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Clinical trials using molecular stratification of pediatric brain tumors
Samuel Z Hanz1, Oluwaseyi Adeuyan1, Grace Lieberman2
1Department of Neurological Surgery, Division of Child Neurology, Weill Cornell Medicine, New York, NY, USA.
Abstract:
Brain cancer is now the leading cause of cancer death in children and adolescents, surpassing leukemia. The heterogeneity and invasiveness of pediatric brain tumors have historically made them difficult to treat. Although surgical intervention and standard of care therapies such as radiation and chemotherapy have improved the outlook for those affected, results are often transient and lend themselves to tumor recurrence or resistance. There also still exists a subset of brain tumors which remain unresponsive to treatment altogether. Therefore, there is great need for new therapeutic approaches. With the recent advent of molecularly-driven technologies, many of these complex tumors can now be classified by integrating molecular profiling data with clinical information such as demographics and outcomes. This new knowledge has allowed for the molecular stratification of pediatric brain tumors into distinct subgroups and the identification of molecular targets, which is changing how these children are treated, namely in the setting of clinical trials. Notable examples include reduced doses of radiation and chemotherapy in the wingless-activated subgroup of medulloblastoma, which has a favorable prognosis, and novel experimental drugs targeting BRAF alterations in low-grade gliomas and dopamine receptors in high-grade gliomas. In this review, we highlight several key previous and ongoing clinical trials that utilize molecular stratifications and targets for the treatment of pediatric brain tumors.
Insights
Pediatric brain cancer treatments are improving with molecular profiling. New targeted therapies and clinical trials offer hope for better outcomes in children with brain tumors.
Area of Science:
- Oncology
- Pediatrics
- Molecular Biology
Background:
- Pediatric brain tumors are the leading cause of cancer death in children, often resistant to conventional therapies.
- Tumor heterogeneity and invasiveness present significant treatment challenges, leading to recurrence and resistance.
- Existing treatments like surgery, radiation, and chemotherapy offer limited long-term success for many pediatric brain cancers.
Purpose of the Study:
- To review how molecularly-driven technologies are transforming pediatric brain tumor treatment.
- To highlight the importance of molecular stratification in identifying new therapeutic targets.
- To discuss current and past clinical trials using molecular targets for pediatric brain tumors.
Main Methods:
- Integrating molecular profiling data with clinical information for tumor classification.
- Identifying distinct molecular subgroups within pediatric brain tumors.
- Analyzing outcomes from clinical trials targeting specific molecular alterations.
Main Results:
- Molecular stratification enables personalized treatment approaches for pediatric brain tumors.
- Targeted therapies show promise, such as BRAF inhibitors for low-grade gliomas and dopamine receptor drugs for high-grade gliomas.
- Specific subgroups, like wingless-activated medulloblastoma, benefit from modified treatment protocols.
Conclusions:
- Molecularly-driven approaches are revolutionizing pediatric brain tumor treatment paradigms.
- Targeted therapies and molecular stratification are crucial for improving outcomes and overcoming treatment resistance.
- Ongoing clinical trials are essential for validating and advancing novel therapeutic strategies.
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