In this lecture presented by Prof. Sarah Tabrizi, Trabrizi walk us through Huntingtin Lowering and the results of the first Huntingtin Lowering drug trial – which looks very promising!
What is Huntingtin lowering? In December 2017, the drug IONIS-HTTRx (now known as RG6042) showed groundbreaking results. The Ionis trial was the first trial in humans to lower the expression of 'huntingtin' in the brain.
Why is this important? An overproduction of the protein 'huntingtin' is causing Huntington's disease. By lowering this production, researchers think that it might be possible to slow down the progression of Huntington's disease and reduce symptoms.
In April 2018, new data from IONIS-HTTRx showed that after 3 months of treatment, participants actually benefited of the drug. They showed somewhat better motor function and scored better at cognitive tasks.
What's next? The drug needs to be tested in a larger number of patients, over a longer period to know how it works over time. It also needs to be compared against a bigger control (placebo) group in order to figure out the specific clinical benefits.
Read more about Huntingtin lowering here:
- Groundbreaking results: eurohuntington.org/2017/12/11/2328/
- Drug seems to improve symptoms: eurohuntington.org/2018/04/25/amazing-news-from-ionis-the-drug-seems-to-improve-symptoms/