Precision Medicine Clinical Trial for Pediatric Cancer Underway


New clinical trial tests experimental drugs in pediatric and adolescent patients.

The National Cancer Institute (NCI) and the Children’s Oncology Group (COG) recently started enrolling pediatric patients in a precision medicine clinical trial, according to a press release.

The Pediatric Molecular Analysis for Therapy Choice (Pediatric MATCH) trial aims to determine whether targeted treatments can be effective for children and young adults with solid tumors that have specific genetic mutations that have progressed on or after standard treatment.

The phase 2 clinical trial is expected to explore more than 8 different targeted drugs to provide patients with an approach that is tailored to their certain mutations, according to the release.

Currently, a similar trial is exploring targeted treatments for adults. Pediatric MATCH includes patients aged 1 to 21 who have solid tumors that do not respond to, or have recurrence after, standard therapy.

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

Initially, patients will enroll for a screening study in which a tumor sample will undergo DNA and RNA sequencing to detect mutations that can be targeted by the drugs being explored. If there is a mutation identified that corresponds with a targeted treatment, the patient may then enroll in the study, according to the release.

The clinical trial will use 1 sequencing test to screen for molecular abnormalities, which is also being used for the adult trial. The updated version of the test searches for alterations in more than 160 genes that have been linked to cancer.

The investigators report that mutations that match the drugs being studied are only expected to be found in 10% of children and adolescents with cancer. They estimate that they will be screening up to 300 patients per year, with 1000 being screened overall, according to the release. They expect to enroll at least 20 patients per treatment arm.

Patients included in the study will be able to continuously receive the treatment if their tumors remain the same size or shrink.

“Pediatric MATCH is a cutting-edge trial in many ways,” said Peter C. Adamson, MD, COG chair. “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 drugs being investigated are all experimental and contributed by pharmaceutical manufacturers, according to the release.

“This trial would not have been possible without the enthusiastic support of the partnering pharmaceutical companies, as evidenced by their willingness to provide targeted agents for this trial,” said study co-chair Nita Seibel, MD, at NCI’s Division of Cancer Treatment and Diagnosis. “Some of the agents included have not previously been tested in children, so this trial will provide broader access to targeted agents for children and adolescents.”

While a majority of DNA mutations in the study will likely only be presented in the cancerous cells themselves, the authors state that some might have been inherited, according to the release.

“We will also look at whether mutations found in tumors are detected in blood samples and hence were inherited,” said COG study co-chair Will Parsons, MD, PhD. “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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