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NCI-COG Pediatric MATCH Study Finds More Targetable Genetic Changes than Expected


Major advances in precision medicine are helping transform childhood cancer care by focusing on the molecular and cellular changes related to cancerous tumors.

In 2017, we reported that the NCI-COG Pediatric Molecular Analysis for Therapy Choice (Pediatric MATCH) trial, a first-of-its-kind precision medicine trial in childhood cancer, had opened for enrollment. It was expected that about 10 percent of pediatric patients who had their tumors tested for genetic changes would be eligible for the trial.

However, an early report shows that 24 percent of screened patients were eligible to receive one of the targeted therapies, as presented by COG study chair and former PBTF Research Advisory Network member D. Williams Parsons, M.D., Ph.D., of Baylor College of Medicine at a press briefing ahead of the 2019 annual meeting of American Clinical Society of Clinical Oncology (ASCO).

Developed and led by the National Cancer Institute (NCI) and the Children’s Oncology Group (COG), Pediatric MATCH is exploring the effectiveness of targeted therapies in children and adolescents with brain and other solid tumors. The nationwide trial focuses specifically on pediatric patients whose tumors contain certain genetic mutations and have progressed despite standard treatments.

The trial is currently investigating ten different study drugs, and more will be added as the study continues. Each study drug will target a specific, predefined set of mutations to match patients with therapies based on their tumors’ genetic mutations.

On average, pediatric cancers have fewer genetic changes than adult cancers, making researchers initially concerned about the trial’s feasibility. However, genetic changes targeted by the treatments were identified in 29 percent of the screened group, with 24 percent found to be eligible for treatment.

One possible reason for this unexpected result is that children with cancers that have returned or do not respond to standard therapies may develop more genetic changes than they had at diagnosis. Another possibility is that due to genetic testing of tumor samples in children becoming more common, clinicians enrolling patients on Pediatric MATCH may already know that their patients likely have mutations that make them eligible.

Although the trial is currently enrolling only patients whose cancer has not respond to other treatments or has come back, study investigators hope the trial ultimately will lead to the availability of therapies for pediatric patients with less-advanced disease.

Dr. Joanne Salcido, vice president of research and advocacy at the PBTF, says, “Targeted therapies represent smarter shots on goal against cancer. Just how broadly applicable they will be as treatments for children and adolescents fighting brain tumors is rightly under intense investigation. We are hopeful that findings from Pediatric MATCH will significantly illuminate the field and help to maximize success.”

Learn more about the trial and preliminary results in the National Institutes of Health’s press release.