A new drug has been shown to completely suppress the effects of Huntington’s genetic mutation in a groundbreaking trial.
It is the first time that an experimental drug to slow the progression of the devastating genetic illness in a trial. Results have been acclaimed as “enormously significant” in the fight against Huntington, an illness causing irreversible damage to the brain.
Prof 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 … The results of this trial are of ground-breaking importance for Huntington’s disease patients and families,” she said.
So far current treatments are only able to help with the symptoms of the disease but are useless to slow its progression. The new drug, a synthetic strand of DNA, could potentially be adapted to cure other brain disorders such Alzheimer’s and Parkinson’s creating a wave of excitement in the medical world.
Roche, the Swiss pharmaceutical giant, has already paid $45m licence fee to take the drug forward to clinical use.
“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,” said Tabrizi. “This is probably the most significant moment in the history of Huntington’s since the gene [was isolated].”
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