Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
New therapies for disorders like Huntington’s disease are on the way, but getting the drugs to enter brain cells can be a major challenge. A group of scientists has redesigned and tested a harmless virus that can efficiently deliver a 'gene silencing' message throughout the brain in mice, much further than naturally occurring viruses can reach. What's more, it can be given with a simple injection into the blood, offering great potential for research in gene silencing research and beyond.
Today brings news that the first Huntington's Disease patients have been successfully dosed with gene silencing drugs targeting the HD gene. These brave volunteers are the first HD patients to ever be treated with drugs designed to attack HD at its root cause, a treatment approach with huge potential. What about this news has us so excited?
Even though every patient with Huntington's Disease has a mutation in the same gene, the age at which HD patients develop symptoms varies widely. A global consortium of HD researchers has just published a landmark study of genetic differences between people that might explain some of that variability, producing some tantalizing new targets for drug discovery efforts.
Just like it is difficult to predict exactly when a storm will hit, predicting when Huntington’s disease symptoms will arise for any particular person is hard to do. However, new research suggests that tiny changes in the on switch of the Huntington’s gene affect symptom onset – and may provide important information in the search for Huntington’s therapies.
Auspex Pharmaceuticals just announced the results of two clinical trials known as 'First-HD' and 'Arc-HD'. These trials were designed to test a modified version of the approved Huntington's disease drug tetrabenazine, which reduces unwanted movements. The results reveal that Auspex's drug has some advantages compared to tetrabenazine for treating excessive movements in HD.
A new clinical trial just announced for 2015 aims to test a “huntingtin lowering” therapy, called an antisense oligonucleotide (ASO), that attacks mutant huntingtin directly. We’re extremely excited—it’s the first-ever human HD trial to fight HD at the root of the problem, and has shown great promise in animal models. What’s the scoop?
CREST-E, the largest clinical trial of the dietary supplement creatine, has been terminated early because an early analysis of the results to date showed there was no realistic chance it could show positive results. This provides compelling evidence that creatine doesn't slow down progression in Huntington's disease patients.
Our final report from the European HD Network meeting. For the first time, video of many presentations, including our 'EuroBuzz' sessions will be made available online shortly.
Here's Ed and Jeff's live Twitter report from the second day of the EHDN 2014 meeting. Our final report will be tomorrow, and we'll be uploading video of our onstage roundup sessions soon.