In the present study, we have attempted to evaluate the potential of developing acetylshikonin, a major biologically active compound present in Lithospermum erythrorhizon root (Cho, Paik, & Hahn, 1999; Rajasekar et al., 2012), as a TOPK inhibitor and assess its anti‐cancer effects in cultureds of colorectal cancer cells and in patient‐derived xenograft (PDX) tumour models in mice. The gene discussed is PBK; the disease is neoplasm.