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NIH Launches New Childhood Cancer Genomics Program

NEW YORK – The National Cancer Institute has launched a new pediatric genomics effort called the Molecular Characterization Initiative. The program will provide tumor molecular characterization to children, adolescents, and young adults with newly diagnosed central nervous system tumors who are being treated at hospitals that are affiliated with the NCI-sponsored Children's Oncology Group (COG), a web of treatment centers that includes more than 200 US hospitals and other institutions.

Announcing the new program — which falls under the NCI's existing Childhood Cancer Data Initiative, launched in 2019 — the National Institutes of Health said that it is intended to support President Joe Biden's Cancer Moonshot goal of fostering data sharing in cancer research.

Enrollment is initially being offered through participation in Project:EveryChild, a childhood cancer registry maintained by COG. Tumor and blood samples from participants will be sent to an accredited lab for analysis, and the results will be available to patients and their families within 21 days. While the initiative focuses at first on CNS tumors, it will expand later in 2022 to include soft tissue sarcomas and other rare tumors.

The NIH said testing will include both DNA and RNA from tumor and blood samples but did not detail which lab will be providing testing, nor what genomic profiling platforms will be employed.

Program leaders said that while comprehensive tumor molecular characterization has been available for some children in the context of specific clinical trials, and for others being treated at larger institutions with state-of-the-art diagnostics, it hasn't yet been implemented on a comprehensive nationwide level.

In addition, existing local programs usually store data on tumor biomarkers at the hospital or institution where a child was treated, with limited sharing between and amongst institutions.

"The ultimate dream has really been for every child with cancer to have a state-of-the-art diagnosis and the safest and most effective therapy," Brigitte Widemann, special adviser to the NCI director for childhood cancer, said in a statement.

"We're going to understand the patient's disease precisely and comprehensively in a way that we've done [only] piecemeal so far," added Douglas Hawkins, group chair of COG.

The hope is that therapy targets uncovered by mutational profiling will help doctors improve patient outcomes by providing opportunities for more effective and/or less toxic treatments.

Maryam Fouladi, COG's central nervous system tumor disease committee leader, offered the example of gliomas, which she said can be misdiagnosed. Using molecular diagnostics, clinicians may find that a child diagnosed with a high-grade glioma actually has a low-grade glioma or an entirely different tumor, "which may need very different treatments and have a very different outcome," she said.

In addition to aiding precise diagnosis, genomic profiling can also help determine whether a child is eligible for a molecularly defined clinical trial.

Finally, data on the molecular changes seen across childhood cancers is also expected to help researchers better understand molecular causes of disease and accelerate the development of new therapies.

Details on the project's funding and longer-term timeline were not disclosed.