Blocking energy production pathways in an aggressive type of brain cancer in children could be a promising new therapeutic strategy, according to research in mice by Johns Hopkins Kimmel Cancer Center investigators. Read more: https://www.hopkinsmedicine.org/news/newsroom/news-releases/2026/04/blocking-energy-metabolism-may-help-treat-aggressive-pediatric-brain-tumor

Diagnosed with DIPG at age six, Lucas’ doctor Jacques Grill told Lucas’ parents, Cedric and Olesja, that he was unlikely to live very long. Instead of giving up hope, Cedric and Olesja flew Lucas to France to participate in a clinical trial called BIOMEDE, which tested new potential drugs against DIPG. Read more: https://www.upworthy.com/first-person-cured-of-dipg-ex1/

Now, results obtained by a study led by the University of Trento in collaboration with Bambino Gesù Children's Hospital in Rome may pave a path to an organoid that could be used to study two common, aggressive childhood brain cancers. Read more: https://medicalxpress.com/news/2026-03-avatar-pediatric-brain-cancer-reality.html

An international team led by researchers at Baylor College of Medicine, Texas Children’s Hospital, McGill University and University of Pittsburgh School of Medicine has discovered what drives the growth of a lethal pediatric brain tumor called Posterior Fossa Type A (PFA) ependymoma. Read more: https://www.eurekalert.org/news-releases/1121004

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