Johung Tessa B, Monje Michelle
Departments of Neurology, Pediatrics, Pathology, and Neurosurgery, Stanford University School of Medicine, 265 Campus Drive, Room G3077, Stanford, CA 94305, USA.
Curr Neuropharmacol. 2017;15(1):88-97. doi: 10.2174/1570159x14666160509123229.
Diffuse Intrinsic Pontine Glioma (DIPG) is the leading cause of brain tumor-related death in children, with median survival of less than one year. Despite decades of clinical trials, there has been no improvement in prognosis since the introduction of radiotherapy over thirty years ago.
To review the clinical features and current treatment challenges of DIPG, and discuss emerging insights into the unique genomic and epigenomic mechanisms driving DIPG pathogenesis that present new opportunities for the identification of therapeutic targets.
In recent years, an increased availability of biopsy and rapid autopsy tissue samples for preclinical investigation has combined with the advent of new genomic and epigenomic profiling tools to yield remarkable advancements in our understanding of DIPG disease mechanisms. As well, a deeper understanding of the developmental context of DIPG is shedding light on therapeutic targets in the microenvironment of the childhood brain.
弥漫性脑桥内生型胶质瘤（DIPG）是儿童脑肿瘤相关死亡的主要原因，中位生存期不到一年。尽管进行了数十年的临床试验，但自三十多年前引入放射治疗以来，预后并无改善。
回顾DIPG的临床特征和当前的治疗挑战，并讨论对驱动DIPG发病机制的独特基因组和表观基因组机制的新见解，这些机制为确定治疗靶点带来了新机会。
近年来，用于临床前研究的活检和快速尸检组织样本的可用性增加，再加上新的基因组和表观基因组分析工具的出现，使我们对DIPG疾病机制的理解有了显著进展。此外，对DIPG发育背景的更深入理解正在揭示儿童脑微环境中的治疗靶点。
