Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Day two of the conference looks at some of the most promising approaches to fighting Huntington's disease.
After an exciting day of science yesterday, day 2 saw updates on strategies to rid cells of the harmful mutant huntingtin protein and exciting reports on current and planned clinical trials.
Common depictions of HD emphasizing only its movement symptoms paint an incomplete picture of the real disease. HD causes both motor and non-motor symptoms that, together, affect the entire body. Now, scientists are using a broader lens to explore this full set of HD symptoms and determine how symptoms might be related in the disease.
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.
Exciting technologies such as gene silencing are being developed for the treatment of Huntington’s disease. Aside from waiting for disease progression to take place, how will we know whether they are working? This has been a major hurdle for HD researchers, but we now have a super-sensitive method to measure the build-up of harmful huntingtin protein in the nervous systems of HD patients.
In Huntington’s disease, brain cells begin dying long before disease symptoms arise. Unfortunately, good tools for monitoring early brain changes – and testing whether new therapies slow or stop them – have not previously been available. However, a newly developed tool aiming to overcome this problem may mean big changes for the way we track Huntington’s disease.
The brain is a very hungry organ, but does it consume energy differently in Huntington's disease? A team led by David Eidelberg of the Feinstein Institute for Medical Research has been studying the patterns of energy consumption in the brains of people carrying the HD mutation. Changes in how much sugar the brain uses are seen even before the brain starts to change physically, suggesting this might be a useful thing to track in HD clinical trials.
If we find a therapy that we hope can slow down Huntington's disease, how can we prove that it works in patients? What tests should we do and how long should we follow people up after treatment in order to see any real benefits? A major new paper from Sarah Tabrizi and colleagues, reporting the final outcomes of the TRACK-HD study, provides information that will help us better design trials of new therapies in HD as well as understand how the disease progresses.
A specific kind of damage called 'oxidative stress' may contribute to cells getting sick and dying in Huntington's disease. Previous reports had suggested that blood levels of a chemical marker of oxidative stress could be a 'biomarker' for HD clinical trials. But a newly-published work strongly suggests that it isn't a useful biomarker after all. Is this bad news?