Researchers Make Breakthrough in Treatment of Devastating Neurodegenerative Disease
Part of an ongoing end-of-year look at major breakthroughs this past year in health and medical science
David Royse | LedeTree
Researchers made a significant breakthrough this year in the treatment of the neurodegenerative disease known as Huntington’s Disease, finding a drug that for the first time reduced the level of a protein that causes the disease.
The researchers, at University College London in England, and Britain’s National Hospital for Neurology and Neurosurgery, said a drug trial, sponsored by Ionis Pharmaceuticals, that was begun 2015, has shown success in suppressing the effects of the genetic mutation and is being called “enormously significant.”
Huntington’s, which is caused by an error in a particular gene, causes deterioration of motor skills and brain function, and while sufferers often live more than a decade with the disease, it usually ultimately ends in early death within about 20 years of onset of the symptoms.
About 30,000 people in the United States suffer from Huntington’s, while more than 200,000 Americans are at risk of having inherited it.
University College London said the new drug, IONIS-HTTRx, produced a significant lowering of the level of the mutant gene, and now will be used in wider trials. The drug trial used just 46 men and women from Canada, the UK and Germany, with early stage Huntington’s Disease, UCL said.
“The results of this trial are of ground-breaking importance for Huntington’s disease patients and families” said Sarah Tabrizi, the director of UCL’s Huntington’s Disease Center and leader of the study. “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.”
John Hardy, an Alzheimer’s and Parkinson’s researcher who wasn’t involved in the research, said on the UCL website that the approach might be viable in treatment of other neurodegenerative diseases characterized by the build-up of toxic proteins in the brain.
“I really think this is, potentially, the biggest breakthrough in neurodegenerative disease in the past 50 years,” Hardy said.
Ionis made a $45 million licensing agreement for the drug with Roche, which will take over development and commercialization of the therapy.
The test took place at the Leonard Wolfson Experimental Neurology Centre at UCL.