The encouraging study outcome including achievement of cCR in eleven patients and downregulation of biomarkers in the post-treatment tissues confirmed the previously described mechanism (suppression of yes-associated protein 1/SRY-box transcription factor 9 (YAP1/SOX9) signaling axis) in vitro as well as in a mouse model and suggested AT-101-related downregulation of cancer stem cells. The gene discussed is SOX9; the disease is cancer.