Postcard from Dubrovnik
In April of 2023, more than 400 scientists from around the world met in Dubrovnik Croatia for the annual HD Therapeutics conference hosted by CHDI Foundation. Each year CHDI creates a video summarizing the conference that is directed at non-scientists. Charles Sabine, British journalist, gene carrier, and HD advocate, hosts the video, which features interviews with scientists and updates on HD therapeutics research. Major topics include the GENERATION HD2 trial of tominersen, uniQure’s trial of gene therapy AMT-130, biomarkers, and novel approaches to treating HD. Check it out here.
This Week in HD History: Observational Research Studies
August of 2002 marked the beginning of an important observational study called PREDICT-HD. Based initially in Iowa, it was one of the largest early studies to track the development of HD symptoms in thousands of individuals over time, and the data is still being used to better understand early HD. In fact, its current iteration, PREVENT-HD, is ongoing at the University of Wisconsin.
Large observational studies are becoming more and more important because the knowledge gained helps to build a case for testing drugs in people with the HD gene before symptoms begin. Studies like Enroll-HD are also magnets for pharma companies to study HD and to recruit individuals who are already participating in research. That’s why HDSA highly encourages participation!
Transplanting Cells to Support the HD Brain
Our brains contain cells called neurons which communicate with one another to drive brain activity. Our brains also contain even more cells called glia which play many important supportive roles. A team of scientists based in Copenhagen and New York recently published a paper showing that it’s possible to replace diseased glia with healthy ones, in mice engineered to have HD. By transplanting “young” cells grown in dishes into state-of-the art genetic mouse models, Dr. Steven Goldman’s team showed that the new cells could replace sick ones and integrate into the brain’s existing network.
Some of the press around this paper emphasizes that human trials are a few years away. It’s always wise to approach those statements with caution, as research timelines can change. This paper is also very focused on the state of the cells in the brain, and it does not include experiments testing changes in HD mouse health or behavior. Further research will need to look at longer term safety and effectiveness. That said, this work represents an exciting potential avenue of research towards cell replacements to treat HD.
