Descriptive "Pharmacogenomics Analysis" of the CBTN PBTA Cohort

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All Brain Tumor Types
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Angela Waanders

Ann & Robert H. Lurie Children’s Hospital of Chicago
Chicago, Illinois, USA



CBTN Participants


Internal funding

About this


Outcomes for pediatric cancer patients have improved over the last few decades due to advancements in treatments, including those for pediatric brain tumors. However, side effects can still have negative effects on patients long after treatment is complete, and some research focus has shifted to combating these effects. One emerging field to address this issue is Pharmacogenomics, the use of genetic information to predict an individual’s response to specific medications. This can help identify patients at increased risk for treatment related toxicities, guiding medical professionals in treatment decisions. An example of the potential impact of research into pharmacogenomics in pediatric cancers is vincristine. Vincristine is a chemotherapy agent that is used in many treatment protocols, including for low-grade gliomas and medulloblastoma. Vincristine is also associated with significant side effects often resulting in dose reductions. Two different studies have identified a genetic variation in the promoter of the CEP72 gene, rs924607, in both adult and pediatric patients with acute lymphoblastic leukemia. This genetic variation is associated with increased rates of vincristine side effects. The role of this variation has not been evaluated in other cancers, such as low-grade gliomas, medulloblastoma, and rhabdomyosarcoma, despite the wide use of vincristine in these treatment regimens. Using the over 900 pediatric tumor genomes sequenced in the Pediatric Brain Tumor Atlas, this study will help to define the frequency of genetic risks for vincristine-related side effects in patients with a spectrum of diagnoses. This research could serve to prevent reductions in therapy due to toxicity, increasing the likelihood of a positive response to treatment.

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What are the goals of this project?

Researchers are looking to identify genetic risk for vincristine-related side effects in pediatric brain cancer patients.

What is the impact of this project?

Many patients experience vincristine related side effects that result in reductions in treatment. Identification of risk factors for these side effects could help guide research in combating such side effects, increasing the likelihood of treatment success.

Why is the CBTN request important to this project?

The dataset provided through the Pediatric Brain Tumor Atlas provides researchers with comprehensive data across many pediatric brain cancer types, greatly increasing the broad impact of this work.

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