Tumor tissue will undergo testing for changes in more than 160 genes. If a patient's tumor has a genetic change that matches one targeted by a drug used in the trial, the patient may be eligible to join the treatment arm targeting that genetic change.

FREDERICK, Md. -- The National Cancer Institute–Children’s Oncology Group trial known as Pediatric MATCH (Molecular Analysis for Therapy Choice) trial launched in 2017, with significant operations and technical support from the Frederick National Laboratory for Cancer Research. Focusing on patients aged 1–21, Pediatric MATCH seeks to determine whether precision medicines that are designed to treat specific cancers with certain mutations can also treat other cancers with the same mutations.

The scientists conducting the study originally estimated that 10 percent of children, adolescent, and young adult cancer patients who enrolled in the trial would test positive for the sought-after genetic mutations and be able to receive treatment. In reality, the number has been more than double that—24 percent.

Donald William “Will” Parsons, M.D., Ph.D., Children’s Oncology Group trial chairperson and associate professor at Baylor College of Medicine, presented the data at the recent American Society of Clinical Oncology meeting.

By the numbers: data collected 7/24/17- 12/31/18, total pediatric patients enrolled (ages 1-21): 422, patients who submitted a tumor sample: 390, patients whose samples were sequenced: (370, 95% of 390), patients with a targetable mutation: 112 (29%), patients who were assigned to treatment: 95 (24%).In total, 390 pediatric cancer patients submitted tumor samples for DNA and RNA sequencing to detect mutations targeted by one of the 10 precision medicines currently used in the trial. Of those patients, 112 were matches, and 95 were subsequently assigned to a treatment.

In addition, patients who “passed” the sequencing screen—in other words, those who had one or more targetable mutations—represented a wide variety of common and uncommon cancer types. If such mutations exist in more cancers than previously thought, precision medicine’s potential applicability in pediatric cancer patients could be significantly expanded.

Frederick National Laboratory’s Role

The Molecular Characterization Laboratory (MoCha) and the Biomedical Applications Development Center at the Frederick National Laboratory helped launch and continue to support the trial.

MoCha led the creation of a laboratory network that developed, harmonized, and validated the sequencing assay used to screen patients’ tumor samples. Its staff is now sequencing those samples in partnership with another network member, the University of Texas MD Anderson Cancer Center. Dartmouth-Hitchcock Medical Center, also a network member, is helping with the trial.

The Biomedical Applications Development Center built and now maintains an intricate software platform, Pediatric MATCHBox, that collects the sequencing data from the tumor samples, analyzes it, and assigns the patients to a precision medicine that can target their cancer. 

Some Biomedical Applications Development Center employees also help to enforce the trial’s protocols in partnership with the scientists and doctors working on the trial.

“[I want to give] my personal thanks to my engineers, especially Shan Yang for her work on the informatics, and to David Sims for his bioinformatics support of my team,” said Brent D. Coffey, the center’s director.

The study’s team hopes results will begin to be available in a few years.

For more information, please see the presentation abstract and the announcement in the ASCO Post.

By Samuel Lopez, staff writer; lead image adapted from original by the National Cancer Institute