We know that keeping up with the latest Huntington’s disease (HD) research and therapeutics can be challenging and confusing, so we’ve prepared a post that brings together resources from a few key sources to help demystify and simplify the latest developments of the past year!
First, we’d recommend HDYO-International’s new video resource from the series Breaking Down Barriers, “Accessing HD Research” (January 2024), which breaks down what types of research are available to people in the HD community (Clinical Trials, Observational Studies, and Surveys and Registries) and how you may access these opportunities:
Secondly, check out CHDI Foundation’s video “Postcards from Dubrovnik”, to hear about the highlights from CHDI’s 18th Annual HD Therapeutics Conference (April 24th-27th 2023), brought to you by Charles Sabine himself and including:
· Tominersen update
· “One-and-done” (adeno-associated virus) from UniQure
· Blood biomarkers for HD (NfL, MRI scans, and cholesterol)
· Novel shots on goal (Arvina’s PROTAC to induce degradation of mutant huntingtin protein and VectorY’s antibody, VTx-003, to mop up mutant huntingtin)
Lastly, for an update on clinical research being conducted in New Zealand, see below the summary provided by Auckland Study Coordinator Christina Buchannan (November 2023):
· Enroll-HD clinics are now operating in Christchurch (laura.paermentier@cdhb.health.nz), Wellington (grace.hodgson@ccdhb.org.nz) and Auckland (cbuchanan@adhb.govt.nz). Enroll-HD is one of the best ways to get involved in HD research, we don't administer any drugs, but we record data and take samples, and keep you up to date with what is going on in HD research.
· HDClarity is a sub-study of Enroll-HD that is taking and storing CSF for HD research. This is already up and going at the Christchurch Enroll-HD site and is soon to start at the Auckland site. CSF is important for measuring brain biomarkers that can not be measured in blood (yet!).
· Gen-HD2 is the continuation of the Roche Gen-HD Tominersen trial (talked about in the postcard video). This is underway in Christchurch and Waikato, but there are strict eligibility for joining this study. If you are part of Enroll-HD, the data we collect there can help decide whether you might be a good candidate for this trial (another good reason to take part in Enroll-HD). If you would like more information about this trial please contact laura.paermentier@cdhb.health.nz or kate.ives@waikatodhb.health.nz
· PTC518 PIVOT study is another huntingtin lowering drug trial that is underway in Christchurch. If you would like more information this trial please contact laura.paermentier@cdhb.health.nz
· Pūnaha Io - the New Zealand Neuro-Genetic Registry and Biobank is also up and going and storing data and samples from people with a wide range of Neuro-Genetic disorders, including Huntington's disease. This is managed through Dr Richard Roxburgh's team in Auckland. If you would like more information this study please contact cbuchanan@adhb.govt.nz
· The Neurological Foundation Human Brain Bank - If you are interested in learning more about the process, or registering your interest, go to: https://www.brainbank.ac.nz/the-process/ . Many Enroll-HD participants will have opted for "future contact" from the Brain Bank as part of their Enroll-HD consent, but the Brain Bank will never contact you unless you contact them first.
The above may seem like a lot of information, but a lot is GOOD, as it represents the growing interest and novel prospects in the HD field which are hopefully inching us closer and closer to a cure. I’ll leave you with a quote from Charles Sabine:
“15 years ago when I came to my first Therapeutics Conference, there was just 1 company represented. This year, no less than 35 are here, and that statistic alone gives us more reason than ever to be hopeful of finding ways to manage our disease” (Charles Sabine, Postcards from Dubrovnik 2023).
