An excerpt from the Fall 2014 Donor Report
For four decades, the Huntington Society of Canada (HSC) has strived for excellence by providing practical help for families struggling with Huntington disease (HD) and investing in research to slow and prevent this disease that destroys both mind and body.
Together we are carving a new path, a path that will bring us closer to a meaningful treatment for Huntington disease (HD). Our community has supported our efforts to ensure clinical trials of safe potential treatments proceed as quickly as possible.
For the past two years the Huntington Society of Canada (HSC) has played a key role in bridging the relationship between researchers and individuals by educating Canadians on the importance of the clinical trial process; how they can get involved and why their participation is so crucial.
On October 23, 2014, partnering with Canada’s Research-Based Pharmaceutical Companies (Rx&D), the Society will host the Be Brave, Be Bold, Be Ready: Clinical Trial Preparation project to continue to build a national HD clinical trial strategy in Canada. This initiative is timely, as the first HD clinical trial is scheduled to begin in mid-2015.
We are pleased to share the details of this exciting initiative. A small pharmaceutical firm, Isis, has partnered with pharmaceutical giant Roche to launch Phase I clinical trials of a drug currently known as ISIS-HTTRx.
By targeting the messenger RNA that holds instructions for making the faulty HD protein, ISIS-HTTRx attacks the root of the problem. Isis and Roche believe their gene-silencing approach could mean treatments for the thousands of Canadians diagnosed with Huntington disease and for the thousands more at-risk of developing it.
In 2012, Isis researchers reported that their approach not only delayed the progression of the disease in a mouse model of HD, it actually reversed the symptoms. Now they are ready to explore moving into human trials, ultimately aiming to see whether it can achieve similar results. In spring 2015, Isis and Roche will launch Phase I clinical trials in a few selected sites in Canada and Europe.
The launch of Phase I trials is an exciting step towards potential treatments for Huntington disease. However, what works in mice may not work in humans. Even if the drug does prove effective, it will take several years to get to market. The goal of a Phase I trial is simply to test the safety of a new drug. If ISIS-HTTRx proves safe, it then moves into Phase II and Phase III trials to assess how well it works. It is a time-consuming process.
HSC is very proud that those efforts have paid off and that Canadian sites may be selected for Phase I trials of ISIS-HTTRx.
That means if this drug proves effective, Canadians will have access to it more quickly.
This has been a fortunate year as we have been in the unique position to fund all of the fundable research projects that were submitted through HSC’s NAVIGATOR and NEW PATHWAYS granting programs. This was made possible because of our very generous donors and their commitment to research. We will be funding some new and exciting research this year and a project that is a continuation of the work we have funded previously.
We continue to fund excellent evidence-based research, thanks to the generosity of our donors. We are proud of the accomplishments that have been made to date and are looking forward to sharing the outcomes.