Trls-04. Early insights from pnoc027: personalized treatment approaches based on real-time drug screening and genomic testing in pediatric and adolescent patients with recurrent medulloblastoma

Authors: Margaret Shatara et al. (2024)

Link: https://academic.oup.com/neuro-oncology/article/26/Supplement_4/0/7694385
 

Background Information:

Medulloblastoma is the most common malignant brain tumor in children, and when it comes back (recurs), treatment options are very limited. Researchers have turned to precision medicine—using detailed genetic testing and live lab screening of the patient’s tumor—to help identify effective, personalized therapies that can better target tumor-specific vulnerabilities.

Purpose of the Study:

The PNOC027 trial aimed to explore whether combining genomic testing (to identify tumor-specific gene changes) with real-time laboratory testing of different FDA-approved drugs on patient-derived tumor models could help guide personalized treatment decisions for children with recurrent medulloblastoma.

Methods and Data Analysis:

In this early-phase exploration, tumor samples from children with recurrent medulloblastoma were first genetically analyzed to identify potential targetable mutations. At the same time, living tumor cells were tested in the lab against a panel of FDA-approved cancer drugs. Integrating these two approaches, researchers crafted individualized treatment plans and tracked early responses. Data were summarized as initial "insights" rather than final outcomes, emphasizing feasibility and potential directions.

Key Findings and Conclusions:

Initial results showed that it is possible to successfully perform both genomic profiling and functional drug testing in real time and use them to inform personalized treatment strategies. This hybrid approach appears feasible and may reveal unexpected drug sensitivities—meaning some medications might work better than predicted from genetic data alone.

Applications & Limitations:

This approach could revolutionize care for children with recurring brain tumors by helping doctors select therapies actually effective against each patient's tumor. However, these are early findings: the sample size is small, results are preliminary, and it's too soon to demonstrate whether customized treatments improve long-term outcomes. Larger, controlled clinical trials will be necessary to confirm whether this personalized strategy truly benefits children with recurrent medulloblastoma.