Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Our second update from the Annual Huntington's Disease Therapeutics Conference.
The first of our special reports from the Annual Huntington's Disease Therapeutics Conference in Palm Springs – the biggest meeting of HD scientists focused on developing treatments to prevent or slow Huntington's disease.
The growth factor 'BDNF' usually sends a “Survive!” signal to brain cells. In Huntington’s Disease (HD), this system doesn’t work as it should, so scientists have been looking for ways to boost the signal. Enter one of nature’s most useful tools: the antibody. Usually antibodies play an important role in the immune system, but researchers have identified two antibodies, produced by the company Pfizer, that can act like a set of spare keys to activate the TrkB receptor. This unlocks the door to determining whether a boost in TrkB activity is enough to prevent neurons from dying, in hopes of slowing the progression of HD.
In Huntington’s Disease, one of the many problems arising from the disease mutation is that DNA gets folded incorrectly. A new study in mice reveals that a drug changing the way DNA folds may have beneficial effects in Huntington’s – even for the untreated offspring of treated individuals. This discovery could affect how we think about drug therapies for Huntington’s Disease.
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?