The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMXT 1501 to treat malignant gliomas , including a particularly aggressive childhood brain tumor called diffuse intrinsic pontine glioma (DIPG). Read more: https://rarecancernews.com/news/fda-grants-amxt-1501-orphan-drug-status-malignant-gliomas/

Immunotherapy has improved how doctors treat cancers all over the body, but so far it hasn’t been able to help children and young people with brain tumours. A discovery from researchers at the Cancer Research UK Cambridge Institute could finally change that. Read more: https://news.cancerresearchuk.org/2026/03/18/scientists-find-a-way-to-use-immunotherapy-to-treat-brain-tumours-in-children/

New research published in Nature finds that tumor cells within supratentorial ependymomas (SE)—an aggressive childhood brain cancer—cluster into distinct tumor cell populations. Read more: https://medicalxpress.com/news/2026-03-distinct-tumor-neighborhoods-treatments-aggressive.html

Researchers at The Institute of Cancer Research, London, led work to develop a large panel of patient-derived mouse models of paediatric-type diffuse high-grade glioma (PDHGG) – a group of brain tumours with poor survival rates. Using magnetic resonance imaging (MRI), the research team demonstrated that these models reproduce key biological and radiological features of the human disease. Read more: https://www.icr.ac.uk/about-us/icr-news/detail/advanced-imaging-reveals-how-ch

Research uncovering the origin of pineoblastoma, a rare pediatric brain tumor, has also revealed a dependency across multiple brain tumor types that share a similar molecular program. Read more: https://www.technologynetworks.com/tn/news/three-childhood-cancers-have-a-shared-dependence-on-light-sensing-genes-410398

Research from St. Jude led by Heather Conklin, PhD, Department of Psychology & Biobehavioral Sciences, demonstrated that radiation therapy was a significant individual contributor to hearing loss, a risk directly linked to cognitive health, in children treated for ependymoma. Read more: https://www.eurekalert.org/news-releases/1117443

Researchers have uncovered the mechanisms behind three unique subtypes of mismatch repair deficient high-grade gliomas. The findings provide a clearer understanding of how these tumors develop, explain why patients respond differently to immunotherapy, and are already helping guide more precise therapies. Read more: https://medicalxpress.com/news/2026-02-discovery-unique-brain-tumor-subtypes.html

In a study published in Nature , University of Michigan researchers have found that a specific molecule called itaconate drives the development of ependymomas. Their findings can help future drug development for children with this type of malignant brain tumor. Read more: https://www.michiganmedicine.org/health-lab/itaconate-identified-new-target-pediatric-brain-tumors-called-ependymomas

A world-first trial of a personalised vaccine for children with deadly brain cancers will be rolled out in eight hospitals throughout Australia later this year.  Read more: https://www.abc.net.au/news/2026-02-03/brain-cancer-vaccine-trial-for-children/106294532

A new research collaboration aims to transform the treatment of children with brain tumors by identifying hidden genetic drivers that current diagnostic technologies often miss. Read more: https://jagwire.augusta.edu/revolutionary-genome-mapping-tech-targets-childhood-brain-cancers/