HDSA’s President and CEO Louise Vetter spoke on Friday, March 13th with James Valvano of We Have a Face, an organization dedicated to broadening global awareness of Huntington’s disease. On their podcast, “We Have a Voice” Radio, James interviews people who are shaping care, research, and advocacy for HD. This week’s topic was HD Trialfinder, HDSA’s clinical trial database and matching service (www.hdtrialfinder.org). Louise and James spoke about the story behind creating HD Trialfinder, its important security and privacy features, and the extraordinary ability of the HD community to persevere, unite, and inspire.
HDBuzz on Shrinking CAG Repeats
People with HD have an extra stretch of CAG repeats in their huntingtin gene, and these tend to expand even further in some cells of the brain and body as they age. In the past few years, research has suggested that this further expansion could lead to earlier symptoms. Therefore, many academic researchers and companies have begun to focus on ways to shrink CAG repeats. Recently a team of Japanese and Canadian researchers used a drug in different laboratory models of HD to successfully stop the expansion, and possibly shrink CAG repeats. HD Buzz does a great job this week covering the story in detail. There’s a lot of work to be done before this could be developed into a therapy – including figuring out how to get the drug to the brain – but it’s a promising early step in research to boost DNA repair.
Designing a Therapy to Create New Neurons
A Pennsylvania-based company called NeuExcell recently published a study in which they used a gene therapy delivered by a harmless virus to create new neurons in the brains of HD mice. They shared a press release this week. The drug, delivered via a surgery to the mouse brain, is able to convert other supportive cells into new neurons, which seem to integrate into the brain’s networks and function like regular neurons. The HD mice then showed improvements in their behavior compared to those that were not treated. This is an exciting advancement in gene therapy technology that will need to be further tested in larger animals before it can be considered as a human therapy.
Brain Awareness Week
Brain Awareness Week, coordinated by the Dana Foundation, is a global campaign to promote awareness enthusiasm, and support for brain science. All around the world for a week in March, community events and activities celebrate and share the wonders of the brain. Thanks for reading this blog and staying up to date on HD research!