CLINICAL TRIAL: PYRIMETHAMINE FOR TREATMENT OF SOD1 MEDIATED AMYLOTROPHIC LATERA

点击翻译按钮获取中文摘要

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Amyotrophic lateral sclerosis is a degenerative disorder of motor neurons that is untreatable and uniformly fatal. Some cases are caused by mutations in the gene encoding cytosolic superoxide dismutase (SOD1). The purpose of this study is to test the hypothesis that the drug pyrimethamine can inhibit the expression of SOD1 in five patients with SOD1-mediated ALS. This study is predicated on the observation that pyrimethamine depresses SOD1 expression in cells in vitro and in a transgenic mouse model of ALS.