Table 1.
2007 | 2017 | Interventions | |
---|---|---|---|
Researcher community | Small, disconnected | >300 active researchers; robust collaboration | International Chordoma Research Workshops; networking events |
Patient community | None | >1,800 patients | Facebook group; website; online advertising and search engine optimization; educational materials; Chordoma Community Conferences |
Tumor tissue | Scarce, no centralized source | Centralized repository with tissue from >200 cases | Tumor donation program allows patients to donate tumor from any US hospital; CF Biobank stores and distributes tumor samples |
Genome characterization | None | 35 cases sequenced | Chordoma Genome Project, in partnership with academic labs |
Cell lines | 1 | 17 | Cell line prize; cell line validation pipeline; genomic characterization; publicly accessible cell-line repository |
Patient-derived xenografts (PDX) | None | 5 | Tumor donation program coupled with rapid implantation in vivo; PDX prize; PDX validation pipeline; publicly accessible PDX repository |
Small-molecule screening | None | >10,000 compounds screened, including all FDA-approved drugs | Collaboration with NIH Chemical Genomics Center; grants to academic investigators; collaborations with industry |
Functional genomic screening | None | Genome-wide CRISPR/Cas9 screens; targeted shRNA screen | Grants to academic investigators |
In vivo evaluation | None | >20 drugs evaluated | CF drug screening pipeline provides in vivo evaluation as a service to academic and industry collaborators |
Clinical trials | 1 completed clinical trial | Pipeline of 7 new clinical trials | Clinical trials program supports trials with: funding, trial design, patient education, and outreach |
Source material: Chordoma Foundation (J. Sommer, personal communication) and www.chordomafoundation.org.