Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Thinking problems in Huntington’s disease take a huge toll from early in the disease. Now, new work suggests that a drug already approved by the FDA to treat another brain disease – multiple sclerosis – may stave off these problems in HD mice. Could these results be real, or are they too good to be true?
The growth factor 'BDNF' usually sends a “Survive!” signal to brain cells. In Huntington’s Disease (HD), this system doesn’t work as it should, so scientists have been looking for ways to boost the signal. Enter one of nature’s most useful tools: the antibody. Usually antibodies play an important role in the immune system, but researchers have identified two antibodies, produced by the company Pfizer, that can act like a set of spare keys to activate the TrkB receptor. This unlocks the door to determining whether a boost in TrkB activity is enough to prevent neurons from dying, in hopes of slowing the progression of HD.
Cells in the brain depend on support from one another to stay alive. Nutrients called trophic factors act like brain fertilizer, keeping neighboring brain cells healthy. This process has long been thought to go wrong in HD, and exciting new mouse research paints a very clear picture of exactly what's happening.
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.
A recent headline from the Society for Neuroscience (SfN) describes work by Stanford researchers with a drug that “reduces brain changes and motor deficits associated with Huntington's disease." The drug works by mimicking a chemical that acts as brain fertilizer called "BDNF", which has an important job keeping brain cells healthy. While this new drug did improve some symptoms of HD in a mouse, it is still a long way from the clinic. What exactly was found?
Our third and final daily report from CHDI’s annual HD therapies meeting in Palm Springs, covering chemicals that can help neurons to survive, and insights into CHDI’s most advanced experimental drug programs.