Is a therapy for Alexander disease already FDA-approved?

描述（由申请人提供）：亚历山大病是一种罕见的、通常致命的中枢神经系统疾病，主要影响幼儿。 最近的遗传学研究已经确定星形胶质细胞中间丝胶质细胞酸性蛋白（GFAP）的杂合错义突变是这种疾病的主要原因。 为了开始寻找这种毁灭性疾病的潜在治疗方法，研究人员建议筛选一个已经获得FDA批准的药物库，以确定抑制GFAP表达的化合物。 他将评估胶质瘤细胞系（特异性目标1）、星形胶质细胞原代培养物（特异性目标2）和脊髓器官型外植体培养物（特异性目标3）中GFAP表达在蛋白质和基因转录水平上的变化。 在未来的几年中，使用表达与人类患者相同的突变GFAP的小鼠（作为另一个项目的一部分产生），然后可以测试作为该筛选的一部分确定的候选药物。 通过将重点限制在FDA批准的清单上，成功鉴定GFAP抑制药物可以为亚历山大病患者提供立即的治疗益处。

DESCRIPTION (provided by applicant): Alexander disease is a rare and generally fatal disorder of the CNS that primarily affects young children. Recent genetic studies have identified heterozygous missense mutations in the astrocyte intermediate filament glial fibrillary acidic protein (GFAP) as the major cause of this disease. To begin a search for potential treatments of this devastating disease, the investigator proposes to screen a library of drugs that are already FDA-approved to identify compounds that suppress expression of GFAP. He will evaluate changes in GFAP expression at the level of both protein and gene transcription, in glioma cell lines (Specific Aim 1), primary cultures of astrocytes (Specific Aim 2), and organotypic explant cultures of spinal cord (Specific Aim 3). In future years using mice that express the same mutant GFAPs found in human patients (generated as part of another project) can then be tested candidate drugs identified as part of this screen. By restricting the focus to the FDA-approved list, successful identification of GFAP-suppressing drugs could offer immediate therapeutic benefit to Alexander disease patients.