- Ionis-HTTRx is the first drug to reduce levels of the toxic protein behind Huntington's
- It's hoped this form of drug could be adapted to target proteins in other brain disorders
(CNN)For the first time, an experimental drug has reduced levels of the toxic protein that causes Huntington's disease in humans.
Huntington's disease is an inherited disorder in which mutant forms of the huntingtin protein damage nerve cells in the brain.
The new drug, Ionis-HTTRx, was shown to be safe in humans and lowered levels of mutant huntingtin protein in the nervous systems of trial participants.
"The results of this trial are of groundbreaking importance for Huntington's disease patients and families," said Sarah Tabrizi, director of University College London's Huntington's Disease Centre, who led the trial. "The key now is to move quickly to a larger trial to test whether the drug slows disease progression."
The trial involved 46 patien