The purpose of our Drug Screening initiative is to identify compounds that are likely to be beneficial to chordoma patients by evaluating their efficacy in preclinical models of chordoma. Large libraries of compounds will be screened against chordoma cell lines and the compounds that show the greatest activity will be tested in chordoma xenograft models to evaluate their ability to slow or stop the growth of chordoma tumors. Initially, screening will focus on approved drugs; compounds already on the market, and, therefore, available to patients immediately. Results from this screening could provide the rationale needed to justify new clinical trials, and could quickly impact the treatment of chordoma patients.
There are over 2,800 FDA-approved drugs, however only four have ever been tested in clinical trials for chordoma patients. To identify possibly effective drugs, we worked with the NIH Chemical Genomics Center (NCGC) to test all of these approved drugs against chordoma cell lines. From this screening, over 40 promising drugs have been found that selectively inhibit the growth of chordoma cells.
We are deeply grateful to the nearly 400 donors who contributed $200,000 needed to test the top 10 approved drugs!
For each additional $20,000 that is raised we will be able to screen one more drug.
We are currently funding Dr. Gary Gallia at Johns Hopkins University to test the most promising drugs identified by the NCGC screen in newly-developed chordoma mouse models. Dr. Gallia developed the first primary skull-base chordoma xenograft, and has an extensive track record of conducting experiments in other primary xenograft models.
Initially Dr. Gallia is testing the top 10 drugs in two chordoma mouse models. However, to validate the results, the drugs need to be tested in more than two mouse models. As funding becomes available, we will screen additional models and additional drugs, with the goal of testing at least 20 drugs in at least three mouse models.