In September, the European Huntington’s Disease Network brought together scientists and stakeholders from around the world for updates from experts in all things HD research. Presentations included an update from Roche on the logistics of data analysis from GENERATION-HD1, a report from Wave Life Sciences on the latest from their approach for selective-lowering on mHTT that will be studied in the SELECT-HD trial of WVE-003, news from Novartis and PTC on upcoming clinical trials expected to launch by early 2022, if not sooner, and much more. For the full recap, read the latest article from HDBuzz here.
Endpoint News on ASO’s: What’s next for HD?
Antisense oligonucleotides, or ASOs, are a class of drugs that are used to switch off a gene. You’re probably familiar with ASOs from Roche’s GENERATION HD-1 trial of tominersen and Wave Life Sciences’ trials that halted in quick succession earlier this year. ASOs have shown promise for treatment of spinal muscular atrophy (SMA), but why haven’t we seen success in HD? Endpoint News recently published on the differences of ASOs for treatment of SMA, HD, and other neurodegenerative diseases. To learn about the nuances in this complex and powerful class of drugs, read the full article here.
This Week in HD History
Early November holds two historic milestones in HD research. The first occurred in 1985 before the discovery of the gene that causes HD; neurologist Jean-Paul Vonsattel, MD, and his colleagues published a classification system to understand how HD affects the brain. Using donated brain tissue, the researchers were able to develop a standardized way to link symptoms with signs of brain damage. Brain and organ donation remain critical tools to improve our understanding of HD. If you’re interested in learning more, click here, or visit HDTrialfinder.org.
The second achievement came to fruition in 1996 when Dr. Gill Bates and her lab in England published the first successful mouse model of HD. By genetically modifying a mouse with a small piece of the human huntingtin gene that contained the disease-causing CAG-repeat mutation, they were able to induce HD-like symptoms. This mouse-model is still used today, and paved the way for many animal models that followed in a diverse array of species to help us understand HD.