Sarah Tabrizi, the lead researcher and director of the Huntington's Disease Centre at UCL, said: "I've been seeing patients in clinic for almost 20 years, I've seen many of my patients over that time die".
Injected directly into spinal fluid, the drug has been shown to actually lower the levels of the toxic huntingtins protein that devastates the nervous system.
A landmark trial for Huntington's disease has announced positive results, suggesting that an experimental drug could become the first to slow the progression of the devastating genetic illness. Therefore, the main aim of the UCL study was determining whether the experimental drug - given to the majority of the 46 men and women from the UK, Germany and Canada with early stages Huntington's who took part - is safe.
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.
Huntington's symptoms typically manifest in middle age and include involuntary movements, changes in personality, mood swings and dementia-like cognitive problems. But the drug manufacturer Roche is seeking to bring the drug to market and has paid $45 million for development rights. We need a larger study to see whether genetically modifying production of Huntington protein translates into slowing down of the degenerative disease process.
Now treatments only exist for the symptoms of Huntington's, which usually begin to appear between the ages of 30 and 50, leading to their gradual decline over the next 10 to 25 years.
Prof Sarah Tabrizi, the lead researcher and director of the Huntington's Disease Centre at UCL, told the BBC: "I've been seeing patients in clinic for almost 20 years, I've seen many of my patients over that time die".
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There is now no effective disease-modifying treatment for the condition, with existing medicines focused only on managing disease symptoms. There is a problem at the genetic level especially at the huntingtin gene that codes for the huntingtin protein. The authors report that these injections-the first of their kind tested in humans-lowered the amount of damaging huntingtin protein in participants' brains.
"This is probably the most significant moment in the history of Huntington's since the gene", added Prof Tabrizi.
BBC quoted Sarah Tabrizi, lead researcher, as saying, "I've been seeing patients in clinic for almost 20 years, I've seen many of my patients over that time die..."
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.
The trial was to assess the safety as well as a the effectiveness of the new drug.
The therapy was developed by Ionis Pharmaceuticals, which said the drug had "substantially exceeded" expectations, and the licence has now been sold to Roche. "You can target [any protein]". "I can say to my children we've gone this long hard road, with no hope, but now for the first time ever there is hope", she told RTÉ's Morning Ireland.