Medindia

X

Huntington's Disease Drug Found Effective in Mice, Monkeys

by Bidita Debnath on  February 29, 2016 at 12:02 AM Research News   - G J E 4
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.
 Huntington's Disease Drug Found Effective in Mice, Monkeys
Huntington's Disease Drug Found Effective in Mice, Monkeys
Advertisement

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.

‘A new drug IONIS-HTTRx was found to be well-tolerated without any dose-limiting side effects, in the experiments conducted on rodents and monkeys.’
Advertisement
"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 findings will be presented at the American Academy of Neurology's 68th Annual Meeting in Vancouver, Canada, starting from April 15. Huntington's disease is passed from parent to child through a mutation in the HTT gene.

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.

Source: IANS
Advertisement

Post your Comments

Comments should be on the topic and should not be abusive. The editorial team reserves the right to review and moderate the comments posted on the site.
User Avatar
* Your comment can be maximum of 2500 characters
Notify me when reply is posted I agree to the terms and conditions

You May Also Like

Advertisement
View All