about what the study means for future therapies with lab-made genetic material.
Finding a cure for any disease is a complex process.
The progressive neurological disorder, which now affects an estimated 10,000 people in the United Kingdom, is incurable - but an experimental drug, which has been shown to lower levels of the harmful protein responsible, could be a "game-changer".
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