Precision Medicine in Pediatric Cancer

Recognizing these challenges, the National Cancer Institute (NCI) has called for increased emphasis on advancing precision medicine for pediatric cancer into clinical care. It convened a workshop last year, as its follow-up summary of the 2-day gathering explained, “to discuss the influence of genomic discoveries on the future of childhood cancer research.”³

To speed progress, the workshop’s members concluded, researchers needed a centralized means of sharing information to “facilitate collaboration across research teams and to facilitate the identification and clinical relevance of low-frequency genomic alterations.”

Just over a year later, in June 2016, the NCI launched the Genomic Data Commons (GDC), which it describes as “a unified data repository that enables data sharing across cancer genomic studies in support of precision medicine.”

To specifically further advances in precision medicine for pediatric patients, source information for the GDC includes research data from the NCI’s Therapeutically Applicable Research to Generate Effective Treatments (TARGET) program aimed at characterizing the genomes and transcriptomes of a variety of childhood cancers including subtypes of acute lymphoblastic leukemia, acute myeloid leukemia, neuroblastoma, osteosarcoma, and some types of kidney tumors.

The continuing studies and sharing of information offer encouraging possibilities for researchers. As the authors of the pediatric oncology review wrote, “both tumor and germline alterations identified in these studies target a diverse set of genes, including many that were not previously known to be associated with the patient’s cancer type or in pediatric cancer, emphasizing the potential yield of genome-scale testing for these patients.”

The hope is that continuing research in genomic sequencing and targeted treatment for pediatric cancer will produce results similar to the progress seen with adult cancers.

“Ultimately, patients may have individually tailored dosing regimens based on their specific physiology. Such prospects have the possibility of dramatically changing the way medicine is practiced,” the review’s authors concluded.

References

1. Mody RJ, Prensner JR, Everett J, Parsons DW, Chinnaiyan AM. Precision medicine in pediatric oncology: Lessons learned and next steps. Pediatr Blood Cancer. 2016 Oct 17. doi: 10.1002/pbc.26288 [Epub ahead of print]
2. Harris MH, DuBois SG, Glade Bender JL. Multicenter feasibility study of tumor molecular profiling to inform therapeutic decisions in advanced pediatric solid tumors: The Individualized Cancer Therapy (iCat) Study. JAMA Oncol. 2016 Jan 28. doi: 10.1001/jamaoncol.2015.5689 [Epub ahead of print]
3. Childhood cancer genomics gaps and opportunities: identification of research priorities. National Cancer Institute website. https://www.cancer.gov/types/childhood-cancers/research/childhood-genomics-workshop-summary.pdf. Updated June 15, 2015. Accessed November, 2016.