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。

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

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 ...

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 first-ever clinical test of the targeted therapy avapritinib in pediatric ...

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 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 ...

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 ...

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

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