Unmet Need
A large number of cancers, including glioblastoma (GBM), lack effective treatment due to inaccessibility of the primary tumor to pharmaceutical interventions. GBM is highly malignant, leading to 5-year survival rates below 10%. The inability to treat GBM tumors effectively is largely due to the blood brain barrier (BBB). Common approaches to increase drug loading at the site of the tumor rely on nanoparticle formulations, but these delivery mechanisms still result in subclinical levels of drug at the intended site. Thus, there is a large unmet need for mechanisms that improve drug delivery to the site of GBM and other brain cancers.
Technology Overview
The investigators have described a mechanism to deliver chemotherapeutic drugs using single stranded DNA (ssDNA) nanotubes. The engineered nanotubes have been demonstrated to cross the BBB and reach the target GBM tissue following systemic injection in mice. Furthermore, the concentration of nanoparticles present in the tumor 24 hours following administration is up to 10 times higher than that for other nanoparticle formulations in development. In vitro and in vivo studies have demonstrated that they bind to and are internalized by glioma cells, but not healthy astrocyte cells. The nanotubes have shown a robust safety profile in mouse studies. The described studies in vitro and in mice reveal that these nanotubes are an attractive vehicle for delivery of chemotherapeutic drugs to GBM tumors. Additionally, this technology could potentially be applied to other tumor types where drug loading at the tumor site is a major barrier to pharmaceutical treatment.
Stage of Development
The investigators have characterized these nanotubes in vitro, and have performed in vivo studies in a mouse model of brain cancer.
