A combination of genomic assays could be an effective precision medicine approach for clinical diagnosis, prognosis, and treatment guidance of pediatric brain tumors, according to new research published in Neuro-Oncology.
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Brain tumors are responsible for 25% of all pediatric deaths attributed to cancer. There has been significant progress in recent years in improving survival rates for children with cancer; yet, progress in pediatric brain cancer has been slower. New approaches for treating brain cancer in children has become an urgent priority, especially because many standard-of-care therapies can result in long-term cognitive or physical adverse events.
Researchers led by Pratiti Bandopadhayay, MBBS, PhD, Dana-Farber/Boston’s Children’s Cancer and Blood Disorders Center, turned to genetic testing and precision medicine as a means of progressing treatment for pediatric brain cancer. The study analyzed cancer genomes of 203 pediatric brain tumors across subtypes, including 117 samples analyzed by OncoPanel (a multiplexed targeted exome-sequencing platform that includes 300 cancer-causing genes); 146 by OncoCopy (a clinical genome-wide array comparative genomic hybridization assay); and 60 tumors subjected to both, which allowed researchers to see if combining the two tests was more powerful than performing each alone.
Results of the study showed clinically relevant alterations (44 cancer mutations, 20 rearrangements) in 56% of sampled patients that could impact a patient’s diagnosis or course of treatment. These included BRAF alterations and rearrangements in MYB-QKI, MYBL1, BRAF, and FGFR1. The combination of OncoPanel and OncoCopy in the 60 sampled tumors identified subgroup-specific alterations in 89% of medulloblastomas.
Researchers concluded that combining OncoPanel and OncoCopy genomic assays successfully help identify critical diagnostic, prognostic, and treatment-relevant alterations. The combination also signifies an effective precision medicine approach for further clinical evaluation of pediatric brain tumors.
"The importance of genomic profiling in the diagnosis and treatment of pediatric brain cancers is reflected in the World Health Organization's recent decision to classify such tumors by the genetic alterations within them, rather than by broad tumor type" said study co-author Susan Chi, MD, Dana-Farber/Boston’s Children’s Cancer and Blood Disorders Center. "Targeted therapies are likely to be most effective when they're matched to specific abnormalities within tumor cells. Our findings show that precision medicine for pediatric brain tumors can now be a reality."
