Christopher Austin (NCATS)
Ruili Huang (NCATS)
This technology includes a group of 20 drugs that can be further developed as a treatment for chordoma. Chordoma is a rare, slow-growing malignant tumor arising from remnants of the fetal notochord, with a high recurrence rate and no effective chemotherapy agents. These 20 drugs inhibited chordoma cell growth, with potencies ranging from 10 to 370 nM in the chordoma cell line UCH1 cells. These agents also had significant inhibitory effects on chordoma patient cells, C24, C25, and C32. Furthermore, a combination treatment of proteasome inhibitors, such as bortezomib and MG-132, with topoisomerase inhibitors, such as topotecan, rubitecan, camptothecin, doxorubicin, and daunomycin, significantly increased the therapeutic potency in the human chordoma cell line, UCH2, and primary chordoma patient cells, C24, C25, and C32.
The individual drugs and drug combinations in this technology may be further developed for chordoma treatment.
Currently, there are no effective chemotherapy agents available for treating chordoma and since many of these agents have been previously tested or approved for use in humans or animals, they are good candidates as chemotherapeutic agents for chordoma.