After an unexpected dosing halt in the GENERATION-HD1 study nearly 6 months ago, Roche is gearing up to increase their commitment to HD research. Spark Therapeutics, a member of the Roche group, recently announced a partnership with NeuExcell to develop new treatments for HD. Many experimental HD therapeutics thus far have sought to lessen effects of the disease by slowing its progression. NeuExcell has instead been developing a platform to regenerate the brain networks that are ravaged by the disease. This experimental gene therapy causes glial cells, an abundant type of brain cell that provides structural support and insulation, to become neurons, the signaling cells in the brain are most affected by HD. To learn more, you can check out a 2020 article from HDBuzz on this topic.
Founder’s Day of Giving SATURDAY!
Join HDSA in celebrating our founder, Marjorie Guthrie, in Founder’s Day of Giving on September 18th! Your contribution will help HDSA carry on its mission work, including our activities supporting and growing the field of HD Research, to improve the lives of everyone affected by HD and their families.
Any amount makes a difference, and better yet—a passionate HD family is generously matching all donations made up to $20,000, meaning your gift will have double the impact for families living with HD.
Visit the Founder’s Day of Giving web page to learn more about how you can show pride and gratitude for the impact HDSA has on each of us, in our families and communities!
This Week in HD History: First Animal Models of HD
Animal models of HD are a critical piece of the research process; they allow us to gain a better understanding of HD and explore potential new avenues to prevent or treat it. Even before the discovery of the gene causing HD in 1993, researchers were determined to come up with ways to study the disease in the living brain. Before it was possible to create and study mice that had the HD mutation, scientists understood that people with HD had a very particular kind of damage to the brain. The area known as the striatum is responsible for movement, motivation, and mood, and in HD, these cells begin to get sick and disappear.
In 1976, researchers were able to recreate this type of brain tissue damage in mice and observe “symptoms” that appeared similar to Huntington’s disease. This type of modeling was used for almost two decades until the first genetic mouse models were created in the mid 1990s. Today there are many different animal models used to study HD, from worms, flies, and slime mold, to rodents, sheep, pigs, and primates. We owe a great debt of gratitude to these animals for the research progress of today. These animals provide researchers with essential insights into the progression of HD that can help us make predictions about how the disease will present in humans. Through observational research studies like Enroll-HD, HD scientists are able to better understand Huntington’s Disease as it occurs in humans, make new observations around disease progression, and confirm discoveries made in animals.