NEW RESEARCH THAT may prevent the development of Huntington’s disease is being described as potentially the biggest breakthrough in neurodegenerative diseases for 50 years.
A new trial at University College London has been shown to successfully lower the level of the harmful huntingtin protein in the nervous system.
The trial involved enrolling 46 patients with early Huntington’s disease at nine study centres in the UK, Germany and Canada.
This trial began in late 2015 and each patient received four doses of either the drug or a placebo. The drug is administered by injection into the spinal fluid to enable it to reach the brain.
After the trial was monitored since it began, today researchers have said that the drug has shown to be both effective and safe for patients.
“The results of this trial are of ground-breaking importance for Huntington’s disease patients and families,” said Professor Sarah Tabrizi, Director of the UCL Huntington’s Disease Centre
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. The key now is to move quickly to a larger trial to test whether the drug slows disease progression.
Huntington’s is a devastating disease that can cause a particularly difficult death.
It has been described as being like Alzheimer’s, Parkinson’s and schizophrenia rolled into one and can be inherited.
It has also been associated for centuries with stigmas that have led to social exclusion and hampered efforts to contain the disease.
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