Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
What happens when you have a broken part in a machine? You fix it! A new study shows that increasing the activity a critical piece of machinery called ‘mTORC1’ in a mouse model of Huntington’s disease leads to improved motor problems and brain abnormalities associated with the disease. These recently published findings may offer scientists a new target for therapeutic development in HD.
The brains of Huntington's disease patients seem to have problems making enough energy. Giving brains an energy boost might help with HD, but getting extra fuel into the brain is difficult. A new study from France reveals that feeding HD patients a special kind of fat improved their brain's energy levels. This opens the door for new studies to test whether this improvement in energy could help with HD symptoms.
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.
Scientists can now reprogram human skin cells to make working cells that resemble 'medium spiny neurons', the type of brain cell that is most affected early in Huntington's disease. We're still a long way off from being able to replace the brain cells that are being lost in HD, but this research is an important step down that path, and is a great tool to study 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.
New work in brain diseases like Alzheimer's suggests that brain cells called neurons might be 'catching' the sickness from their neighbors. A recently published paper suggests that, in very specific lab conditions, this might also happen in Huntington's disease. What does this mean for what we know about HD, and how to treat it?
Ed and Jeff present the second day's science at the EHDN meeting in Barcelona. Features a clinical trials roundup, an exclusive interview with Prof Sarah Tabrizi about the first trial of a huntingtin lowering 'gene silencing' drug, and a surprise for EHDN president Prof Bernhard Landwehrmeyer.
We present the video of Ed and Jeff's review of first day's science at the European HD Network meeting in Barcelona.