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Genetic profiling feasible, useful in pediatric brain tumors
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Genomic profiling of brain tumors in children is clinically feasible and useful for guiding treatment, according to study results published in Neuro-Oncology.
New approaches for the treatment of pediatric brain cancer are urgently needed, the researchers said in an accompanying press release.
“Although there has been a great deal of progress over the past 30 years in improving survival rates for children with cancer, advances in pediatric brain cancer haven’t been as dramatic,” Pratiti Bandopadhayay, MBBS, PhD, pediatric neuro-oncologist at Dana-Farber Cancer Institute and Boston Children’s Hospital, said in the press release. “In [another] study, brain tumors accounted for 25 percent of all pediatric deaths attributed to cancer. In addition, many of the [available] therapies can result in long-term difficulties in cognitive or physical functioning.”
Pediatric and adult brain tumors show key differences, particularly in incidence rates and driver events.
“In order to translate these advances into clinical practice, physicians require a means by which to obtain accurate clinically relevant genomic data for these tumors,” the researchers wrote.
The researchers used OncoPanel (n = 117 samples), a multiplexed targeted exome-sequencing platform, and OncoCopy (n = 146), a clinical genome-wide array comparative genomic hybridization assay, or both (n = 60) to identify genetic alterations in 203 pediatric brain tumors. The researchers annotated alterations in four tiers by clinical significance, in which tier 1 indicated well-established clinical utility.
OncoPanel identified clinically significant alterations in 56% of patients — 44 cancer mutations and 20 rearrangements — including BRAF alterations, which direct the use of targeted inhibitors in treatment. Researchers also detected rearrangements in MYB-QKI, MYBL1, BRAF and FGFR1.
Use of both OncoPanel and OncoCopy identified subgroup-specific alterations in 89% of tumors.
“The importance of genomic profiling in the diagnosis and treatment of pediatric brain cancers is reflected in the World Health Organization’s … decision to classify such tumors by the genetic alterations within them, rather than by broad tumor type,” Susan Chi, MD, director of the pediatric brain tumor clinical trials program at Dana-Farber Cancer Institute, said in the press release. “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.” – by Andy Polhamus
Disclosure: The researchers report no relevant financial disclosures.
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