Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
A new analysis of clinical data from the TRACK-HD and COHORT studies proposes a way to design of clinical trials designed to delay the onset of HD, rather than treating symptoms after they occur.
Nearly a thousand HD family members converged on Baltimore, Maryland for the 2016 Huntington’s Disease Society of America’s Annual Convention. We normally don’t write reports from patient and family conferences, but there was something special about the atmosphere of this year’s Convention that compelled us to pen a brief update.
The family and friends of individuals with HD often tell doctors that they began to notice changes in behavior long before a diagnosis was made. To better understand these early signs, researchers analyzed a psychological questionnaire filled out yearly for a decade by thousands of HD mutation carriers and their companions. The companions were more likely to perceive worsening symptoms over time.
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.
The Huntington's disease genetic test involves counting the number of CAGs at the start of the HD gene. Some results are in a 'gray area' - larger than normal but smaller than the range that causes HD. Now, by closely studying people with CAG counts in the intermediate range, the PHAROS study has found some subtle differences in mood and behavior. These results don't mean that intermediate CAG counts cause HD, but they do suggest they may have some effects on the brain, which need to be studied in more detail.
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.
Our daily report from the annual Huntington's Disease Therapeutics Conference in Venice, Italy on the third and final day of the conference.
With gene silencing therapies heading towards the clinic, a new question has arisen - how will we know if they work? How can we tell if the amount of huntingtin protein is lowered in people? New work from London and Basel demonstrates that the huntingtin protein is detectable in blood samples, and that its levels change during the course of HD.
Jeff Carroll and Ed Wild present EuroBuzz episode 1 - bringing the European HD Network Meeting direct to you, in plain language. Watch online!