Molecular guided therapy leading to exceptional response in relapsed osteosarcoma. (PubMed, Front Pharmacol)
RNA transcriptome sequencing of the subject's tumor showed overexpression of SLC29A11 (Z-score = 3.3) indicating sensitivity to gemcitabine as well as activation of the biological pathways mTOR, CSF1R, EPHA2, SLC29A1, suggesting possible beneficial treatment with a combination of everolimus, gemcitabine, doxycycline and dasatinib. The subject responded to the novel drug combination, continuing medications for 5 years with some modifications, and remained on everolimus alone for an additional 4 years with a complete response, no serious adverse events, and excellent quality of life. In conclusion, Molecular Guided Therapy with tumor board recommendations resulted in a novel therapeutic approach leading to long term survival which correlated to response in vitro.