Those living with Huntington’s Disease have reason to celebrate today after scientists suppressed the defect that causes the neurodegenerative disease for the first time.
Researchers from University College London injected an experimental drug into the spinal fluid and found it safely lowered levels of toxic proteins linked to Huntington’s in the brain, the BBC reports.
The team says it’s the biggest breakthrough in treatment for the devastating disease in 50 years.
Huntington’s Disease is a inherited condition in which nerve cells in the brain break down over time.
It is sometimes described as a mix of Parkinson’s and Alzheimer’s and motor neurone disease rolled into one and leaves victims with physical, cognitive and psychiatric symptoms.
The disease usually manifests during middle age. Some people die from the disease within decades.
Prof Sarah Tabrizi, director of University College London’s Huntington’s Disease Centre who led the phase 1 trial, told The Guardian 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.
The trial involved 46 men and women from the UK, Germany and Canada. They were given four spinal injections one month apart, with the dose increased each time.
Researchers found the drug decreased the concentration of the harmful protein in the spinal fluid in proportion with the strength of the dose, indicating the drug was effective.
“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,” Tabrizi told The Guardian.
“This is probably the most significant moment in the history of Huntington’s since the gene [was isolated].”