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ROCHE RG6042 Update

For more information on this trial click here.

New England Journal of Medicine: Huntington Lowering in HD Patients

May 7, 2019

On May 6, 2019, the New England Journal of Medicine (NEJM) published the first manuscript describing a study concerning the lowering of huntingtin protein in disease patients.  The NEJM paper focuses on the results of the Phase I/IIa study sponsored by Ionis (and now led by Roche Pharmaceuticals).

The paper provides an important overview of previous results shared – as well as some new information.  HDBuzz provides a comprehensive recap, which you can find here.

The publication of clinical study results is always a significant step, and publication in the NEJM in particular – arguably one of the premiere peer-reviewed journals – is positive indeed for the HD Community.

Canada is a global leader in HD research, including this important clinical trial.  Brave Canadians stepped forward, receiving the first dose of the Phase I/IIa study at the University of British Columbia (UBC) under the leadership of Dr. Blair Leavitt.  Dr. Leavitt is one of the principal investigators on this trial – and one of the authors of the aforementioned paper.   Thanks to our generous donors, HSC is proud to fund research and a clinical fellow to support this important work in Dr. Leavitt’s laboratory at UBC.

Publication in the New England Journal of Medicine is positive news.  It shows the high calibre of work being undertaken and the high quality of the researchers, clinicians and community involved.  Coverage in a prestigious publication such as this will also help with awareness across Canada and the global medical community.

We have many reasons to feel optimistic about meaningful treatments.  Right now there are 10 clinical trials and observational studies active in Canada – and more that we believe to be on the way.  But, we should also remember that trials are not therapies.  The journey from clinical trial to commercially available treatments is an multi-faceted process that can take several years. There is no guarantee that drugs currently being tested will prove effective against HD.

In the meantime, families impacted by HD will need support and we are there for them. We will continue to stay in touch and keep you updated as more information is made available.  For our family members, if you have specific questions about participation in clinical trials we recommend that you contact your local neurologist, movement disorders or Huntington disease clinic.



For the full history of updates on this trial, click here.

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