Figure 21.

Nanoscopic formulations for the delivery of therapeutics to the brain. NU-0129 is a gold nanoparticle formulation whose surface is conjugated with of polyethylene glycol chains and small interfering RNAs (siRNAs) that target the Bcl-2-like protein. NU-0129 is currently under clinical trial for its antineoplastic activity and potential in the treatment of recurrent glioblastoma multiforme. SGT-53 is a cationic liposome in complex with its cargo, a genetic therapeutic material. The liposomal surface is conjugated with an anti-transferrin receptor single-chain antibody fragment that serves as the targeting vector. SGT-53 is currently under clinical trial for its potential to induce apoptosis in patients with recurrent glioblastoma. Onzeald ^® (or etirinotecan pegol) is a pegylated irinotecan formulation with a long-acting effect. Onzeald ^TM is currently under clinical trial to evaluate its efficacy at the control of brain metastases. Onivyde^® and Doxil^® are liposomal formulations that encapsulate irinotecan and doxorubicin, respectively, in an aqueous space. The liposomal surface of Onivyde^® and Doxil^® is conjugated with chains of polyethylene glycol to improve their colloidal stability and non-immunogenicity. Because of their long-acting effect, Onivyde^® and Doxil^® are under clinical trials to evaluate their efficacy at treating patients with glioblastoma. Doxil^® is also under pre-clinical investigation to assess its efficiency at the treatment glioblastoma after transient disruption of the BBB induced by microbubble-assisted focused ultrasound (MB-FUS).