UPDATE FROM WESTMEAD HD SERVICE & HNSW&ACT

On Monday, 11th December, 2017 Huntington’s disease was back in the international spotlight – the last time the disease hit the headlines was when the gene was discovered in 1993. Social media has been buzzing with news of a research breakthrough. A study showed that a drug, Ionis-HTTRx, could lower the amount of the mutant protein “huntingtin” (mHTT) in individuals with early stage HD. This is important because toxic mHTT is believed to be part of the mechanism leading to HD symptoms. 

When testing new drugs, the very first thing we need to know is that they are safe - and this study found no evidence that patients experienced unpleasant or dangerous side-effects or other adverse events.

There is no doubt that this is extremely exciting news. This first study of Ionis-HTTRx, which began in July, 2015, had 46 participants, recruited from 9 centres in Great Britain, Canada and Germany. The trial was not designed to address the question of whether the drug has an effect on the onset or the course of the disease, and a further, larger trial will be needed to demonstrate this.

The press release issued by the company which ran the study, Ionis Pharmaceuticals, reported that the next phase of research will be handled by the pharmaceutical company Roche. The only information provided by Roche about the next stage of research is that “future studies for the program will be conducted globally” and “information will be provided as soon as possible.”

Like others around the world, we are eagerly awaiting announcements about the next stage of the study. Until then, there is nowhere in Australia, or anywhere else in the world, where one can join an Ionis-HTTRx study.

The Westmead HD Service and Huntington’s NSW & ACT work extremely closely together. We will ensure that any news about new trials will be distributed immediately through the Association’s website, Facebook page or directly through emails (if you are a member or client of the Association).

Watch this space!


Dr Clement Loy                    Robyn Kapp OAM
Director & Neurologist          Executive Officer
HD Service                           HNSW & ACT

Westmead Hospital              www.huntingtonsnsw.org.au                                                                                                      

For a good, reliable source of information about this study, including an informative Q&A about the research and most HD research results, we suggest you look at the HD-Buzz website:  https://en.hdbuzz.net

If you or anyone you know have any concerns about HD, you can make an appointment to attend the Westmead Hospital HD clinic. To discuss what a clinic visit involves, you can ring the Outreach Team 8890 9960, or to make an appointment ring 8890 6544.

If you are interested in participating in research, you may also want to consider the global Enroll-HD study (https://www.enroll-hd.org) This is an observational study (no drug treatment) for HD families. You can find out more about Enroll-HD by contacting the researchers at Westmead Hospital, on 8890 6310 or 0438 604 719 (Mondays and Thursdays).

 

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