Identification of cantharidin as a drug candidate for glioblastoma by using a Connectivity Map–based approach

Glioblastoma (GBM) is the most common brain tumor in adults. Although the surgical and chemoradiotherapy approaches for treatment have improved, the prognosis of patients with GBM is still poor and novel drugs are urgently required. Therefore, we investigated small molecular inhibitors to target GBM on the basis of gene expression data by using a Connectivity Map (CMAP)–based approach. Using meta-analysis performed with publically available gene expression data, we identified the gene expression signature of GBM. The CMAP analysis identified 15 candidate drugs for GBM treatment. We confirmed the anticancer cell proliferation activity of cantharidin as one of the top 15 drugs with high negative enrichment scores in CMAP analysis by using GBM cell lines. Our results indicate the potential utility of CMAP to discover the potent drugs in the GBM treatment. This approach can be applied to other malignancies than GBM.