An integral first important step in this targeted therapeutic research initiative is to build an integrated comprehensive database that would ‘house’ the genetic tumor information for subsequent comparative analysis.
It is now technically and financially feasible to generate extensive genome-wide data sets on individual human subjects, including extensive clinical and laboratory data, 500k SNP chips for CGH and genetic association studies, mRNA expression and splicing arrays, quantitative proteomic profiling, and re-sequencing data.
Essential to collaborative molecular pediatric cancer research is the development of an integrated database structure that is “standardized” both in data input, data fields, and data integration. Such databases would put more focus on quality control and standard operating procedures, with an effort to provide consistency in data structures that would enable more powerful data analysis tools. It is believed by experts in the field that the development of an integrated database would more quickly and more accurately achieve the molecular tissues analysis necessary to move this essential drug development forward.
To begin collaborations on this necessary research initiative, Candlelighters initiated a 2 day work shop funded by the American Cancer Society and the National Cancer Institute in May 2004. We subsequently hosted a workshop of interested childhood cancer organizations at the World Cancer Congress held in July 2006. This led to collaborations with St. Baldrick’s Foundation and Children’s National Medical Center to study the existing molecular database structures worldwide.
The study showed that caBIG, and especially caIntegrator were the most mature of those databases analyzed currently available for integrating clinical data, mRNA profiling, CGH, and resequencing data. The caBIG resource, while primarily developed at the NCI, and also initially hosted there, was built to be purposefully ‘de-centralized,’ with ‘adopters’ at different centers nationally. The caBIG resource was also providing support to NCI-funded pediatric cancer initiatives (leukemias, and more recently Dr. Maris’ TARGET neuroblastoma project). To support multiple pediatric cancer projects using caIntegrator, one or more external, non-NCI (University or biotech) adopters of caIntegrator (and other caBIG resources) should be developed for pediatric cancers.
To move ACCO into the research realm, we were proud to be recipients of the funding raised through the gala ‘Naomi’s Hope for a Cure’ (www.hope4acure.net). All proceeds from this gala event were donated to Candlelighters for our Targeted Therapies Research Initiative.
Next Steps
To encourage further research focused on improving translational medicine information systems, American Childhood Cancer Organization, in collaboration with St. Baldricks Foundation (www.stbaldricks.org) and Alex's Lemonade Foundation (www.alexslemonade.org) will provide funds to Lombardi Comprehensive Cancer Center at Georgetown University to host and conduct a workshop in May 2009, which aims to bring together experts from research, professional, and government sectors. The workshop will dedicate one day to individual and panel presentations to identify and prioritize key challenges. We plan to invite experts with substantial track records in the area, industry experts, program managers in NIH, and other key leaders in the field of cancer informatics, and build upon the current NCI TARGET initiative.
In the “omics” era, clinical research relies heavily on database-supported bioinformatics. Integrating our knowledge about disease phenotypes and finding preventive and treatment interventions are central to how we approach informatics in support of large-scale studies with high-dimensional datasets such as those generated by high throughput genomics and proteomics platforms. Under the broad theme of "informatics in support of translational research in childhood cancers," in this workshop, we plan to explore research challenges and emerging solutions for handling translational medicine data from the point of origin (i.e., data collection) to its presentation and manipulation using user-friendly data portals. Workshop participants will explore a wide spectrum of health data from childhood cancer studies ranging from–genomics and cellular level, to the patients’ level and ultimately to the population level.
Upon conclusion of this workshop it is anticipated that American Childhood Cancer Organization, St. Baldricks, Alex's Lemonade Foundation and other childhood cancer non-profits will take the lead in a new national initiative through the development of an integrated pediatric oncology molecular database.
Through the development of targeted therapeutic drugs, children with cancer world-wide could be given hope for a cure, and hope for long term survival that would be exempt from life-threatening late effects of current toxic treatment.