Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Thinking problems in Huntington’s disease take a huge toll from early in the disease. Now, new work suggests that a drug already approved by the FDA to treat another brain disease – multiple sclerosis – may stave off these problems in HD mice. Could these results be real, or are they too good to be true?
Though many scientists have focused on damage to a part of the brain called the striatum as a source of HD symptoms, this is a narrow picture of what changes in the brain during HD. A new book provides a summary of many research techniques over a hundred years that have led to a more complete image of HD as a disease affecting the entire brain.
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?
The last few years have been full of announcements about the results of clinical trials for HD drugs, but it can be surprisingly hard to understand what these results actually mean. What seems like a simple question – did a drug work or not – turns out to be more complicated than you'd expect. HDBuzz is here to help HD families untangle hope from hype when it comes to clinical trial announcements.
It’s like gardening gone wrong: scientists can sprinkle Huntington’s protein on the outside of laboratory-grown brain cells and make sticky, potentially harmful protein clumps grow inside the cells. Now, new research showing that human brain fluid does the same thing could help us monitor Huntington's disease.
When patients participate in clinical trials, there needs to be some type of readout to determine whether the new treatment worked. It’s important to know two key things: What to measure and how to measure it. In the case of HD, these obstacles have vexed scientists and doctors for years. The latest research comes up with a clever new approach to overcome both challenges in a new way. These results could offer a valuable tool to study new HD therapeutics entering clinical trials.
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.
It takes a lot of research to decide whether a new treatment is ready to test in humans. A few years ago, we reported that a 'brain fat' called GM1 is reduced in HD, and replacement therapy showed potential in mice. Despite rumors you may have heard, it’s not ready to try in the clinic. But if preclinical research continues positively, there may be a surprising potential source of GM1: the brains of sheep.
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.