PEDT-19 RECENT PROGRESS IN CHILDHOOD BRAIN TUMORS RESEARCH

The multidisciplinary standard of care for medulloblastoma, a typical pediatric malignant brain tumor of cerebellar primary origin, has been established by large RCTs in the United States. The results of the Phase III RCTs sponsored by Children's Oncology Group's for newly-diagnosed medulloblastoma in children 3 years of age and older, ACNS0331, which attempted radiotherapy reduction for the average-risk group, and ACNS0332, which attempted intensified treatment with carboplatin as a radiosensitizer and isotretinoin as an apoptosis inducer for high-risk group are discussed.It has recently been reported that some infantile glioma cells have activating fusion genes with tyrosine kinase receptor genes, including NTRK, and that TRK inhibitors are effective against these tumors, and two TRK inhibitors have been used with cancer genome panel tests as companion diagnostics, Two TRK inhibitors can now be available for glioma treatment in Japanese practice. The results of clinical trials on the efficacy of these targeting agents in brain tumors with NTRK mutations will be presented.CAR-T cell therapy was developed for GD2 following HER2, which had attracted attention as an immunotherapeutic target for pediatric glioma, and its efficacy was demonstrated in clinical trials. We will examine the results of clinical trials of this immunotherapy, a promising treatment for DMS/DIPG, the disease with absolutely poor prognosis with no effective drug therapy, although it is used in a small number of cases.