Posted on May 24, 2018
If you are negative for the HD gene, currently participate in Enroll-HD, and will be attending HDSA’s Convention in June, consider volunteering for a research study on Friday, June 8th at the Convention hotel. Dr. Madeleine Sharp is studying how HD affects cognition and she is looking for control participants (negative for HD) to contribute to her research. The study involves a neurological exam and some paperwork. If you are able to set aside less than an hour at Convention to help an HDSA-funded researcher, contact firstname.lastname@example.org to sign up.
Early Research on HD Gene Therapy at Voyager Therapeutics
Voyager Therapeutics, a Massachusetts-based biotech company, shared a press release this week about their work presented at the annual meeting of the American Society of Gene and Cell Therapy. The company is working on the development of gene therapies for neurological diseases like Parkinson’s, ALS, and HD. Their approach is to design a virus called an AAV that can correct or silence a genetic mistake in brain cells. Voyager’s drug for Parkinson’s disease has so far shown promise in a small safety study in humans. Their Huntington’s Disease drug candidate, called VY-HTT01, is at an earlier stage of the pipeline, delivery and safety testing in non-human primates. Recent data showed that the drug can be injected into the brain once and lower huntingtin by 54% in areas affected by HD, and that no major safety issues were observed in primates after 5 weeks. Although this research is still in the early stages, we’re glad to hear that Voyager and other companies are invested in gene therapy approaches to HD. You can read more about the Voyager Therapeutics approach here on their website.
This week HDSA President and CEO Louise Vetter and Senior Director of Mission and Scientific Affairs George Dr. Yohrling attended the first Enroll-HD Congress in Quebec City, Canada. Enroll-HD is a worldwide study that measures symptoms and progression of HD. It is observational, meaning there are no drugs involved, and it’s not necessary to be tested for the HD gene to participate – anyone in an HD family is eligible. It involves yearly 1-2 hour study visits that include a neurological exam, blood sample, and questionnaires and tests about thinking, behaviors, feelings, and lifestyle. Enroll is helping scientists to better understand what types of genetic and lifestyle factors contribute to the onset and progression of HD. This week’s congress provided researchers and HD organizations a space to highlight advances in clinical research, discuss modifications to the patient protocol, and connect about data and the future of the study. To learn more and participate, visit https://www.enroll-hd.org/.