Breakthrough genetic treatment slows the progression of Huntingdons Disease

WHY THIS MATTERS IN BRIEF

Huntingdons Disease is a debilitating condition that slowly kills a patients brain cells and there is no cure for it, now though a new treatment slows down its progression, and one day it might halt it altogether.

 

This week people suffering from Huntington’s Disease were offered new hope after a ground breaking trial showed that a revolutionary genetic treatment could slow down the illness, and one day, potentially stop it in its tracks.

 

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Today most of the medications that are available on the market only treat the symptoms of the disease rather than the underlying condition itself which gradually eats away at the patient’s brain, robbing them of movement, speech and, ultimately their dignity. But a small trial of 46 men and women showed that the new drug can silence the genetic mutation that causes Huntington’s in the first place.

Professor Sarah Tabrizi, Director of University College London’s Huntington’s Disease Centre who led the Phase 1 trial, said the results were “Beyond what I’d ever hoped,” adding that it might eventually be possible to stop the disease before irreversible damage to the brain had occurred.

“The results of this trial are of ground breaking importance for Huntington’s disease patients and families,” she said, “for the first time we have the potential, we have the hope, of a therapy that one day may slow or prevent Huntington’s disease.”

 

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Huntington’s is an incurable degenerative disease caused by a single defect in the Huntingtin gene which turns a usually helpful protein into a lethal brain cell killer, and it affects around 10,000 people in Britain.

The drug that the team developed, which is 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, and stopping Huntington’s in its tracks.

Although the trial was too small, and not long enough, to show whether patients’ clinical symptoms improved, it showed the drug was safe, well tolerated by patients and crucially that it reduced the levels of Huntingtin in the brain.

“This is probably the most significant moment in the history of Huntington’s since the gene,” added Tabrizi.

 

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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, and it’s thought that the treatment could potentially be adapted to target other incurable brain disorders such as Alzheimer’s and Parkinson’s, which recently saw it’s own breakthrough when researchers managed to turn a patients Parkinson’s disease on and off again using a genetic switch.

Since the trial the Swiss pharmaceutical giant Roche has paid $45 million for the rights to the drug, and they’re expected to launch a major trial to test its clinical effectiveness in the coming months. Meanwhile charities have welcome the results, saying, again, that it was of “ground breaking importance to families affected by Huntington’s.”

“This is a great day for the Huntington’s disease community,” said Chief Executive Cath Stanley, “this is a big step forward in Huntington’s disease research and although there is still some way to go before the overall results are known, this is a big step forward.”

 

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“I would say that the results are encouraging as it shows delivering therapeutic agents of this type is feasible and well tolerated and the treatment has done what it said it would,  namely drop the relevant protein levels in the CSF,” said Professor Roger Barker, Professor of Clinical Neuroscience at the University of Cambridge, who had six patients in the trial, “the question is whether this is enough to make a difference to patients and their clinical course and that we will have to wait for bigger trials.”

However some experts urged caution until the research was published.

“The details of this study have not been peer reviewed or published yet, so the scientific community simply does not know yet how robust the findings are,” said Professor Tara Spires-Jones of the UK Dementia Research Institute, “with that caveat in mind, the approach used in this trial has excellent potential to prevent Huntington’s disease, and if the next stage of larger trials live up to their promise, this will be a breakthrough.”

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