Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Nearly a thousand HD family members converged on Baltimore, Maryland for the 2016 Huntington’s Disease Society of America’s Annual Convention. We normally don’t write reports from patient and family conferences, but there was something special about the atmosphere of this year’s Convention that compelled us to pen a brief update.
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.
According to the Huntington Study Group’s HD Insights Publication, there are currently thirteen clinical trials recruiting or in progress in Huntington’s 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 we’ve 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 Pfizer’s 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. Teva’s Legato-HD study of laquinimod, for example, is testing a drug that it’s hoped will slow the progression of Huntington’s disease but still needs more volunteers. The same is true of several other trials.
Ed Wild gave an update on the trial of Ionis-HTTRx, the first ‘gene silencing’ or ‘huntingtin-lowering’ drug that’s 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 trial’s 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.
How can HD-impacted people get into a clinical trial? In the US and Canada, the answer is HDSA’s 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.
Clinical trials can be hard to get into because each one has strict criteria for who’s eligible and who’s not. If you find there’s no drug trial near you that you’re able to take part in, don’t lose heart. Here’s our 3-step plan for finding the silver lining.
“We need to recruit all active trials as quickly as possible. ”
Although the HDSA convention isn’t a science meeting, we were surprised by the amount and quality of innovative new projects we heard about there. Our personal highlights were:
In our regular research roundup, we compared Huntington’s disease research to a tree, rather than the ‘pipeline’ you may have heard about before.
The roots of the tree are the global HD community; the trunk is the ‘basic’ 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 Huntington’s 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.