Novel Glioblastoma Drug Can Cross The Blood-brain Barrier
An experimental spherical nucleic acid (SNA) drug was able to penetrate the blood-brain barrier and trigger glioblastoma tumour cell death in an early clinical trial.
Glioblastoma is the most common and aggressive brain tumour, accounting for 16% of cases. It affects 3.2 per 100 000 people, at an average age of 64 years although it can appear at any time.
The new drug, NU-0129, is the first SNA drug developed for systemic use. The SNA groups RNA or DNA around a nanoparticle. A revolutionary new class of drugs, it can be adapted to a number of neurological diseases such as Parkinson’s.
“We showed the drug, NU-0129, even at very small doses, causes tumour cells to undergo what’s called apoptosis or programmed cell death,” said lead investigator Dr Priya Kumthekar, associate professor of neurology at Northwestern University Feinberg School of Medicine and a Northwestern Medicine physician. “It’s a remarkable finding in humans that confirms what we had previously seen in our animal studies.”
The study participants received the drug intravenously prior to surgery to remove the tumour. The researchers team studied the tumours to determine how well the drug crossed the blood-brain barrier and its effect on their cells.
“This unique 3D design has the ability to infiltrate tumor cells to correct the genes inside and make them susceptible for therapy-induced killing,” said senior author Alexander Stegh, an associate professor of neurology at Northwestern.
Unusually, the drug was developed entirely within the university without involving pharmaceutical licensing.
“We want to move the technology forward as quickly as possible because there are patients with a disease with no current cure,” Kumthekar said.
Dr Leon Platanias, director of the Lurie Cancer Center, said, “These exciting findings for the first time support the potential of spherical nucleic acids for drug delivery to brain tumors. They may prove to have important long-term translational implications for the treatment of these tumours.”
Source: Medical Xpress
Journal information: P. Kumthekar el al., “A first-in-human phase 0 clinical study of RNA interference–based spherical nucleic acids in patients with recurrent glioblastoma,” Science Translational Medicine (2021). stm.sciencemag.org/lookup/doi/ … scitranslmed.abb3945
