First Huntington’s disease treatment is possible
Jeff Lagerquist, CTVNews.ca Staff For the first time ever, an experimental drug has showed promise in lowering levels of brain toxins caused by Huntington’s disease.
It’s an early-stage breakthrough that could guide researchers towards an effective treatment for the fatal inherited brain disorder.
Huntington’s disease affects an estimated 21,000 people in Canada. It has been described as Parkinson’s and Alzheimer’s rolled into one — parts of the patient’s brain die as it progresses, leading to incapacitation and eventually death.
Huntington’s patient Michelle Dardengo, of Vancouver, B.C., watched six members of her family die of the disorder. She was diagnosed three years ago.
“It worries me that I am going to change, and people around me are going to be put through what I went through with my father. I don’t want to do that to them,” she told CTV News.
The condition is caused by a mutation in the huntingtin gene, which incorrectly encodes a protein vital to nerve-cell communication.
Symptoms like depression and uncontrollable movements typically begin between the ages of 30 and 50. Most people diagnosed with the currently untreatable disorder require professional nursing before the fatal effects take hold. Patients have a 50 per cent chance of passing the disease on to each of their children.
Dardengo was the first patient in this study. She doesn’t know if she received the drug or placebo injections, but she is going to receive the actual medication starting February. Even though the research is in its infancy, she said she’s optimistic.
“I really hoped that something would come up and it has,” she said of the drug.
“My family and my children, they are very excited, and very supportive.”
Prof. Sarah Tabrizi of the University College London Institute of Neurology led a 46-patient trialinvolving Huntington’s patients at nine study centres in Canada, the U.K., and Germany. They were given four spinal injection doses of an experimental drug called IONIS-HTTRx, or four placebo shots.
“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 said in a release on Monday. “The results of this trial are of groundbreaking importance for Huntington’s disease patients and families.”
The drug, which is made of artificial DNA, is designed to silence the problem gene. It works by interfering with its ability to cause cells to produce the toxic proteins that damage the brain.
The researchers report significantly lower levels of brain toxins in those given the highest doses of the drug, but there is little proof at this time that it slows the overall progress of the disease.
“We wish we could get these therapies to patients faster, but we have to be careful and ensure they are safe,” said Dr. Blair Leavitt, a neurologist with the University of British Columbia.
Tabrizi began this first-ever human trial of a huntingtin-lowering drug in late 2015, following over a decade of pre-clinical development.
The full results of the IONIS-HTTRx program have yet to be released in detail or published in a peer-reviewed journal.
Swiss healthcare giant Roche did, however, find the results promising enough to exercise its option to license IONIS-HTTRx from California-based Ionis Pharmaceuticals, who sponsored the trial. Roche will assume responsibility for further development of the drug. More studies are planned next year.
Researchers hope the findings could one day translate to treatments for other degenerative diseases, such as Alzheimer’s and Parkinson’s.
“The key now is to move quickly to a larger trial to test whether the drug slows disease progression,” Tabrizi said.
By Jeff Lagerquist, CTVNews.ca Staff
Photo and video – from the original article website.