A drug that would be the first to target the cause of Huntington's disease (HD) -- a rare, hereditary disease -- has been found effective and safe when tested in mice and monkeys.
The new drug, called IONIS-HTTRx, is an anti-sense drug that acts as a "gene silencer" to inhibit the production of Huntingtin (HTT) protein in people with Huntington's disease that can cause uncontrolled movements, loss of intellectual abilities, emotional problems and eventually death.
"It is very exciting to have the possibility of a treatment that could alter the course of this devastating disease," said principal investigator Blair Leavitt from the University of British Columbia in Vancouver, Canada.
The mutation results in the production of a disease-causing HTT protein. Each child has a 50/50 chance of inheriting the gene mutation. Everyone who inherits the mutated gene would eventually develop the disease.
In the study, IONIS-HTTRx was found to be well-tolerated without any dose-limiting side effects, in the experiments conducted on rodents and monkeys. The drug is now in the initial phase of its clinical trial.