Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Significant news for the Huntington's disease community this week, as the USA's drug regulator, the FDA, has formally approved Austedo, also known as deutetrabenazine, for prescribing in HD. This modified form of tetrabenazine helps control chorea, the jerky movements often found in HD patients, but is taken twice rather than three times a day.
A scientific paper declares positive results for a trial of deutetrabenazine in Huntington's disease, but the headlines report the FDA has rejected the drug. Confusing stuff! The reality is positive overall for this new way of treating uncontrollable movements in HD, but patience will be needed to see where it all ends up.
Auspex Pharmaceuticals just announced the results of two clinical trials known as 'First-HD' and 'Arc-HD'. These trials were designed to test a modified version of the approved Huntington's disease drug tetrabenazine, which reduces unwanted movements. The results reveal that Auspex's drug has some advantages compared to tetrabenazine for treating excessive movements in HD.
A news article reports that a "breakthrough" program of physical, mental, and social stimulation could "halt Huntington's progression". Sounds pretty exciting — but does the science back up the hype?
Here's Buzzilia, video 2: highlights and interviews from the World Congress on Huntington's disease 2013 in Rio de Janeiro. Jeff and Ed discuss biomarkers and talk to Dr Ralf Reilmann about quantitative motor assessment, and Dr Julie Stout about cognitive problems.
A proper balance of electrically charged particles is required for muscle cells to respond appropriately to stimuli. However, a recent report by a group of scientists from California State Polytechnic University concludes that disruption of this balance occurs in Huntington's disease – causing HD mouse muscle fibers to contract more easily than they should.
A new paper in the journal Movement Disorders reports the findings of the HART study of pridopidine, also known as Huntexil - a new drug aimed at improving movements in people with Huntington's disease. Unfortunately this publication doesn't change much - a new, larger trial is still needed before we will know whether Huntexil works.
The results of a two-year study of HD mutation carriers, called TRACK-HD, have just been released. These results prove that a number of changes, including thinking ability and brain changes, occur early in people carrying the HD mutation. Most importantly, these changes are suitable for use as endpoints in future clinical trials for drugs to prevent or delay the onset of HD.
Scientists have successfully used viruses to deliver genes to the brains of Parkinson’s Disease patients. The gene carried by the viruses improved the movement symptoms of patients receiving injections. This proves that gene therapy in the brain can work, providing hope for similar therapies in HD.