Summary
- Leaving a Legacy: Brain Donation in HD Webinar available online
- Delivering HD Drugs to the brain: scientists explore new methods
- An Advocate’s take: FDA approves Valbenazine for treatment of HD chorea
Leaving a Legacy: Brain Donation in HD Webinar available online
Last week, HDSA was joined by leading researchers, a neurologist specializing in HD, and an HD family member and community advocate for a powerful webinar covering all things brain donation. Each speaker shared their unique perspectives on the importance of brain donation for HD, and expertise in navigating the decision of donation. You can read more about brain donation on HDSA’s website, or watch the full webinar on HDSA’s YouTube channel here.
Delivering HD Drugs to the brain: scientists explore new methods
Antisense oligonucleotides, or ASOs, are a class of drugs that are often used to treat a genetic disease by switching off a gene. These drugs have a lot of promise as a tool to turn off the mutant copy of the huntingtin gene in people with HD. However, it is difficult for ASOs to cross the blood brain barrier, which requires them to be delivered directly into the central nervous system. One solution to this challenge is to deliver ASOs with a spinal tap so that they can enter the fluid that bathes the brain, but research has shown that this results in limited distribution through brain tissue.
Researchers at University of British Columbia, including 2019 HDSA Berman-Topper Fellow, Dr. Nick Caron, are studying new ways to deliver ASOs that may allow them to circulate better through the brain. In a recently published paper, they show that a “nanodisk” system can be used to deliver an ASO into the noses of HD mice. The drug successfully reached the deep areas of the brain most affected by HD. This group is also exploring IV delivery and moving the work into primate models.
An Advocate’s take: FDA approves Valbenazine for treatment of HD chorea
As mentioned in last week’s blog, August 18th marked the FDA approval of INGREZZA, a drug developed by Neurocrine Biosciences that can now be prescribed in the US to treat chorea associated with Huntington’s disease. HD advocate and community member, Ken Serbin, also known as Gene Veritas, discusses what this news means for the HD community in his most recent blog post. Read his take here.
