Screening for Recently Defined Genetic Lesions in Poor Risk Adult and Childhood ALL, and Developing Treatment Approaches to Target Causative Pathways.

筛查低危成人和儿童 ALL 中最近确定的遗传病变，并开发针对致病途径的治疗方法。

• 批准号: nhmrc : 1044884
• 负责人: A/Pr Rosemary Sutton
• 金额: $ 49.67万
• 依托单位: The University of Adelaide
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2013
• 资助国家: 澳大利亚
• 起止时间: 2013-01-01 至 2015-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/screening-recently-defined-causative-pathways/111696
• 关键词: Screening; Recently; Defined; Genetic; Lesions

Most adults and 20% of children with ALL relapse and die of their disease. Chromosomal changes resulting in the formation of new proteins have been recently identified in a significant number of cases. Importantly, drugs targeting these proteins are in clinical practice for other diseases. We will develop new tests to rapidly identify these patients at diagnosis, and assess the efficacy of adding these drugs to first line treatment in a clinical trial. The outcome for these patients will likely improve significantly using this approach

大多数成人和20%的儿童ALL复发并死于疾病。最近在大量病例中发现了导致新蛋白质形成的染色体变化。重要的是，靶向这些蛋白质的药物在临床实践中用于其他疾病。我们将开发新的检测方法，以在诊断时快速识别这些患者，并在临床试验中评估将这些药物添加到一线治疗的疗效。使用这种方法，这些患者的结局可能会显著改善