International Childhood Cancer Day 2026: Advances in Pediatric Oncology Research and Care

February 15 is recognized worldwide as International Childhood Cancer Day (ICCD). This day aims to raise awareness of cancers affecting children and adolescents, and to highlight ongoing gaps in diagnosis, treatment and survival worldwide.

Childhood cancer encompasses a diverse group of malignancies, including leukemias, brain and other central nervous system tumors, lymphomas and solid tumors such as neuroblastoma, Wilms tumor and bone cancers. These cancers often progress quickly and require specialized, age-appropriate care. Treatment approaches for children are usually different from those used for adults.

The theme for ICCD 2026, “Demonstrating Impact: From Challenge to Change,” highlights a focus on measurable progress. This includes regulatory approvals, late-stage clinical trials and policy initiatives that aim to improve access to care.

On International Childhood Cancer Day 2026, let’s take a look at several recent developments in childhood cancer research and the policy shifts surrounding it.

Progress in Childhood Cancer Treatment

Dordaviprone for Diffuse Midline Glioma Enters Confirmatory Testing

In August 2025, the FDA granted accelerated approval to Jazz Pharma’s Modeyso (dordaviprone) for patients with H3 K27M-mutant diffuse midline glioma, a rare and aggressive pediatric brain tumor. The decision was based on data from early-phase clinical studies using surrogate endpoints considered reasonably likely to predict clinical benefit. Confirmatory Phase III trials are required to further evaluate outcomes such as overall survival and durability of response.

Selumetinib Expands Approved Use to Younger Children With NF1-Associated Tumors

In September 2025, the FDA approved AstraZeneca’s Koselugo (selumetinib) for pediatric patients aged 1 year and older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas. The approval followed results from Phase II clinical studies in children, extending previous approvals in older pediatric populations. The expanded labeling allows younger children access to a targeted therapy intended to help manage pain, disfigurement and functional impairment associated with tumor growth.

Revumenib Gains Pediatric-Inclusive Approval in Relapsed or Refractory AML

In October 2025, the FDA approved Syndax Pharma’s Revuforj (revumenib), a menin inhibitor, for relapsed or refractory acute myeloid leukemia (AML) with a susceptible NPM1 mutation in patients aged 1 year and older who have no satisfactory treatment options. The approval was based on findings from Phase II clinical trials evaluating responses in patients whose leukemia carries this specific genetic change. Additional studies are ongoing to better understand long-term outcomes and evaluate use in other treatment settings.

CAR T-Cell Therapy Continues to Advance in Pediatric Brain Tumors

In 2025, researchers at Stanford Medicine reported results from a Phase I clinical trial evaluating a GD2-targeted CAR T-cell therapy in children and young adults with diffuse midline glioma, including diffuse intrinsic pontine glioma (DIPG). GD2 is being studied as a target because it is expressed on several aggressive pediatric tumors. The therapy received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, and while the approach remains investigational, expanded Phase I/II studies are planned to further evaluate safety and early signs of clinical activity.

Efforts Underway to Expand Pediatric Trial Access

While scientific and regulatory advances in childhood cancer continue to emerge, access to clinical trials and care does not always move in parallel.

In late 2025, pediatric oncology programs in Canada reported pauses in enrollment for several US-led clinical trials following changes in US funding and policy affecting support for non-US trial sites. Clinicians noted that for children with rare or treatment-refractory cancers — particularly certain brain tumors — clinical trials may represent the only remaining option, making even temporary interruptions consequential.

This uneven access reflects broader structural gaps. A recent WHO analysis of global cancer trials found that only about 3% explicitly enroll children, showing longstanding challenges in pediatric trial inclusion and access.

At the same time, however, efforts to expand access have not halted. In Canada, Kingston Health Sciences Centre announced steps to broaden clinical trial access for children with cancer, including participation in additional pediatric oncology studies aimed at reducing the need for families to travel long distances for experimental care.

In the US, access efforts are also taking shape at the state level. In November 2025, Johns Hopkins All Children’s Hospital received a $7.5 million renewable grant (2025-2026) through Florida’s Cancer Connect Collaborative Research Incubator program. The initiative will support pediatric cancer research across four specialty children’s hospitals and focuses on expanding clinical trial access, building statewide trial-matching infrastructure and accelerating the translation of research into care.

In recent global initiatives, the WHO also partnered with the St. Jude Children’s Research Hospital to continue to improve the availability of essential childhood cancer medicines, particularly in low- and middle-income countries.

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