Weathers Shiao-Pei S, Gilbert Mark R
Department of Neuro-Oncology, University of Texas MD Anderson Cancer Center, 1400 Holcombe Blvd., Unit 431, Houston, TX, 77030, USA.
Neuro-Oncology Branch, National Cancer Institute, National Institutes of Health, Bloch Bldg. 82, Rm. 235, 9030 Old Georgetown Road, Bethesda, MD, 20892, USA.
Neurotherapeutics. 2017 Apr;14(2):256-264. doi: 10.1007/s13311-016-0496-5.
In neuro-oncology, there has been a movement towards personalized medicine, or tailoring treatment to the individual patient. Ideally, tumor and patient evaluations would lead to the selection of the best treatment (based on tumor characterization) and the right dosing schedule (based on patient characterization). The recent advances in the molecular analysis of glioblastoma have created optimism that personalized targeted therapy is within reach. Although our understanding of the molecular complexity of glioblastoma has increased over the years, the path to developing effective targeted therapeutic strategies is wrought with many challenges, as described in this review. These challenges include disease heterogeneity, clinical and genomic patient variability, limited number of effective treatments, clinical trial inefficiency, drug delivery, and clinical trial support and accrual. To confront these challenges, it will be imperative to devise innovative and adaptive clinical trials in order to accelerate our efforts in improving the outcomes for our patients who have been in desperate need.
在神经肿瘤学领域，已出现朝着个性化医疗发展的趋势，即针对个体患者量身定制治疗方案。理想情况下，肿瘤和患者评估应能促成最佳治疗方案的选择（基于肿瘤特征）以及合适的给药方案（基于患者特征）。胶质母细胞瘤分子分析方面的最新进展让人们乐观地认为个性化靶向治疗已触手可及。尽管多年来我们对胶质母细胞瘤分子复杂性的理解有所加深，但如本综述所述，开发有效的靶向治疗策略之路充满诸多挑战。这些挑战包括疾病异质性、临床和基因组层面的患者变异性、有效治疗手段数量有限、临床试验效率低下、药物递送以及临床试验支持和入组情况。为应对这些挑战，必须设计创新型和适应性临床试验，以加快我们改善急需治疗的患者治疗效果的努力。
