1800 244 735

Helpline (02) 9874 9777

EuroBuzz 2014: day three

Our final report from the European HD Network meeting. For the first time, video of many presentations, including our ‘EuroBuzz’ sessions will be made available online shortly.

08:09 – This mornings EHDN session focuses on one of the most exciting therapeutic options of all – Huntingtin lowering

08:10 – Leslie Thompson is interested in how small changes to the huntingtin protein itself control it’s turnover. If we can understand how cells normally get rid of huntingtin, maybe we can increase the process with drugs. Thompson’s team is exploring a key pathway that cells use to get rid of huntingtin called “sumo-ylation” (really!). Another way to benefit cells with the HD mutation is to help them make proteins more carefully. Cells make proteins to do most of their work, which have to fold into complex shapes, this process can sometimes go wrong. A normal part of the cell protein-folding machinery is called TRIC. Giving cells more TRIC protects them from the HD mutation. Thompson is now exploring different ways to get TRIC into the brain, to understand whether this could be a therapeutic option for HD.

08:27 – Doug Macdonald of the CHDI foundation has been working for a long time on Huntingtin lowering therapeutics. Huntingtin lowering is one of the most exciting potential therapies for HD. The mutant HD gene gets used by the cell to make a copy we call a “message RNA”, which gets turned into a protein. Confused? Surprisingly, there are now therapies that can attack each of these levels – gene, message and protein, to try and get rid of it. So which approach will work best to reduce the symptoms of HD? CHDI is, with other investigators, trying a number of approaches. One issue with all these approaches is how will we know if we’ve reduced huntingtin levels? Amazing new approaches allow researchers to count individual copies of the huntingtin protein. Because they’re so sensitive, these techniques let scientists measure huntingtin in cerebrospinal fluid, which bathes the brain. If we can get drugs into the brain to lower HTT, maybe we’ll be able to ensure it’s working by collecting spinal fluid. Donating spinal fluid is not trivial, but it’s a lot easier than donating your brain! CHDI is working to develop high tech measures of brain function that work in HD mice, in hopes they’ll also work in people.

08:51 – Jang-Ho Cha, of Merck and the HDSA, addresses the conference on the challenge of clinical trials in HD. “Our finish line: Treatments for HD. What are we going to require to get to this point?” Without a path through clinical trials, we won’t get effective treatments. There’s two kinds of research we need to get to clinical trials – “clinical” work in people, and “pre-clinical” work in the lab. Along the way, there’ll be impossible seeming gaps, but if we’re clever we’ll get across. Once we figure out how to solve impossible seeming problems for one trial, the solutions will help speed future ones. The first phase of testing a drug is a ‘phase 1’ trial. These trials are just to establish a drug is safe and well-tolerated. Biomarkers, measurable traits that can be measured in people, help keep HD drug developers ‘on the trail’. Drug development for multiple sclerosis was accelerated by the development of MRI-based biomarkers. This rapid speeding of trials in MS has led to 14 treatments for this previously untreatable disease. What is ‘phase 2’ study? A study designed to provide some “proof of concept” that a drug works. We have to think differently about drugs designed to improve HD symptoms and those we think might actually prevent the disease. Better ways of quantifying HD symptoms will lead to smaller, faster and cheaper trials. A ‘phase 3’ trial is designed to provide ‘pivotal’ evidence that a drug works, and can lead to its approval. “No patients, no trials”. An engaged and informed HD community is required to complete the clinical trials we need.

10:10 – Prof Landwehrmeyer’s thoughts on coping with disappointment and frustration, quoting Churchill – “The route to success is to go from failure to failure with undiminished enthusiasm”

10:22 – Landwehrmeyer tells us: We’ve been saying “The drugs are coming” for years – it’s finally happening

Share on facebook
Share on twitter
Share on pinterest
Share on email

Latest Research Articles

Scientists identify precisely how pridopidine works in models of Huntington’s disease

Published date: 12 June, 2021

Pridopidine is a drug developed to treat Huntington’s disease (HD) and now scientists have a clearer understanding of how it works in the body and brain. In a series of academic papers, researchers figured out that pridopidine is working by targeting a particular receptor protein called S1R. With this new understanding, the researchers believe pridopidine ... Read more

Huntington's disease therapeutics conference 2021 – Day 3

Published date: 29 April, 2021

We are back for the last day of the virtual 2021 CHDI Therapeutics conference. This article summarises our live Twitter updates on the exciting science being presented, which you can find with the hashtag #HDTC2021. The final session of the conference will provide the latest news on more Huntington’s disease clinical programs. A new way ... Read more

Huntington's disease therapeutics conference 2021 – Day 2

Published date: 28 April, 2021

We are back with Day 2 of the virtual 2021 CHDI Therapeutics conference. This article summarises our live Twitter updates on the exciting science being presented, which you can continue to follow with the hashtag #HDTC2021. The morning session focussed on promising HD therapeutics that are in preclinical development and the afternoon session covered different ... Read more

Huntington’s disease therapeutics conference 2021 – Day 1

Published date: 28 April, 2021

The CHDI Therapeutics Conference 2021 kicked off today. This article summarizes our live Twitter updates on the exciting science being presented, which you can continue to follow with the hashtag #HDTC2021. The morning of Day 1 focused on clinical trial updates, while the afternoon explored genetic modifiers of HD and how they might be harnessed ... Read more

Huntington’s disease clinical trial round up

Published date: 26 April, 2021

It seems like the HD community has been inundated with updates from different companies and clinical trials recently. The news is far from complete doom and gloom; although there have been some real disappointments from some of the frontrunner trials, there are also positive updates from many different companies and lots of reasons for us ... Read more

Good news from uniQure: gene therapy trial on track, and promising data in animals

Published date: 15 April, 2021

Two recent press releases from uniQure provide welcome good news: the first ever HD gene therapy, known as AMT-130, has been administered via brain surgery to a small set of participants in an early safety trial. At the same time, uniQure has published findings in HD animal models that increase confidence in the drug’s ability ... Read more