Gene-silencing (otherwise known as huntingtin lowering therapy) offers a promising way to slow or stop Huntington disease (HD). By using anti-sense oligonucleotides (ASOs) to prevent the HD gene from producing huntingtin protein, this approach targets the disease right at its root.
Thanks to HSC donors, Canadians have never had more opportunities to take part in clinical trials for potential HD treatments.
Donor support allowed for the creation of the Clinical Trials Consortium which first convened in 2015. Since then this group has worked together to connect clinicians, share best practices and maximize the opportunities for people affected by HD to participate in clinical trials.
HSC donors helped to lay the groundwork for these trials to take place and that will only prove more beneficial in the future as more trials become available.
RG6042 (formerly known as IONIS-HTTrx)
The initial Phase I/IIa RG6042 trial has completed. That study demonstrated that the drug was initially safe and that higher doses reduced the huntingtin protein by 40 to 60 per cent. The next step is to run a trial that answers several key questions: What are the effects of lowering huntingtin over a longer period of time? Do any unexpected safety concerns emerge? And, of course, does sustained treatment slow or stop the progression of HD? The Canadian HD Community has been leading the charge. Canadians are a part of this clinical trial helping us all to get to this important point in our history.
Dr. Mark Guttman described in his Symposium presentation in October that two more potential HD treatments have reached the point of clinical trials, and his Toronto clinic is the first in the world to be testing them. The first doses have already been given to courageous trail blazers in Toronto! There’s a long list of criteria they need to meet to be eligible, however. They have to be in the early stages of HD, under the age of 65, and otherwise in good health. They have to live within a 90-minute drive of the clinic, and they have to have one or both SNPs in the right location.