There is a new kind of Huntington’s disease drug which is right now undergoing clinical trials. This drug pridopidine has shown results in the form of altering the gait problems in mice suffering from Rett syndrome. This disease has no treatment as of now. Its various symptoms are problems in breathing, autism, and cognitive disability. The death rate with this disease is quite high and almost 50% of the women who have this disease don’t cross the age of 50.
The boys who have this disease meet with death in infancy only. Michal Geva who is the Pharmacology Project Leader in Teva Pharmaceutical industries, which has been conducting trials on this drug had to say that, “[Pridopidine] is a safe, novel drug with encouraging potential to improve the life of Rett patients, who currently have no treatment.” After showing such effective results, pridopidine is now undergoing the testing for approval by US FDA where it has already been approved in initial tests determining its efficacy and safety.
This drug has also been tested on 408 individuals suffering from Huntington’s disease last year and the results were that the drug didn’t lead to any improvement in the motor problems experienced by such patients,although there was improvement in their wellbeing. However, this drug has led to improvement in motor symptoms in Huntington’s disease mice and that’s why it was used for testing for gait improvement in mice suffering from Rett syndrome.
As far as the testing of this drug on male mice was concerned, the test subjects were segregated into two groups, 60 male mice were taken out of which 40 were given this drug and the rest 20 a placebo. All the mice taken as test subjects were 4 years old. However, when the mice who were given the drugs turned 6, it was seen that there were improvements in terms of their gait. The mice were also tested for the strength of their motor skills via a test called NueroCube in which they showed improvement also.
The drug was also given to 40 female mice aged 5.5 weeks who were suffering from Rett syndrome. Out of these 40 females, 20 were given a placebo and the rest were given the drug. As per the results, there was a tremendous improvement by 45% in the gait of females who were given this drug by the time they turned 8 weeks old and by 55% when their age was 12 weeks.
However, the drug didn’t lead to any improvement in the death rate of the mice, they still meet their demise when they are 8-10 weeks old, but the symptoms did go down. As per Geva “We still need to understand the mechanism of action of pridopidine.” The researchers plan to go forward with the testing of the drug for its consumption by children. There are common features between the Huntington’s disease and Rett syndrome both the disorders include gait problems because the patients can’t have coordination between the leg muscles and joints.
Source URL: https://spectrumnews.org/news/experimental-huntingtons-drug-improves-gait-rett-mice/