The implementation of functional precision medicine , which combines genomic profiling with drug sensitivity testing of patient-derived tumor cells, has potential to identify treatment options when standard-of-care is exhausted. The goal of this prospective observational study was to generate FPM data for pediatric patients with relapsed or refractory cancer.
Overall, the addition of functional drug testing to current personalized medicine platforms has promising potential to expand treatment options when limited alternatives exist. This is especially valuable when assessing drugs whose mechanisms of action are poorly understood or not robustly characterized.
Sequencing was performed by BGI using the DNBSeq G400 sequencer and by Novogene using the Illumina NovaSeq6000 sequencer, and data were analyzed using previously established analysis pipelines based on best practices). To call variants from RNA sequencing data, post-quality-control FASTQ files were aligned to the GRCh38 human reference genome using STAR v.2.7.10b and processed using GATK v.4.
Division of Pediatric Hematology Oncology, Department of Pediatrics, Nicklaus Children’s Hospital, Miami, FL, USACenter for Precision Medicine, Nicklaus Children’s Hospital, Miami, FL, USAD.J.A. and D.S. conceived and designed the study. M.F., Z.K., O.M.M. and H.A., members of the FPMTB, collected and provided patient tissues used during the study. D.J.A., N.E.B., P.S.E., D.S., A.M.A.D.L.R., E.R.C., C.M.A.F., J.G., Y.V., A.D., A.J., V.R., B.H., Y.G., L.R. and T.R.G. collected and assembled data.
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