RNA interference for glioblastoma therapy: Innovation ladder from the bench to clinical trials

Figure 1. This diagram summarizes the most important steps in the development of RNAi-based therapies for GBM treatment. The first step involves the identification of potential targets for the RNAi approach; and the evaluation of the efficacy of the RNAi-based therapy in GBM cell lines and animal models. The second step, involves the design of nanocarriers for RNAi delivery into the brain. These carriers, when administered systemically, should be able to cross the blood brain barrier (BBB) and reach GBM tumor tissue. In this step, RNAi-nanocarriers should be characterized in vivo and evaluated for tissue distribution, safety, and efficacy using additional animal models. Finally, clinical trials evaluating overall survival, progression free survival, and safety RNAi doses should be performed.167