Abstract
Cancer Glioblastoma (GBM) is a difficult cancer to treat, in part because of the blood–brain and blood–tumor barriers. Kumthekar et al. studied the drug NU-0129, which consists of a gold nanoparticle core conjugated to small interfering RNA oligonucleotides targeting the oncogene Bcl2L12 , for the treatment of GBM. After performing toxicology studies in cynomolgus monkeys, the authors conducted a first-in-human single-arm clinical trial of very-low-dose NU-0129 in eight patients with recurrent GBM. By analyzing gold accumulation, the authors found that NU-0129 passed through the blood–brain barrier and accumulated in the tumor, where it reduced Bcl2L12 protein abundance. These results suggest that spherical nucleic acid conjugates can potentially treat GBM. Sci. Transl. Med. 13 , eabb3945 (2021).
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