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Small-molecule screen reveals synergy of cell cycle checkpoint kinase inhibitors with DNA-damaging chemotherapies in medulloblastomaMedulloblastoma (MB) consists of four core molecular subgroups with distinct clinical features and prognoses. Treatment consists of surgery, followed by radiotherapy and cytotoxic chemotherapy. Despite this intensive approach, outcome remains dismal for patients with certain subtypes of MB, namely, MYC-amplified Group 3 and TP53-mutated SHH. Using high-throughput assays, six human MB cell lines were screened against a library of 3208 unique compounds. We identified 45 effective compounds from the screen and found that cell cycle checkpoint kinase (CHK1/2) inhibition synergistically enhanced the cytotoxic activity of clinically used chemotherapeutics cyclophosphamide, cisplatin, and gemcitabine.
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Efficacy of acute myeloid leukemia therapy without stem-cell transplantation in a child with blastic plasmacytoid dendritic cell neoplasmOur case demonstrates that AML therapy, without HSCT, can be sufficient to treat this rare disease in children.
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Identification of suitable endogenous control genes for microRNA expression profiling of childhood medulloblastoma and human neural stem cellsMedulloblastoma (MB) is the most common type of malignant childhood brain tumour.
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Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene FusionsTropomyosin receptor kinase (TRK) fusions are detected in less than 2% of central nervous system tumors. There are limited data on the clinical course of affected patients.
We strive for a future where no child will die from brain cancer because we have developed new therapies that will cure their disease.