Scientists have recently carried out a trial to see if gene silencing drugs are safe to use on patients with Amyotrophic lateral sclerosis (ALS) a motor neurone disease that also goes by the name Lou Gehrig’s disease in the US.
Gene silencing drugs can be used to instruct cells to stop making the huntingtin protein, and if the delivery method is deemed to be safe in these trials, the next step may be to start testing the drugs for Huntington’s disease.
According to the Huntington’s Disease Association, the trial involved 21 patients with ALS, separated into four different groups. Each group received a different dose of the medication, with one set being administered a placebo. The test was mainly to see if the drug could be administered safely to patients, and whether it was successfully ‘crossing the brain’s checkpoint system’.
The next step will be to increase the dose gradually to ensure that higher levels are safe to administer to ALS patients. If all goes well, similar therapies for Huntington’s disease may begin clinical trials.
Source: Barchester Healthcare