A new drug has been found to be safe and effective in combating Huntington's disease when tested in mice and monkeys.

Results of a new study of the drug, which would be the first to target the cause of the brain disorder, are slated for release at the American Academy of Neurology's 68th Annual Meeting in Vancouver, next month.

Researchers said they have now begun a study to test the drug — called IONIS-HTTRx — in humans with Huntington’s. It acts as a "gene silencer" to block the production of what is known as “huntingtin protein” in people with Huntington's disease.

"It is very exciting to have the possibility of a treatment that could alter the course of this devastating disease," said clinical study principal investigator Dr. Blair R. Leavitt, of the University of British Columbia in Vancouver. "Right now we only have treatments that work on the symptoms of the disease."

Leavitt notes the drug is still years away from being used in human clinical practice, but is showing promise in early testing.

Huntington's is a rare, hereditary disease that causes uncontrolled movements, loss of intellectual abilities, emotional problems, and eventually death.