A drug that acts like a growth-promoting protein in the brain reduces degeneration and motor deficits associated with Huntington’s disease in two mouse models of the disorder.

Previous studies of people with Huntington’s disease point to a link between low levels of a neurotrophin called brain-derived neurotrophic factor (BDNF) and symptoms of the disorder.

In the current study, which appeared in the Journal of Neuroscience, Frank Longo, MD, PhD, and others at Stanford University, tested LM22A-4, a drug that specifically binds to and activates the BDNF receptor TrkB on nerve cells, in mice that model the disorder. They found LM22A-4 reduces abnormal protein accumulation, delays nerve cell degeneration, and improves motor skills in the animals.

The findings add to a growing body of evidence that protecting or boosting neurotrophins – the molecules that support the survival and function of nerve cells – may slow the progression of Huntington’s disease and other neurodegenerative disorders.



doi: 10.1523/JNEUROSCI.1310-13.2013

December 2, 2013