Because pediatric cancers are rare, there is less data to work with regarding tumor genomics, and fewer available targeted drugs than for adult patients. Despite these limitations, Dr. Katherine Janeway of the Dana-Farber Cancer Institute (DCFI) has been working to bring personalized, molecularly targeted treatments to children with cancer. The results of her first study, called Individualized Cancer Therapy (iCat), were published in 2016 and showed that bringing clinical genomic sequencing to pediatric oncology practice is feasible.
Dr. Janeway and colleagues are now conducting a follow-up study, the Genomic Assessment Improves Novel Therapy (GAIN) Consortium study. The GAIN Consortium includes more than twenty pediatric oncology academic medical centers, led by DCFI, and its work runs on a platform developed by the CRI.
The primary goal of GAIN is to offer molecular diagnostics to children with solid tumors and leukemia. Member institutions enroll eligible patients and take samples of their tumors, which are transported to DFCI and the Broad Institute for sequencing. Based on this analysis, a comprehensive report is generated for the referring physician. This report identifies any changes in the genes of the tumor that are associated with the success of a particular therapy, allowing the doctor to develop an individualized treatment plan for the patient that takes into account the genomics of their tumor. The study will then track each patient to study the effect these individualized plans have on treatment outcomes.
In order to carry out the many steps of this study, the GAIN project has complex technological needs. To begin, it requires a highly regulated and integrated system for enrolling patients and collecting, centralizing, and tracking samples for processing and analysis. After this phase, the molecular pathologists performing the analysis require an advanced, integrated knowledge management system with which to develop diagnostic reports, capable of bringing together multiple databases for knowledge extraction, genomic variant calling, and report generation.
Thanks to our history of developing highly customized and integrated knowledge management solutions for multi-institution research, the CRI was selected to lead the development, deployment, and management of the platform for the GAIN study. Our expertise across several areas allowed us to build a unified system supporting acquisition, analysis, and storage of biospecimens that exceeded the consortium’s design specifications.
Our Custom Applications team, led by Brian Furner, built an innovative web-based case and specimen tracking system. This system moved into production in June 2016 and will coordinate with the GAIN iCAT system developed by our Bioinformatics Core, which will be used for the analysis portion of the study. This integrated research platform runs on our secure VMware infrastructure. The CRI is proud to contribute the diverse abilities of three of our major service lines to this promising study.