The field has exceptional scientific expertise, experienced clinicians, specialized cancer centers, research networks, pharmaceutical capabilities, regulatory mechanisms, and philanthropic support. The problem is that they are not always connected at the points where they could have the greatest influence on whether a promising program becomes a medicine. Read more: https://www.clinicalleader.com/doc/turning-pediatric-cancer-discoveries-into-medicines-requires-better-connecti

A new, high-quality study reveals that the cardioprotective drug dexrazoxane significantly reduces the heart damage caused by chemotherapy received by pediatric cancer patients 15 years or more after treatment ends. Read more: https://news.ufl.edu/2026/08/chemotherapy-heart-protecting-drug/

Survivors of childhood cancer have a higher risk for antidepressant use and for combining these medications with other psychotropic drugs compared with individually-matched population references, according to findings published in EJC Paediatric Oncology. Read more: https://www.cancertherapyadvisor.com/news/childhood-cancer-survivors-antidepressants-other-psychotropics/

"The biology has been pointing in this direction for a decade. The regulatory architecture is in place. The incentives exist. What happens in the next generation of molecularly targeted oncology programs will depend on whether protocol teams treat that nine-year-old’s NTRK fusion as a scheduling problem for post-approval — or as the enrollment criterion she was always supposed to be." Read more: https://www.clinicaltrialvanguard.com/opinion/pediatric-oncology-keeps-getting-tr

Most anticancer drugs that enter clinical testing in children and adolescents fail to reach late-phase trials or earn pediatric regulatory approval, according to the authors of new research. Read more: https://www.medscape.com/viewarticle/few-pediatric-cancer-drugs-reach-phase-3-or-approval-2026a1000fsh?src=rss&form=fpf

A research team identified three genes connected to a rare form of childhood leukaemia and found that clinically available drugs had a positive influence on these genes in tests on mice, increasing rates of survival. Read more: https://www.innovationnewsnetwork.com/repurposed-drugs-could-improve-treatment-for-rare-infant-leukaemia/68964/

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/

The FDA approved a larger nelarabine vial to improve dosing flexibility and simplify treatment for adult and pediatric patients with T-ALL and T-LBL. Read more: https://www.curetoday.com/view/fda-approves-larger-nelarabine-vial-for-some-leukemias-and-lymphomas

A discovery from Australian researchers could lead to better treatment for children with neuroblastoma, a cancer that currently claims 9 out of 10 young patients who experience recurrence. The team at the Garvan Institute of Medical Research in Sydney, Australia, found a drug combination that can bypass the cellular defenses these tumors develop that lead to relapse. Read more: https://medicalxpress.com/news/2025-11-drug-combination-sidesteps-resistance-aggressive.html

Cancer treatment balances on a razor’s edge. On one side, physicians must use enough of a drug to kill tumor cells effectively. On the other side, a high drug concentration can be harmful to a patient’s normal cells. In children, the difference between the two, called the therapeutic index, is small. Read more: https://www.stjude.org/research/progress/2025/finding-balance-between-efficacy-and-toxicity-in-pediatric-cancer-treatment.html