Human clinical trials of a drug that claims to treat Huntington’s disease is all set to begin. The first-of-its-kind medicine has already proven safe and successful on animal models.
The new medicine, IONIS-HTTRx, is an antisense drug that makes the protein responsible for causing the disease inactive. In other words, this drug works as a “gene silencer” that prohibits the Huntington protein that causes the disease in patients from occurring.
"It is very exciting to have the possibility of a treatment that could alter the course of this devastating disease," says study principal author Dr. Blair Leavitt from the University of British Columbia in Vancouver, Canada, reports Tech Times
The first tests were done on mice and monkeys. In both the animal models, IONIS-HTTRx did not lead to side effects and the subjects were tolerant of the drugs. The study on the animal models will be presented at in 68th Annual Meeting of American Academy of Neurology in Canada.
With the success experienced in the animal trials, they will now be applied on humans too. Since this drug is unable to pass through the blood-brain blockade, it must be administered in the spinal canal of human participants. For now, it will be dispensed in four doses, in monthly intervals. However, for the safety purposes, the researchers will analyze different doses.
The researchers will also study the impact of the drug on specific disease indicators. Keeping all the processes in mind, it will take a few more years before the drug can be put to clinical practice, says Leavitt.
Through their experiments, scientists are happy to see that it is not only possible to stop the disease but can also reverse the damage done.
"It is very exciting to have the possibility of a treatment that could alter the course of this devastating disease," said Leavitt. "Right now we only have treatments that work on the symptoms of the disease."
