On February 15, International Childhood Cancer Day 2025 highlights the challenges faced by children with cancer and their families.
What started as an awareness campaign has evolved into a global movement advocating for better treatment and care. Established by Childhood Cancer International (CCI) and supported by the International Society of Paediatric Oncology (SIOP), International Childhood Cancer Day emphasizes that childhood cancer is not just a medical issue but a societal one.
Pediatric cancer care has traditionally relied on chemotherapy, radiation and surgery, but emerging therapies are expanding possibilities. Gene therapy has shown promise in treating cancers that are particularly difficult to manage — such as neuroblastoma and Wilms tumor — using CAR-T cell therapy, CRISPR-Cas9 gene editing and RNA-based interventions to target genetic drivers of disease.
While obstacles such as delivery barriers, immune responses and regulatory approvals remain, clinical trials are refining these approaches, bringing hope for more effective and less toxic therapies.
International Childhood Cancer Day 2025 enters the second phase of its three-year campaign, “Equal Access to Care for All Children with Cancer.” The initiative aims to expose challenges (2024), highlight solutions (2025) and showcase impact (2026). It encourages regional efforts to ensure sustainable progress in childhood cancer care.
This year’s theme, “Inspiring Action,” moves beyond raising awareness to focusing on real-world solutions.
XTALKS WEBINAR: Pioneering Progress for Rare Lives: Cell and Gene Therapy Trials in Pediatric Rare Disease Populations
Live and On-Demand: Monday, February 24, 2025, at 10am EST (4pm CET/EU-Central)
Register for this free webinar to gain insights on improving outcomes through early treatment and addressing regulatory requirements for pediatric rare diseases.
The Burden of Childhood Cancer
According to the WHO, an estimated 400,000 children and adolescents aged 0 to 19 develop cancer annually. The most common types include leukemia, brain tumors, lymphomas, neuroblastoma and Wilms tumor.
While over 80% of children survive in high-income countries, survival drops below 30% in low- and middle-income countries due to misdiagnosis, lack of access to care and treatment abandonment.
Another WHO report highlights that childhood cancer is the leading cause of disease-related death in small European nations, where families often travel abroad for treatment due to a lack of specialized pediatric oncology services. Additionally, access to essential medicines and trained specialists remains inconsistent, reinforcing the need for policy improvements and international collaboration.
Beyond physical health, childhood cancer survivors face long-term psychological burdens. A JAMA Pediatrics meta-analysis found that children, adolescents and young adults diagnosed with cancer have higher lifetime risks of depression, anxiety and psychotic disorders compared to non-cancer peers.
Depression worsens in survivors over 30, while anxiety is more pronounced past 25. Older adolescents (15 to 19 years) and those with central nervous system tumors face a higher suicide risk.
Socioeconomic factors like lower education and income increase distress, while strong family support improves outcomes.
XTALKS WEBINAR: Best Practices for Operationalizing Global, Early-Phase Oncology Trials
Live and On-Demand: Wednesday, March 19, 2025, at 11am EDT (4pm CET/EU-Central)
Register for this free webinar to gain actionable strategies, lessons learned and best practices to enhance the success of complex early-phase oncology trials.
Advances in Research and Treatment
Recent efforts by global pharma companies reflect a renewed commitment to childhood cancer.
Sanofi, for instance, has launched a dedicated R&D program to accelerate novel therapies for pediatric cancers. By advancing targeted therapies, expanding clinical trial accessibility and forming partnerships with pediatric oncology research centers, Sanofi aims to expand treatment options and improve survival outcomes.
A significant advancement in treating B-cell acute lymphoblastic leukemia (B-ALL) was achieved through research at Children’s Hospital Colorado. Their study found that adding blinatumomab to standard chemotherapy significantly improves three-year disease-free survival rates.
Similarly, Seattle Children’s Research Institute demonstrated that incorporating blinatumomab into standard regimens for B-ALL reduces relapse rates, a crucial development as relapse remains a leading cause of pediatric cancer mortality.
For pediatric brain tumors, particularly diffuse intrinsic pontine glioma (DIPG), BrainChild Bio has advanced BCB-276, an investigational CAR-T cell therapy targeting the immune checkpoint B7-H3. This engineered CAR T-cell therapy, delivered directly to the brain, has shown promising early clinical results. If successful in upcoming trials, it could become the first approved treatment for DIPG, a currently incurable cancer.
Regulatory Approvals and Expanding Treatment Options
Last year, the FDA’s accelerated approval of tovorafenib marked a milestone for pediatric low-grade glioma, as it is the first systemic therapy targeting BRAF-altered tumors in children. With a high response rate and a manageable safety profile, tovorafenib offers new hope for patients with relapsed or refractory disease.
Meanwhile, MAIA Biotechnology’s investigational therapy THIO, has also received a Rare Pediatric Disease designation for treating high-grade gliomas. By activating the immune system while evading tumor immunosuppression, THIO presents a novel approach to these aggressive cancers.
Further expanding treatment options, Senhwa Biosciences initiated a Phase I/II trial evaluating silmitasertib — an investigational oral small-molecule inhibitor with FDA’s Orphan Drug designation — combined with chemotherapy in children and young adults with relapsed or refractory solid tumors.
The Road Ahead for Childhood Cancer
While research progress is promising, the financial and emotional toll on families remains significant. Travel costs, lost income and out-of-pocket expenses create overwhelming burdens. Ensuring access to financial protection and national support programs can help alleviate these challenges, allowing families to focus on their child’s care rather than financial survival.
At the same time, pharma companies and non-profits continue expanding pediatric oncology research, while global health organizations drive collaborations to improve treatment access. Under the Global Initiative for Childhood Cancer (GICC), the WHO is supporting Sri Lanka by enhancing cancer registries at treatment centers and implementing strategies to achieve a 60% survival rate for children with cancer by 2030.
While efforts are underway, pediatric drug development still lags due to limited clinical trials and funding. Strengthening clinical research, improving regulatory pathways and increasing financial support could be essential to bridge these gaps.
As International Childhood Cancer Day 2025 emphasizes solutions, sustained efforts from policymakers, researchers and industry leaders are vital to ensuring that childhood cancer treatment continues to evolve and reach those in need.
Join or login to leave a comment
JOIN LOGIN