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News and reporting on pediatric cancer.
The company's approach combines whole-genome sequencing, CRISPR, liquid biopsy, and gene therapy to target cancer cells with gene fusions.
Use of the drug resulted in an encouraging response when an infant patient received it off-label in a first-line setting.
Comprehensive analysis by a team led by Elaine Mardis has given 93 percent of around 150 pediatric cancer patients at least one medically actionable finding.
An analysis of pediatric cancer survivors suggests subsequent neoplasm risk can increase after certain genotoxic treatments in those with DNA repair gene changes.
The INFORM study echoed the 2019 interim results from the NCI Pediatric MATCH trial, which found that 24 percent of participants were eligible for targeted treatment.
The effort will compare clinical sequencing strategies and explore best practices for educating participants and applying the results to patient care.
The guidance is meant to help provide the pharma industry, researchers, and IRBs with information to facilitate studies of oncology drugs for pediatric patients.
By looking at genes and pathways with altered expression in tumor samples, researchers uncovered potential treatment targets beyond those provided by tumor DNA testing.
Whether they're analyzing checkpoint inhibitors, CAR T cells, or combination regimens, pediatric cancer researchers must carefully consider which targets and drugs to trial.
At the NCI's Childhood Cancer Data Initiative Symposium, speakers emphasized measures such as data harmonization and the need for longitudinal data collection.