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NCI Begins Enrollment in Precision Medicine Trial for Childhood Cancers

NEW YORK (GenomeWeb) – The National Cancer Institute and its Children's Oncology Group (COG) announced today that they have begun enrollment in a Phase II trial that will test a variety of targeted treatments for childhood cancers.

Called Pediatric MATCH — short for Molecular Analysis for Therapy Choice — the study has been in the works since 2015 as the pediatric counterpart to an ongoing precision medicine study in cancer patients 18 years and older. The NCI's plans for Pediatric MATCH were first reported last year.

The trial will initially comprise six treatment arms with one targeted drug being evaluated in each, although the number of arms may be expanded to eight or more as the study progresses. Enrollment will be open to patients aged one to 21 with certain solid tumors — including non-Hodgkin lymphomas, brain tumors, and histiocytoses — that no longer respond to standard treatment or have recurred after treatment.

All patients will initially have their relapsed tumors sequenced using an NCI-developed test to detect genetic abnormalities targeted by one or more of the drugs being studied. Archived tumor samples may be used if they were obtained after the tumor progressed following initial treatment, the NCI noted. Patients whose tumors carry genetic abnormalities may then be enrolled in the appropriate treatment arm.

The NCI said that it expects to find cancer mutations matching at least one of the drugs in the study in about 10 percent of tumors from child and adolescent patients. Therefore, trial investigators anticipate screening a total of 1,000 patients — 200 to 300 a year — in order to find eligible patients for each treatment arm. At least 20 patients will be targeted for enrollment in each arm.

"Pediatric MATCH is a cutting-edge trial in many ways," COG Chairman and Children’s Hospital of Philadelphia researcher Peter Adamson said in a statement. "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."

Treatments being tested in the trial include Loxo Oncology's larotrectinib, which targets tropomyosin receptor kinases; Epizyme's tazemetostat, which targets enhancer of zeste homolog 2; AstraZeneca's selumetinib, which targets mitogen-activated protein kinase kinase; Xcovery's ensartinib, which targets anaplastic lymphoma kinase; Genentech's vemurafenib, which targets BRAF; and AstraZeneca's olaparib, which targets poly ADP ribose polymerase.

All of the drugs are being donated by their developers, and tumor sequencing and treatment will be available to study participants free of charge. Trial enrollees will be able to receive treatment as long as their tumors remain stable in size or get smaller.

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