HD Buzz on the Brain, Vico begins dosing, New Podcast

How does an HD brain find balance? HD Buzz covers the latest clues 

It is well known that Huntington’s disease is caused by a mutation in one copy of a person’s huntingtin gene, which leads to production of expanded, mutant huntingtin protein (mHTT) alongside unexpanded huntingtin protein (HTT). A group of researchers at French National Institute for Health and Medical Research explored how presence and absence of both mHTT and HTT affect the way that brain cells communicate with each other. Their findings revealed amazing insights about how HD affects communication between cells, and how the brain may compensate for changes brought on by the disease. In their latest article, HD Buzz breaks down the highlights of this exciting paper—read the full write-up here.  

Vico Therapeutics announces first patient dosed in study of VO659 

This week, a Netherlands-based pharmaceutical company, Vico Therapeutics, announced they have begun dosing patients in a phase 1/2a clinical trial of VO659 in people with HD and Spinocerebellar Ataxia (SCA) types 1 and 3. Like HD, SCA 1 and 3 are CAG-repeat expansion diseases. VO659 is an ASO drug that targets CAG-repeat expansion by preventing copies of expanded genes from becoming mutant proteins. By reducing levels of mutant proteins that are in circulation in the body, the goal is that this drug will have the power to stop or reverse progression of HD, SCA 1, and SCA 3. Vico Therapeutics will share more on this drug and their trial at the upcoming CHDI conference later this month—stay tuned for more coverage, and in the meantime, read the full press release here.   

This Month on The HDSA Podcast 

Every month, HDSA President & CEO, Louise Vetter, gives listeners the opportunity to meet members of the HD community and get a behind-the-scenes look at the Huntington’s Disease Society of America on the HDSA podcast. In this month’s episode, Louise is joined first by HDSA’s Manager of Youth & Community Services, Maryann Emmerick, MSW, LCSW, to discuss her deep connection to the HD community and passion for connecting young people who are impacted by HD. Next, Louise is joined by two individuals who contributed to the discovery of the HD gene 30 years ago, Drs. James Gusella and Marcy MacDonald. The trio discuss the evolution of HD research and the next challenge to overcome since the discovery of the HD gene—how to treat the disease. To listen to the full episode, click here.