Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
A new clinical trial just announced for 2015 aims to test a “huntingtin lowering” therapy, called an antisense oligonucleotide (ASO), that attacks mutant huntingtin directly. We’re extremely excited—it’s the first-ever human HD trial to fight HD at the root of the problem, and has shown great promise in animal models. What’s the scoop?
CREST-E, the largest clinical trial of the dietary supplement creatine, has been terminated early because an early analysis of the results to date showed there was no realistic chance it could show positive results. This provides compelling evidence that creatine doesn't slow down progression in Huntington's disease patients.
The largest ever therapeutic trial for Huntington's disease was halted early this week because an analysis of the results to date showed that it was very unlikely to show positive results. The study, called 2CARE, was designed to test whether a treatment called coenzyme Q10 could slow the progression of HD.
The goal of everyone in the HD community is to come up with effective therapies for the disease. A recent publication describes a study in an HD mouse model that comprehensively shows that a proposed therapeutic approach doesn't work. Why are we excited about this bad news?
A chemical called cysteamine has long been of interest as a potential therapy for Huntington's disease. Now, Raptor Pharmaceuticals has announced the interim results of a study of cysteamine in HD patients. The trial failed to meet its pre-specified goal, but there are some interesting details in the data suggesting the ongoing trial deserves attention.
The results are in from the Reach2HD study, which was designed to test the experimental drug PBT2 for early and mid-stage Huntington's disease. The drug seems safe and well-tolerated at the doses that were tested, but we have major concerns about the way the results have been reported.
The results of a new study called PRECREST, investigating whether the nutritional supplement creatine can slow Huntington's disease progression, have just been published. Uniquely, this studied the effects of high-dose creatine supplementation in people carrying the HD mutation, but without clear disease symptoms.
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?
Deep brain stimulation - a procedure in which miniature electrodes that release pulses of electricity are implanted into the brain - sounds like something out of a scary science fiction movie. However, this sci-fi-like treatment may prove useful for reducing symptoms of uncontrollable movement known as chorea in patients with HD.