Glioblastoma is a highly heterogenous and devastating CNS tumour for which no cure is presently available. The brain patterning gene Emx2 normally inhibits proliferation of astrocyte progenitors. Moreover, it is poorly expressed by human glioblastomas compared to surrounding healthy tissue. Interestingly, we found that Emx2 overexpression induced the collapse of nine out of nine in vitro tested glioblastoma cell lines, antagonizing their proliferation and stimulating apoptosis. Next, it suppressed four out of four of these lines in vivo, upon their transplantation into neocortical parenchyma of immunocompetent mouse neonates. Finally, it increased the survival rate of juvenile immunodefective mice, orthotopically transplanted by U87MG glioblastoma cells.

As proven by dedicated rescue assays, Emx2 activity originated from Emx2 impact on a number of distinct metabolic nodes, which accounts for the robustness of such activity despite glioblastoma heterogeneity. Last but not least,  in two out of two tested lines, the tumor culture collapse was also achieved when Emx2 was driven by a neural stem cell-specific promoter, mainly firing in tumor-initiating cells.

At the moment we are addressing molecular mechanisms mediating Emx2 antiglioblastoma activity. Moreover,  we are working at the development of a therapeutic design based on Emx2 overexpression, suitable for treatment of glioblastoma and prevention of its recurrencies.