The results of the first trial are “a triumph” for Huntington’s patients and their families since existing medications only help to manage the symptoms of the disease, one of the researchers told DW.
Scientists at the University College London (UCL) on Monday announced that for the first time a drug has been used to successfully lower the level of a protein known to cause Huntington’s disease in the nervous system of patients.
“We were pleasantly surprised by the results,” Dr. Edward Wild, a senior investigator on the UCL drug trial, told DW.
The Ionis Pharmaceuticals drug IONIS-HTTRx managed to reduce the level of the mutant huntingtin protein during the trial and researchers were excited to discover that these reductions were “dose-dependent.”
“In other words, the bigger the dose of the drug that was given, the bigger the reduction in the huntingtin protein level,” Wild explained. “So that really was a home run for this trial.”
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Professor Sarah Tabrizi, director of the UCL Huntington’s Disease Centre, said in a statement that not only was it the first time a drug managed to lower the levels of the toxic protein, but that IONIS-HTTRx was “safe and well-tolerated.”
“The results of this trial are of ground-breaking importance for Huntington’s disease patients and families,” Tabrizi said.
Hope for those with Huntington’s
Currently, medications on the market for those with Huntington’s focus solely on managing the disease’s symptoms and there is no effective disease-modifying treatment for the condition.
“What’s special about this drug is that it’s targeting the known cause of the disease,” Wild told DW.
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The first human trial started in 2015 after more than a decade of pre-clinical development. A total of 46 patients with early Huntington’s disease were enrolled in the trial at nine study centers in the UK, Germany and Canada.
For more read the full of article at The Dw
