In support of President Biden’s Cancer Moonshot goal of fostering data sharing in cancer research, the National Cancer Institute (NCI), which is part of the National Institutes of Health, has launched the Molecular Characterization Initiative for pediatric tumors. This program offers tumor molecular characterization (also known as biomarker testing) to children, adolescents, and young adults with newly diagnosed central nervous system tumors who are being treated at hospitals that are affiliated with the Children’s Oncology Group (COG), an NCI-supported clinical trials group that includes more than 200 hospitals and institutions that treat most children diagnosed with cancer in the United States.
The Molecular Characterization Initiative is offered through NCI’s Childhood Cancer Data Initiative, which was launched in 2019 to promote data sharing and collection of new data among researchers who study childhood cancers.
Molecular Characterization Initiative
Children, adolescents, and young adults diagnosed with a central nervous system cancer across the United States will be eligible to receive molecular characterization of their tumors free of charge through this voluntary program. DNA and RNA from tumor and blood samples will be analyzed to help make an accurate diagnosis and to understand what is causing or driving the cancer.
“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,” said Brigitte C.Widemann, MD, Special Advisor to the NCI Director for Childhood Cancer.
Brigitte C. Widemann, MD
Having a precise diagnosis based on the molecular characteristics of a patient’s tumor can help clinicians choose the most effective and potentially least toxic treatment for each child. Data on the molecular changes seen across childhood cancers can also help researchers better understand the molecular causes of childhood cancers and accelerate the development of new, more effective, and potentially fewer toxic treatments, especially for rare childhood cancers for which treatment options are limited.
“The game-changer for patients is that we’re going to understand the patient’s disease precisely and comprehensively in a way that we’ve done piecemeal so far,” said Douglas S. Hawkins, MD, Group Chair of COG.
Douglas S. Hawkins, MD
Data on tumor biomarkers were stored exclusively at the hospital or institution where a child was treated, with limited sharing of data among institutions. The new program will make tumor molecular characterization broadly available for children throughout the country. Moreover, the data collected will be available in a central location so childhood cancer researchers can learn from the data and use them to inform future studies.
“We can help make molecular characterization available throughout the country so it will be a standard of care that every child can get,” said Maryam Fouladi, MD, COG’s Central Nervous System Tumor Disease Committee Leader. “An accurate molecular diagnosis can inform optimal treatment for every child.”
In addition to providing detailed information about a cancer to use in making an accurate diagnosis, the data can also be used to determine whether a child is eligible for a clinical trial. Molecular characterization can reveal, for example, whether a child has a specific cancer subtype that is eligible for a clinical trial evaluating a new treatment explicitly designed for that subtype.
Maryam Fouladi, MD
Enrollment in the Molecular Characterization Initiative is initially offered through participation in Project:EveryChildExit Disclaimer, a childhood cancer registry maintained by COG (APEC14B1). Initial participants will include newly diagnosed children, adolescents, and young adults (up to age 25 years) at the time of diagnosis. Young adults older than 25 who are being screened for eligibility into a COG clinical trial may also be included.
