Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
The largest ever therapeutic trial for Huntington's disease was halted early this week because an analysis of the results to date showed that it was very unlikely to show positive results. The study, called 2CARE, was designed to test whether a treatment called coenzyme Q10 could slow the progression of HD.
Cells in the brain depend on support from one another to stay alive. Nutrients called trophic factors act like brain fertilizer, keeping neighboring brain cells healthy. This process has long been thought to go wrong in HD, and exciting new mouse research paints a very clear picture of exactly what's happening.
We know those famous cells called neurons are important in Huntington's disease. But the brain has other cell types with 'supporting actor' roles. New research has shown that brain cells called astrocytes may misbehave in HD, allowing the neurons to malfunction.
The goal of everyone in the HD community is to come up with effective therapies for the disease. A recent publication describes a study in an HD mouse model that comprehensively shows that a proposed therapeutic approach doesn't work. Why are we excited about this bad news?
Huntington’s disease (HD) progression is a long process in which the first changes in the brain happen well before we even see symptoms in patients. It makes sense to focus our efforts on treating the earliest changes, to nip the problem in the bud. But what are these changes and how can we target them? A recent study has literally shed some light on this question. By creating HD mice with glowing brain cells, researchers at the University of Nottingham Medical School and the Babraham Institute in the UK have found that some of the earliest changes happen before these cells start to die, in a region of the brain where HD researchers have never before thought to look.
Huntington's disease is caused by the malfunctioning and early death of brain cells. Replacing those dead and dying cells with stem cells has long been a goal of some HD scientists. A new study investigates the long-term health of some of the earliest cell transplants into HD patient brains — and finds a surprising result.
The symptoms of HD are caused by damage to the brain, but not all parts of the brain are affected equally. This raises an important question - if we had a treatment that could help only a small part of the brain, which part would we pick? A new mouse study from William Yang, at UCLA, attempts to answer this question.
Huntington’s disease is caused by the accumulation of the toxic mutant huntingtin (mHTT) protein. This means that decreasing levels of mHTT, by boosting its breakdown, could be therapeutically beneficial. Palacino and colleagues have identified a possible contender for this role: negative regulator of ubiquitin-like protein 1, better known as NUB1.
All the proteins in our body are made of tiny chemical building blocks, called amino acids. The internet was recently buzzing about a newly discovered link between one of these amino acids, cysteine, and Huntington's Disease. Is it true, as some headlines suggested, that "Brain Degeneration In Huntington’s Disease Caused By Amino Acid Deficiency"?
A chemical called cysteamine has long been of interest as a potential therapy for Huntington's disease. Now, Raptor Pharmaceuticals has announced the interim results of a study of cysteamine in HD patients. The trial failed to meet its pre-specified goal, but there are some interesting details in the data suggesting the ongoing trial deserves attention.