Published: Wed, December 13, 2017
Culture | By Henry Herrera

New drug could offer first treatment for irreversible Huntington's disease

New drug could offer first treatment for irreversible Huntington's disease

The condition is caused by a mutation in the huntingtin gene, which incorrectly encodes a protein vital to nerve-cell communication.

Scientists have for the first time corrected a protein defect that causes Huntington's disease, by injecting a drug into the spine, offering new hope for patients with the devastating genetic disease.

The Phase 1/2a evaluated the first therapy in clinical development meant to target the underlying cause of Huntington's Disease (HD).

Researchers say the synthetic strand could be customised to target other proteins in the treatment of other forms of dementia like Alzheimer's. The patients were given four spinal injections one month apart and the drug dose was increased at each session; roughly a quarter of participants had a placebo injection.

Still, the reduction in the protein is notable, according to Huntington's specialists and patient advocates, especially since existing Huntington's therapies only tackle its symptoms rather than its causes.

The outcomes have been hailed as "tremendously huge" in light of the fact that it is the first run through any medication has been appeared to smother the impacts of the Huntington's change that makes irreversible harm the cerebrum.

Among people receiving Ionis-HTTRx, the team saw a significant decline in levels of mutant huntingtin, which causes symptoms of the disease, with higher doses having greater impact.

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Peter Allen, 51, is in the early stages of Huntington's and took part in the trial: "You end up in nearly a vegetative state, it's a disgusting end".

Prof John Hardy, a neuroscientist at UCL who was not involved in the trial, said: "If I'd have been asked five years ago if this could work, I would have absolutely said no. It's not a total cure but it's the next best thing at the moment", she said.

A breakthrough procedure that could halt the progress of Huntington's disease is being heralded as potentially the biggest development in neurodegenerative medicine in half a century. "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".

Huntington's disease progressively robs you of your mental and physical faculties. "You can target [any protein]". This builds upon earlier research into Alzheimer's disease and urea published previous year. "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?" At every step patient safety and well being was assessed along with the effectiveness of the drug.

"Obviously, there will be much enthusiasm into whether it can be connected to the treatment of other neurodegenerative diseases, as Alzheimer's", she included. We need a larger study to see whether genetically modifying production of Huntington protein translates into slowing down of the degenerative disease process. For instance, Alnylam Pharmaceuticals' own patisiran has driven the company's stock up more than 250% this year thanks to strong, later-stage clinical trial results.

"This is of groundbreaking importance for patients and families".

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