MASSACHUSETTS GENERAL HOSPITAL, THE

This proposal is for a Rare Cancer Genetics Registry (RCGR). The proposal is being submitted for Challenge Area: 07: Enhancing Clinical Trials and specific Challenge Topic: Rare Disease Genetic Patient Registry: 07-OD(ORDR)-102. We will develop a registry of individuals with rare cancers who are interested in participating in clinical research. We propose to rapidly recruit a large number of individuals with rare cancers and maintain their data in a secure web-based database. Registrants will give consent to be re-contacted for participation in studies and to provide updates to their clinical information. They will give samples from which DNA will be extracted and stored in a central bank. To develop this infrastructure we will leverage the resources and scientific community of the Cancer Genetics Network (CGN). The CGN Steering Committee will guide decisions on who and what data should be in the Registry, recruit registrants from their cancer clinics, and review all requests for access to resources of the registry. We will build and maintain a web-based resource for communication about the Registry with researchers, including a query tool which can be used to characterize the registrants and samples available for studies. We will provide researchers who contact the RCGR with biostatistical support to plan and design studies using registry subjects. This project will fill an important gap in the research infrastructure, providing a resource to recruit sufficient numbers of cases needed to perform important clinical and translational research such as exploring the genetic determinants of disease and response to therapy. Such studies are often infeasible because the subject pool is small, but have the potential to reveal the etiologic mechanisms not only of specific rare cancers, but possibly of more common and complex tumors. By utilizing the existing infrastructure of the CGN, we will be able to quickly and economically develop this infrastructure within the two years of this Challenge Grant, which can subsequently be supported by research grants from NIH or other agencies. This project also serves the intent of the Challenge Grants related to stimulus of the economy, as it will require hiring personnel at the clinical sites and at the coordinating center who are not currently funded or employed. Thus, this project will be a boost to the economy while improving the research infrastructure, increasing the opportunity for new insights into the causes of and treatment of cancer. This project will fill an important gap in the research infrastructure, providing a resource to recruit sufficient numbers of cases needed to perform important clinical research including clinical trials and analyses of the genetic determinants of disease and treatment efficacy in these rare diseases. Such studies are currently difficult to perform, but have the potential to reveal the etiologic mechanisms not only of specific rare cancers, but possibly more common and complex tumors. By utilizing the existing infrastructure of the CGN, we will be able to quickly and economically develop this infrastructure within the two years of this Challenge Grant, thus rapidly building an important tool for research into rare and under-studied cancers.

Clarification of Codes