NIH launches program to offer a molecular characterization of childhood cancers

In support of United States President Biden’s goal of fostering data sharing in cancer research, the National Cancer Institute (NCI) has launched the Molecular Characterization Initiative for pediatric tumors. This program will offer tumor molecular characterization, also known as biomarker testing, to young people – children, adolescents, and young adults with recently diagnosed central nervous system tumors that are being treated at hospitals affiliated with the Children’s Oncology Group (COG).

National Cancer Institute (NCI) is part of the National Institutes of Health (NIH).

Children’s Oncology Group (COG) is a National Cancer Institute (NCI)-supported clinical trials group which includes more than 200 hospitals and institutions that will treat most children diagnosed with cancer in different parts of the United States.

The Molecular Characterization Initiative is offered through National Cancer Institute (NCI) ’s Childhood Cancer Data Initiative. NCI launched the initiative in 2019 to promote data sharing and collection of new data among various researchers who study childhood cancers.

Children, adolescents, and young adults diagnosed with central nervous system cancer will now be eligible to receive molecular characterization of the tumors free of charge under this voluntary program. DNA and RNA from the tumor and blood samples can be analyzed to assist in getting a more accurate diagnosis and thus understand what is causing or driving this cancer. The Molecular Characterization Initiative will expand later in 2022 to include other rare tumors like soft tissue sarcomas.

“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,” commented Brigitte C. Widemann, M.D. and a special advisor to the National Cancer Institute (NCI) director for childhood cancer. “The Molecular Characterization Initiative is a transformative collaboration that will entail participation of the entire community.”

Having a precise diagnosis based on these molecular characteristics of the tumor can help choose the most effective and potentially least toxic of different treatment options for each patient. Data on the molecular changes observed across childhood cancers may also help researchers better understand the molecular causes of these childhood cancers and thus accelerate the development of novel, more effective, and less risky treatments, especially in cases of such rare childhood cancers where 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,” asserted Douglas S. Hawkins. He is an M.D. and a group chair of COG.

“We can help make molecular characterization available throughout the country so that it will be a standard of care that every child can get,” reflected Maryam Fouladi, another M.D. at COG. “An accurate molecular diagnosis can inform optimal treatment for every child.”

Dr. Fouladi explained that some childhood cancers, such as gliomas, can be easily misdiagnosed. “We can apply molecular diagnostics to a child diagnosed with a high-grade glioma and find out that it is a low-grade glioma or an entirely different tumor, which may need very different treatments and have a very different outcome,” she said. “Molecular diagnostics can really contribute to getting the correct diagnosis, offering the optimal treatment and, ultimately, improving the patient’s outcome.”

Dr. Widemann commented, “To be able to apply the best tools to make the most accurate diagnosis so that the most effective treatment can be prescribed, that’s a goal that I think physicians and families can all align around.”

The National Cancer Institute (NCI) leads the country’s National Cancer Programme and NIH’s efforts to reduce the prevalence of various types of cancer and thus improve the quality of the lives of cancer patients and their caretakers with research into prevention and cancer biology as well as the development of new interventions.