Government Begins Study to Test Targeted Therapies in Childhood Cancer

James Radke

The National Cancer Institute (NCI) and the Children’s Oncology Group (COG) are working together to study targeted therapies in pediatric cancers.
The project, called the Pediatric Molecular Analysis for Therapy Choice (Pediatric MATCH), is enrolling children and adolescents with solid tumors who have progressed either during or after standard therapy.
Each patient will be matched, based on their own genetic mutations to 1 of 8 different drugs, each one targeting a predefined set of genetic mutations.
According to, the pediatric solid tumors being considered include:
The study is underway with 6 treatment arms using drugs provided by the pharmaceutical industry, and will eventually expand to 8 or more drugs. Seven of the drugs are currently listed on They are:
Patients in the trial will first undergo a screening procedure where a tumor sample will be sequenced to detect genetic abnormalities. If 1 of the experimental drugs can target a discovered abnormality, the patient will be enrolled in that arm of the study. For example, patients with an ALK or ROS1 gene alteration would receive ensartinib, whereas patients with an EZH2, SMARCB1, or SMARCA4 gene mutation receive tazemetostat.
Researchers are hoping to screen 200 t0 300 patients per year for a total of 1000 patients screened. It is expected that approximately 10% of screened patients will have genetic mutations that can be grouped with 1 of the targeted therapies being studied.
Pediatric MATCH is a very special trial,” said acting director of the NCI Douglas R. Lowy, M.D. in a news release. “There aren’t any other cancer trials of this scale exploring targeted treatments for children whose cancers have specific genetic abnormalities. Precision medicine trials like Pediatric MATCH have the potential to accelerate progress in identifying more effective treatments for children with cancer.”
“Pediatric MATCH is a cutting-edge trial in many ways,” said COG chair Peter C. Adamson, M.D., of the Children’s Hospital of Philadelphia. “It will bring molecular analysis, coupled to a portfolio of new targeted agents, to children and adolescents with relapsed cancer across the United States. Importantly, it will also help us learn more about relapsed cancer in pediatric patients, catalyzing research aimed at developing better treatments.”
The study will also examine the possible role that inheritance plays in pediatric cancers.
“We will also look at whether mutations found in tumors are detected in blood samples and hence were inherited.” COG Study co-chair Will Parsons, M.D., Ph.D., of Baylor College of Medicine in Houston, Texas said. “This will allow us to provide the treating physician with guidance for the patient’s family regarding the need for formal genetic testing, counseling, and follow-up care.”
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