In Huntington's disease (HD), a neurodegenerative disorder, abnormal nerve cell (neuron) death occurs. French researchers at the Institut Curie, Centre National de la Recherche Scientifique (CNRS), and Inserm have shown in mice with HD that the drug cysteamine prevents the death of neurons. Cysteamine, used to treat cystinosis, raises the level of brain-derived neurotrophic factor (BDNF), which is subnormal in individuals with Huntington's disease. BDNF also blocks the effect of mutant huntingtin protein found in the disorder. A multicenter trial to test the effects of cysteamine in about 100 individuals with Huntington's disease is scheduled to begin by the end of 2006. The research was published in the May 2006 issue of Journal of Clinical Investigation.
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