Targeted therapy in pediatric central nervous system tumors: a review from the National Pediatric Cancer Foundation

https://www.frontiersin.org/journals/oncology/articles/10.3389/fonc.2025.1504803/full

Siegel BI, Patil P, Prakash A, Klawinski DM and Hwang EI (2025)

Front. Oncol. 15:1504803. doi: 10.3389/fonc.2025.1504803
 

The article titled "Targeted Therapy in Pediatric Central Nervous System Tumors: A Review from the National Pediatric Cancer Foundation" (DOI: 10.3389/fonc.2025.1504803) provides an in-depth analysis of recent advancements in targeted therapies for pediatric central nervous system (CNS) tumors. These tumors are the leading cause of cancer-related mortality in children, and traditional treatments like surgery, radiation, and chemotherapy often have limited efficacy and significant side effects.

In summary, the review underscores the transformative potential of targeted therapies in pediatric neuro-oncology, highlighting the importance of continued research and personalized approaches to treatment.

Key Points:

* Molecular Pathways and Targeted Therapies:

• The review highlights the significance of the mitogen-activated protein kinase (MAPK) pathway in pediatric low-grade gliomas (pLGGs), noting that over 85% of pLGGs exhibit molecular aberrations of RAF, leading to upregulation of the MAPK pathway.
• Targeted therapies, such as BRAF and MEK inhibitors, have shown promise in treating tumors with specific genetic alterations.

* Clinical Trials Landscape:

• The article provides an overview of completed and ongoing clinical trials focusing on molecular targeted therapies for pediatric CNS tumors, emphasizing the need for continued research and development in this area.

* Challenges and Future Directions:

• While targeted therapies offer hope, the review acknowledges challenges such as drug resistance and the complexity of tumor biology.
• It advocates for combinatorial approaches and real-time drug screening to personalize treatment plans effectively.

Implications for Human Health and Society:

• The advancements discussed in this review have several implications for improving human health:
• Enhanced Treatment Efficacy: By targeting specific molecular pathways, therapies can become more effective against certain tumor types.
• Reduced Side Effects: Targeted therapies aim to minimize damage to healthy tissues, potentially leading to fewer side effects compared to traditional treatments.
• Personalized Medicine: The emphasis on real-time drug screening and understanding individual tumor biology paves the way for personalized treatment plans, increasing the likelihood of successful outcomes.
• Improved Survival Rates: As targeted therapies become more refined, they hold the promise of improving survival rates for children with CNS tumors.