Huntington's News

UPDATE FROM WESTMEAD HD SERVICE & HNSW&ACT

Posted: 3rd January 2018

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).

 

BIGGEST BREAKTHROUGH SINCE THE DISCOVERY OF THE HD GENE IN 1993

Posted: 12th December 2017

In an announcement likely to stand as one of the biggest breakthroughs in Huntington's disease since the discovery of the HD gene in 1993, Ionis and Roche have announced that the first human trial of a huntingtin-lowering drug, IONIS-HTTRx, demonstartes that it reduces mutant huntingtin in the nervous system, and is safe and well-tolerated. More information can be found at HDBuzz

 

HUNTINGTON'S NSW YOUTH CONNECTION

Posted: 5th September 2017

PGD AT A MORE AFFORDABLE COST

Posted: 20th April 2017

Westmead Fertility Centre (WFC) which is affiliated with Westmead Hospital and the University of Sydney offers PGD at a more affordable cost for those who would like to have an HD-free child.

The following is an approximate guide to the type of treatment costs, which are all inclusive, you can expect to pay.

Treatment fees

An IVF cycle costs between $1,735–$5,085 at WFC but after Medicare refunds, out-of-pocket expenses are usually between $317–$1,586 for a first cycle. Additional Health Fund Insurance is not required to have treatment at WFC.

In addition, PGD charges billed by the Genetics Laboratory range from $2,250 – $3,000. 

For more specific information please contact Westmead Fertility Centre on 9845 7484 or visit the website https://www.westmeadivf.com.au/

 

 

 

 

ENROLL-HD NOW AT WESTMEAD HOSPITAL

Posted: 9th December 2015

Huntington’s NSW is excited to announce that the Enroll-HD study is now up and running at Westmead Hospital. This international study presents an opportunity for all family members affected by HD to be involved in research. If you are interested in participating, contact  the study coordinator, Therese Alting, telephone 9845 6310 or 0438 604 719 or email  therese.alting@health.nsw.gov.au. This is an observational study, and not a drug trial. For more information about the study visit www.enroll-hd.org

Dantrolene protects neurons from Huntington's disease

Posted: 30th November 2011

Huntington's disease (HD) is characterized by ongoing destruction of specific neurons within the brain. It affects a person's ability to walk, talk, and think - leading to involuntary movement and loss of muscle co-ordination. New research published in BioMed Central's open access journal Molecular Neurodegeneration shows that the RyanR inhibitor Dantrolene is able to reduce the severity of walking and balance problems in a mouse model of HD.

Read the full story on the EurekAlert! website

Prana's Planned Huntington's Trial Receives Strong Interest From Patients and Researchers

Posted: 11th October 2011

Prana Biotechnology Limited received a very positive reaction from international patient groups and researchers at this week's World Congress on Huntington's Disease in Melbourne. The company had a major presence at the Congress and the Prana team has held numerous meetings and conversations with patient groups and researchers.

Read the full story on the Market Watch website.

Getting The Data Out - A New Scientific Journal Just For HD

Posted: 3rd July 2011

The search for better treatments for HD requires a lot of effort by researchers across the globe. Time is of the essence: the ideal time for a treatment for HD is yesterday. Data produced by all these researchers need to be made available sooner rather than later, so that others can build on what is already known. PloS is a new journal launched to speed up the process of scientific discovery in HD.

The traditional way to publish new findings is for researchers to send data with a ‘story’ to a scientific journal. A panel of experts then reviews that story, to judge whether the data and story are solid, and also whether the story is important enough to be interesting to the journal’s audience. This process is known as ‘peer review’.

This approach has advantages: it ensures that what is published is scientifically sound. Once published, articles can be retrieved via the internet sites like PubMed, and used by other scientists to guide and advance their own research.

However, there are several drawbacks, too. Firstly, the time between doing the research and the story being published can be very long. Sometimes the story has to be offered to several journals, one after the other, before it is eventually published. Several years can pass like this.

Secondly, most journals survive by selling copies, so they have to rate the importance of each story. Anything that might not be interesting to that journal’s audience is likely to be rejected, even if the story is scientifically sound.

That introduces bias to what’s available in the scientific literature. It favours exciting stories, but makes it difficult to publish solid, well-conducted scientific research if the results are less glamorous – for instance, if they show that a particular approach, idea or experiment has not helped. These are known as ‘negative results’.

Another problem is that a complete scientific ‘story’ might take five or ten years to research from start to finish. Along the way, interesting data might be produced, but because they don’t tell a complete story, they are unlikely to be published and seen by other researchers.

“Negative data”, or data that don’t make a complete story, can still be really useful to other researchers. For science to make progress, knowing what doesn’t work can be as useful as knowing what does.

Imagine ten researchers in different places, working on similar scientific projects, that could have been shown to be pointless already, if someone had published a single negative result. All that effort, time and money could have been put to much better use.

A new platform for publishing was launched in September 2010, aiming to make HD research more efficient by publishing the results that would otherwise never be seen, and shortening the time it takes to get data published.

This platform is called Public Library of Science (PloS) Current Huntington Disease and is supported by the CHDI Foundation.

The HD Outreach Service Has Moved

Posted: 3rd July 2011

The HD Outreach Service has now moved from Lottie Stewart Hospital at Dundas to Westmead Hospital. Please note their new contact details below:

Westmead Hospital HD Service,
Westmead Hospital,
Hawkesbury Road,
Westmead 2145
Phone: 02 98459960

Neutrons provide first sub-nanoscale snapshots of Huntington's disease protein

Posted: 20th May 2011

Researchers at the Department of Energy's Oak Ridge National Laboratory and the University of Tennessee have for the first time successfully characterized the earliest structural formation of the disease type of the protein that causes Huntington's disease. The incurable, hereditary neurological disorder is always fatal and affects one in 10,000 Americans.

Pages

This website is certified by Health On the Net Foundation. Click to verify. This site complies with the HONcode standard for trustworthy health information:
verify here.