Huntington's disease drug clears initial hurdles - raising hopes

Representational Image

Results from the five-year study show that the Huntington's sheep model, which are symptom-free but carry the mutant form of the human Huntingtin gene, have the same increase in urea in the brain as the post-mortem brains of people who have died of Huntington's.

Huntington's is a disorder that causes irreversible damage to the brain, affecting patients' movements, behaviour and ability to think clearly.

The trial's lead investigators, Dr Sarah Tabrizi, professor of clinical neurology and director of UCL's Huntington Centre, said: "The results of this trial are of ground-breaking importance for Huntington's disease patients and families".

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. Using an ultra-sensitive assay, concentrations of the protein were measured in each patient's spinal fluid before and after treatment.

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 probably the most significant moment in the history of Huntington's since the gene [was isolated]".

UCL sicentist Ed Wild, who administered the drug in the trial, said he was quietly optimistic the drug could potentially be used to treat the amyloid proteins in Alzheimer's sufferers.

"The key now is to move quickly to a larger trial to test whether IONIS-HTT (Rx) slows disease progression", she said.

Each patient received four doses of either IONIS-HTTRx or placebo, given by injection into the spinal fluid to enable it to reach the brain.

About 8,500 people in the UK have Huntington's, while 30,000 people have it in the United States.

The drug, Ionis-HTTRx, works by intercepting the messenger molecule and destroying it before the harmful protein can be made, effectively silencing the effects of the mutant gene. "One day we want to prevent the disease". "You can target any protein".

"I don't want to overstate this too much, but if it works for one, why cant it work for a lot of them?" They still need vital long-term data to show whether lowering levels of Huntington will change the course of the disease.

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.

"Alzheimer's and Huntington's are at opposite ends of the dementia spectrum - so if this holds true for these types, then I believe it is highly likely it will hold true for all the major age-related dementias", Cooper said, in the paper published in the Proceedings of the National Academy of Sciences.

"The case for these is not as clear-cut as for Huntington's disease, they are more complex and less well understood".

About 10,000 people in the United Kingdom have the condition and about 25,000 are at risk.

Most of the patients get it inherited from their ancestors but surprisingly every 1 in 5 Huntington's patients is not inherited from his/her ancestors.

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