由Dana-Farber癌症研究所的内科科学家领导的一个国际临床合作者团队，首次对患有高级胶质瘤的儿科和年轻患者进行了靶向治疗avapritinib的临床试验。他们发现，这种已经获得fda批准用于治疗某些成人癌症的药物，总体上是安全的，在脑部扫描中，7名患者中有3名的肿瘤缩小了，临床效果也得到了改善。

文章中说，血小板源性生长因子受体α（PDGFRA）对肿瘤的发生至关重要，在高级别胶质瘤（HGG）中经常发生基因组改变。在包含 261 例儿童高级别胶质瘤的综合数据集中，研究者检测到 15% 的病例存在 PDGFRA 突变和/或扩增，这表明 ...

High-grade glioma, an aggressive form of pediatric and adult brain cancer, is challenging to treat given the tumor location, ...

本文研究发现 Avapritinib 对 PDGFRA 改变的儿童高级别胶质瘤（HGG）有治疗潜力，值得关注。 ### 儿童高级别胶质瘤治疗现状与挑战 儿童高级别胶质瘤（pediatric high-grade gliomas，pHGGs），如 H3K27M 弥漫性中线胶质瘤（diffuse midline gliomas，DMGs），是一类致命的疾病 ...

An international team of clinical collaborators, led by physician scientists from Dana-Farber Cancer Institute, performed a ...

An international team of clinical collaborators, led by physician scientists from Dana-Farber Cancer Institute, performed a ...

Though this work is preliminary, Koschmann is hopeful that avapritinib could be an additional tool to help patients. "We know a single drug is not going to be enough for this disease," he said.

A new therapy offers hope for treating pediatric brain tumors, targeting cancer cells while minimizing side effects for young ...

High-grade glioma, a difficult-to-treat and aggressive form of pediatric and adult brain cancer, responded to FDA-approved avapritinib. | Drug Discovery And Development ...

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A study, published in Cancer Cell, shows that high-grade glioma tumor cells harboring DNA alterations in the gene PDGFRA responded to the drug avapritinib, which is already approved by the United ...