Since 2012, George Yohrling, PhD has served as the Senior Director, Mission and Scientific Affairs for the Huntington’s Disease Society of America (HDSA). In this video, he discusses current treatment options for patients, as well as therapies in the pipeline and why it’s such an exciting time to be a part of the Huntington’s Disease (HD) research community.
Yohrling: In the future, and hopefully within the next couple of years, we’ll see an onslaught of other Huntingtin-lowering technologies coming into the clinic. Voyager and Genzyme-Sanofi, UniQure, Spark Therapeutics in Philadelphia – All of them are going after a viral-mediated approach using RNAi, so this would involve a direct injection of an adeno-associated virus into the brain of HD patients to deliver an RNA-interfering compound or drug, which would then, hopefully, have the same result as the antisense oligonucleotides. They’re a few years away from the clinic, but there are other therapies currently in the clinic, but they are primarily looking to affect some of the symptoms associated with HD.
It used to be called “Huntington’s Chorea.” Now, it’s not called that anymore, it’s Huntington’s disease because we know that HD is far more than just a movement disorder. It’s a triad of symptoms – it’s motor, it’s cognitive, and it’s psychiatric. Families and caregivers know that it’s really not the motor that’s the most debilitating. It’s that cognitive, and it’s the psychiatric; those are the symptoms that are most debilitating. Those are the ones that keep families or patients from working and maintaining a job. Those will get them out of the house and into a facility. We want to develop drugs that could hopefully impact those symptoms. There’s a company called Azovan Pharmaceuticals that’s testing a compound that’s a vasopressin 1A antagonist. It’s called the STAIR study, and it’s currently recruiting in the United States. This is to test their compound to see if it can impact the irritability and aggression commonly seen in HD patients.
There’s a genuine sense of optimism that I haven’t seen before. I truly believe we’re at the cusp of something fantastic for HD patients. With the initiation of these potential disease-modifying trials for HD, and more to come, I hope that I’m not here 5 years from now talking to you. I hope that we can be at a point where we’re the disease is maintained or we can prevent symptoms from even occurring. It’s a really exciting time for the HD research community.
—
For the latest video interviews with experts from within the rare disease community, follow Rare Disease Report on Facebook and Twitter.
For more information about Huntington’s disease and the work of the Huntington’s Disease Society of America, please visit HDSA.org.
