July 24, 2017
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NCI, Children’s Oncology Group collaborate on trial to evaluate targeted drugs for pediatric cancers

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Peter C. Adamson
Douglas R. Lowy

NCI and the Children’s Oncology Group today announced enrollment opened for a phase 2 study designed to evaluate targeted therapies in children and adolescents with solid tumors.

The Pediatric Molecular Analysis for Therapy Choice (Pediatric MATCH) trial — similar to the NCI-MATCH trial being conducted in adults — will evaluate investigational drugs that target a predefined set of genetic mutations.

“Pediatric MATCH is a cutting-edge trial in many ways,” Peter C. Adamson, MD, Children’s Oncology Group chair and professor of pediatrics at Children’s Hospital of Philadelphia, said in an NIH-issued press release. “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 trial, focused on patients aged 1 to 21 years with cancers that progressed during or after standard therapy, is intended to open with approximately six study arms and expand to eight or more.

Each patient will undergo DNA and RNA sequencing through a test developed by the Molecular Characterization Laboratory at the NCI-sponsored Frederick National Laboratory for Cancer Research in Maryland.

Sequencing will detect genetic abnormalities that could be targeted by one or more of the study drugs. If an eligible patient is found to have a genetic abnormality and a drug under investigation in the trial targets that abnormality, the patient may enroll in the corresponding treatment arm.

Approximately 200 to 300 patients will be screened per year. Target enrollment is at least 20 patients in each study arm, and additional treatment arms will be added as the trial progresses.

“Pediatric MATCH is a very special trial,” Douglas R. Lowy, MD, acting director of NCI, said in the 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.”

The trial also will investigate inherited genetic abnormalities in this patient population.

“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, MD, PhD, associate professor of pediatrics at Baylor College of Medicine, said in the release. “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.”