This week marks a major milestone in HD research: the first two patients were dosed in the world’s first gene therapy study for HD. On June 19th, uniQure announced in a press release that two brave participants had undergone the brain surgery required to deliver the experimental huntingtin-lowering therapy AMT-130. The surgeries took place at the Ohio State University’s Wexner Medical Center, which is a designated HDSA Center of Excellence. The drug is a harmless virus that contains the instructions to create a genetic weapon, a micro-RNA, that stops the huntingtin message from creating new toxic protein. This is a small and slow-moving safety trial, with 26 planned participants who will be followed closely for up to five years. The first two patients, one who received the drug, and one who received an imitation surgery, are recovering and will be followed very closely for 90 days before the surgery is attempted in any more people.
The HD field is very excited about this trial beginning after a slight delay due to COVID-19, and we are hopeful that what we learn from these brave individuals will carry us forward into a new age of HD therapy. You can learn more about how AMT-130 works from a great HDBuzz article published in early 2019, and you can read more about the clinical trial on uniQure’s website, on www.hdtrialfinder.org, and at clinicaltrials.gov.
HDBuzz on Brain Cells Changing Jobs
Viral delivery approaches are also being used to explore HD therapies that are different from huntingtin-lowering. A group of HD scientists recently demonstrated a new potential approach to replacing the neurons lost in HD. They showed that they could use a virus to deliver instructions that would turn some of the brain’s supportive cells, known as astrocytes, into new, functional neurons. This cell replacement strategy was successful in HD mice and caused them to perform better on learning and movement tasks. Check out the whole story on brain cells getting new jobs here on HDBuzz.
Podcast and Courses from the Huntington Study Group
The Huntington Study Group (HSG)’s HD Insights Podcast presents long-form interviews of people who are shaping HD care and research today. The most recent episode features Dr. Ignacio Muñoz-Sanjuán, Vice President of Translational Biology at CHDI Foundation. He speaks about his work as President and Founder of FACTOR-H, a non-profit that focuses on the Huntington’s disease community in Latin America, and about the situation of the Venezuelan HD community that was essential to the discovery of the gene more than 25 years ago.
HSG also recently announced a new online learning series for medical professionals as part of its CME4HDTM curriculum. Using interactive case studies, the course builds on a series of lectures that helps providers to learn more about caring for HD patients while earning continuing medical education credits. Initiatives like these will help the medical field to prepare for an influx of new HD patients if and when disease-modifying treatments become available.
