For the first time, scientists are testing gold nanoparticles loaded with drugs in glioblastoma patients.
The study is a first-in-human phase 0 clinical trial testing the efficacy and safety of a treatment using gold nanoparticles for glioblastoma. The gold particles function as carrier of gene-inhibiting molecules, called small interfering RNA (siRNA), targeting the gene Bcl2L12 in patients suffering from recurrent glioblastoma.
The innovative approach was tested in eight patients and in non-human primates and showed that the gold nanoparticles managed to enter the brain and target the cancer-related gene.
Glioblastoma and other brain cancer are particularly difficult to treat, in great part due to the presence of blood-brain and blood-tumour barriers, which complicates the delivery of drugs.
In Australia, according to data from the Australian Institute of Health and Welfare, around 1,879 cases of brain cancer were diagnosed in 2020. Most of them will die from their condition – for the same year, the AIHW reported 1,518 deaths due to brain cancer.
There is a dire need to develop new and more effective ways to treat brain cancer.
A golden idea
To address this issue, a team of American researchers has created nanoparticles called RNA interference–based spherical nucleic acids or SNAs. Each of these nanoparticles is made of a gold nanoparticle, densely coated with radially oriented small interfering RNA (siRNA) molecules.
siRNAs, also known as silencing RNA, are small pieces of double-stranded RNA that can stop the translation of genes into proteins, effectively “silencing” genes. siRNA is an integral part of all human, as well as mammalian and plant, cells, where they help with the regulation of gene function, specifically silencing target genes through the process of RNA interference.
In recent years, scientists have used siRNAs to target genes involved with diseases, such as cancer.
In this new study, siRNA is used to stop the expression of the gene Bcl2L12, a gene involved in many cancers. Their approach is backed by previous pre-clinical data in animal models, generated by the same research team, which demonstrated the efficacy of their technique.
Now, in this novel human clinical trial, researchers show that their technique is safe for humans and accomplishes the goal of reaching the brain, overcoming all barriers, and effectively deliver the loaded gold nanoparticles and inhibit the target cancer-causing gene.
The findings of this study stand as a promise for the development of a new, safe, and effective treatment for a deadly and hard to treat brain cancer.
[Credit: Kumthekar et al.]