
Planting trees together: The 2016 Huntington's Disease Society of America Convention
HDBuzz summarizes the science from an unusually large and energized @HDSA convention in Baltimore

Nearly a thousand HD family members converged on Baltimore, Maryland for the 2016 Huntingtonâs Disease Society of Americaâs Annual Convention. We normally donât write reports from patient and family conferences, but there was something special about the atmosphere of this yearâs Convention that compelled us to pen a brief update.
The Convention
This was the second-biggest convention in the HDSA’s history, and HDBuzz co-founders Jeff and Ed were there to talk science. You can see a video of our presentation on YouTube and hear the audio in the latest edition of our podcast, HDBuzzCast. Many of the other talks are featured on the HDSA website.
Clinical trials
According to the Huntington Study Groupâs HD Insights Publication, there are currently thirteen clinical trials recruiting or in progress in Huntingtonâs disease, some of which are testing treatments specifically designed with HD in mind, rather than drugs thought to be generally good for brains. This is an incredible time for HD families.

Two trials weâve been watching closely had important updates. The Teva PRIDE-HD study of pridopidine, a new drug to treat movement problems in HD, is fully recruited. The same is true of Pfizerâs Amaryllis study of their PDE-10 inhibitor drug that aims to improve communication between neurons. Delivering efficient trial recruitment is great news and a testament to the ability of the HD community to rise to the challenge.
There is no room for complacency though. We need to recruit all active trials as quickly as possible. Tevaâs Legato-HD study of laquinimod, for example, is testing a drug that itâs hoped will slow the progression of Huntingtonâs disease but still needs more volunteers. The same is true of several other trials.
Good news from the âgene silencingâ trial
Ed Wild gave an update on the trial of Ionis-HTTRx, the first âgene silencingâ or âhuntingtin-loweringâ drug thatâs ever been tested in human HD patients. Speaking on behalf of the sponsor, Ionis Pharmaceuticals, Ed reported that the trial began in September 2015 and that 5 clinical sites are now up and running. In an exclusive HDSA update, he announced that there have been no safety issues with the drug so far, and the trialâs independent safety committee had just approved the start of the third of four dosing levels. This is the best news we could have expected at this stage in the trial, and we eagerly await further updates.
Where do I sign up?
How can HD-impacted people get into a clinical trial? In the US and Canada, the answer is HDSAâs TrialFinder system. You enter a few basic details and within a minute you will see a customized list of what studies you may be eligible for in your area, with links to the research teams.
What if Iâm not eligible?
Clinical trials can be hard to get into because each one has strict criteria for whoâs eligible and whoâs not. If you find thereâs no drug trial near you that youâre able to take part in, donât lose heart. Hereâs our 3-step plan for finding the silver lining.
âWe need to recruit all active trials as quickly as possible.â
- Sign up for Enroll-HD, a platform for understanding HD and a database for recruitment into future clinical trials. Virtually all HD family members are eligible, even people who havenât had a genetic test.
- Be a trial wingman: spread the word about trials and research on Social Media and in person to your friends and family. Can you volunteer to help someone else be in a trial, by accompanying them to appointments or helping them complete trial activities?
- Take part in observational studies â thatâs HD research that doesnât involve testing new treatments. These studies are essential because they help bring about the next generation of treatments and trials.
Innovation
Although the HDSA convention isnât a science meeting, we were surprised by the amount and quality of innovative new projects we heard about there. Our personal highlights were:
- CHDI Foundation linking up with IBMâs Watson supercomputer platform to understand HD, like modeling the huntingtin protein and how itâs affected by the HD mutation
- Hearing directly from Teva Pharmaceuticalsâ Dr David Stamler that they remain committed to getting SD809 licensed for HD. SD809 is a slow-acting form of tetrabenazine for HD movements that the FDA recently requested more information about. Stamler was unequivocal: âThe FDA did not reject the application ⊠they asked for addition information and analyses ⊠There were no safety concerns that were raised by the Agency ⊠Teva is doing everything in its power to bring this medicine to the market as quickly as possible.â
- Azevan Pharmaceuticalsâ STAIR trial, investigating whether their drug SRX246 can help with the common and very challenging symptom of irritability in HD.
- Wave Life Sciences, engaging early with the HD community to talk about their programs to develop new âgene silencingâ drugs to lower production of the mutant huntingtin protein. Waveâs approach aims to take advantage of a quirk in chemistry. Most drugs are actually a mixture of âregularâ and âmirror-imageâ versions of the same drug that have the same basic structure but can behave slightly differently. Wave hopes that by eliminating the mirror-image versions, their silencing drugs will be more powerful. If this works itâll be pretty cool.
Planting trees
In our regular research roundup, we compared Huntingtonâs disease research to a tree, rather than the âpipelineâ you may have heard about before.
The roots of the tree are the global HD community; the trunk is the âbasicâ laboratory research that happens every day and helps to keep new drugs coming; the branches are the observational research involving human volunteers that helps us understand HD and develop new drugs; and the leaves are the clinical treatment trials that let us test those drugs.

We like this image because all parts of the tree are interconnected and depend on each other to produce the fruit we all need: effective treatments for Huntingtonâs disease. Everyone in the HD community has a responsibility to look after each bit of the tree if we want those treatments as soon as possible.
Our presentation ended with a Chinese proverb:
The best time to plant a tree is twenty years ago. The second best time is today.
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