This week uniQure shared a press release stating that another two patients have entered their HD clinical trial. The drug, AMT-130, is an experimental gene therapy that aims to lower harmful huntingtin protein in the brains of people with HD. It is delivered via a one-time brain surgery procedure, and because this type of treatment is so new, uniQure is proceeding carefully and slowly to ensure that it is safe. This means that it is being tested in a small group of people (26) with early symptoms of HD, and participation is widely spaced out at the beginning. The first two trial participants, who underwent surgery in June, were closely followed for several months, and when no dangerous side effects were noted after 90 days, the next two patients then were approved to have the surgery in September. If these first four participants don’t experience any hazardous or life-threatening side effects, uniQure will recruit up to 10 patients by the middle of 2021. It’s exciting to see this first HD gene therapy trial continue to move forward. 

Voyager Therapeutics is a gene therapy company focused on neurodegenerative disorders that is working on a treatment for HD. They also shared a press release this week with an update on their drug VY-HTT01. They had submitted an Investigational New Drug (IND) application to the FDA, which all companies must do in order to move forward with clinical trials. They announced in their press release that the FDA has placed a “clinical hold” on their application. You may have seen some buzz in the news about this, but it essentially means that Voyager will need to resolve some of the FDA’s questions about the chemistry of the drug and how they will manufacture it. This happens frequently during the process of creating a new drug for human testing, and they expect to hear more from the FDA within about a month, so that they can resolve any issues and move forward with a clinical trial.   

At-home Tele-Exercise Study  

Researchers at Burke Neurological Institute are conducting a research study of an at-home exercise program. It is designed for people with chronic neurological injuries or brain diseases (like HD) and is done virtually, from a seated position. It involves boxing, intervals, and exercises for posture, which have all been adapted for those who have movement symptoms. For more information and to contact the study coordinators, check out this flyer. 

HD Insights Podcast 

The Huntingtin Study Group hosts the HD Insights Podcast, which features long-form interviews with people shaping HD care and research all over the world. The most recent episode is a conversation with Dr. Dietrich Haubenberger, Medical Director at Neurocrine Biosciences. Neurocrine is conducting the KINECT-HD study, a clinical trial of a drug to treat HD movement symptoms (chorea). The drug, valbenazine, is approved for people with tardive dyskinesia, a condition that also causes involuntary movements of the face and limbs. Dr. Haubenberger talks about his own career path, the “village” of doctors, researchers, and families required to run a clinical trial, and the importance of getting input from patients on what interventions and improvements are meaningful. He also shares details about the KINECT-HD study.